Clinical Trials - Gene Therapy Trial Browser
The Gene Therapy Trial Browser represents a unique publicly accessible, free database for the benefit of users seeking information on gene therapy development. The information within integrates various sources, including clinicaltrials.gov, publications, sponsor press releases, patent applications, and more to give a comprehensive overview of the gene therapy clinical trial landscape.
Showing all 380 results ... in ClinicalTrials
SCGE Platform Gene Therapy Clinical Trials downloaded on: 2025/05/28 16:33:41; Please cite the Somatic Cell Genome Editing Consortium Platform when using publicly accessible data in formal presentation or publication.
Definitions
Abbreviation
| AAV | Adeno-associated virus, e.g. AAV2, AAV5, AAV2/5 indicates virus containing the genome of serotype 2 packaged in the capsid from serotype 5 |
| ABE | Adenine base editor |
| Ad | Adenovirus |
| Cas9 | CRISPR associated protein 9 |
| CBE | Cytosine base editor |
| CRISPR | Clustered regularly interspaced short palindromic repeats |
| Gc | Genome copies |
| HIV | Human immunodeficiency virus |
| HSV | Herpes simplex virus |
| LNP | Lipid nanoparticle |
| LV | Lentivirus |
| MRNA | Messenger ribonucleic acid |
| ODD | Orphan Drug Designation |
| PFU | Plaque forming units |
| RMAT | Regenerative Medicine Advanced Therapy |
| RNP | Ribonucleoprotein |
| RPDD | Rare Pediatric Disease Designation |
| RV | Retrovirus |
| START | Support for Clinical Trials Advancing Rare Disease Therapeutics |
| Vg | Vector genomes |
| VSV-G | Vesicular stomatitis virus G |
Delivery Type
| Electroporation | Cell membrane permeablized by electrical field to allow gene therapy to enter the cell |
| Lipid encapsulation | Any lipid nanoparticle used to deliver editor, corrected gene |
| Microinjection | Drug is injected into individual cells |
| Plasmid | Any plasmid used to deliver editor, corrected gene |
| Viral transduction | Any virus used to deliver editor, corrected gene, etc. |
FDA Designation
| Accelerated Approval | These regulations allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint |
| Breakthrough Therapy | A process designed to expedite the development and review of drugs which may demonstrate substantial improvement over available therapy. |
| Fast Track | Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need |
| Orphan Drug | This designation is for drugs intended to treat rare diseases or conditions that affect a small number of people, offering incentives like tax credits and market exclusivity |
| Priority Review | A Priority Review designation means FDA’s goal is to take action on an application within 6 months. |
| Rare Pediatric Disease | The rare pediatric disease PRV program aims to incentivize drug development for rare pediatric diseases. |
| Regenerative Medicine Advanced Therapy | Facilitates the development and expedites the review of regenerative medicine therapies, including cell therapies, therapeutic tissue engineering products, and human cell and tissue products, for serious or life-threatening conditions. |
| Support for Clinical Trials Advancing Rare Disease Therapeutics | (START) Pilot Program is a program designed to accelerate the development of novel drug and biological products for rare diseases by providing sponsors with enhanced communication and guidance from FDA staff. |
Funder Type
| Industry | All for-profit entities |
| NIH | U.S. National Institutes of Health |
| Other Non-Profit | Includes individuals, universities, community-based organizations |
Route Of Administration
| CED | Convection enhanced delivery to the brain |
| Inhalational | Delivered to the lungs in the form of a fine spray |
| Intraarterial | Into the lumen of an artery |
| Intraarticular | Into a joint space |
| Intracerebroventricular | Into the ventricles of the brain (ICV) |
| Intracisterna magna | Into the cisterna magna of the brain |
| Intracochlear | Into the cochlea of the ear |
| Intradermal | Into the dermal layer of the skin |
| Intragastric | Into the stomach |
| Intramuscular | Into a skeletal muscle |
| Intraocular | Administered into the eye |
| Intraparenchymal | Into the brain |
| Intraperitoneal | Into the peritoneal cavity |
| Intrastromal | Into the stroma of the cornea |
| Intrathecal | Into the spinal canal |
| Intravenous | Into a vein |
| Intravesicular | Into the bladder |
| Intravitreal | Into the eye (IVT) |
| Subcutaneous | Under the skin |
| Subretinal | Under the sensory retina |
| Suprachoroidal | Into the suprachoroidal space between the sclera and the choroid of the eye |
| Topical | Applied to the outer layer of the skin |
Stages
| Early Phase I | Describes exploratory trials conducted before traditional Phase I trials to investigate how or whether a drug affects the body, have no therapeutic or diagnostic goals |
| Phase I | Describes clinical trials that focus on the safety of a drug |
| Phase II | Describes clinical trials that gather preliminary data on effectiveness, continue to monitor safety |
| Phase I/II | Combination Phase I/Phase II clinical trial |
| Phase III | Pivotal experiments to gather data on safety and effectiveness |
| Phase II/III | Combination Phase II/Phase III clinical trial |
Study Status
| Active, Not Recruiting | Study has ongoing, but is not enrolling new participants |
| Completed | Study has concluded normally |
| Not Yet Recruiting | Study has not started enrollment |
| Recruiting | Study is actively looking for participants |
| Suspended | Study halted prematurely but has the potential to resume |
| Terminated | Study was halted prematurely and will not resume, participants are no longer recieving intervention |
| Unknown | Study has passed its completion date, but last known status was not listed as Completed, Terminated or Withdrawn. Status has not been verified within the past 2 years. Studies with an unknown status are considered closed studies |
Table Column Header
| Actual Study Start Date (m/d/y) | The actual date on which the first participant was enrolled in a clinical study |
| Adult/Pediatric/Both | Variable on whether trial accepts patients who are adults, pediatric (<18 years of age) or both |
| Ages Eligible for Study | More specific age ranges eligible for the study |
| Clinical Centers in USA? | Binary variable on whether trial sites are located in the USA (Y) or not (N) |
| Clinical Publications | URL connections to clinical data on human subjects |
| Compound Name | The interventional compound given to the study subjects |
| Countries | List of countries that contain at least 1 clinical trial site |
| Current Stage | The stage of the clinical trial, determined based on the studies' objective |
| Date of Last Update | The most recent date on which changes to a study record were made available on ClinicalTrials.gov |
| Delivery System | Describes the nature of the drug substance or process that is used to deliver the editor or corrected gene |
| Development Status | Indicates whether or not the clinical development program is ongoing, or if the drug is approved |
| Dose levels (up to 5) | List of doses given in the indicated clinical trial, may be expressed in specific units, or a range of possible doses |
| Drug Product Type | Describes the nature of the drug product |
| Editor Type | For gene editing type therapies, the protein that will perform the gene correction |
| Estimated Primary Completion Date (m/d/y) | The anticipated date that the primary outcome measure data will be complete (last participant data collected) |
| FDA Designations | List of special FDA designations granted to the Sponsor for the development program (i.e. Orphan Drug Designation, Fast Track, Rare Pediatric Disease Designation, RMAT, etc.) |
| Funder Type | Describes the organization that provides support for the clinical study |
| Grants | URL connections to funding used to conduct preclinical or clinical studies, granted by NIH or other US institution |
| Has US IND? | Binary variable indicating whether the drug product is regulated by an approved Investigational New Drug application by the Food and Drug Administration of the United States |
| Indication | The disease, disorder, syndrome, illness, or injury that is being studied |
| Locations | List of countries that contain at least 1 clinical trial site |
| Mechanism of Action | Simplified description of how the drug product works |
| NCT Number | Unique identification code given to each clinical study upon registration at ClinicalTrials.gov |
| News and Press Releases | URL connections to press releases generated by drug product Sponsor |
| N trial sites | Number of clinical sites where the clinical trial is conducted |
| Patents | URL connections to patents, intellectual property related to the drug product |
| Phases | The stage of the clinical trial, determined based on the studies' objective |
| Preclinical Publications | URL connections to preclinical data (in vitro, animal data) |
| Protocols | URL connections to clinical trial protocols, study design papers |
| Recent Regulatory Updates | News, updates on recent or anticipated regulatory milestones |
| Recruitment Status | Indicates the current recruitment status or the expanded access status |
| Results Posted | Indicates if summary results are posted to the clinical trial record |
| Route of Administration | How the drug product is introduced to the body |
| Sexes Eligible for Study | A type of eligibility criteria that indicates the sex of people who may participate in a clinical study (all, female, male) |
| Sponsor | The organization or person who initiates the study and who has authority and control over the study |
| Sponsor Class | Describes the organization that provides support for the clinical study |
| Standard Ages | Variable on whether trial accepts patients who are adults, pediatric (<18 years of age) or both |
| Target Gene or Variant | The symbol of the gene that is corrected or replaced by the drug compound |
| Target Tissue or Cell | Lists target cells if the therapy is directed at a particular cell type (either by ex-vivo enrichment, or tissue-specific regulatory elements) |
| Therapy Route | Describes whether the gene therapy is introduced to cells in-vivo or ex-vivo |
| Therapy Type | Describes the nature of the gene therapy |
| Trial Enrollment | Number of study subjects planned or actually enrolled in the study |
| Vector Type | Gives additional specifics (if known) about delivery system (e.g. AAV serotype) |
Therapy Route
| Ex-vivo | When the cells are modified outside the body |
| In-vivo | When the cells are modified inside the body |
Therapy Type
| Gene editing | A gene therapy where disease-causing variant is corrected via a gene editor |
| Recent Regulatory Updates | A gene therapy where a corrected gene is administered to relevant cells/tissues via a delivery system |
| Trial ID | Indication | FDA Designation | Compound Name | Sponsor | Funder Type | Target Gene/Variant | Therapy Type | Therapy Route | Mechanism of Action | Route of Administration > | Drug Product Type | Target Tissue/Cell | Delivery System | Vector Type | Editor Type | Dosage/s | Current Stage | Recruitment Status | Development Status | Submit Date > | Completion Date | Last_Update | Eligibility Age | Enrollment Count | No. of Trial Sites | Locations | Has US IND | Patents | Recent Updates | Resources/Links |
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| Trial ID | Indication | FDA Designation | Compound Name | Sponsor | Funder Type | Target Gene/Variant | Therapy Type | Therapy Route | Mechanism of Action | Route of Administration > | Drug Product Type | Target Tissue/Cell | Delivery System | Vector Type | Editor Type | Dosage/s | Current Stage | Recruitment Status | Development Status | Submit Date > | Completion Date | Last_Update | Eligibility Age | Enrollment Count | No. of Trial Sites | Locations | Has US IND | Patents | Recent Updates | Resources/Links |
| NCT04483440 | Choroideremia | Orphan Drug Designation | 4D-110 | 4D Molecular Therapeutics | Industry | CHM | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV R100 | Dose 1: 3E11 vg/eye | Dose 2: 1E12 vg/eye | Phase1 | Active not recruiting | Inactive | 2020-07-20 | 2027-06 | 2024-05-09 | >= 18 Years | 13 | 2 | United States | January 2025: Sponsor announced they would terminate development of this program | |||||
| NCT05248230 | Cystic Fibrosis | Orphan Drug Designation, Rare Pediatric Disease Designation | 4D-710 | 4D Molecular Therapeutics | Industry | CFTR | Gene transfer | In-vivo | Functional gene replacement | Inhalational | Viral vector | Airway epithelial cells | Viral transduction | AAV A101 | Dose 1: 2.5E14 vg | Dose 2: 5E14 vg | Dose 3: 1E15 vg (maximum tolerated dose) | Dose 4: 2E15 vg (discontinued due to high transduction to interstitium) | Phase2, Phase1 | Recruiting | Active | 2022-02-10 | 2030-01 | 2025-02-21 | >= 18 Years | 40 | 17 | United States | US11499166B2; WO2023172873A2 | Phase 1 AEROW enrollment completed in November 2024 (Cohorts 3 & 4 fully enrolled with n=3 each), follow-up ongoing; Interim data update expected to be presented in mid-2025 |
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| NCT04519749 | Fabry Disease | Fast Track, Orphan Drug Designation | 4D-310 | 4D Molecular Therapeutics | Industry | GLA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Cardiomyocyte | Viral transduction | AAV C102 | 1E13 vg/kg | Phase2, Phase1 | Active not recruiting | Active | 2020-08-14 | 2030-06 | 2024-04-08 | >= 18 Years | 18 | 4 | United States | WO2021222094A1 | No further significant investment is expected on this program, pending additional financing or partnerships |
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| NCT05930561 | Diabetic Macular Edema, Diabetic Retinopathy | 4D-150 | 4D Molecular Therapeutics | Industry | Anti-VEGF | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV | Dose 1: Dose range: 5E9 -3E10 vg/eye | Dose 2: Planned Phase 3 dose: 3E10 vg/eye | Phase2 | Recruiting | Active | 2023-06-26 | 2028-07-01 | 2024-12-04 | >= 18 Years | 72 | 15 |
Locations:United States + 1 more
Puerto Rico, United States
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52-week interim data update expected at a scientific conference in mid-2025 |
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| NCT05197270 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | Regenerative Medicine Advanced Therapy | 4D-150 | 4D Molecular Therapeutics | Industry | Anti-VEGF | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV | Dose 1: 1E10 vg/eye | Dose 2: 3E10 vg/eye (planned phase 3 dose) | Phase2, Phase1 | Recruiting | Active | 2022-01-05 | 2026-11 | 2024-11-05 | >= 50 Years | 215 | 24 |
Locations:United States + 1 more
Puerto Rico, United States
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US11766489B2; US11613766B2; US20240226336A9 | Phase III trial to begin Q1 2025 |
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| NCT06864988 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | Regenerative Medicine Advanced Therapy | 4D-150 | 4D Molecular Therapeutics | Industry | Anti-VEGF | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV | Dose 1: 1E10 vg/eye | Dose 2: 3E10 vg/eye (planned phase 3 dose) | Phase3 | Recruiting | Active | 2025-03-04 | 2028-06 | 2025-03-07 | >= 50 Years | 400 | 96 |
Locations:United States + 2 more
Canada, Puerto Rico, United States
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Phase III trial to begin Q1 2025 |
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| NCT04517149 | X-Linked Retinitis Pigmentosa | Fast Track, Orphan Drug Designation | 4D-125 | 4D Molecular Therapeutics | Industry | RPGR | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV | Dose 1: 3E11 vg/eye | Dose 2: 1E12 vg/eye | Phase2, Phase1 | Active not recruiting | Inactive | 2020-08-14 | 2029-05 | 2025-03-21 | >= 12 Years | 21 | 8 | United States | US11613766B2; US20220290181A1 | January 2025: Sponsor announced they would terminate development of this program | ||||
| NCT04676048 | Hemophilia A | Fast Track, Orphan Drug Designation | ASC618 | ASC Therapeutics | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/8 | Undisclosed dose 1 | Phase2, Phase1 | Recruiting | Active | 2020-12-15 | 2026-12 | 2023-02-01 | >= 18 Years | 12 | 1 | United States | First patient dosed January 2024 |
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| NCT03454893 | Fabry Disease | Orphan Drug Designation | AVR-RD-01 | AVROBIO | Industry | GLA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Dose 1: Target range: 3-20E6 CD34+ cells/kg | Dose 2: Dose range: 3.1 - 13.8E6 CD34+ cells/kg (reported in IIT trial) | Phase2, Phase1 | Terminated | Inactive | 2017-12-20 | 2022-03-14 | 2024-01-05 | 16 Years - 50 Years | 15 | 5 |
Locations:United States + 2 more
Australia, Brazil, United States
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Fabry program was discontinued in Jan 2022 |
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| NCT04145037 | Gaucher Disease, Type 1 | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | AVR-RD-02 | AVROBIO | Industry | GBA1 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Target range: 3-10E6 CD34+ cells/kg | Phase2, Phase1 | Terminated | Inactive | 2019-08-27 | 2023-08-21 | 2024-01-18 | 18 Years - 50 Years | 8 | 5 |
Locations:United States + 1 more
Canada, United States
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Program development was halted in July 2023 |
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| NCT02556736 | Advanced Retinitis Pigmentosa | Orphan Drug Designation | RST-001 | AbbVie | Industry | ChR2 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Retinal ganglion | Viral transduction | AAV2.7m8 | 3 undisclosed dose levels | Phase2, Phase1 | Completed | Active | 2015-09-21 | 2024-10-21 | 2024-11-08 | >= 18 Years | 14 | 4 | United States | |||||
| NCT04704921 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | ABBV-RGX-314 | AbbVie | Industry | Anti-VEGF | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: 2.5E11 GC/eye | Dose 2: 5.0E11 GC/eye | Dose 3: 1.0E12 GC/eye | Dose 4: 1.5E12 GC/eye (suprachoroidal delivery) | Phase3, Phase2 | Recruiting | Active | 2021-01-08 | 2026-05 | 2025-01-29 | 50 Years - 89 Years | 540 | 91 | United States | US20230057519A1; US20200093939A1; | Pivotal data expected 2026 |
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| NCT05407636 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | ABBV-RGX-314 | AbbVie | Industry | Anti-VEGF | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: 2.5E11 GC/eye | Dose 2: 5.0E11 GC/eye | Dose 3: 1.0E12 GC/eye | Dose 4: 1.5E12 GC/eye (suprachoroidal delivery) | Phase3 | Recruiting | Active | 2022-01-28 | 2026-11 | 2024-10-16 | 50 Years - 89 Years | 660 | 128 |
Locations:United States + 9 more
Canada, France, Germany, Hungary, Italy, Japan, Puerto Rico, Spain, United Kingdom, United States
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Pivotal data expected 2026 |
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| NCT05725018 | Recessive Dystrophic Epidermolysis Bullosa (RDEB) | Breakthrough Therapy, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | EB-101 | Abeona Therapeutics, Inc | Industry | COL7A1 | Gene transfer | Ex-vivo | Functional gene replacement | Skin graft | Autologous cells | Keratinocytes | Viral transduction | LZRSE | Transduced 35cm^2 keratinocyte sheets, up to 6 wound sites | Phase3 | Recruiting | Active | 2023-01-23 | 2025-06-30 | 2024-06-27 | >= 12 Months | 12 | 2 | United States | US20240067926A1; US20230045590A1 | BLA resubmission October 2024, new PDUFA date 4/29/25 |
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| NCT03315182 | Mucopolysaccharidosis Type 3 B | ABO-101 | Abeona Therapeutics, Inc | Industry | NAGLU | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 2E13 vg/kg | Dose 2: 5E13 vg/kg | Dose 3: 1E14 vg/kg | Phase2, Phase1 | Terminated | Inactive | 2017-10-10 | 2022-04-07 | 2022-05-05 | 11 | 4 |
Locations:United States + 3 more
France, Germany, Spain, United States
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Abeona decided to discontinue this program due to lack of drug supply |
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| NCT04783181 | Congenital Adrenal Hyperplasia | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | BBP-631 | Adrenas Therapeutics Inc | Industry | CYP21A2 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV5 | Dose 1: 1.5E13 vg/kg | Dose 2: 3.0E13 vg/kg | Dose 3: 6.0E13 vg/kg | Phase2, Phase1 | Active not recruiting | Inactive | 2021-03-01 | 2029-02 | 2024-09-26 | >= 18 Years | 8 | 5 | United States | Bridge Bio is cutting this program, lack of sufficient efficacy |
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| NCT02168686 | Alpha-1 Antitrypsin Deficiency (AATD) | ADVM-043 | Adverum Biotechnologies, Inc. | Industry | SERPINA1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh10 | Dose 1: 8E13 vg | Dose 2: 4E14 vg | Dose 3: 1.2E15 vg | Phase2, Phase1 | Completed | Inactive | 2014-06-10 | 2019-08-29 | 2023-10-05 | >= 18 Years | 6 | 2 | United States | Product failed to show efficacy or dose-response effect; Product development was discontinued in August 2019 |
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| NCT05536973 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | Fast Track, Regenerative Medicine Advanced Therapy | ADVM-022 | Adverum Biotechnologies, Inc. | Industry | Codon optimized aflibercept | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV.7m8 | Dose 1: 6E10 vg/eye (selected as pivotal dose) | Dose 2: 2E11 vg/eye | Phase2 | Active not recruiting | Active | 2022-09-08 | 2028-08 | 2025-03-03 | >= 50 Years | 69 | 39 |
Locations:United States + 2 more
France, United Kingdom, United States
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US20220265740A1; US20230322911A1 | Phase 3 non-inferiority study will evaluate a broad patient population; initiated March 2025 |
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| NCT06856577 | Neovascular Age-Related Macular Degeneration (nAMD) | Fast Track, Regenerative Medicine Advanced Therapy | ADVM-022 | Adverum Biotechnologies, Inc. | Industry | Codon optimized aflibercept | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV.7m8 | Dose 1: 6E10 vg/eye (selected as pivotal dose) | Dose 2: 2E11 vg/eye | Phase3 | Recruiting | Active | 2025-02-26 | 2030-03-25 | 2025-03-28 | >= 50 Years | 284 | 6 | United States | Phase 3 non-inferiority study will evaluate a broad patient population; initiated March 2025 |
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| NCT05821959 | Sensorineural Hearing Loss, Bilateral | Orphan Drug Designation, Rare Pediatric Disease Designation | AK-OTOF | Akouos, Inc. | Industry | OTOF | Gene transfer | In-vivo | Functional gene replacement | Intracochlear | Viral vector | Hair cell | Viral transduction | dual Anc80L65 | Dose 1: 4.1E11 vg/cochlea | Dose 2: 8.1E11 vg/cochlea | Phase2, Phase1 | Recruiting | Active | 2023-02-08 | 2028-10 | 2025-02-06 | 14 | 6 |
Locations:United States + 2 more
Taiwan, United Kingdom, United States
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US20210017235A1; US11104885B2; US20240011039A1; EP4063510A1 |
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| NCT06517888 | Vestibular Schwannoma | AAVAnc80-antiVEGF | Akouos, Inc. | Industry | Anti-VEGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intracochlear | Viral vector | Viral transduction | AAVAnc80 | Dose 1: Undisclosed dose 1 | Dose 2: Undisclosed dose 2 | Dose 3: Undisclosed dose 3 | Phase2, Phase1 | Recruiting | Active | 2024-07-19 | 2029-08 | 2025-03-12 | >= 18 Years | 27 | 3 | United States | Eli Lilly acquired this company in 2022 |
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| NCT02725580 | Variant Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease) | Orphan Drug Designation, Rare Pediatric Disease Designation | AT-GTX-501 | Amicus Therapeutics | Industry | CLN6 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | 1.5E13 vg | Phase2, Phase1 | Completed | Inactive | 2016-03-16 | 2021-10-27 | 2023-03-08 | >= 1 Year | 13 | 1 | United States | Program was discontinued due to lack of sustained efficacy in LTFU study, Amicus returned development rights to Abigail Wexner Research Institute at Nationwide Children's Hospital in January 2024 |
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| NCT03770572 | CLN3 Batten Disease, Juvenile Neuronal Ceroid Lipofuscinosis | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | AT-GTX-502 | Amicus Therapeutics | Industry | CLN3 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | Dose 1: 6E13 vg (no more than 5mL) | Dose 2: 1.2E14 vg (no more than 10mL) | Phase2, Phase1 | Active not recruiting | Inactive | 2018-12-07 | 2024-09-30 | 2023-08-29 | 3 Years - 10 Years | 7 | 1 | United States | Amicus returned development rights to Abigail Wexner Research Institute at Nationwide Children's Hospital in January 2024 |
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| NCT00891306 | Familial Lipoprotein Lipase Deficiency | Orphan Drug Designation | GLYBERA | Amsterdam Molecular Therapeutics | Industry | LPL | Gene transfer | In-vivo | Functional gene replacement | Intramuscular | Viral vector | Viral transduction | AAV1 | Dose 1: 1E11 gc/kg | Dose 2: 3E11 gc/kg | Dose 3: 1E12 gc/kg (approved dose) | Phase3, Phase2 | Completed | EMA approved - product is unavailable | 2009-04-30 | 2011-04 | 2011-09-29 | >= 18 Years | 5 | 2 | Canada | Product was acquired by uniQure, uniQure did not renew their EMA marketing application when it expired 10/25/17, and Glybera is no longer commercially available |
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| NCT02144610 | Critical Limb Ischemia | COLLATEGENE | AnGes USA, Inc. | Industry | HGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramuscular | Plasmid | None (naked plasmid) | Dose 1: 0.4mg (3X: day 0, 14, 28) | Dose 2: 4.0mg (2X: day 0, day 28) | Dose 3: 4.0mg (3X: day 0, day 14, day 28) | Phase3 | Terminated | Inactive | 2014-05-20 | 2016-11-28 | 2019-08-13 | 40 Years - 90 Years | 46 | 60 |
Locations:United States + 10 more
Belgium, Canada, Denmark, Finland, France, Hungary, Italy, Netherlands, Poland, Sweden, United States
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Product was approved in Japan in 2019, marketing application has since been withdrawn |
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| NCT06839235 | Primary Hyperoxaluria Type 1 (PH1) | Orphan Drug Designation, Rare Pediatric Disease Designation | ABO-101 | Arbor Biotechnologies | Industry | HAO1 | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Lipid encapsulation | LNP | Cas12i2 | Undisclosed dose escalation | Phase2, Phase1 | Recruiting | Active | 2025-02-17 | 2043-02 | 2025-04-02 | 6 Years - 64 Years | 23 | 1 | United States | IND accepted 12/19/24 |
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| NCT06467344 | Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration | Fast Track, Rare Pediatric Disease Designation | ACDN-01 | Ascidian Therapeutics, Inc | Industry | ABCA4 pre-mRNA | Gene editing | In-vivo | Exon skipping/splice editor | Subretinal | Viral vector | Viral transduction | AAV | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2024-05-31 | 2030-12-01 | 2025-01-22 | >= 18 Years | 13 | 10 | United States | US20240091381A1; WO2020214990A1 | Rare Pediatric Disease designation granted 4/25/24 |
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| NCT06285643 | Parkinson Disease | Fast Track | AB-1005 | AskBio Inc | Industry | GDNF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Convection enhanced delivery into the putamen | Viral vector | Viral transduction | AAV2 | Up to 1.8mL (3.3E12 vg/ml/gadoteridol 2mM co-infusion) | Phase2 | Recruiting | Active | 2024-02-14 | 2027-11-30 | 2025-04-24 | 45 Years - 75 Years | 87 | 33 |
Locations:United States + 2 more
Poland, United Kingdom, United States
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US20180140672A1; US20180344199A1; WO2023183594A2 | Received FDA Fast Track designation |
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| NCT05230459 | Limb-Girdle Muscular Dystrophy, Type 2I/R9 | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | AB-1003 | AskBio Inc | Industry | FKRP | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed dose 1 | Dose 2: Undisclosed dose 2 | Phase2, Phase1 | Recruiting | Active | 2022-01-27 | 2028-12 | 2024-11-29 | 18 Years - 65 Years | 10 | 6 | United States | US20200061092A1; US20190008881A1 | First patient dosed (8/3/23) |
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| NCT03533673 | Pompe Disease | ACTUS-101 | AskBio Inc | Industry | GAA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Hepatocyte | Viral transduction | AAV2/8 | Dose 1: 1.6E12 vg/kg | Dose 2: Undisclosed dose 2 | Dose 3: Undisclosed dose 3 | Phase2, Phase1 | Active not recruiting | Active | 2018-04-13 | 2026-03 | 2023-06-28 | >= 18 Years | 7 | 1 | United States | US20180371440A1; US20220389399A1; US20230210884A1; | First patient dosed 1/22/19, most recent data presented at ASGCT in May 2022 |
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| NCT05598333 | Congestive Heart Failure | Fast Track | AB-1002 | AskBio Inc | Industry | PPP1R1A | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarterial | Viral vector | Cardiomyocyte | Viral transduction | AAV2i8 | Dose 1: 3.25E13 vg | Dose 2: 6.5E13 vg | Phase2 | Recruiting | Active | 2022-10-25 | 2030-12-31 | 2025-04-09 | >= 18 Years | 150 | 43 |
Locations:United States + 5 more
Austria, Germany, Netherlands, Spain, United Kingdom, United States
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US20230340528A1; AU2021320244A1 | Received Fast Track designation 4/18/24 |
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| NCT04998396 | Canavan Disease | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | BBP-812 | Aspa Therapeutics | Industry | ASPA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed dose escalation, 2 levels | Dose 2: Undisclosed expansion dose | Phase2, Phase1 | Recruiting | Inactive | 2021-08-05 | 2030-10-08 | 2024-10-22 | <= 30 Months | 26 | 4 | United States | Program terminated, effective Feb 2025 |
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| NCT05071222 | Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency | ARTEGENE | Assistance Publique - Hôpitaux de Paris | Other | DCLRE1C | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells, range: 1.8-3.7E6 CD34+ cells/kg | Phase2, Phase1 | Recruiting | Active | 2021-08-27 | 2041-07-19 | 2023-11-27 | <= 47 Months | 5 | 1 | France |
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| NCT06736080 | Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3) | Autologous Cells Transduced Ex Vivo with LV-UNC13D | Assistance Publique - Hôpitaux de Paris | Other | UNC13D | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells/T-CD3+ cells | Viral transduction | LV | Dose 1: 2-20E6 CD34+ cells/kg | Dose 2: 1.1E4 - 5.1E6 T-CD3+ cells/kg (as needed) | Phase2, Phase1 | Not yet recruiting | Active | 2024-12-04 | 2029-01 | 2024-12-16 | 3 Months - 17 Years | 5 | 1 | France |
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| NCT01410019 | X-linked Severe Combined Immunodeficiency (XSCID) | Autologous CD34+ cells transduced with the self-inactivating (SIN) GAMMARETROVIRAL vector pSRS11.EFS.IL2RG.pre | Assistance Publique - Hôpitaux de Paris | Other | IL2RG | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | RV | Median dose: 7.58E6 transduced CD34+ cells/kg (n=11) | Phase2, Phase1 | Completed | Inactive | 2011-06-21 | 2015-06-16 | 2021-09-28 | <= 12 Months | 5 | 1 | France |
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| NCT03964792 | Sickle Cell Disease | DREPAGLOBE | Assistance Publique - Hôpitaux de Paris | Other | HBB | Gene transfer | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells (range: 6 - 8.1E6 cells/kg) | Phase2, Phase1 | Active not recruiting | Active | 2019-05-14 | 2024-01 | 2024-01-09 | 12 Years - 20 Years | 6 | 1 | France | US20200190536A1 | Uses the same βAS3-globin as NCT02247843; vector performed poorly |
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| NCT03199469 | X-Linked Myotubular Myopathy | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | AT132 | Astellas Gene Therapies | Industry | MTM1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: 1.3E14 vg/kg | Dose 2: 3.5E14 vg/kg | Phase3, Phase2 | Active not recruiting | Active | 2017-06-21 | 2030-03-31 | 2025-03-17 | <= 5 Years | 27 | 6 |
Locations:United States + 3 more
Canada, France, Germany, United States
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US20220411819A1; WO2023196853A1; WO2022251208A1 | On clinical hold since 2021 |
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| NCT06483802 | Friedreich Ataxia, Cardiomyopathy | ASP2016 | Astellas Gene Therapies | Industry | FXN | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Undisclosed dose escalation | Phase1 | Suspended | Inactive | 2024-06-26 | 2031-01-31 | 2025-01-08 | 18 Years - 40 Years | 14 | 5 | United States | WO2023044424A1; AR127081A1; EP4401756A1 | Sponsor announced termination of the program February 2025, no patients were enrolled in the study |
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| NCT04174105 | Pompe Disease (Late-onset) | AT845 | Astellas Gene Therapies | Industry | GAA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: 3E13 vg/kg | Dose 2: 6E13 vg/kg | Dose 3: 1E14 vg/kg | Phase2, Phase1 | Active not recruiting | Active | 2019-11-13 | 2030-02-28 | 2025-03-25 | 18 Years - 80 Years | 11 | 4 |
Locations:United States + 1 more
United Kingdom, United States
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US20210162073A1; US20220411819A1; US20220387562A1; WO2023150688A8 | Anticipated PoC judgement timing: 2H/FY2025 |
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| NCT05224505 | Limb-Girdle Muscular Dystrophy, Type 2I/R9 | Fast Track | GNT0006 | Atamyo Therapeutics | Industry | FKRP | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/9 | Dose 1: 9.0E12 vg/kg | Dose 2: 2.7E13 vg/kg | Phase2, Phase1 | Recruiting | Active | 2021-12-15 | 2030-10 | 2024-07-31 | 16 Years - 99 Years | 39 | 3 |
Locations:Denmark + 2 more
Denmark, France, United Kingdom
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US20230321277A1; EP4031673A1; EP4198047A1 | Dose-escalation (Phase 1b) recruitment completed September 2024 |
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| NCT05973630 | Limb-Girdle Muscular Dystrophy, Type 2C/R5 | Orphan Drug Designation, Rare Pediatric Disease Designation | ATA-200 | Atamyo Therapeutics | Industry | SGCG | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/8 | Dose 1: 1.0E14 vg/kg | Dose 2: 3.0E14 vg/kg | Phase2, Phase1 | Recruiting | Active | 2023-07-24 | 2032-01-31 | 2025-02-14 | 6 Years - 13 Years | 6 | 3 |
Locations:United States + 2 more
France, Italy, United States
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EP4198134A1 | US IND cleared 11/12/24 |
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| NCT03920007 | Leber Congenital Amaurosis, LCA, LCA1 | Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | ATSN-101 | Atsena Therapeutics Inc. | Industry | GUCY2D | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/5 | Dose 1: 1.0E10 vg/eye (300ul) | Dose 2: 3.0E10 vg/eye (300ul) | Dose 3: 1.0E11 vg/eye (300ul); expansion dose | Phase2, Phase1 | Active not recruiting | Active | 2019-04-10 | 2027-05-19 | 2024-02-20 | >= 6 Years | 15 | 2 | United States |
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| NCT05878860 | X-Linked Retinoschisis | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | ATSN-201 | Atsena Therapeutics Inc. | Industry | RS1 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV.SPR | Dose 1: 1.5E10 vg/eye | Dose 2: 5.0E10 vg/eye | Dose 3: 1.7E11 vg/eye | Phase2, Phase1 | Recruiting | Active | 2023-05-12 | 2029-10 | 2025-02-19 | >= 6 Years | 21 | 4 | United States | FDA granted fast Track designation |
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| NCT03223194 | Crigler-Najjar Syndrome | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | AT342 | Audentes Therapeutics | Industry | UGT1A1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: 1.5E12 vg/kg | Dose 2: Planned medium dose: 6.0E12 vg/kg (never administered) | Dose 3: Planned high dose: 1.5E12 vg/kg (never administered) | Phase1 | Terminated | Inactive | 2017-07-17 | 2021-02-11 | 2022-05-18 | >= 1 Year | 1 | 4 |
Locations:United States + 2 more
Israel, United Kingdom, United States
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Sponsor terminated development, only 1 patient was enrolled in the study prior to termination and received the lowest dose which was well-tolerated but did not demonstrate efficacy beyond a few weeks |
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| NCT06064890 | Frontotemporal Dementia, FTD, FTD-GRN, Dementia, Frontotemporal | Fast Track, Orphan Drug Designation | AVB-101 | AviadoBio Ltd | Industry | GRN | Gene transfer | In-vivo | Functional gene replacement | Intrathalamic | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2023-09-12 | 2030-10-31 | 2025-04-22 | 30 Years - 75 Years | 9 | 15 |
Locations:United States + 5 more
Netherlands, Poland, Spain, Sweden, United Kingdom, United States
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Partnership with Astellas for clinical development of this product announced October 2024 |
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| NCT00076557 | Hemophilia B | Orphan Drug Designation | AAV2-hFIX16 | Avigen | Industry | F9 | Gene transfer | In-vivo | Functional gene replacement | Hepatic artery infusion | Viral vector | Hepatocyte | Viral transduction | AAV2 | Dose 1: 8E10 vg/kg | Dose 2: 4E11 vg/kg | Dose 3: 2E12 vg/kg | Phase2, Phase1 | Terminated | Inactive | 2004-01-26 | null | 2007-04-04 | >= 18 Years | 15 | 3 | United States | Avigen suspended enrollment in May 2004; transferred AAV-based product rights to Genzyme Corporation in December 2005, LTFU was sponsored by CHOP from 2009-2013, then Spark Therapeutics afterwards |
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| NCT06550011 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | ABI-110 | Avirmax Biopharma Inc | Industry | VEGFtrap | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Macula | Viral transduction | AAV.N54 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2024-07-25 | 2030-02 | 2024-10-02 | 50 Years - 89 Years | 18 | 4 | United States | First cohort completion announced February 2025 | |||||
| NCT05241444 | Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome (IPEX) | CD4^LVFOXP3 | Bacchetta, Rosa, MD | Other | FOXP3 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD4+ T cells | Viral transduction | LV | Dose 1: 1.0E6 CD4+ cells/kg | Dose 2: 3.0E6 CD4+ cells/kg | Dose 3: 1.0E7 CD4+ cells/kg | Phase1 | Recruiting | Active | 2022-01-12 | 2037-02 | 2025-04-21 | 4 Months - 35 Years | 30 | 1 | United States | US20220273712A1; EP3672617A4; US20230183804A1 |
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| NCT04394286 | Hemophilia B | SHP648 | Baxalta now part of Shire | Industry | F9 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: 2E11 vg/kg | Dose 2: 1.0E12 vg/kg | Dose 3: 3E12 vg/kg | Phase2, Phase1 | Terminated | Inactive | 2020-05-15 | 2021-05-03 | 2022-05-19 | 18 Years - 75 Years | 2 | 2 |
Locations:Spain + 1 more
Spain, Turkey
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Takeda decided to terminate the SHP648 (TAK-748) program prior to dosing additional subjects. In the previous clinical trial, there was little evidence of sustained efficacy |
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| NCT03588299 | Hemophilia A | DTX201 | Bayer | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVhu37 | Dose 1: 0.5E13 GC/kg | Dose 2: 1E13 GC/kg | Dose 3: 2E13 GC/kg | Dose 4: 4E13 GC/kg | Phase2, Phase1 | Active not recruiting | Active | 2018-07-06 | 2026-11-03 | 2025-04-08 | >= 18 Years | 11 | 13 |
Locations:United States + 5 more
Bulgaria, France, Germany, Netherlands, United Kingdom, United States
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License reverted to Ultragenyx in 2022, uncertain development status |
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| NCT06611436 | Hemophilia B | Orphan Drug Designation | BE-101 | Be Biopharma | Industry | F9 | Gene editing | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | B cells | Viral transduction | AAV6 | Cas9 RNP | Dose 1: Undisclosed dose 1 | Dose 2: Undisclosed dose 2 | Dose 3: Undisclosed dose 3 | Phase2, Phase1 | Recruiting | Active | 2024-09-18 | 2027-07 | 2025-03-18 | >= 18 Years | 24 | 3 | United States |
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| NCT00749957 | Leber Congenital Amaurosis-Type 2 | RAAV2-CB-hRPE65 | Beacon Therapeutics | Industry | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: 1.8E11 vg/eye | Dose 2: 6E11 vg/eye | Phase2, Phase1 | Completed | Inactive | 2008-09-08 | 2017-09-22 | 2017-12-28 | >= 6 Years | 12 | 2 | United States | Company discontinued development due to competing therapies for the same indication |
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| NCT02599922 | Achromatopsia | AGTC-401 | Beacon Therapeutics | Industry | CNGB3 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Cone cells | Viral transduction | AAV2tYF | Dose 1: 2E11 vg/ml | Dose 2: 4E10 vg/ml | Dose 3: 1.2E11 vg/ml | Dose 4: 3.6E11 vg/ml | Dose 5: 1.1E12 vg/ml; Dose 6: 3.2E12 | Phase2, Phase1 | Active not recruiting | Inactive | 2015-11-05 | 2026-07 | 2022-07-22 | >= 4 Years | 32 | 8 | United States | Development discontinued by Sponsor |
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| NCT02935517 | Achromatopsia | Orphan Drug Designation | AGTC-402 | Beacon Therapeutics | Industry | CNGA3 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Cone cells | Viral transduction | AAV2tYF | Dose 1: 4.0E10 vg/mL | Dose 2: 1.2E11 vg/mL | Dose 3: 3.6E11 vg/mL | Dose 4: 1.1E12 vg/mL | Dose 5: 3.2E12 vg/mL | Phase2, Phase1 | Active not recruiting | Inactive | 2016-10-13 | 2026-08 | 2022-07-22 | >= 4 Years | 24 | 6 |
Locations:United States + 1 more
Israel, United States
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Development discontinued by Sponsor |
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| NCT02416622 | X-linked Retinoschisis | Orphan Drug Designation | RAAV2tYF-CB-hRS1 | Beacon Therapeutics | Industry | RS1 | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV2tYF | Dose 1: 1E11 vg/eye | Dose 2: 3E11 vg/eye | Dose 3: 6E11 vg/eye | Phase2, Phase1 | Completed | Active | 2015-04-02 | 2023-05-09 | 2023-06-12 | >= 6 Years | 27 | 9 | United States | The product was well tolerated, but no signs of clinical activity were observed at six months post treatment, development discontinued in 2018; product was transferred to TeamedOn |
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| NCT04850118 | X-Linked Retinitis Pigmentosa | Fast Track, Regenerative Medicine Advanced Therapy | AGTC-501 | Beacon Therapeutics | Industry | RPGR | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2tYF | 6.8E11 vg/eye | Phase3, Phase2 | Recruiting | Active | 2021-04-05 | 2029-10 | 2025-02-11 | 12 Years - 50 Years | 75 | 21 |
Locations:United States + 2 more
Australia, United Kingdom, United States
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First patient dosed in registrational trial 6/12/24, enrollment ongoing |
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| NCT01054339 | Alpha-1 Antitrypsin Deficiency (AATD) | AGTC-0106 | Beacon Therapeutics | Industry | SERPINA1 | Gene transfer | In-vivo | Functional gene replacement | Intramuscular | Viral vector | Viral transduction | AAV1 | Dose 1: 6E11 vg/kg | Dose 2: 1.9E12 vg/kg | Dose 3: 6E12 vg/kg | Phase2 | Completed | Inactive | 2010-01-21 | 2015-10 | 2019-03-28 | 18 Years - 75 Years | 9 | 4 |
Locations:United States + 1 more
Ireland, United States
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IM route resulted in poor efficacy, product development abandoned |
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| NCT06735755 | Glycogen Storage Disease Type Ia | BEAM-301 | Beam Therapeutics Inc. | Industry | G6PC1 (p.R83C) | Gene editing | In-vivo | Mutation correction | Intravenous | MRNA, LNP | Lipid encapsulation | LNP | ABE | Undisclosed dose escalation | Phase2, Phase1 | Recruiting | Active | 2024-12-06 | 2027-12-30 | 2025-04-24 | >= 18 Years | 36 | 2 | United States | Dosing anticipated to commence in early 2025 |
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| NCT06389877 | Alpha-1 Antitrypsin Deficiency (AATD) | BEAM-302 | Beam Therapeutics Inc. | Industry | SERPINA1 (p.Glu366Lys) | Gene editing | In-vivo | Mutation correction | Intravenous | MRNA, LNP | Liver | Lipid encapsulation | LNP | ABE | Dose 1: 15mg | Dose 2: 30mg | Dose 3: 60mg | Phase2, Phase1 | Recruiting | Active | 2024-04-22 | 2027-08 | 2025-01-10 | 18 Years - 70 Years | 106 | 5 |
Locations:Australia + 3 more
Australia, Netherlands, New Zealand, United Kingdom
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US20200399626A1; US20210371858A1; US20230080198A1 | Beam Therapeutics Announces Positive Initial Data; IND cleared by FDA March 2025 |
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| NCT05456880 | Sickle Cell Disease | BEAM-101 | Beam Therapeutics Inc. | Industry | HBG1/HBG2 | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Electroporation | none | ABE8 | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | Active | 2022-06-23 | 2027-02-01 | 2025-01-10 | 18 Years - 35 Years | 15 | 20 | United States | US20200399626A1; US11142760B2; US20230080198A1 | Beam expects to dose 30 patients by mid-2025, data update expected mid-2025; 1 patient death reported November 2024, deemed related to busulfan conditioning |
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| NCT05968118 | Peripheral Artery Disease | NL003 | Beijing Northland Biotech. Co., Ltd. | Industry | HGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramuscular | Plasmid | None (naked plasmid) | Concentration: 0.5mg/1ml (32 sites x 0.5ml), Total 8mg | Phase3 | Recruiting | Active | 2023-01-04 | 2023-10-31 | 2023-08-01 | 18 Years - 80 Years | 36 | 1 | China | Same product as VM202 | |||||||
| NCT04275323 | Critical Limb Ischemia | NL003 | Beijing Northland Biotech. Co., Ltd. | Industry | HGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramuscular | Plasmid | None (naked plasmid) | Dose 1: Concentration: 0.5mg/1ml (32 sites x 0.5ml), Total 8mg | Dose 2: Total dose: 12mg | Dose 3: Total dose: 18mg | Dose 4: Total dose: 24mg | Phase3 | Completed | Active | 2020-01-18 | 2024-06-03 | 2024-09-19 | 20 Years - 80 Years | 302 | 13 | China | Same product as VM202 |
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| NCT06761183 | Ischemic Stroke | NXL-001 | Beijing Tiantan Hospital | Other | NeuroD1 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intracerebral | Viral vector | Astrocyte | Viral transduction | AAV9 | Dose escalation with 3 levels, unknown concentrations | Early phase1 | Not yet recruiting | Active | 2024-12-27 | 2026-12-31 | 2025-01-07 | 40 Years - 75 Years | 9 | 1 | China |
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| NCT06185673 | Oculopharyngeal Muscular Dystrophy | Orphan Drug Designation | BB-301 | Benitec Biopharma, Inc. | Industry | PABPN1 | Gene transfer | In-vivo | Functional gene replacement/gene inactivation | Intramuscular (pharyngeal constrictor muscles) | Viral vector | Viral transduction | AAV9 | Dose 1: 1.2E13 vg | Dose 2: 3.6E13 vg | Dose 3: 5.4E13 vg | Phase2, Phase1 | Recruiting | Active | 2023-12-15 | 2040-11 | 2025-02-05 | <= 65 Years | 30 | 1 | United States | 5th subject was treated in February 2025 |
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| NCT04737460 | CLN7 Batten Disease | AAV9/CLN7 | Benjamin Greenberg | Other | MFSD8 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | Dose 1: 5E14 vg | Dose 2: 1E15 vg | Phase1 | Active not recruiting | Active | 2021-01-15 | 2029-02-01 | 2024-12-13 | 1 Year - 18 Years | 4 | 1 | United States | WO2020033833A1; US11753655B2 |
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| NCT05121376 | Hereditary Angioedema | Orphan Drug Designation | BMN 331 | BioMarin Pharmaceutical | Industry | SERPING1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAV5 | Dose 1: 6E13 vg/kg | Dose 2: Other unknown doses | Phase2, Phase1 | Active not recruiting | Inactive | 2021-10-28 | 2028-11 | 2024-05-16 | >= 18 Years | 44 | 16 |
Locations:United States + 2 more
Australia, Spain, United States
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Biomarin announced they were discontinuing this program April 2024 | ||||
| NCT04480567 | Phenylketonuria (PKU) | Fast Track, Orphan Drug Designation | BMN 307 | BioMarin Pharmaceutical | Industry | PAH | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV5 | Dose escalation with 3 levels, unknown concentrations | Phase2, Phase1 | Active not recruiting | Inactive | 2020-07-08 | 2027-12 | 2024-12-12 | >= 15 Years | 100 | 3 |
Locations:United States + 1 more
United Kingdom, United States
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BioMarin (developer of pegvaliase) has made no official announcement of discontinuation. Clinical hold was placed when mice in preclinical studies developed tumors, and Biomarin was sued on allegations that Biomarin had overstated BMN 307's clinical and commercial prospects. | |||||
| NCT04323098 | Hemophilia A | Breakthrough Therapy, Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy | ROCTAVIAN | BioMarin Pharmaceutical | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Hepatocyte | Viral transduction | AAV5 | 6E13 vg/kg | Phase3 | Active not recruiting | Approved | 2020-03-24 | 2027-01 | 2024-04-04 | >= 18 Years | 22 | 12 |
Locations:United States + 3 more
Australia, Brazil, Taiwan, United States
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FDA approved 6/29/23, price/treatment $2.9M |
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| NCT03116113 | X-Linked Retinitis Pigmentosa | BIIB112 | Biogen | Industry | RPGR | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: Dose escalation: 5E10 gp/ml - 5E12 gp/ml (6 levels) | Dose 2: Expansion doses: 5E10 vg/eye, 2.5E11 vg/eye | Phase2, Phase1 | Completed | Inactive | 2017-03-29 | 2020-11-18 | 2024-01-18 | >= 10 Years | 50 | 8 |
Locations:United States + 1 more
United Kingdom, United States
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Phase 2/3 study did not meet its primary endpoint, Company suspended development in 2021 |
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| NCT03496012 | Choroideremia | Regenerative Medicine Advanced Therapy | BIIB111 | Biogen | Industry | CHM | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: 1.0E10 gp | Dose 2: 1.0E11 gp | Phase3 | Completed | Inactive | 2018-03-07 | 2020-12-01 | 2023-12-07 | >= 18 Years | 169 | 18 |
Locations:United States + 7 more
Canada, Denmark, Finland, France, Germany, Netherlands, United Kingdom, United States
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Phase III study did not meet its primary endpoint or demonstrate efficacy on key secondary endpoints |
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| NCT04278131 | Retinitis Pigmentosa | Regenerative Medicine Advanced Therapy | BS01 | Bionic Sight LLC | Industry | ChronosFP | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Retinal ganglion | Viral transduction | AAV2 | Dose escalation with 4 levels, unknown concentrations | Phase2, Phase1 | Recruiting | Active | 2020-02-13 | 2029-12-30 | 2023-05-03 | >= 18 Years | 20 | 1 | United States | Gene therapy product is coupled with a neural circuit, retinal prosthesis |
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| NCT06515002 | Cystic Fibrosis | BI 3720931 | Boehringer Ingelheim | Industry | CFTR | Gene transfer | In-vivo | Functional gene replacement | Inhalational | Viral vector | Viral transduction | rSIV.F/HN | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2024-07-18 | 2027-04-13 | 2025-04-16 | >= 18 Years | 36 | 10 |
Locations:France + 4 more
France, Italy, Netherlands, Spain, United Kingdom
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| NCT00272857 | Fanconi Anemia | MSCV-FANCA | Boston Children's Hospital | Other | FANCA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | MSCV | 3.5-4.5E5 CD34+ cells/kg | Phase1 | Completed | Inactive | 2006-01-04 | 2007-10 | 2017-06-23 | 1 Year - 35 Years | 3 | 1 | United States | Clinical study did not demonstrate long-term engraftment or selective advantage |
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| NCT04680065 | Multiple System Atrophy | AB-1005 | Brain Neurotherapy Bio, Inc. | Industry | GDNF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal | Viral vector | Viral transduction | AAV2 | Undisclosed dose 1 | Phase1 | Recruiting | Active | 2020-12-15 | 2028-12 | 2025-02-10 | 35 Years - 75 Years | 9 | 7 | United States | US20180140672A1; US20180344199A1; WO2023183594A2 | First patient randomized 11/17/23 | |||||
| NCT05541627 | Huntington's Disease | AB-1001 | Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio) | Industry | CYP46A1 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal | Viral vector | Viral transduction | AAVrh10 | Dose 1: 4E8 vg/uL | Dose 2: 1.1E9 vg/uL | Phase2, Phase1 | Active not recruiting | Inactive | 2022-09-13 | 2029-12-31 | 2024-08-06 | 18 Years - 65 Years | 5 | 1 | France | WO2012049314A1 | Bayer announced the discontinuation of this program November 2024 |
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| NCT02161380 | Leber's Hereditary Optic Neuropathy | ScAAV2-P1ND4v2 | Byron Lam | Other | ND4 | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV2 | Dose 1: 5.0E8 vg | Dose 2: 2.5E9 vg | Dose 3: 1.0E10 vg | Phase1 | Active not recruiting | Inactive | 2014-06-06 | 2025-03-31 | 2024-09-04 | >= 15 Years | 28 | 1 | United States | Safe but low efficacy, researchers did not advance development |
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| NCT04091737 | Sickle Cell Disease | Orphan Drug Designation | CSL200 | CSL Behring | Industry | HBG1/HBG2 | Gene transfer | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase1 | Terminated | Inactive | 2019-09-13 | 2021-05-05 | 2021-06-18 | 18 Years - 45 Years | 1 | 1 | United States | Trial was terminated due to unanticipated delays, only enrolled 1 patient |
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| NCT03569891 | Hemophilia B | Breakthrough Therapy, Priority Review, Orphan Drug Designation | HEMGENIX | CSL Behring | Industry | F9R338L | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAV5 | 2E13 vg/kg | Phase3 | Active not recruiting | Approved | 2018-06-14 | 2025-03 | 2025-03-21 | >= 18 Years | 67 | 33 |
Locations:United States + 7 more
Belgium, Denmark, Germany, Ireland, Netherlands, Sweden, United Kingdom, United States
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FDA approved 11/22/22; Price/treatment: $3.5M |
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| NCT06650319 | Wilson Disease | Orphan Drug Designation, Rare Pediatric Disease Designation | LY-M003 | Chaohui Yu | Other | ATP7B | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transdution | AAV8 | Undisclosed dose escalation, 3 levels | Early phase1 | Recruiting | Active | 2024-10-15 | 2030-03-30 | 2025-02-13 | 18 Years - 60 Years | 10 | 1 | China |
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| NCT06458595 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | KH658 | Chengdu Origen Biotechnology Co., Ltd. | Industry | Anti-VEGF | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Suprachoroidal | Viral vector | Viral transduction | AAV | Undisclosed dose | Phase2, Phase1 | Recruiting | Active | 2024-06-09 | 2026-12-28 | 2024-11-29 | 50 Years - 85 Years | 44 | 1 | China | WO2023236964A1 |
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| NCT05672121 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | KH631 | Chengdu Origen Biotechnology Co., Ltd. | Industry | VEGFR1/R2 fusion protein | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV8 | 5 undisclosed dose levels (200ul/dose) | Phase2, Phase1 | Recruiting | Active | 2022-12-21 | 2026-12-28 | 2024-11-27 | 50 Years - 85 Years | 42 | 1 | China |
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| NCT05657301 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | KH631 | Chengdu Origen Biotechnology Co., Ltd. | Industry | VEGFR1/R2 fusion protein | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV8 | 5 undisclosed dose levels (200ul/dose) | Phase1 | Recruiting | Active | 2022-12-10 | 2027-09 | 2024-02-26 | 50 Years - 85 Years | 25 | 7 | United States | WO2022051537A1 | 1st patient dosed 11/20/23 |
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| NCT05832684 | Bietti Crystalline Dystrophy | ZVS101e | Chigenovo Co., Ltd | Network | CYP4V2 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/8 | 7.5E10 vg | Phase2, Phase1 | Active not recruiting | Active | 2023-04-02 | 2028-12 | 2024-08-28 | >= 18 Years | 24 | 3 | China |
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| NCT05761899 | Hereditary Pulmonary Alveolar Proteinosis | Gene-Corrected Macrophages | Children's Hospital Medical Center, Cincinnati | Other | CSF2RA | Gene transfer | Ex-vivo | Functional gene replacement | Broncoscopy | Viral vector | CD34+ cells | Viral transduction | LV | 11.1E6 cells/kg ideal body weight | Phase2, Phase1 | Recruiting | Active | 2023-02-28 | 2038-10-01 | 2025-01-30 | >= 18 Years | 3 | 1 | United States | US20220315900A1; US20200199623A1 |
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| NCT02186418 | Sickle Cell Disease | ARU-1801 | Children's Hospital Medical Center, Cincinnati | Other | HBG(G16D) | Gene transfer | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells (dose range: 1.4-7.1E6 cells/kg) | Phase2, Phase1 | Terminated | Inactive | 2014-06-30 | 2022-10-31 | 2024-04-12 | 18 Years - 45 Years | 7 | 1 | United States | Clinical development was discontinued in June 2022 |
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| NCT04286815 | Severe Combined Immunodeficiency, X-linked | Lentiviral vector/IL2RG | Children's Hospital of Chongqing Medical University | Other | IL2RG | Gene transfer | Undisclosed | Functional gene replacement | Undisclosed | Undisclosed | Undisclosed | undisclosed | Undisclosed | Na | Recruiting | Active | 2020-02-23 | 2025-05-01 | 2020-03-27 | <= 18 Years | 10 | 1 | China |
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| NCT06207552 | Fabry Disease | BBM-F101 | Children's Hospital of Fudan University | Other | GLA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV | Undisclosed dose | Early phase1 | Recruiting | Active | 2023-12-18 | 2029-06 | 2024-07-09 | 7 Years - 18 Years | 6 | 1 | China | WO2022222869A1 |
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| NCT06364774 | Beta-Thalassemia Major | CHOP-ALS20 | Children's Hospital of Philadelphia | Other | HBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | Active | 2024-03-26 | 2027-12-31 | 2025-04-23 | 18 Years - 35 Years | 12 | 1 | United States | ||||||
| NCT05265767 | Hemophilia A | CD68-ET3-LV | Christian Medical College, Vellore, India | Other | F8 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | 5.0-6.1E6 CD34+ cells/kg | Phase1 | Completed | Active | 2022-02-16 | 2024-06-28 | 2024-07-12 | 18 Years - 45 Years | 6 | 1 | India |
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| NCT06291961 | Beta-Thalassemia Major | CS-101 | CorrectSequence Therapeutics Co., Ltd | Industry | BCL11A | Gene editing | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Electroporation | none | transformer BE RNP | Transduced CD34+ cells | Phase1 | Recruiting | Active | 2024-02-23 | 2025-07 | 2024-07-01 | 12 Years - 35 Years | 8 | 3 | China | US11384353B2; WO2020156575 |
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| NCT06647979 | Sickle Cell Disease, Beta-Thalassemia | Autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein | Daniel Bauer | Other | BCL11A | Gene editing | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Electroporation | SpCas9 mRNA | Transduced CD34+ cells | Phase1 | Not yet recruiting | Active | 2024-10-11 | 2030-12 | 2025-02-12 | 13 Years - 40 Years | 10 | 1 | United States | Includes genotyping for Variant rs114518452 as part of the inclusion/exclusion criteria |
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| NCT03311503 | X-linked Severe Combined Immunodeficiency (XSCID) | G2SCID lentiviral vector transduced CD34+ cells | David Williams | Other | IL2RG | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | HIV | Transduced CD34+ cells (minimum dose: 2.5E6 cells/kg) | Phase2, Phase1 | Recruiting | Active | 2017-10-12 | 2028-10-01 | 2025-01-06 | 0 Years - 5 Years | 12 | 4 | United States |
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| NCT05353647 | Sickle Cell Disease | Autologous CD34+ HSC cells transduced with the lentiviral vector containing BCL11a-targeted shRNA | David Williams | Other | BCL11A | Gene transfer | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | VSV-G | Transduced CD34+ cells | Phase2 | Active not recruiting | Active | 2022-04-21 | 2027-07 | 2025-04-18 | 13 Years - 40 Years | 25 | 9 | United States |
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| NCT02082860 | Acute Intermittent Porphyria | RAAV2/5-PBGD | Digna Biotech S.L. | Industry | PBGD | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/5 | Dose 1: 5.0E11 gc/kg | Dose 2: 2E12 gc/kg | Dose 3: 6E12 gc/kg | Dose 4: 1.8E13 gc/kg | Phase1 | Completed | Inactive | 2014-02-28 | 2014-11 | 2014-12-18 | 18 Years - 64 Years | 8 | 2 | Spain | Therapy was safe, but failed to show efficacy, this product did not advance to Phase II, original developers working on alternatives |
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| NCT02247843 | Sickle Cell Disease | Lenti | Donald B. Kohn, M.D. | Other | HBB | Gene transfer | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase2, Phase1 | Active not recruiting | Active | 2014-09-20 | 2025-12 | 2024-05-16 | >= 18 Years | 4 | 1 | United States | WO2020168004A1 | Uses the same βAS3-globin as NCT03964792 |
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| NCT04798235 | Infantile GM2 Gangliosidosis | Orphan Drug Designation, Rare Pediatric Disease Designation | TSHA-101 | Dr. Anupam Sehgal | Network | HEXA/HEXB | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | 5E14 vg (n=3) | Phase2, Phase1 | Active not recruiting | Active | 2021-02-22 | 2027-03-12 | 2023-05-09 | <= 15 Months | 3 | 1 | Canada | The Company announced they had transferred rights back to Queen's University in February 2024 | |||||
| NCT05444894 | Beta-Thalassemia Major | Regenerative Medicine Advanced Therapy | EDIT-301 | Editas Medicine, Inc. | Industry | HBG1/HBG2 | Gene editing | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | ASCas12a mRNA | Dose 1: Min: 5.7E6 CD34+ cells/kg | Dose 2: Max: 11.9E6 CD34+ cells/kg | Phase2, Phase1 | Active not recruiting | Inactive | 2022-06-27 | 2025-12 | 2025-04-02 | 18 Years - 35 Years | 9 | 8 |
Locations:United States + 1 more
Canada, United States
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WO2017160890A1 | Ended development of this program due to inability to locate a financial partner |
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| NCT04853576 | Sickle Cell Disease | EDIT-301 | Editas Medicine, Inc. | Industry | HBG1/HBG2 | Gene editing | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | ASCas12a | Dose 1: Min: 2.9E6 CD34+ cells/kg | Dose 2: Max: 10.0E6 CD34+ cells/kg | Phase2, Phase1 | Active not recruiting | Inactive | 2021-04-16 | 2025-08 | 2025-01-31 | 12 Years - 50 Years | 45 | 24 |
Locations:United States + 1 more
Canada, United States
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US12031132B2; US20200299661A1; | Ended development of this program due to inability to locate financial partner |
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| NCT06692712 | Hereditary Spastic Paraplegia Type 50 | MELPIDA | Elpida Therapeutics SPC | Industry | AP4M1 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | 1E15 vg | Phase3 | Not yet recruiting | Active | 2024-11-14 | 2032-06-01 | 2024-11-18 | 21 Months - 78 Months | 24 | 0 |
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| NCT05518188 | Hereditary Spastic Paraplegia Type 50 | MELPIDA | Elpida Therapeutics SPC | Industry | AP4M1 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | 1E15 vg | Phase2, Phase1 | Recruiting | Active | 2022-08-24 | 2030-10-01 | 2024-10-08 | 4 Months - 10 Years | 4 | 1 | United States | WO2022226183A3 | Phase III Study approved, begins August 2024 |
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| NCT05419492 | Dravet Syndrome | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | ETX101 | Encoded Therapeutics | Industry | SCN1A | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intracerebroventricular | Viral vector | GABAergic inhibitory interneurons | Viral transduction | AAV9 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2022-06-10 | 2031-04 | 2025-01-31 | 6 Months - 47 Months | 22 | 3 | United States | US20230203531A1; US20200397917A1; US20220136009A1; US20220168449A1; US20220170910A1 | Dosing and preliminary safety and efficacy data expected in 2H25 |
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| NCT06399107 | Sickle Cell Disease, Sickle-Cell Disease With Crisis | BAH243 | Essen Biotech | Other | HBB | Gene transfer | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | Active | 2024-04-29 | 2025-12-28 | 2024-11-05 | 2 Years - 90 Years | 85 | 1 | China |
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| NCT05144386 | HIV-1-infection | Fast Track, Regenerative Medicine Advanced Therapy | EBT-101 | Excision BioTherapeutics | Industry | HIV genes | Gene editing | In-vivo | Gene excision | Intravenous | Viral vector | Lymphoid tissues | Viral transduction | AAV9 | Cas9 | Dose 1: 0.9E12 vg/kg | Dose 2: 3.0E12 vg/kg | Phase1 | Completed | Active | 2021-11-22 | 2024-11-14 | 2024-12-02 | 18 Years - 70 Years | 6 | 3 | United States | US20190367924A1; US20200392487A1; US20240139294A1 | Safe, but does not provide long-term viral suppression |
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| NCT05903794 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | EXG102-031 | Exegenesis Bio | Industry | ABD-VEGFR | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: Undisclosed dose 1 | Dose 2: Undisclosed dose 2 | Phase1 | Active not recruiting | Active | 2023-06-06 | 2026-02-28 | 2025-04-17 | >= 50 Years | 12 | 2 | United States | US20240190943A1 | FDA cleared IND 1/18/23 |
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| NCT04418414 | Hemophilia A | CD68-ET3 | Expression Therapeutics, LLC | Industry | F8 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase1 | Not yet recruiting | Active | 2020-05-28 | 2039-08 | 2024-02-20 | >= 18 Years | 7 | 0 | WO2022165390A1 |
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| NCT06641154 | Crigler-Najjar Syndrome Type I | GT-UGT1A1-AAV8-02 | Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare | Other gov | UGT1A1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | rAAV2/8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2024-10-11 | 2029-11-01 | 2025-01-13 | 3 Months - 10 Years | 5 | 2 | Russian Federation |
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| NCT06545955 | Non-muscle Invasive Bladder Cancer With Carcinoma in Situ | ADSTILADRIN | Ferring Pharmaceuticals | Industry | IFNa2b | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravesicular | Viral vector | Viral transduction | Ad5 | Dose: 3E11 vp/ml, volume: 75ml, frequency: every 3 months | Phase2 | Recruiting | Approved | 2024-08-06 | 2030-12-31 | 2025-04-23 | >= 18 Years | 150 | 10 | United States | FDA approved on 12/16/22 for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. |
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| NCT05762510 | Beta-Thalassemia Major | GMCN-508B | First Affiliated Hospital of Guangxi Medical University | Other | HBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Early phase1 | Recruiting | Active | 2022-01-06 | 2030-10-31 | 2023-05-09 | 5 Years - 35 Years | 5 | 1 | China |
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| NCT05757245 | Transfusion-dependent Alpha-Thalassemia | GMCN-508A | First Affiliated Hospital of Guangxi Medical University | Other | HBA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase1 | Recruiting | Active | 2023-02-24 | 2030-12-31 | 2023-04-18 | 5 Years - 35 Years | 5 | 1 | China |
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| NCT03173521 | Mucopolysaccharidosis Type VI | AAV2/8.TBG.hARSB | Fondazione Telethon | Other | ARSB | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/8 | Dose 1: 6E11 gc/kg | Dose 2: 2E12 gc/kg | Dose 3: 6E12 gc/kg | Phase2, Phase1 | Completed | Active | 2017-04-12 | 2024-07-16 | 2024-11-22 | 4 Years - 65 Years | 9 | 2 |
Locations:Italy + 1 more
Italy, Turkey
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US20190343929A1 |
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| NCT00598481 | Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) | STRIMVELIS | Fondazione Telethon | Other | ADA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | MoMLV (RV) | Dose 1: Median dose: 11.6E6 CD34+ cells/kg (19 patients) | Dose 2: Median dose: 9.2E6 CD34+ cells/kg (22 patients) | Phase2 | Completed | Approved | 2008-01-10 | 2019-06-19 | 2024-01-29 | <= 17 Years | 12 | 2 |
Locations:Israel + 1 more
Israel, Italy
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Product was initially developed by SR-TIGET, in-licensed by GlaxoSmithKline in 2010. EMA approval in May 2016. GSK sold Strimvelis in March 2018 to Orchard Therapeutics. Orchard discontinued the program in March 2022. Product license was transferred to Fondazione Telethon in July 2023 |
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| NCT03837483 | Wiskott-Aldrich Syndrome | Etuvetidigene autotemcel | Fondazione Telethon | Other | WAS | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells (4.4 - 14.5E6 cells/kg) | Phase3 | Active not recruiting | Active | 2019-02-08 | 2027-09 | 2024-01-31 | <= 65 Years | 10 | 2 |
Locations:United States + 1 more
Italy, United States
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EP3973996A1 | BLA submitted 3/11/25 |
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| NCT05739643 | Krabbe Disease | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | FBX-101 | Forge Biologics, Inc | Industry | GALC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh10 | Dose 1: 1.6E13 gc/kg | Dose 2: Undisclosed dose 2 | Phase2, Phase1 | Active not recruiting | Active | 2023-02-13 | 2026-11 | 2025-01-29 | <= 18 Years | 9 | 3 | United States | WO2018136710; WO2020132385A1 | Dose escalation to highest dose approved, will start 2Q24 |
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| NCT06492863 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | FT-003 | Frontera Therapeutics | Industry | Aflibercept | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV2.7m8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2024-07-01 | 2028-10-15 | 2024-07-09 | 50 Years - 80 Years | 78 | 1 | China | US20230295243A1 | FDA clearance for Phase 2 trial received 11/11/24 | |||||
| NCT05858983 | Biallelic RPE65 Mutation-associated Retinal Dystrophy | FT-001 | Frontera Therapeutics | Industry | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: 1.5E10 vg/eye | Dose 2: 7.5E10 vg/eye | Dose 3: 15E10 vg/eye | Phase2, Phase1 | Recruiting | Active | 2023-04-29 | 2029-11-30 | 2023-05-15 | 8 Years - 45 Years | 9 | 1 | China | US20230321280A1 |
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| NCT06492876 | Diabetic Macular Edema | FT-003 | Frontera Therapeutics | Industry | Aflibercept | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV2.7m8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2024-07-01 | 2028-11-15 | 2024-07-09 | 18 Years - 74 Years | 78 | 1 | China | US20230295243A1 | IND cleared 12/30/24 |
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| NCT06492850 | X-Linked Retinitis Pigmentosa (XLRP) | Fast Track, Orphan Drug Designation | FT-002 | Frontera Therapeutics | Industry | RPGRORF15 | Gene transfer | In-vivo | Functional gene replacement | Intraocular | Viral vector | Photoreceptors | Viral transduction | AAV5 | Dose 1: 5E10 vg/eye | Dose 2: 10E10 vg/eye | Dose 3: 20E10 vg/eye | Phase2, Phase1 | Recruiting | Active | 2024-07-01 | 2026-02-01 | 2024-07-09 | 8 Years - 45 Years | 32 | 1 | China | CA3186826A1 | FDA clears IND for Phase 2 clinical trials 9/23/24 |
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| NCT02652767 | Leber Hereditary Optic Neuropathy (LHON) | LUMEVOQ | GenSight Biologics | Industry | ND4G11778A | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV2 | 9E10 vg in 90uL | Phase3 | Completed | Active | 2016-01-07 | 2019-07-04 | 2022-07-29 | >= 15 Years | 39 | 7 |
Locations:United States + 4 more
France, Germany, Italy, United Kingdom, United States
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WO2019241206A1 | EAP due to commence Q3 2024, France only; MAA in UK goal in 2026 |
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| NCT03326336 | Non-syndromic Retinitis Pigmentosa | GS030 | GenSight Biologics | Industry | ChR-tdT | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Retinal ganglion | Viral transduction | AAV2.7m8 | Dose 1: 5E10 vg/eye | Dose 2: 1.5E11 vg/eye | Dose 3: 5E11 vg/eye | Phase2, Phase1 | Recruiting | Active | 2017-10-11 | 2025-12 | 2022-07-26 | 18 Years - 75 Years | 15 | 3 |
Locations:United States + 2 more
France, United Kingdom, United States
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US20240238613A1; US20240165198A1 |
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| NCT05835895 | Osteoarthritis, Knee | Fast Track | GNSC-001 | Genascence Corporation | Industry | IL1RN | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarticular | Viral vector | Viral transduction | AAV2.5 | Dose 1: 1E11 vg/knee | Dose 2: 1E12 vg/knee | Dose 3: 1E13 vg/knee | Phase1 | Active not recruiting | Active | 2023-04-19 | 2029-05 | 2024-12-27 | 40 Years - 75 Years | 67 | 9 | United States | US20220016213A1 | Initial data for Phase 1b trial expected Q4 2024 |
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| NCT05860569 | Hypertriglyceridemia | GC304 | GeneCradle Inc | Industry | GPIHBP1 or LPL | Gene transfer | In-vivo | Functional gene replacement, overexpression of protective allele/gene | Intravenous | Viral vector | Viral transduction | AAV5 | Dose 1: 3.0E12 vg/kg | Dose 2: 1.0E13 vg/kg | Dose 3: 3.0E13 vg/kg | Phase1 | Recruiting | Active | 2023-04-23 | 2028-12 | 2025-03-13 | 18 Years - 60 Years | 7 | 1 | China | Recruitment started October 2024 |
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| NCT06391736 | Pompe Disease (Late-onset) | Orphan Drug Designation | GC301 | GeneCradle Inc | Industry | GAA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 3.0E13 vg/kg | Dose 2: 6.0E13 vg/kg | Dose 3: 1.2E14 vg/kg (IIT study) | Phase2, Phase1 | Recruiting | Active | 2024-04-25 | 2026-12 | 2024-04-30 | >= 6 Years | 33 | 1 | China | First subject dosed August 2024 |
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| NCT05824169 | Spinal Muscular Atrophy | GC101 | GeneCradle Inc | Industry | SMN1 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | Dose 1: 2.4E14 vg | Dose 2: 4.8E14 vg | Phase2, Phase1 | Recruiting | Active | 2023-04-10 | 2025-12 | 2023-04-25 | 0 Months - 6 Months | 18 | 4 | China |
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| NCT06739434 | Rett Syndrome | GCB-002 | Genecombio Ltd. | Other | MECP2 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | scAAV9 | 2 cohort dose escalation, undisclosed concentrations | Na | Enrolling by invitation | Active | 2024-12-12 | 2030-12 | 2025-03-11 | 2 Years - 10 Years | 6 | 1 | China |
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| NCT01344798 | Limb-Girdle Muscular Dystrophy, Type 2C/R5 | AAV1.DES.hSGCG | Genethon | Other | SGCG | Gene transfer | In-vivo | Functional gene replacement | Intramuscular (carpi radialis) | Viral vector | Viral transduction | AAV1 | Dose 1: 3E9 vg/100ul | Dose 2: 1.5E10 vg/100ul | Dose 3: 4.5E10 vg/300ul | Phase1 | Completed | Inactive | 2011-03-17 | 2010-06 | 2011-04-29 | >= 15 Years | 9 | 1 | France | Vector was well tolerated but expression of transgene was not detectable in most patients after 30 days | ||||||
| NCT03466463 | Crigler-Najjar Syndrome | GNT0003 | Genethon | Other | UGT1A1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAV8 | Dose 1: 2E12 vg/kg | Dose 2: 5E12 vg/kg | Na | Recruiting | Active | 2018-02-01 | 2030-03-30 | 2023-03-28 | >= 9 Years | 17 | 4 |
Locations:France + 2 more
France, Italy, Netherlands
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| NCT01024998 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | RAAV2-sFLT01 | Genzyme, a Sanofi Company | Industry | SFLT01 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Viral transduction | AAV2 | Dose 1: 2E8 vg/eye | Dose 2: 2E9 vg/eye | Dose 3: 6E9 vg/eye | Dose 4: 2E10 vg/eye | Phase1 | Completed | Inactive | 2009-12-02 | 2018-07 | 2018-08-22 | >= 50 Years | 19 | 4 | United States | Sanofi acquired Genzyme in 2011, product was well tolerated but showed limited efficacy and never moved to Phase 2 |
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| NCT06199531 | NGLY1 Deficiency | Support for Clinical Trials Advancing Rare Disease Therapeutics | GS-100 | Grace Science, LLC | Industry | NGLY1 | Gene transfer | In-vivo | Functional gene replacement | Intracerebroventricular | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2023-11-21 | 2028-01-31 | 2025-04-24 | 2 Years - 18 Years | 6 | 3 | United States | EP4329824A1 | Selected for START program (6/3/24) |
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| NCT05207657 | Autosomal Recessive Chronic Granulomatous Disease (CGD) | PCHIM-p47 | Great Ormond Street Hospital for Children NHS Foundation Trust | Other | NCF1 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells, minimum dose: 3E6 CD34+ cells/kg | Phase2, Phase1 | Recruiting | Active | 2021-06-24 | 2029-04-01 | 2023-05-16 | >= 23 Months | 5 | 1 | United Kingdom | Updated NCTID is NCT06253507 (NIH-sponsored trial) |
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| NCT06833983 | Hemophilia A | GS1191-0445 | Gritgen Therapeutics Co., Ltd. | Industry | F8 (BDD variant) | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Hepatocyte | Viral transduction | AAV8 | Dose 1: 2E12 vg/kg | Dose 2: 4E12 vg/kg | Phase3 | Recruiting | Active | 2025-01-28 | 2030-11-30 | 2025-04-24 | 18 Years - 65 Years | 50 | 12 | China | First patient dosed November 2023 | |||||
| NCT06856759 | Rett Syndrome | AAV-MECP2 | Guangzhou Women and Children's Medical Center | Other | MECP2 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | Dose 1: 7E14 vg | Dose 2: 1.5E15 vg | Phase2, Phase1 | Recruiting | Active | 2024-12-29 | 2029-10-23 | 2025-03-04 | 4 Years - 10 Years | 6 | 1 | China | |||||||
| NCT04566445 | Dry Age-related Macular Degeneration | GT005 | Gyroscope Therapeutics Limited | Industry | CFI | Gene transfer | In-vivo | Overexpression of protective allele/gene | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: Undisclosed dose 1 | Dose 2: Undisclosed dose 2 | Dose 3: Undisclosed dose 3 | Phase2 | Terminated | Inactive | 2020-09-09 | 2024-06-10 | 2024-07-08 | >= 55 Years | 255 | 62 |
Locations:United States + 6 more
Australia, France, Germany, Poland, Spain, United Kingdom, United States
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Novartis acquired the company; Study was terminated due to interim analysis demonstrating lack of efficacy |
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| NCT00821340 | Leber Congenital Amaurosis-Type 2 | RAAV2-hRPE65 | Hadassah Medical Organization | Other | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Undisclosed | Phase1 | Completed | Inactive | 2009-01-12 | 2017-01-01 | 2018-04-10 | >= 8 Years | 3 | 1 | Israel | Enrolled 3/10 planned patients | ||||||
| NCT00787059 | Congestive Heart Failure | Fast Track | RT-100 | Hammond, H. Kirk, M.D. | Indiv | ADCY6 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intracoronary | Viral vector | Myocardium | Viral transduction | Ad5 | Dose 1: 3.2E9 vp (n=6) | Dose 2: 3.2E10 vp (n=6) | Dose 3: 1E10 vp (n=6) | Dose 4: 3.2E11 vp (n=12) | Dose 5: 1E12 vp (n=12) | Phase2, Phase1 | Completed | Active | 2008-11-06 | 2017-11-16 | 2018-02-09 | 18 Years - 80 Years | 56 | 7 | United States | Company is preparing to submit a plan to the FDA for the Phase 2b/3 study |
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| NCT06819514 | Fabry Disease | Orphan Drug Designation | EXG110 | Hangzhou Jiayin Biotech Ltd | Industry | GLA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | proprietary AAV | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Phase2, Phase1 | Not yet recruiting | Active | 2025-01-22 | 2028-03-15 | 2025-02-11 | >= 18 Years | 16 | 3 | China | First patient has been dosed in China, company plans to initiate US clinical trials |
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| NCT05361031 | Charcot-Marie-Tooth Disease 1A | ENGENSIS | Helixmith Co., Ltd. | Industry | HGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramuscular | Plasmid | None (naked plasmid) | 0.25mg/0.5ml (56 injections on days 0,14,90,104) | Phase2, Phase1 | Completed | Active | 2022-03-31 | 2021-09-09 | 2022-05-04 | 19 Years - 65 Years | 12 | 1 |
Locations:Korea + 1 more
Korea, Republic of
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Same product as NL003 |
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| NCT01064440 | Critical Limb Ischemia | ENGENSIS | Helixmith Co., Ltd. | Industry | HGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramuscular (gastrocnemius) | Plasmid | None (naked plasmid) | Dose 1: 2,4,8,16 mg total dose | Dose 2: Total dose: 8mg, 16mg | Dose 3: Total dose: 4mg/8ml, 8mg/16ml, 12mg/24ml, 16mg/32ml; concentration: 0.25/0.5ml, injections/day x 2 days: 8, 16, 24, 32 | Phase2 | Completed | Active | 2010-02-04 | 2023-11-17 | 2025-03-11 | 18 Years - 90 Years | 52 | 16 |
Locations:United States + 2 more
Korea, Republic of, United States
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Same product as NL003 |
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| NCT04469270 | Diabetic Neuropathy | Regenerative Medicine Advanced Therapy | ENGENSIS | Helixmith Co., Ltd. | Industry | HGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramuscular (gastrocnemius) | Plasmid | None (naked plasmid) | Dose 1: Total dose: 4mg or 8mg or 16mg (injections divided on day 0 and day 14) | Dose 2: Total dose: 16mg/32 x 0.5ml injections/gastrocnemius | Dose 3: Total dose: 32mg/32 x 0.5ml injections/gastrocnemius | Dose 4: Total dose: 32mg, concentration: 0.25mg/0.5ml, 16injections/day, 4 days: 0, 14 ,90, 104, bilateral gastroc. | Phase3 | Completed | Active | 2020-06-25 | 2024-07-31 | 2025-01-20 | >= 18 Years | 162 | 16 | United States | Same product as NL003 |
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| NCT02563522 | Diabetic Foot Ulcer | ENGENSIS | Helixmith Co., Ltd. | Industry | HGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramuscular (gastrocnemius) | Plasmid | None (naked plasmid) | 4mg (dose/day) * 4 treatment days | Phase3 | Terminated | Active | 2015-09-28 | 2019-09-24 | 2025-03-28 | 18 Years - 80 Years | 44 | 22 | United States | Same product as NL003 | |||||||
| NCT05176093 | Amyotrophic Lateral Sclerosis | ENGENSIS | Helixmith Co., Ltd. | Industry | HGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramuscular | Plasmid | None (naked plasmid) | 64mg/cycle, 3 cycles/patient, 2 days/cycle, 128 injections/cycle | Phase2 | Completed | Active | 2021-11-05 | 2022-12-29 | 2023-02-02 | 18 Years - 80 Years | 8 | 2 |
Locations:United States + 2 more
Korea, Republic of, United States
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Same product as NL003 |
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| NCT02984085 | Recessive Dystrophic Epidermolysis Bullosa | HOLOGENE7 | Holostem s.r.l. | Industry | COL7A1 | Gene transfer | Ex-vivo | Functional gene replacement | Skin graft | Autologous cells | Epidermal cells | Viral transduction | RV | Transduced autologous skin grafts | Phase2, Phase1 | Terminated | Inactive | 2016-12-02 | 2018-08-06 | 2022-02-16 | 6 Years - 54 Years | 3 | 1 | Austria | 3 patients enrolled, study terminated, to be replaced by a new study |
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| NCT05222178 | Phenylketonuria (PKU) | Fast Track | HMI-103 | Homology Medicines, Inc | Industry | PAH | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Hepatocyte | Viral transduction | AAVHSC15 | 6E13 vg/kg | Phase1 | Terminated | Inactive | 2021-12-21 | 2023-09-14 | 2023-10-10 | 18 Years - 55 Years | 3 | 2 | United States | Homology Medicines has discontinued development of HMI-103 |
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| NCT06178432 | Pompe Disease (Late-onset) | BBM-G102 | Huashan Hospital | Other | GAA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV | Undisclosed dose 1 | Early phase1 | Not yet recruiting | Active | 2023-11-30 | 2028-12 | 2023-12-21 | >= 18 Years | 6 | 1 | China | This investigator-initiated trial is funded by Belief-Delivery BioMed Co. |
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| NCT05906953 | Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations | Orphan Drug Designation, Rare Pediatric Disease Designation | HG004 | HuidaGene Therapeutics Co., Ltd. | Industry | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2023-05-09 | 2025-12 | 2024-09-19 | 6 Years - 50 Years | 20 | 3 |
Locations:United States + 1 more
China, United States
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First patient dosed November 2023 |
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| NCT06031727 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | HG202 | HuidaGene Therapeutics Co., Ltd. | Industry | VEGFA mRNA | Gene editing | In-vivo | Gene inactivation | Subretinal | Viral vector | Viral transduction | AAV | hfCas13Y | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Early phase1 | Recruiting | Active | 2023-09-01 | 2026-06-30 | 2025-03-17 | 50 Years - 80 Years | 12 | 2 | China | WO2023185878A1 | FDA cleared IND November 2024 |
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| NCT06615206 | MECP2 Duplication Syndrome | Orphan Drug Designation, Rare Pediatric Disease Designation | HG204 | HuidaGene Therapeutics Co., Ltd. | Industry | MECP2 | Gene editing | In-vivo | Gene inactivation | Intracerebroventricular | Viral vector | Viral transduction | AAV | hfCas13Y | Up to 2 dose cohorts, undisclosed concentration | Na | Recruiting | Active | 2024-09-04 | 2026-10-31 | 2024-11-26 | 2 Years - 18 Years | 6 | 1 | China | First patient dosed announced in December 2024 |
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| NCT06594094 | Duchenne Muscular Dystrophy (DMD) | Orphan Drug Designation, Rare Pediatric Disease Designation | HG302 | HuidaGene Therapeutics Co., Ltd. | Industry | DMD (exon 51 splice site) | Gene editing | In-vivo | Exon skipping/splice editor | Intravenous | Viral vector | Viral transduction | AAV | hfCas12Max | Up to 2 dose cohorts, undisclosed concentration | Na | Recruiting | Active | 2024-09-10 | 2026-09-30 | 2024-11-25 | 4 Years - 8 Years | 6 | 1 | China | First patient dosed announced in December 2024 |
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| NCT06623279 | Neovascular Age-Related Macular Degeneration (nAMD) | HG202 | HuidaGene Therapeutics Co., Ltd. | Industry | VEGFA mRNA | Gene editing | In-vivo | Gene inactivation | Subretinal | Viral vector | Viral transduction | AAV | hfCas13Y | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase1 | Not yet recruiting | Active | 2024-09-30 | 2031-02-01 | 2024-10-02 | 50 Years - 85 Years | 15 | 0 | FDA cleared IND November 2024 |
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| NCT05454566 | Osteoarthritis, Knee | ICM-203 | ICM Co. Ltd. | Industry | NKX3-2 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarticular | Viral vector | Viral transduction | AAV5.2 | Dose 1: 6E12 vg | Dose 2: 2E13 vg | Dose 3: 6E13 vg | Phase2, Phase1 | Not yet recruiting | Active | 2022-07-04 | 2026-11 | 2024-10-15 | 50 Years - 80 Years | 18 | 0 | Update on Phase 1/2a study given at ASGCT 2024 |
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| NCT02453477 | Beta-Thalassemia Major | OTL-300 | IRCCS San Raffaele | Other | HBB | Gene transfer | Ex-vivo | Functional gene replacement | Intraosseous | Autologous cells | CD34+ cells | Viral transduction | LV | Dose 1: Target dose: 5E6 CD34+ cells/kg | Dose 2: Minimum dose: 2E6 CD34+ cells/kg | Dose 3: Maximum dose: 20E6 CD34+ cells/kg | Phase2, Phase1 | Unknown | Inactive | 2015-05-15 | 2019-08 | 2019-06-28 | 3 Years - 64 Years | 10 | 1 | Italy | Program discontinued by Orchard |
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| NCT01621867 | Cystic Fibrosis | PGM169/GL67A | Imperial College London | Other | CFTR | Gene transfer | In-vivo | Functional gene replacement | Inhalational | Encapsulated plasmid | Liposome | GL67A | Dose 1: Concentration: 26.5mg pDNA/10ml | Dose 2: 5ml (n=8) selected as optimal dose, n=78 for phase 2b | Dose 3: 10ml (n=10) | Dose 4: 20ml (n=17) was discontinued due to adverse effects | Phase2 | Completed | Inactive | 2012-06-14 | 2014-05 | 2015-10-22 | >= 12 Years | 130 | 3 | United Kingdom | Benefits were too small to enable Phase 3 development, partnered with Boehringer Ingelheim to develop a new strategy using viral delivery. Clinical trial (NCT06515002) is ongoing |
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| NCT06289452 | Retinoschisis, Retinal Disease, Retinal Degeneration, Eye Diseases | Rare Pediatric Disease Designation | IVB102 | InnoVec Biotherapeutics Inc. | Industry | RS1 | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Early phase1 | Recruiting | Active | 2024-02-25 | 2029-12-31 | 2024-05-07 | >= 8 Years | 18 | 1 | China | WO2023143606A1 |
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| NCT06196827 | Inherited Retinal Dystrophy Associated With RPE65 Mutations | LX101 | Innostellar Biotherapeutics Co.,Ltd | Industry | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: Undisclosed low dose (0.3mL/eye) | Dose 2: Undisclosed high dose (0.3mL/eye) | Phase2, Phase1 | Active not recruiting | Active | 2023-12-25 | 2027-12 | 2024-01-09 | >= 6 Years | 9 | 2 | China | US11970519B2 |
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| NCT06196840 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | LX102 | Innostellar Biotherapeutics Co.,Ltd | Industry | VEGF-trap | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: 2E10 vg | Dose 2: Undisclosed dose 2 | Phase2 | Recruiting | Active | 2023-12-25 | 2029-05 | 2024-01-09 | 50 Years - 89 Years | 50 | 3 | China | |||||||
| NCT06817382 | Duchenne Muscular Dystrophy (DMD) | INS1201 | Insmed Gene Therapy LLC | Industry | Micro-dystrophin | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV | Undisclosed dose escalation, 2 levels | Phase1 | Recruiting | Active | 2025-02-04 | 2028-03-31 | 2025-04-24 | 2 Years - 4 Years | 12 | 1 | United States | IND cleared in 4Q2024, Clinical trial initiation planned in 1H2025 |
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| NCT01801709 | Metachromatic Leukodystrophy | AAVrh.10-CAG/cuARSA | Institut National de la Santé Et de la Recherche Médicale, France | Other gov | ARSA | Gene transfer | In-vivo | Functional gene replacement | Intracerebral | Viral vector | Viral transduction | AAVrh.10 | Dose 1: 1E12 vg | Dose 2: 4E12 vg | Phase2, Phase1 | Completed | Inactive | 2013-01-28 | 2022-12-20 | 2025-02-04 | 6 Months - 5 Years | 5 | 1 | France | INSERM is developing a new therapy with a different AAV (PMID: 34093126) |
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| NCT04186650 | Recessive Dystrophic Epidermolysis Bullosa (RDEB) | EF1a-COL7A1-SIN retroviral vector transduced autologous skin | Institut National de la Santé Et de la Recherche Médicale, France | Other gov | COL7A1 | Gene transfer | Ex-vivo | Functional gene replacement | Skin graft | Autologous cells | Keratinocytes | Viral transduction | RV | Up to 6 grafts of 50 cm^2 each | Phase2, Phase1 | Active not recruiting | Active | 2019-11-25 | 2027-06-09 | 2025-01-29 | >= 18 Years | 3 | 1 | France | EP2766480B1; WO2022122900A1 |
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| NCT04728841 | Hemophilia A | GS001 | Institute of Hematology & Blood Diseases Hospital, China | Other | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: 2E12 vg/kg | Dose 2: 4E12 vg/kg | Dose 3: Up to 2E13 vg/kg | Na | Recruiting | Active | 2021-01-25 | 2028-07-31 | 2025-02-24 | >= 18 Years | 12 | 1 | China | |||||||
| NCT05641610 | Hemophilia B | ZS801 | Institute of Hematology & Blood Diseases Hospital, China | Other | F9 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV | Dose 1: 2.0E12 vg/kg | Dose 2: 5.0E12 vg/kg | Dose 3: 1.0E13 vg/kg | Phase2, Phase1 | Recruiting | Active | 2022-11-25 | 2028-12 | 2025-02-24 | >= 18 Years | 21 | 1 | China |
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| NCT06238908 | Hemophilia A | NGGT003 | Institute of Hematology & Blood Diseases Hospital, China | Other | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: 4E11 vg/kg | Dose 2: 1E12 vg/kg | Dose 3: 2.5E12 vg/kg | Early phase1 | Recruiting | Active | 2024-01-26 | 2030-01-31 | 2025-02-21 | >= 18 Years | 6 | 1 | China | CN116715752A | ||||||
| NCT06634420 | Hereditary Angioedema | NTLA-2002 | Intellia Therapeutics | Industry | KLKB1 | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Hepatocyte | Lipid encapsulation | LDLR | Cas9 mRNA | 50 mg | Phase3 | Recruiting | Active | 2024-10-07 | 2027-09 | 2025-04-22 | >= 16 Years | 60 | 27 |
Locations:United States + 8 more
Australia, Canada, France, Germany, Netherlands, New Zealand, South Africa, United Kingdom, United States
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US20230203480A1; US20240018496A1; WO2024011206A1 | First patient dosed January 2025; BLA submission planned H2 2026; link to NCT05120830 |
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| NCT06128629 | Transthyretin Amyloidosis (ATTR) with Cardiomyopathy | NTLA-2001 | Intellia Therapeutics | Industry | TTR | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Hepatocyte | Lipid encapsulation | LNP | Cas9 mRNA | 55mg | Phase3 | Recruiting | Active | 2023-11-08 | 2028-04 | 2025-03-06 | 18 Years - 90 Years | 765 | 92 |
Locations:United States + 22 more
Argentina, Australia, Austria, Brazil, Canada, Czechia, Denmark, France, Germany, Hungary, Israel, Italy, Korea, Netherlands, New Zealand, Portugal, Republic of, Singapore, Spain, Sweden, Taiwan, United Kingdom, United States
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US20230257747A1; WO2017173054A1; US20240076636A1; US20230287400A1; US20210087568A1; WO2020219876A1; US20220009878A1 | Plan to dose the first patient in the pivotal Phase 3 MAGNITUDE-2 trial for ATTRv-PN in 1Q25. |
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| NCT06622668 | Alpha-1 Antitrypsin Deficiency (AATD) | NTLA-3001 | Intellia Therapeutics | Industry | SERPINA1 | Gene editing | In-vivo | Functional gene replacement | Intravenous | MRNA, LNP | Liver | Lipid encapsulation | LNP | Cas9 mRNA | 3 undisclosed dose levels | Phase2, Phase1 | Withdrawn | Inactive | 2024-09-30 | 2025-01-09 | 2025-01-17 | 18 Years - 75 Years | 0 | 1 | New Zealand | Sponsor discontinued this program, no subjects were enrolled and the trial was withdrawn from clinicaltrials.gov | ||||
| NCT05120830 | Hereditary Angioedema | NTLA-2002 | Intellia Therapeutics | Industry | KLKB1 | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Hepatocyte | Lipid encapsulation | LDLR | Cas9 mRNA | Dose 1: 25 mg | Dose 2: 50 mg (pivotal dose) | Dose 3: 75 mg | Phase2, Phase1 | Active not recruiting | Active | 2021-11-03 | 2026-03-31 | 2024-09-19 | >= 18 Years | 37 | 9 |
Locations:Australia + 5 more
Australia, France, Germany, Netherlands, New Zealand, United Kingdom
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US20230203480A1; US20240018496A1; WO2024011206A1 | BLA submission planned 2026; Phase II study met all primary and secondary endpoints, selected pivotal dose |
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| NCT06662188 | SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome | JAG201 | Jaguar Gene Therapy, LLC | Industry | SHANK3 | Gene transfer | In-vivo | Functional gene replacement | Intracerebroventricular | Viral vector | Viral transduction | AAV2/9 | Undisclosed dose escalation | Phase2, Phase1 | Recruiting | Active | 2024-10-16 | 2031-06 | 2025-03-05 | 2 Years - 9 Years | 6 | 2 | United States | Clinical sites are now open |
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| NCT05811351 | Geographic Atrophy | JNJ-1887 | Janssen Research & Development, LLC | Industry | CD59 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Viral transduction | AAV2 | Dose 1: 3.56E10 vg/eye | Dose 2: 1.071E11 vg/mL | Dose 3: 3.56E11 vg/eye | Phase2 | Active not recruiting | Active | 2023-03-07 | 2026-05-19 | 2025-04-25 | >= 60 Years | 305 | 162 |
Locations:United States + 16 more
Australia, Belgium, Canada, Czechia, Denmark, Germany, Hungary, Italy, Netherlands, Poland, Portugal, Spain, Sweden, Switzerland, Turkey, United Kingdom, United States
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WO2023089564A1 |
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| NCT04671433 | X-Linked Retinitis Pigmentosa | Fast Track, Orphan Drug Designation | Botaretigene sparoparvovec | Janssen Research & Development, LLC | Industry | RPGR | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/5 | Dose 1: 2.0E10 vg/mL | Dose 2: 2.0E11 vg/mL | Dose 3: 2.0E12 vg/mL | Phase3 | Completed | Active | 2020-11-05 | 2024-09-30 | 2025-04-25 | >= 3 Years | 97 | 27 |
Locations:United States + 10 more
Belgium, Canada, Denmark, France, Israel, Italy, Netherlands, Spain, Switzerland, United Kingdom, United States
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WO2024079655A1; US11045558B2 | Phase 3 dosing complete, potential BLA filing in 2025 |
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| NCT02354781 | Duchenne Muscular Dystrophy (DMD) | RAAV1.CMV.huFollistatin344 | Jerry R. Mendell | Other | FST (FS344) | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramuscular (gluteus, quadriceps, tibialis anterior) | Viral vector | Viral transduction | AAV1 | 2.4E12 vg/kg | Phase2, Phase1 | Completed | Inactive | 2015-01-26 | 2017-11 | 2023-10-11 | >= 7 Years | 3 | 1 | United States | Study drug was well tolerated, was not tested further | ||||||
| NCT04819841 | Sickle Cell Disease | KMAU-001 | Kamau Therapeutics | Industry | HBB | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Viral transduction | AAV6 | Cas9 mRNA | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | Active | 2021-03-24 | 2027-07-31 | 2025-04-13 | 12 Years - 40 Years | 15 | 3 | United States | US11851652B2 | Product was previously developed by Graphite Bio |
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| NCT06280378 | Beta-Thalassemia Major | KL003 | Kanglin Biotechnology (Hangzhou) Co., Ltd. | Industry | HBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | Active | 2024-02-20 | 2027-05 | 2025-03-10 | 3 Years - 35 Years | 41 | 2 | China | Received NMPA approval on 1/3/24 | |||||
| NCT03333590 | Duchenne Muscular Dystrophy (DMD) | RAAVrh74.MCK.GALGT2 | Kevin Flanigan | Other | GALGT2 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Isolated limb infusion (femoral artery) | Viral vector | Viral transduction | AAVrh74 | Dose 1: 2.5E13 vg/kg/leg | Dose 2: 5E13 vg/kg/leg | Phase2, Phase1 | Completed | Inactive | 2017-11-01 | 2023-12-31 | 2025-03-13 | >= 4 Years | 2 | 1 | United States | Licensed to Sarepta in December 2015, only 2 patients were dosed out of 6 planned, efficacy data was not compelling |
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| NCT01482195 | Retinal Disease, Retinitis Pigmentosa | AAV2-VMD2-hMERTK | King Khaled Eye Specialist Hospital | Other gov | MERTK | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: 5.96E10 vg/150ul (n=2) | Dose 2: 1.788E11 vg/450ul (n=4) | Phase1 | Completed | Inactive | 2011-09-28 | 2019-08 | 2022-01-26 | 14 Years - 70 Years | 6 | 1 | Saudi Arabia | Therapy was well tolerated, 3/6 patients displayed improved visual acuity, but the effects persisted less than 2 years in 2 of the 3 |
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| NCT06049082 | Alpha-1 Antitrypsin Deficiency (AATD) | Orphan Drug Designation | KB-408 | Krystal Biotech, Inc. | Industry | SERPINA1 | Gene transfer | In-vivo | Functional gene replacement | Inhalational | Viral vector | Lung | Viral transduction | HSV-1 | Dose 1: 10^9 PFU | Dose 2: 10^10 PFU | Dose 3: 10^11 PFU | Phase1 | Recruiting | Active | 2023-09-15 | 2026-05 | 2024-12-16 | 18 Years - 70 Years | 12 | 2 | United States | Company expects additional updates in 2025, will open cohort 3 (highest dose) |
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| NCT04917874 | Recessive Dystrophic Epidermolysis Bullosa (RDEB) | Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | VYJUVEK | Krystal Biotech, Inc. | Industry | COL7A1 | Gene transfer | In-vivo | Functional gene replacement | Topical | Viral vector | Viral transduction | HSV1 | Dose 1: Maximum weekly dose: 1.6E9 PFU (6 months to < 3 years) | Dose 2: Maximum weekly dose: 3.2E9 PFU (over 3 years old) | Phase3 | Completed | Approved | 2021-06-02 | 2023-07-31 | 2024-04-09 | >= 2 Months | 47 | 6 | United States | FDA approved 5/19/23, estimated price $600K/year |
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| NCT05735158 | Autosomal Recessive Congenital Ichthyosis | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | KB105 | Krystal Biotech, Inc. | Industry | TGM1 | Gene transfer | In-vivo | Functional gene replacement | Topical | Viral vector | Viral transduction | HSV-1 | Undisclosed single dose formulated as a topical gel, weekly application | Phase2 | Unknown | Active | 2023-02-09 | 2024-04 | 2023-02-23 | >= 6 Months | 15 | 1 | United States | WO2019200163A1; EP3377637B1; US11717547B2; US20200101123A1 | Initiate Phase 2 cohort in 2026 |
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| NCT05504837 | Cystic Fibrosis | Orphan Drug Designation, Rare Pediatric Disease Designation | KB407 | Krystal Biotech, Inc. | Industry | CFTR | Gene transfer | In-vivo | Functional gene replacement | Inhalational | Viral vector | Viral transduction | HSV-1 | Dose 1: 10E9 PFU (single administration | Dose 2: 10E9 PFU (2 administrations) | Dose 3: 10E9 PFU (4 administrations) | Phase1 | Recruiting | Active | 2022-08-15 | 2025-07 | 2025-02-04 | >= 18 Years | 12 | 3 | United States | WO2020163703A1; WO2021127524A1 | Interim data expected for cohort 3 mid-2025 |
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| NCT02852213 | Aromatic L-amino Acid Decarboxylase (AADC) Deficiency | AAV2-hAADC | Krzysztof Bankiewicz | Other | DDC | Gene transfer | In-vivo | Functional gene replacement | CED | Viral vector | Substantia nigra & ventral tegmental area | Viral transduction | AAV2 | Dose 1: Dose: 1.3E11 vg (<160uL); concentration of 8.3E11 vg/mL | Dose 2: 4.2E11 vg (160ul) | Dose 3: 1.6E12 vg (60uL) | Dose 4: 1.3E12 vg (500uL) | Phase1 | Recruiting | Active | 2016-07-20 | 2031-07 | 2024-12-20 | >= 24 Months | 42 | 3 | United States | US20230330267A1 |
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| NCT06191354 | Spinal Muscular Atrophy | SKG0201 | Kun Sun | Other | SMN1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV | Undisclosed dose escalation | Na | Recruiting | Active | 2023-12-19 | 2025-12 | 2024-05-01 | <= 180 Days | 12 | 3 | China | |||||||
| NCT03612869 | Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome) | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | LYS-SAF301 | LYSOGENE | Industry | SGSH | Gene transfer | In-vivo | Functional gene replacement | Intracerebral | Viral vector | Viral transduction | AAV2rh.10 | Dose 1: 7.2E11 vg (used in Phase 1/2) | Dose 2: 7.2E12 vg | Phase3, Phase2 | Unknown | Inactive | 2018-07-04 | 2022-03 | 2021-08-31 | >= 6 Months | 20 | 8 |
Locations:United States + 4 more
France, Germany, Netherlands, United Kingdom, United States
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Phase 2/3 study failed primary efficacy endpoint |
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| NCT04273269 | GM1 Gangliosidosis | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | LYS-GM101 | LYSOGENE | Industry | GLB1 | Gene transfer | In-vivo | Functional gene replacement | Intracisternal | Viral vector | Viral transduction | AAVrh.10 | 8E12 vg/kg | Phase2, Phase1 | Terminated | Inactive | 2020-01-21 | 2023-05-22 | 2023-06-09 | <= 3 Years | 5 | 3 |
Locations:United States + 2 more
France, United Kingdom, United States
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2 participants died due to progression of disease, little evidence of potential efficacy |
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| NCT06288230 | Spinal Muscular Atrophy | Vesemnogene lantuparvovec | Lantu Biopharma | Industry | SMN1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV | Undisclosed dose | Phase2, Phase1 | Recruiting | Active | 2024-02-18 | 2027-10-30 | 2024-10-09 | 6 | 1 | China |
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| NCT06308159 | Beta-Thalassemia Major | Vebeglogene autotemcel | Lantu Biopharma | Industry | HBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Minimum dose: 5.0E6 CD34+ cells/kg | Phase2, Phase1 | Recruiting | Active | 2024-03-06 | 2027-08-01 | 2024-10-09 | <= 35 Years | 6 | 2 | China |
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| NCT04797260 | Severe Combined Immunodeficiency Due to RAG1 Deficiency | Autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector | Leiden University Medical Center | Other | RAG1 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Na | Recruiting | Active | 2021-03-11 | 2029-12-31 | 2024-04-18 | 8 Weeks - 24 Months | 10 | 7 |
Locations:Australia + 6 more
Australia, Italy, Netherlands, Poland, Spain, Turkey, United Kingdom
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| NCT05445323 | Friedreich Ataxia, Cardiomyopathy, Secondary | Fast Track | LX2006 | Lexeo Therapeutics | Industry | FXN | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh10 | Dose 1: 1.8E11 vg/kg | Dose 2: 5.6E11 vg/kg | Dose 3: 1.2E12 vg/kg | Phase2, Phase1 | Active not recruiting | Active | 2022-06-23 | 2029-09 | 2024-11-15 | 18 Years - 50 Years | 8 | 3 | United States | WO2023150563A1 | Program update expected mid 2025, potential to initiate registrational study by end of 2025/early 2026 |
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| NCT03634007 | Alzheimer Disease, Early Onset Alzheimer Disease | Fast Track | LX1001 | Lexeo Therapeutics | Industry | APOE2 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intrathecal | Viral vector | Viral transduction | AAVrh10 | Dose 1: 1.4E10 gc/mL CSF | Dose 2: 4.4E10 gc/mL CSF | Dose 3: 1.4E11 gc/mL CSF | Dose 4: 1.4E14 gc (fixed dose) | Phase2, Phase1 | Completed | Active | 2018-08-03 | 2024-11-07 | 2024-11-22 | >= 50 Years | 15 | 4 | United States | WO2021108809A1; WO2024011237A1; WO2021076941A1; WO2021022208A1 | Lexeo is pursuing partnership opportunities for continued development |
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| NCT06109181 | Arrhythmogenic Right Ventricular Cardiomyopathy | Fast Track, Orphan Drug Designation | LX2020 | Lexeo Therapeutics | Industry | PKP2 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh10 | Starting dose: 2.0E13 gc/kg | Phase2, Phase1 | Recruiting | Active | 2023-10-25 | 2027-02 | 2025-01-23 | 18 Years - 65 Years | 10 | 5 | United States | Initial clinical data expected late Q1/ early Q2 of 2025 |
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| NCT06818838 | Gaucher Disease Type 1 | LY-M001 | Lingyi Biotech Co., Ltd. | Industry | GBA1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAV8 | Dose 1: Starting dose: 1.5E13 vg/kg | Dose 2: High dose: 3.0E13 vg/kg | Dose 3: Backdose: 5E12 vg/kg | Phase2, Phase1 | Recruiting | Active | 2025-01-24 | 2031-07-30 | 2025-02-13 | 18 Years - 60 Years | 18 | 1 | China | IND granted in January 2024 |
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| NCT01494805 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | RAAV.sFlt-1 | Lions Eye Institute, Perth, Western Australia | Other | Soluble VEGFR1 | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Subretinal | Viral vector | Viral transduction | AAV | Dose 1: 1E10 vg (n=3) | Dose 2: 1E11 vg (n=3) | Dose 3: 1E11 vg (n=21) | Phase2, Phase1 | Completed | Inactive | 2011-12-14 | 2017-08 | 2017-09-01 | >= 55 Years | 40 | 1 | Australia | Product was well tolerated, unable to determine efficacy |
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| NCT04581785 | Methylmalonic Acidemia | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | LB-001 | LogicBio Therapeutics, Inc | Industry | MMUT | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Hepatocyte | Viral transduction | AAV-LK03 | Dose 1: 5E13 vg/kg | Dose 2: 1E14 vg/kg | Phase2, Phase1 | Terminated | Inactive | 2020-10-02 | 2023-01-10 | 2024-02-23 | 6 Months - 12 Years | 4 | 4 | United States | Program was terminated 4/25/23 due to low likelihood of clinical benefit |
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| NCT05040217 | Alzheimer's Disease, Mild Cognitive Impairment | AAV2-BDNF | Mark Tuszynski | Other | BDNF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal | Viral vector | Entorhinal cortex & hippocampus | Viral transduction | AAV2 | Dose 1: 3E11 vg/mL | Dose 2: 1E12 vg/mL | Phase1 | Recruiting | Active | 2021-06-21 | 2027-10-01 | 2024-08-27 | 50 Years - 80 Years | 12 | 2 | United States | US6683058B1; US20030124095A1; WO2023196575A1 | Surgical treatment of the AD patients will be complete in December 2024, and the MCI cohort is expected be completed in 2025 |
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| NCT04774536 | Sickle Cell Disease | CRISPR_SCD001 | Mark Walters, MD | Other | HBB | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Electroporation | none | Cas9 mRNA | Transduced CD34+ cells (range: 3 - 20E6 cells/kg) | Phase2, Phase1 | Recruiting | Active | 2021-02-17 | 2029-03-01 | 2024-09-20 | 12 Years - 35 Years | 9 | 2 | United States |
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| NCT03818763 | Hemophilia A | Pleightlet | Medical College of Wisconsin | Other | F8 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | Megakaryocytes | Viral transduction | VSV-G | Transduced CD34+ cells (not to exceed 20ml/kg body weight) | Phase1 | Recruiting | Active | 2019-01-15 | 2033-05-01 | 2025-02-07 | >= 18 Years | 5 | 1 | United States | WO2014066663A1 | First patient enrolled in March 2022, only 2 patients dosed so far 8/16/24 |
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| NCT04240314 | Duchenne Muscular Dystrophy (DMD) | AT702 | Megan Waldrop | Other | DMD (exon 2) | Gene transfer | In-vivo | Exon skipping/splice editor | Intravenous | Viral vector | Viral transduction | AAV9 | 3E13 vg/kg | Phase2, Phase1 | Active not recruiting | Inactive | 2020-01-22 | 2025-11-19 | 2023-02-09 | 6 Months - 13 Years | 3 | 1 | United States | Product was licensed to Audentes/Astellas Therapeutics in 2019, discontinuation was announced in April 2022 |
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| NCT05152823 | Spinal Muscular Atrophy with Respiratory Distress | AAV9-IGHMBP2 | Megan Waldrop | Other | IGHMBP2 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | Undisclosed | Phase2, Phase1 | Enrolling by invitation | Active | 2021-11-25 | 2028-11 | 2024-08-21 | 2 Months - 14 Years | 10 | 1 | United States | |||||||
| NCT03001310 | Achromatopsia | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | AAV-CNGB3 | MeiraGTx UK II Ltd | Industry | CNGB3 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Cone cells | Viral transduction | AAV2/8 | Dose 1: 0.1E12 vg/mL; 0.5 mL | Dose 2: 0.3E12 vg/mL; 0.5mL | Dose 3: 0.6E12 vg/mL; 0.5mL | Dose 4: 1.0E12 vg/mL; 0.5mL | Phase2, Phase1 | Completed | Active | 2016-11-22 | 2019-10-25 | 2023-03-08 | >= 3 Years | 23 | 2 |
Locations:United States + 1 more
United Kingdom, United States
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| NCT03758404 | Achromatopsia | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | AAV-CNGA3 | MeiraGTx UK II Ltd | Industry | CNGA3 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Cone cells | Viral transduction | AAV2/8 | 3 undisclosed dose levels | Phase2, Phase1 | Completed | Active | 2018-11-27 | 2021-06-10 | 2022-12-01 | >= 3 Years | 11 | 2 |
Locations:United States + 1 more
United Kingdom, United States
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| NCT02781480 | Leber Congenital Amaurosis | Orphan Drug Designation, Rare Pediatric Disease Designation | Cevaretigene ritoparvovec | MeiraGTx UK II Ltd | Industry | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/5 | Dose 1: 1.0E11 vg/mL | Dose 2: 3.0E11 vg/mL | Dose 3: 1.0E12 vg/mL | Phase2, Phase1 | Completed | Active | 2016-04-28 | 2018-12 | 2021-07-12 | >= 3 Years | 15 | 2 |
Locations:United States + 1 more
United Kingdom, United States
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WO2024079655A1 |
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| NCT05926765 | Grade 2 and 3 Radiation-Induced Late Xerostomia | Orphan Drug Designation, Regenerative Medicine Advanced Therapy | AAV-hAQP1 | MeiraGTx, LLC | Industry | AQP1 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Parotid | Viral vector | Viral transduction | AAV2 | Dose 1: 0.4E12 vg/gland | Dose 2: 1.2E12 vg/gland | Phase2 | Recruiting | Active | 2023-06-22 | 2025-07 | 2024-10-08 | >= 18 Years | 120 | 22 |
Locations:United States + 2 more
Canada, United Kingdom, United States
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WO2024079655A1; WO2024079657A1 | FDA in support of Phase II study as pivotal for BLA submission |
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| NCT05603312 | Parkinson Disease | AAV-GAD | MeiraGTx, LLC | Industry | GAD1/2 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal (subthalamic nucleus) | Viral vector | Viral transduction | AAV2 | Dose 1: 7.0E10 vg | Dose 2: 21E10 vg | Phase2, Phase1 | Completed | Active | 2022-10-12 | 2024-09-06 | 2024-10-08 | 25 Years - 85 Years | 14 | 6 | United States | WO2024079655A1; WO2021119615A1; US8017385B2 | Initiation of Phase III design discussions in H2 2024 |
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| NCT04833907 | Canavan Disease | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | MYR101 | Myrtelle Inc. | Industry | ASPA | Gene transfer | In-vivo | Functional gene replacement | Intracerebroventricular | Viral vector | Oligodendrocytes | Viral transduction | AAVOlig001 | 3.7E13 vg | Phase2, Phase1 | Recruiting | Active | 2021-03-24 | 2027-08-31 | 2025-01-09 | 3 Months - 60 Months | 24 | 1 | United States | US9636370B2; US10532110B2 | Selected for START program 7/2024 |
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| NCT06706427 | Bietti Crystalline Dystrophy | Orphan Drug Designation | NGGT001 | NGGT (Suzhou) Biotechnology Co., Ltd. | Industry | CYP4V2 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: 1.5E11 vg/eye | Dose 2: 3.0E11 vg/eye | Phase2, Phase1 | Active not recruiting | Active | 2024-11-14 | 2029-09-26 | 2024-11-26 | >= 18 Years | 12 | 2 | China |
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| NCT06332807 | Phenylketonuria (PKU) | Orphan Drug Designation | NGGT002 | NGGT INC. | Industry | PAH | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: 6.0E12 vg/kg | Dose 2: 1.0E13 vg/kg | Dose 3: 2.0E13 vg/kg | Phase2, Phase1 | Recruiting | Active | 2024-02-07 | 2030-12-30 | 2025-01-08 | 18 Years - 55 Years | 12 | 2 | United States | WO2024094044A1 | Recieved Orphan Drug Designation from the FDA in Jan 2023 |
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| NCT04945772 | Retinitis Pigmentosa, Retinal Dystrophies, Retinal Degeneration | Fast Track, Orphan Drug Designation | MCO-010 | Nanoscope Therapeutics Inc. | Industry | MCO | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Bipolar cells | Viral transduction | AAV2 | Dose 1: 0.9E11 gc/eye | Dose 2: 1.2E11 gc/eye | Phase2 | Completed | Active | 2021-06-15 | 2024-01-18 | 2024-03-22 | >= 18 Years | 27 | 6 |
Locations:United States + 1 more
Puerto Rico, United States
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BLA submission planned in Q1 2025 |
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| NCT05417126 | Stargardt Disease | Fast Track, Orphan Drug Designation | MCO-010 | Nanoscope Therapeutics Inc. | Industry | MCO | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Bipolar cells | Viral transduction | AAV2 | 1.2E11 gc/eye | Phase2 | Completed | Active | 2022-06-09 | 2023-09-28 | 2023-11-09 | >= 16 Years | 6 | 2 | United States | Phase III Study expected to begin Q1 2025 |
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| NCT01496040 | Leber Congenital Amaurosis | RAAV2/4.hRPE65 | Nantes University Hospital | Other | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/4 | Dose 1: 1.22E10 -2E10 vg | Dose 2: 3.27E10-4.8E10 vg | Phase2, Phase1 | Completed | Inactive | 2011-11-24 | 2014-08 | 2015-10-07 | 6 Years - 50 Years | 9 | 1 | France | Clinical study demonstrated safety, inconsistent findings on long-term efficacy |
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| NCT03952637 | GM1 Gangliosidosis | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | AXO-AAV-GM1 | National Human Genome Research Institute (NHGRI) | NIH | GLB1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 1.5E13 vg/kg | Dose 2: 4.5E13 vg/kg | Phase2, Phase1 | Recruiting | Active | 2019-05-15 | 2028-01-01 | 2024-11-27 | 6 Months - 12 Years | 45 | 1 | United States |
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| NCT06851767 | X-linked Severe Combined Immunodeficiency (XSCID) | BE-HSPC-IL2RG | National Institute of Allergy and Infectious Diseases (NIAID) | NIH | IL2RG (p.Q144X) | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Undisclosed | base editor | >5E6 cells/kg | Phase2, Phase1 | Not yet recruiting | Active | 2025-02-26 | 2034-12-31 | 2025-04-25 | 3 Years - 99 Years | 18 | 1 | United States |
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| NCT00394316 | X-Linked Chronic Granulomatous Disease | MFGS-gp91phox | National Institute of Allergy and Infectious Diseases (NIAID) | NIH | CYBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | MFGS RV | Transduced CD34+ cells | Early phase1 | Terminated | Inactive | 2006-10-31 | 2014-04-08 | 2018-07-05 | 3 Years - 55 Years | 3 | 1 | United States | Enrolled 3/6 planned patients, study was terminated in April 2014 |
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| NCT06253507 | Autosomal Recessive Chronic Granulomatous Disease (CGD) | PCHIM-p47 | National Institute of Allergy and Infectious Diseases (NIAID) | NIH | NCF1 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells, minimum dose: 3E6 CD34+ cells/kg | Phase2, Phase1 | Enrolling by invitation | Active | 2024-02-09 | 2027-03-31 | 2025-04-10 | 3 Years - 65 Years | 10 | 1 | United States |
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| NCT06325709 | X-Linked Chronic Granulomatous Disease | Base-edited autologous CD34+ cells | National Institute of Allergy and Infectious Diseases (NIAID) | NIH | CYBB c.676 C>T | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Electroporation | none | ABE8e-SpRY | Transduced CD34+ cells | Phase2, Phase1 | Suspended | Active | 2024-03-21 | 2032-12-31 | 2025-04-24 | 18 Years - 75 Years | 1 | 1 | United States | Trial stoppage rules triggered due to a serious adverse event |
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| NCT01306019 | X-linked Severe Combined Immunodeficiency (XSCID) | VSV-G pseudotyped CL20-i4-EF1α-hγc-OPT vector | National Institute of Allergy and Infectious Diseases (NIAID) | NIH | IL2RG | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | VSV-G | Transduced CD34+ cells (median dose: 6.73E6 cells/kg; range: 4.46-15.10E6 cells/kg) | Phase2, Phase1 | Recruiting | Inactive | 2011-02-26 | 2032-12-31 | 2025-04-20 | 2 Years - 50 Years | 40 | 1 | United States | Developed in partnership with St. Jude, and licensed to Mustang Bio, Mustang Bio discontinued this program |
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| NCT00028236 | Severe Combined Immunodeficiency (XSCID) | GALV MFGS-gc retrovirus | National Institute of Allergy and Infectious Diseases (NIAID) | NIH | IL2RG | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | MFGS/GALV | Dose: 30E6 transduced CD34+ cells/kg | Phase1 | Completed | Inactive | 2001-12-17 | 2011-07-25 | 2017-07-02 | 18 Months - 20 Years | 3 | 1 | United States | Enrolled 3/8 planned patients |
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| NCT00372320 | Parotid Salivary Dysfunction | AdhAQP1 | National Institute of Dental and Craniofacial Research (NIDCR) | NIH | AQP1 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Parotid | Viral vector | Ductal cells | Viral transduction | Ad5 | Dose 1: 4.8E7 vp/gland (n=3) | Dose 2: 2.9E8 vp/gland (n=3) | Dose 3: 1.3E9 vp/gland (n=3) | Dose 4: 5.8E9 vp/gland (n=2) | Phase1 | Completed | Inactive | 2006-09-02 | 2018-09-05 | 2018-09-11 | >= 18 Years | 17 | 1 | United States | Therapy was safe, but does not provide long-term benefit and redosing is not indicated with adenoviral vector. This program was transferred to MeiraGTx, who changed the vector to AAV2 |
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| NCT02362438 | Giant Axonal Neuropathy | ScAAV9/JeT-GAN | National Institute of Neurological Disorders and Stroke (NINDS) | NIH | GAN | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | Dose 1: 3.5E13 vg | Dose 2: 1.2E14 vg | Dose 3: 1.8E14 vg | Dose 4: 3.5E14 vg | Phase1 | Recruiting | Active | 2015-02-12 | 2035-04-01 | 2025-04-24 | 3 Years - 99 Years | 21 | 1 | United States | US20240102050A1 | Taysha discontinued development in September 2023, transferred development rights to NIH |
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| NCT00494195 | Limb-Girdle Muscular Dystrophy, Type 2D/R3 | RAAV1.tMCK.human-alpha-sarcoglycan | Nationwide Children's Hospital | Other | SGCA | Gene transfer | In-vivo | Functional gene replacement | Intramuscular (extensor digitorum brevis) | Viral vector | Muscle | Viral transduction | AAV1 | 3.25E11 vg | Phase1 | Completed | Inactive | 2007-06-27 | 2011-08 | 2013-02-05 | >= 5 Years | 6 | 1 | United States | Study drug was well tolerated by 3/3 subjects, protein was detectable in all subjects at 6 months post-injection. Clinical development of this program moved to Sarepta Therapeutics with optimized AAV capsid and modified intramuscular delivery |
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| NCT00428935 | Duchenne Muscular Dystrophy (DMD) | RAAV2.5-CMV-mini-Dystrophin | Nationwide Children's Hospital | Other | Micro-dystrophin | Gene transfer | In-vivo | Functional gene replacement | Intramuscular (bicep) | Viral vector | Viral transduction | AAV2.5 | Dose 1: 6E11 vg | Dose 2: 3.0E12 vg | Phase1 | Completed | Inactive | 2007-01-26 | 2010-07 | 2013-02-05 | 5 Years - 15 Years | 6 | 1 | United States | Study drug was well tolerated by 6/6 subjects, limited efficacy: protein was detectable in 2/6 subjects at d43 post administration | ||||||
| NCT01519349 | Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis | RAAV1.CMV.huFollistatin344 | Nationwide Children's Hospital | Other | FST (FS344) | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramuscular | Viral vector | Viral transduction | AAV1 | Dose 1: 2E11 vg/kg (single leg) | Dose 2: 3E11 vg/kg/quad | Dose 3: 6E11 vg/kg/quad | Phase1 | Completed | Inactive | 2012-01-23 | 2017-10 | 2023-10-02 | >= 18 Years | 15 | 1 | United States | Study drug was well tolerated, was not tested further |
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| NCT05984927 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | Fast Track | NG101 | Neuracle Genetics, Inc | Industry | Codon optimized aflibercept | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: 1E9 vg | Dose 2: 3E9 vg | Dose 3: 8E9 vg | Phase2, Phase1 | Recruiting | Active | 2023-07-24 | 2030-01 | 2025-03-27 | 50 Years - 89 Years | 18 | 4 |
Locations:United States + 1 more
Canada, United States
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WO2022010277A1; KR20230167312A; | Plans to expand trial into the USA |
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| NCT03562494 | Parkinson's Disease | Regenerative Medicine Advanced Therapy | VY-AADC01 | Neurocrine Biosciences | Industry | DDC | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal (striatum) | Viral vector | Striatum | Viral transduction | AAV2 | Dose 1: Total dose: 9E10 vg/200ul (4 sites) | Dose 2: Total dose: 3E11 vg/200ul (4 sites) | Dose 3: 3.6E12 vg | Phase1 | Completed | Inactive | 2018-05-16 | 2024-10-30 | 2024-12-10 | 40 Years - 75 Years | 14 | 11 | United States | Avigen transferred AAV-based product rights to Genzyme Corporation in December 2005, Genzyme launched a collaboration with Voyager Therapeutics in February 2015, then this collaboration was dissolved by Sanofi-Genzyme in October 2017, Voyager entered a collaborative agreement with Neurocrine Biosciences Inc. in March 2019, Neurocrine terminated this agreement effective August 2021, Voyager announced they were terminating this program in 2022 |
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| NCT05898620 | Rett Syndrome | Support for Clinical Trials Advancing Rare Disease Therapeutics | NGN-401 | Neurogene Inc. | Industry | MECP2 | Gene transfer | In-vivo | Functional gene replacement | Intracerebroventricular | Viral vector | Viral transduction | AAV9 | Dose 1: 1E15 vg | Dose 2: 3E15 vg (discontinued after patient death) | Phase2, Phase1 | Recruiting | Active | 2023-06-01 | 2029-10 | 2024-10-28 | 4 Years - 10 Years | 16 | 8 |
Locations:United States + 2 more
Australia, United Kingdom, United States
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US9415121B2 | Interim data expected 2H:2025; SAE reported for 3E15 vg dose, development will continue using 1E15 vg dose only |
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| NCT05228145 | Neuronal Ceroid Lipofuscinosis CLN5 | NGN-101 | Neurogene Inc. | Industry | CLN5 | Gene transfer | In-vivo | Functional gene replacement | Intracerebroventricular, intravitreal | Viral vector | Viral transduction | AAV9 | Undisclosed dose escalation, 3 levels | Phase2, Phase1 | Active not recruiting | Inactive | 2021-12-17 | 2028-11 | 2024-08-12 | 3 Years - 9 Years | 6 | 2 |
Locations:United States + 1 more
United Kingdom, United States
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RMAT application was denied by FDA, company discontinued the program |
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| NCT05820152 | Leber Hereditary Optic Neuropathy (LHON) | NFS-02 | Neurophth Therapeutics Inc | Other | ND1G3460A | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV2 | Dose 1: 5.0E7 vg, 0.05mL/eye | Dose 2: Starting dose: 1.5E8 vg, 0.05mL/eye | Dose 3: 5.0E8 vg, 0.05mL/eye | Dose 4: 1.5E9 vg, 0.05mL/eye | Phase2, Phase1 | Terminated | Inactive | 2023-04-06 | 2024-06-24 | 2024-09-20 | 18 Years - 75 Years | 11 | 4 |
Locations:United States + 1 more
China, United States
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WO2021115317A1 | Study terminated due to sponsor circumstances and external reasons |
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| NCT05293626 | Leber Hereditary Optic Neuropathy (LHON) | Orphan Drug Designation | OPVIKA | Neurophth Therapeutics Inc | Other | ND4G11778A | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV2 | Dose 1: Dose de-escalation: 0.5E9, 0.05mL | Dose 2: Starting dose: 1.5E9 vg, 0.05ml/eye | Dose 3: Intermediate dose: 3.0E9, 0.05mL | Dose 4: High dose: 4.5E9, 0.05mL | Phase2, Phase1 | Active not recruiting | Active | 2021-12-09 | 2029-12 | 2024-09-04 | 18 Years - 75 Years | 12 | 3 | United States | WO2020038352A1; US11332741B1; EP4382601A1 | Granted ODD by FDA and EMA, completed US enrollment February 2024 |
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| NCT03363165 | Peripheral Artery Disease | ENGENSIS | Northwestern University | Other | HGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramuscular (gastrocnemius) | Plasmid | None (naked plasmid) | 4mg (dose/leg/day) * 4 treatment days | Phase2 | Completed | Active | 2017-11-30 | 2023-09-05 | 2024-05-30 | >= 55 Years | 39 | 1 | United States | Same product as NL003 |
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| NCT04443907 | Sickle Cell Disease | OTQ923 | Novartis Pharmaceuticals | Industry | HBG1/HBG2 | Gene editing | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Electroporation | Cas9 RNP | Dose 1: Minimum dose: 3E6 CD34+ cells/kg | Dose 2: Dose range: 2.8-5.99E6 CD34+ cells/kg | Phase1 | Terminated | Inactive | 2020-04-29 | 2025-01-06 | 2025-02-11 | 2 Years - 40 Years | 4 | 3 | United States | Product was developed in collaboration with Intellia, Novartis opted to discontinue development in February 2023 |
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| NCT03374657 | Retinitis Pigmentosa | CPK850 | Novartis Pharmaceuticals | Industry | RLBP1 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV8 | Dose escalation with 4 levels, unknown concentrations | Phase2, Phase1 | Active not recruiting | Active | 2017-12-11 | 2026-05-11 | 2025-01-10 | 18 Years - 70 Years | 12 | 1 | Sweden | Novartis announced their decision to partner in July 2022 |
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| NCT05073133 | Spinal Muscular Atrophy | Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | ZOLGENSMA | Novartis Pharmaceuticals | Industry | SMN1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | 1.1E14 vg/kg | Phase4 | Completed | Approved | 2021-09-20 | 2023-08-08 | 2024-10-09 | <= 24 Months | 16 | 5 |
Locations:Argentina + 1 more
Argentina, Brazil
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FDA approved 5/24/19, price/treatment $2.1M |
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| NCT02132130 | Unilateral Severe to Profound Hearing Loss OR Bilateral Severe to Profound Hearing Loss | CGF166 | Novartis Pharmaceuticals | Industry | ATOH1 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intra-labyrinthine infusion | Viral vector | GFAP-positive cells | Viral transduction | Ad5 | Undisclosed dose escalation, 4 levels | Phase2, Phase1 | Completed | Inactive | 2014-05-05 | 2019-12-09 | 2021-10-08 | 18 Years - 75 Years | 22 | 4 | United States | Study completed, did not show efficacy | |||||
| NCT06018558 | Geographic Atrophy | OCU410 | Ocugen | Industry | RORA | Gene transfer | In-vivo | Overexpression of protective allele/gene | Subretinal | Viral vector | Viral transduction | AAV5 | Dose 1: 2.5E10 vg/mL | Dose 2: 5E10 vg/mL | Dose 3: 1.5E11 vg/mL | Phase2, Phase1 | Recruiting | Active | 2023-06-30 | 2025-09-23 | 2025-03-20 | >= 50 Years | 63 | 9 | United States | US20210123077A1 | Dosing complete in the Phase 2 clinical trial |
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| NCT05956626 | Stargardt Disease | Orphan Drug Designation | OCU410ST | Ocugen | Industry | RORA | Gene transfer | In-vivo | Overexpression of protective allele/gene | Subretinal | Viral vector | Viral transduction | AAV5 | Dose 1: 3.75E10 vg/mL | Dose 2: 7.5E10 vg/mL | Dose 3: 2.25E11 vg/mL (Maximum Tolerated Dose) | Phase2, Phase1 | Recruiting | Active | 2023-06-30 | 2025-10-28 | 2025-03-20 | 6 Years - 65 Years | 42 | 6 | United States | US20210123077A1 | Reached an alignment with FDA on Phase 2/3 pivotal confirmatory clinical trial |
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| NCT06388200 | Retinitis Pigmentosa | OCU400 | Ocugen | Industry | NR2E3 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Subretinal | Viral vector | Viral transduction | AAV5 | 2.5E10 vg/eye | Phase3 | Recruiting | Active | 2024-04-09 | 2026-10-30 | 2025-02-11 | >= 8 Years | 150 | 15 |
Locations:United States + 1 more
Canada, United States
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US20210123077A1 | OCU400 remains on track to meet 1H 2025 recruitment completion and potential BLA/MAA filings by mid-2026 |
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| NCT05616793 | LCA5 | Rare Pediatric Disease Designation | OPGx-LCA5 | Opus Genetics, Inc | Industry | LCA5 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2022-11-07 | 2028-01-30 | 2024-07-01 | >= 13 Years | 15 | 1 | United States | Early efficacy and safety data expected by Q3 2025 |
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| NCT04283227 | Metachromatic Leukodystrophy | Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | LENMELDY | Orchard Therapeutics | Industry | ARSA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | VSV-G | Dose 1: Minimum dose: 4.2E6 CD34+ cells/kg | Dose 2: Maximum dose: 30E6 CD34+ cells/kg | Phase3 | Active not recruiting | Approved | 2020-02-13 | 2031-03-31 | 2024-01-18 | 6 | 1 | Italy | FDA approval granted 3/18/24 |
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| NCT06149403 | Mucopolysaccharidosis Type I (Hurler Syndrome) | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | OTL-203 | Orchard Therapeutics | Industry | IDUA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Mean dose: 20.9E6 transduced CD34+ cells/kg | Phase3 | Active not recruiting | Active | 2023-11-17 | 2031-03 | 2025-03-27 | 28 Days - 30 Months | 41 | 6 |
Locations:United States + 3 more
Italy, Netherlands, United Kingdom, United States
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First patient randomized 2/2024 |
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| NCT05901480 | DFNB9 | OTOV101 | Otovia Therapeutics | Industry | OTOF | Gene transfer | In-vivo | Functional gene replacement | Intracochlear | Viral vector | Hair cell | Viral transduction | dual AAV | 5.6E11 vg/AAV, 1.2E12 vg total | Na | Recruiting | Active | 2023-05-09 | 2025-02-18 | 2024-05-08 | >= 1 Year | 25 | 23 | China |
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| NCT04903288 | Aromatic L-amino Acid Decarboxylase (AADC) Deficiency | Priority Review, Accelerated Approval, Orphan Drug Designation, Rare Pediatric Disease Designation | KEBILIDI, UPSTAZA | PTC Therapeutics | Industry | DDC | Gene transfer | In-vivo | Functional gene replacement | Intraparenchymal | Viral vector | Putamen | Viral transduction | AAV2 | Dose 1: Low dose: 1.8E11 vg/320ul dose (approved dose) | Dose 2: High dose: 2.37E11 vg (discontinued) | Phase2 | Active not recruiting | Approved | 2021-05-21 | 2028-04-30 | 2025-04-10 | 1 Year - 17 Years | 13 | 6 |
Locations:United States + 2 more
Israel, Taiwan, United States
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US-20190000991-A1; US-10898585-B2; US-20210236653-A1; US-11865188-B2; US-20240100186-A1 | FDA approval granted 11/13/24 |
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| NCT04119687 | Osteoarthritis, Knee | Regenerative Medicine Advanced Therapy | PCRX-201 | Pacira Pharmaceuticals, Inc | Industry | IL1RA | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarticular | Viral vector | Viral transduction | Ad5 | Dose 1: 1.4E10 gc/knee | Dose 2: 1.4E11 gc/knee | Dose 3: 1.4E12 gc/knee | Phase1 | Active not recruiting | Active | 2019-10-03 | 2026-11-28 | 2025-03-17 | 30 Years - 80 Years | 72 | 6 | United States | US20190376080A1 | FDA granted RMAT designation March 2024 |
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| NCT04747431 | Frontotemporal Dementia, FTD, FTD-GRN, C9orf72 | PBFT02 | Passage Bio, Inc. | Industry | GRN | Gene transfer | In-vivo | Functional gene replacement | Intracisterna magna | Viral vector | Viral transduction | AAV1 | Dose 1: 3.3E10 GC/g brain weight | Dose 2: 1.1E11 GC/g brain weight | Dose 3: 2.2E11 GC/g brain weight | Phase2, Phase1 | Recruiting | Active | 2021-02-02 | 2031-08 | 2025-01-24 | 35 Years - 75 Years | 25 | 7 |
Locations:United States + 3 more
Brazil, Canada, Portugal, United States
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WO2024081551A1 | 12-month data from Dose 1 and interim safety and biomarker data from Dose 2 expected in 2H 2025; plan to seek regulatory feedback on FTD-GRN pivotal trial design in 1H 2026 |
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| NCT04713475 | GM1 Gangliosidosis, Beta-Galactosidase-1 (GLB1) Deficiency | PBGM01 | Passage Bio, Inc. | Industry | GLB1 | Gene transfer | In-vivo | Functional gene replacement | Intracisterna magna | Viral vector | Viral transduction | AAVhu68 | Dose 1: 3.3E10 GC/g brain weight | Dose 2: 1.1E11 GC/g brain weight | Dose 3: 2.2E11 GC/g brain weight | Dose 4: Undisclosed dose 4 | Phase2, Phase1 | Active not recruiting | Active | 2021-01-04 | 2029-02 | 2024-05-28 | 1 Month - 24 Months | 26 | 9 |
Locations:United States + 4 more
Brazil, Canada, Turkey, United Kingdom, United States
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WO2024081551A1 | Out-licensed development to Gemma Biotherapeutics, uncertain development status |
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| NCT06749639 | Inherited Retinal Diseases, RDH12 Retinopathy | PUMCH-E101 | Peking Union Medical College Hospital | Other | RDH12 | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Undisclosed | Undisclosed | undisclosed | undisclosed | Undisclosed dose escalation, 2 levels | Early phase1 | Recruiting | Active | 2024-12-19 | 2030-01-04 | 2024-12-31 | 8 Years - 45 Years | 10 | 1 | China | ||||||
| NCT06392724 | Duchenne Muscular Dystrophy (DMD) | GEN6050X | Peking Union Medical College Hospital | Other | DMD | Gene editing | In-vivo | Exon skipping/splice editor | Intravenous | Viral vector | Viral transduction | dual AAV9 | eTAM | 5E13 vg/kg body weight | Early phase1 | Recruiting | Active | 2024-04-26 | 2027-12 | 2024-07-08 | 4 Years - 10 Years | 3 | 1 | China | CA3191505A1; WO2017215619A1 | Drug is being developed by GenAssist Ltd |
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| NCT05805007 | Retinitis Pigmentosa | ZVS203e | Peking University Third Hospital | Other | RHO | Gene editing | In-vivo | Gene inactivation | Subretinal | Viral vector | Viral transduction | AAV | Cas9 mRNA | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Early phase1 | Recruiting | Active | 2023-03-14 | 2026-04 | 2023-10-24 | >= 18 Years | 9 | 1 | China |
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| NCT06645197 | Amyotrophic Lateral Sclerosis (ALS) | SNUG01 | Peking University Third Hospital | Other | TRIM72 | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intrathecal | Viral vector | Viral transduction | AAV9 | 3 undisclosed dose levels | Early phase1 | Not yet recruiting | Active | 2024-10-11 | 2029-10-15 | 2024-10-16 | 18 Years - 80 Years | 7 | 5 | China | FDA cleared IND application | ||||||
| NCT04370054 | Hemophilia A | Giroctogogene fitelparvovec | Pfizer | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAV2/6 | 3E13 vg/kg | Phase3 | Active not recruiting | Active | 2020-04-21 | 2028-10-25 | 2025-04-15 | 18 Years - 64 Years | 76 | 37 |
Locations:United States + 16 more
Australia, Brazil, Canada, France, Germany, Greece, Italy, Japan, Korea, Republic of, Saudi Arabia, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States
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WO2017074526 | Pfizer terminated this program due to marketing considerations, Development rights are being returned to Sangamo |
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| NCT03861273 | Hemophilia B | Breakthrough Therapy, Orphan Drug Designation, Regenerative Medicine Advanced Therapy | BEQVEZ | Pfizer | Industry | F9R338L | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAVrh74 | 5E11 vg/kg | Phase3 | Active not recruiting | Approved (marketing discontinued) | 2019-03-01 | 2031-02-25 | 2025-04-17 | 18 Years - 65 Years | 51 | 60 |
Locations:United States + 16 more
Australia, Brazil, Canada, France, Germany, Greece, Italy, Japan, Korea, Republic of, Saudi Arabia, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States
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FDA approved 4/25/24, Price/treatment $3.5M; Pfizer will no longer market Beqvez |
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| NCT04281485 | Duchenne Muscular Dystrophy (DMD) | PF-06939926 | Pfizer | Industry | Mini-dystrophin | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | 2E14 vg/kg | Phase3 | Active not recruiting | Inactive | 2020-02-11 | 2039-04-15 | 2025-01-13 | 4 Years - 7 Years | 122 | 49 |
Locations:United States + 15 more
Australia, Belgium, Canada, France, Germany, Israel, Italy, Japan, Korea, Republic of, Russian Federation, Spain, Switzerland, Taiwan, United Kingdom, United States
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Phase 3 study did not meet its primary efficacy endpoint, discontinuation was announced Q3 2024 |
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| NCT06680232 | Chronic Hepatitis B | Fast Track | PBGENE-HBV | Precision BioSciences, Inc. | Industry | HBV DNA | Gene editing | In-vivo | Gene inactivation | Intravenous | LNP | Lipid encapsulation | LNP | ARCUS | Dose 1: 0.25mg/kg | Dose 2: Undisclosed medium/high doses | Phase1 | Recruiting | Active | 2024-10-24 | 2026-12 | 2025-02-10 | 18 Years - 70 Years | 45 | 3 |
Locations:Hong Kong + 3 more
Hong Kong, Moldova, New Zealand, Republic of
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IND cleared by FDA in March 2025 |
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| NCT04127578 | Parkinson Disease | Fast Track | PR001 | Prevail Therapeutics | Industry | GBA1 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intracisterna magna | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed dose 1 | Dose 2: Undisclosed dose 2 | Phase2, Phase1 | Recruiting | Active | 2019-10-14 | 2030-12-31 | 2025-04-10 | 35 Years - 80 Years | 20 | 13 |
Locations:United States + 2 more
Israel, Netherlands, United States
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| NCT04408625 | Frontotemporal Dementia | Fast Track, Orphan Drug Designation | LY3884963 | Prevail Therapeutics | Industry | GRN | Gene transfer | In-vivo | Functional gene replacement | Intracisterna magna | Viral vector | Viral transduction | AAV9 | Dose 1: 2.1E13 vg | Dose 2: 4.2E13 vg | Phase2, Phase1 | Recruiting | Active | 2020-05-21 | 2030-04-30 | 2025-03-18 | 30 Years - 85 Years | 30 | 11 |
Locations:United States + 5 more
Australia, Belgium, France, Spain, United Kingdom, United States
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US20210261981A1; WO2022082017A2 |
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| NCT04411654 | Gaucher Disease, Type 2 | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | LY3884961 | Prevail Therapeutics | Industry | GBA1 | Gene transfer | In-vivo | Functional gene replacement | Intracisterna magna | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Phase2, Phase1 | Active not recruiting | Active | 2020-05-11 | 2028-05 | 2025-02-13 | 0 Months - 24 Months | 15 | 5 |
Locations:United States + 1 more
United Kingdom, United States
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WO2022082017A2; US20200318115A1; US11903985B2; US20220211871A1 |
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| NCT06559176 | Chronic Granulomatous Disease | Orphan Drug Designation, Rare Pediatric Disease Designation | PM359 | Prime Medicine, Inc. | Industry | NCF1 (c.75_76delGT) | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Electroporation | prime editor | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | Active | 2024-07-11 | 2030-02 | 2025-04-04 | >= 6 Years | 12 | 5 |
Locations:United States + 2 more
Canada, United Kingdom, United States
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US20230357766A1; US11795452B2; US20230220374A1 | Initial data from Phase 1/2 clinical trial of PM359 for p47phox CGD expected in 2025 |
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| NCT02651675 | Homozygous Familial Hypercholesterolemia (HoFH) | Orphan Drug Designation | RGX-501 | REGENXBIO Inc. | Industry | LDLR | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAV8 | Dose 1: 2.5E12 GC/kg | Dose 2: 7.5E12 GC/kg | Phase2, Phase1 | Terminated | Inactive | 2016-01-04 | 2020-11-27 | 2023-07-13 | >= 18 Years | 9 | 9 |
Locations:United States + 3 more
Canada, Italy, Netherlands, United States
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Terminated by Sponsor for business reasons |
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| NCT03566043 | Mucopolysaccharidosis Type II (Hunter Syndrome) | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | RGX-121 | REGENXBIO Inc. | Industry | IDS | Gene transfer | In-vivo | Functional gene replacement | Intracisterna magna | Viral vector | Viral transduction | AAV9 | Dose 1: 1.3E10 GC/g brain mass | Dose 2: 6.5E10 GC/g brain mass | Dose 3: 2.9E11 GC/g brain mass (pivotal dose) | Phase3, Phase2 | Active not recruiting | Active | 2018-05-01 | 2025-08 | 2025-01-28 | 4 Months - 5 Years | 48 | 5 |
Locations:United States + 1 more
Brazil, United States
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US20180036388A1; US20240108761A1 | Program is now being developed as part of a partnership with Nippon Shinyaku |
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| NCT05693142 | Duchenne Muscular Dystrophy (DMD) | RGX-202 | REGENXBIO Inc. | Industry | Micro-dystrophin | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: 1E14 GC/kg body weight | Dose 2: 2E14 GC/kg body weight (pivotal dose) | Phase3, Phase2 | Recruiting | Active | 2023-01-04 | 2028-08 | 2025-03-12 | >= 1 Year | 65 | 6 | United States | US20230270886A1 | Pivotal trial underway; BLA expected 2026 |
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| NCT03580083 | Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | RGX-111 | REGENXBIO Inc. | Industry | IDUA | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV2/9 | Dose 1: 1E10 GC/g brain mass | Dose 2: 5E10 GC/g brain mass | Phase2, Phase1 | Active not recruiting | Active | 2018-06-18 | 2024-10 | 2023-12-06 | >= 4 Months | 8 | 4 |
Locations:United States + 2 more
Brazil, Israel, United States
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REGENXBIO is partnering with Nippon Shinyaku to continue clinical development |
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| NCT06460844 | Retinitis Pigmentosa, Choroideremia | RTx-015 | Ray Therapeutics, Inc. | Industry | Codon optimized ChR-3M | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Viral transduction | AAV.7m8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase1 | Recruiting | Active | 2024-06-10 | 2026-05 | 2025-03-10 | >= 18 Years | 15 | 3 | United States |
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| NCT05788536 | Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF) | DB-OTO | Regeneron Pharmaceuticals | Industry | OTOF | Gene transfer | In-vivo | Functional gene replacement | Intracochlear | Viral vector | Hair cell | Viral transduction | dual AAV1 | Dose 1: 3E13 vg/ml (unilateral) | Dose 2: Undisclosed high dose (unilateral) | Dose 3: Undisclosed expansion dose (bilateral) | Phase2, Phase1 | Recruiting | Active | 2023-03-15 | 2031-04-19 | 2025-03-17 | <= 17 Years | 22 | 12 |
Locations:United States + 2 more
Spain, United Kingdom, United States
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Phase I/II interim data presented at ASGCT 2024 on 2 patients |
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| NCT06511349 | Type 1 Primary Hyperoxaluria | Orphan Drug Designation, Rare Pediatric Disease Designation | YOLT-203 | RenJi Hospital | Other | HAO1 | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Lipid encapsulation | LNP | YolCas12 | Dose 1: 0.3 mg/kg | Dose 2: 0.6 mg/kg | Dose 3: 1.0 mg/kg | Early phase1 | Recruiting | Active | 2024-07-04 | 2026-01-31 | 2024-12-19 | >= 2 Years | 7 | 1 | China | First patient dosed (8/5/24) |
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| NCT04525352 | Infantile Malignant Osteopetrosis | Fast Track | RP-L401 | Rocket Pharmaceuticals Inc. | Industry | TCIRG1 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase1 | Terminated | Inactive | 2020-08-11 | 2021-05-21 | 2022-07-13 | >= 1 Month | 1 | 1 | United States | Study terminated due to feasibility in December 2021, program returned to academic innovators |
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| NCT06422351 | Pyruvate Kinase Deficiency | Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy | RP-L301 | Rocket Pharmaceuticals Inc. | Industry | PKLR | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | PGK-coPRK-WPRE | Dose range: 2.3E6 - 6.5E6 transduced CD34+ cells/kg | Phase2 | Not yet recruiting | Active | 2024-05-15 | 2029-01 | 2024-05-22 | 8 Years - 55 Years | 10 | 3 |
Locations:United States + 1 more
Spain, United States
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| NCT04248439 | Fanconi Anemia Complementation Group A | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | RP-L102 | Rocket Pharmaceuticals Inc. | Industry | FANCA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Dose range: 2.0E5 - 4.1E6 transduced CD34+ cells/kg | Phase2 | Active not recruiting | Active | 2020-01-24 | 2026-05 | 2024-04-10 | >= 1 Year | 5 | 2 | United States | CA3093708A1; CA3106241A1; WO2020037249A1 | MAA accepted by EMA 4/2/24; Rolling BLA submission initiated November 2024 |
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| NCT04105166 | Pyruvate Kinase Deficiency | Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy | RP-L301 | Rocket Pharmaceuticals Inc. | Industry | PKLR | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | PGK-coPRK-WPRE | Dose range: 2.3E6 - 6.5E6 transduced CD34+ cells/kg | Phase1 | Active not recruiting | Active | 2019-09-24 | 2025-05 | 2024-02-05 | 8 Years - 50 Years | 5 | 3 |
Locations:United States + 1 more
Spain, United States
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CA3106241A1; WO2020037249A1 | Initiated Phase 2 study (NCT06422351) |
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| NCT05885412 | PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) | Fast Track, Orphan Drug Designation | RP-A601 | Rocket Pharmaceuticals Inc. | Industry | PKP2 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh.74 | Starting dose: 8E13 GC/kg | Phase1 | Recruiting | Active | 2023-05-22 | 2026-09 | 2024-10-04 | >= 18 Years | 9 | 3 | United States | Preliminary data from the Phase 1 study is expected in the first half of 2025 |
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| NCT03812263 | Leukocyte Adhesion Defect - Type I | KRESLADI | Rocket Pharmaceuticals Inc. | Industry | ITGB2 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | Chim.hCD18-LV | Dose 1: Dose range: 2.8-10E6 transduced CD34+ cells/kg | Dose 2: Min dose: 2.0E6 CD34+ cells/kg | Phase2, Phase1 | Completed | Active | 2019-01-18 | 2023-09-12 | 2023-11-15 | >= 3 Months | 9 | 3 |
Locations:United States + 2 more
Spain, United Kingdom, United States
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EP3830248A1; CA3106241A1; WO2020037249A1 | FDA review of limited additional CMC information ongoing for KRESLADI; approval anticipated in 2025 |
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| NCT06092034 | Danon Disease | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | RP-A501 | Rocket Pharmaceuticals Inc. | Industry | LAMP2B | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 6.7E13 GC/kg | Dose 2: 1.1E14 GC/kg (dose discontinued) | Phase2 | Active not recruiting | Active | 2023-10-11 | 2029-09 | 2024-09-25 | >= 8 Years | 12 | 6 |
Locations:United States + 3 more
Germany, Italy, United Kingdom, United States
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US10703797B2; US20210379201A1 | Phase II enrollment completed 09/2024 |
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| NCT02610582 | Achromatopsia | RAAV.hCNGA3 | STZ eyetrial | Other | CNGA3 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Cone cells | Viral transduction | AAV8 | 1E11 vg/dose | Phase2, Phase1 | Active not recruiting | Active | 2015-09-18 | 2027-06 | 2024-04-18 | >= 6 Years | 13 | 1 | Germany | US20180353619; WO2017144080A1; WO2016146669A1; WO2018010965A1 |
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| NCT04611503 | Retinitis Pigmentosa | RAAV.hPDE6A | STZ eyetrial | Other | PDE6A | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: 1E9 vg | Dose 2: 5E9 vg | Dose 3: 1E10 vg | Dose 4: 5E10 vg | Phase2, Phase1 | Active not recruiting | Active | 2020-05-20 | 2027-07 | 2024-04-18 | >= 18 Years | 9 | 1 | Germany |
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| NCT03432364 | Beta-Thalassemia Major | ST-400 | Sangamo Therapeutics | Industry | BCL11A | Gene editing | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Electroporation | ZFN mRNA | Dose 1: Minimum dose: 4.5E6 cells/kg | Dose 2: Max dose: 11.4E6 CD34+ cells/kg | Dose 3: Average dose: 7.3E6 CD34+ cells/kg | Phase2, Phase1 | Completed | Inactive | 2018-02-01 | 2022-11-17 | 2023-12-14 | 18 Years - 40 Years | 5 | 6 | United States | Development discontinued by Sponsor November 2021 |
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| NCT00876863 | Alzheimer's Disease | CERE-110 | Sangamo Therapeutics | Industry | NGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal | Viral vector | Nucleus basalis of Meynert | Viral transduction | AAV2 | Dose 1: 1.2E10 vg (Phase I) | Dose 2: 5.8E10 vg (Phase I) | Dose 3: 1.2E11 vg (Phase I) | Dose 4: 2.0E11 vg (Phase II) | Phase2 | Completed | Inactive | 2009-04-03 | 2015-08-13 | 2020-12-21 | 55 Years - 80 Years | 49 | 10 | United States | Program was terminated in April 2015 |
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| NCT02702115 | Mucopolysaccharidosis Type I (Hurler Syndrome) | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | SB-318 | Sangamo Therapeutics | Industry | IDUA | Gene editing | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/6 | ZFN | Dose 1: 1E13 vg/kg | Dose 2: 5E13 vg/kg | Phase2, Phase1 | Terminated | Inactive | 2016-02-29 | 2021-11-03 | 2023-01-26 | >= 5 Years | 3 | 1 | United States | Only 3 subjects were enrolled prior to study termination, Sangamo announced they had stopped development in 2022 |
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| NCT02695160 | Hemophilia B | SB-FIX | Sangamo Therapeutics | Industry | F9 | Gene editing | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/6 | ZFN | 5E13 vg/kg | Phase1 | Terminated | Inactive | 2016-02-24 | 2021-04-19 | 2024-07-19 | >= 18 Years | 1 | 1 | United States | Only 1 subject was enrolled prior to study termination, Sangamo announced they has stopped development in 2022 |
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| NCT04046224 | Fabry Disease | Accelerated Approval, Orphan Drug Designation | ST-920 | Sangamo Therapeutics | Industry | GLA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/6 | Dose 1: 0.26E13 vg/kg | Dose 2: 0.53E13 vg/kg | Dose 3: 1.58E13 vg/kg | Dose 4: 2.63E13 vg/kg (expansion dose) | Phase2, Phase1 | Active not recruiting | Active | 2019-08-01 | 2025-09 | 2024-05-09 | >= 18 Years | 34 | 18 |
Locations:United States + 6 more
Australia, Canada, Germany, Italy, Taiwan, United Kingdom, United States
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US20200231989A1; US9877988B2; | Recent Type B meeting with FDA allowed ongoing Phase 1/2 study to serve as the primary basis for approval under Accelerated Approval Program, BLA submission H2 2025 |
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| NCT03041324 | Mucopolysaccharidosis Type II (Hunter Syndrome) | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | SB-913 | Sangamo Therapeutics | Industry | IDS | Gene editing | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/6 | ZFN | Dose 1: 5E12 vg/kg | Dose 2: 1E13 vg/kg | Dose 3: 5E13 vg/kg | Phase2, Phase1 | Terminated | Inactive | 2017-01-13 | 2021-05-07 | 2022-10-25 | 5 Years - 65 Years | 9 | 5 | United States | Sangamo announced they had stopped development in 2022 |
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| NCT00985517 | Idiopathic Parkinson's Disease | CERE-120 | Sangamo Therapeutics | Industry | NRTN | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal | Viral vector | Putamen, substantia nigra | Viral transduction | AAV2 | Dose 1: Phase 1: 1.3E11 vg (n=6) | Dose 2: Phase 1: 5.4E11 vg (n=6) | Dose 3: Phase 2: 5.4E11 vg (n=38) | Dose 4: Phase 2: 1.0E12 vg (putamen) + 2.0E11 vg (substantia nigra) | Phase2, Phase1 | Completed | Inactive | 2009-09-25 | 2017-11-16 | 2020-04-16 | 35 Years - 70 Years | 57 | 11 | United States | Therapy was originally developed by Ceregene, Inc. Company was acquired by Sangamo Therapeutics in 2013 after CERE-120 failed its Phase 2b trial for Parkinson's disease |
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| NCT05972629 | Phenylketonuria (PKU) | SAR444836 | Sanofi | Industry | PAH | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV SNY001 | Undisclosed dose 1 | Phase2, Phase1 | Recruiting | Inactive | 2023-06-23 | 2027-07-31 | 2025-03-27 | 18 Years - 65 Years | 32 | 9 |
Locations:United States + 4 more
Argentina, Brazil, Israel, Turkey, United States
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Deprioritized by Sponsor |
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| NCT06844214 | Myotonic Dystrophy | SAR446268 | Sanofi | Industry | DMPK | Gene transfer | In-vivo | Gene inactivation | Intravenous | Viral vector | Viral transduction | AAV.SAN011 | 3 cohort dose escalation, undisclosed concentrations | Phase2, Phase1 | Not yet recruiting | Active | 2025-02-19 | 2030-02-28 | 2025-03-12 | 10 Years - 50 Years | 32 | 0 |
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| NCT04703842 | Congestive Heart Failure, Heart Failure With Reduced Ejection Fraction (HFrEF) | SRD-001 | Sardocor Corp. | Industry | SERCA2a | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarterial | Viral vector | Viral transduction | AAV1 | Dose 1: 3E13 vg | Dose 2: 4.5E13 vg | Phase2, Phase1 | Recruiting | Active | 2021-01-07 | 2028-12 | 2024-03-26 | 18 Years - 80 Years | 57 | 5 | United States | WO2011130552A2; WO2020176732A1 | Anticipated Phase 2b in 4Q2024-1Q2025 |
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| NCT06061549 | Heart Failure, Diastolic, Heart Failure With Preserved Ejection Fraction | Fast Track | SRD-001 | Sardocor Corp. | Industry | SERCA2a | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarterial | Viral vector | Viral transduction | AAV1 | 3E13 vg | Phase1 | Recruiting | Active | 2023-07-30 | 2029-08 | 2023-09-29 | >= 50 Years | 10 | 2 | United States | WO2011130552A2; WO2020176732A1 | First patients dosed Q1 2024, product/indication combo granted Fast Track designation |
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| NCT06224660 | DMD-Associated Dilated Cardiomyopathy | Orphan Drug Designation | SRD-001 | Sardocor Corp. | Industry | SERCA2a | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarterial | Viral vector | Viral transduction | AAV1 | Dose 1: 1E13 vg | Dose 2: 3E13 vg | Phase1 | Recruiting | Active | 2024-01-11 | 2030-10 | 2025-02-27 | >= 18 Years | 12 | 3 | United States | Anticipated Phase 2b in 2H2025 |
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| NCT06246513 | Limb-Girdle Muscular Dystrophy, Type 2E/R4 | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | SRP-9003 | Sarepta Therapeutics, Inc. | Industry | SGCB | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh74 | Dose 1: 1.85E13 vg/kg (n=3) | Dose 2: 5E13 vg/kg | Dose 3: 7.41E13 vg/kg (n=3) | Phase3 | Active not recruiting | Active | 2024-01-30 | 2029-11-30 | 2025-04-06 | >= 4 Years | 17 | 5 |
Locations:United States + 1 more
United Kingdom, United States
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US20230241252A1; WO2021257595A1; EP4219726A1 | Data from EMERGENE are expected in the first half of 2025; BLA submission anticipated in 2025 |
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| NCT01976091 | Limb-Girdle Muscular Dystrophy, Type 2D/R3 | SRP-9004 | Sarepta Therapeutics, Inc. | Industry | SGCA | Gene transfer | In-vivo | Functional gene replacement | Isolated limb infusion | Viral vector | Viral transduction | AAVrh74 | Dose 1: 1E12 vg/kg/limb | Dose 2: 3E12 vg/kg/limb | Phase2, Phase1 | Completed | Active | 2013-07-24 | 2019-03-14 | 2023-06-15 | >= 7 Years | 6 | 0 | US20220370639A1; WO2021257595A1 | Phase 1b study initiating in December 2024 (NCT06747273) |
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| NCT06747273 | Limb-Girdle Muscular Dystrophy, Type 2D/R3 | SRP-9004 | Sarepta Therapeutics, Inc. | Industry | SGCA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh74 | Dose 1: Phase 1/2 study: 1E12 vg/kg/single limb (n=1) | Dose 2: Phase 1/2 study: 1E12 vg/kg/limb (n=3), total dose: 2E12 vg/kg | Dose 3: Phase 1/2 study: 3E12 vg/kg/limb (n=2), total dose: 6E12 vg/kg | Phase1 | Enrolling by invitation | Active | 2024-12-18 | 2030-03-29 | 2025-03-03 | >= 4 Years | 4 | 2 | United States | Enrollment and dosing is complete |
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| NCT05906251 | Limb Girdle Muscular Dystrophy, Type 2B/R2 | SRP-6004 | Sarepta Therapeutics, Inc. | Industry | DYSF | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | dual AAVrh74 | Dose 1: 2E12 vg | Dose 2: 6E12 vg | Phase1 | Active not recruiting | Active | 2023-06-07 | 2028-08-31 | 2024-08-20 | 18 Years - 50 Years | 2 | 1 | United States | WO2021257595A1 |
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| NCT05881408 | Duchenne Muscular Dystrophy (DMD) | Priority Review, Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | ELEVIDYS | Sarepta Therapeutics, Inc. | Industry | Micro-dystrophin | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravenous | Viral vector | Cardiac and skeletal muscle | Viral transduction | AAVrh74 | Dose 1: For patients < 70kg: 1.33E14 vg/kg | Dose 2: For patients >70kg: 9.3E15 vg | Phase3 | Recruiting | Approved | 2023-05-19 | 2028-06-30 | 2025-04-15 | 148 | 43 |
Locations:United States + 13 more
Australia, Belgium, Germany, Hong Kong, Israel, Italy, Japan, Korea, Republic of, Spain, Sweden, Taiwan, United Kingdom, United States
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US20230173101A1 | First approval was June 2023, expanded indication to all DMD patients regardless of ambulatory status or age on 6/20/24 |
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| NCT06370351 | OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders, Deafness, Hearing Loss | SENS-501 | Sensorion | Industry | OTOF | Gene transfer | In-vivo | Functional gene replacement | Intracochlear | Viral vector | Viral transduction | dual AAV | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Dose 3: Undisclosed expansion dose | Phase2, Phase1 | Recruiting | Active | 2024-04-09 | 2031-07 | 2024-09-26 | 6 Months - 31 Months | 12 | 2 |
Locations:Australia + 1 more
Australia, France
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First patient dosed Q3 2024 |
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| NCT06465550 | Beta-Thalassemia Major | BD211 | Shanghai BDgene Co., Ltd. | Industry | HBB | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells (> 5E6 cells/kg) | Phase1 | Recruiting | Active | 2024-06-12 | 2026-12 | 2024-06-24 | 3 Years - 35 Years | 9 | 3 | China | WO2023130911A1 | |||||
| NCT06474442 | Herpes Simplex Virus Type I Stromal Keratitis | Orphan Drug Designation | BD111 | Shanghai BDgene Co., Ltd. | Industry | HSV genes | Gene editing | In-vivo | Gene excision | Intrastromal | Viral vector | Viral transduction | LV | SpCas9 mRNA | Dose 1: 1.25E6 TU/eye | Dose 2: 2.5E6 TU/eye | Dose 3: 5.0E6 TU/eye | Dose 4: 10E6 TU/eye | Phase2 | Not yet recruiting | Active | 2024-06-13 | 2026-12 | 2024-06-25 | 18 Years - 70 Years | 40 | 1 | China | WO2024011980A1 |
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| NCT06111638 | Hemophilia A | Orphan Drug Designation | BBM-H803 | Shanghai Belief-Delivery BioMed Co., Ltd | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV | Undisclosed dose 1 | Phase2, Phase1 | Recruiting | Active | 2023-10-27 | 2030-06-30 | 2024-07-23 | >= 18 Years | 12 | 1 | China | WO2022222869A1; WO2021180118A1 | First patient dosed January 2024 |
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| NCT05203679 | Hemophilia B | Orphan Drug Designation, Rare Pediatric Disease Designation | BBM-H901 | Shanghai Belief-Delivery BioMed Co., Ltd | Industry | F9 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV843 | Dose 1: 5E12 vg/kg (IIT and Phase 1 dose) | Dose 2: 1E13 vg/kg | Phase3 | Active not recruiting | Active | 2021-12-29 | 2028-06-30 | 2025-02-26 | >= 18 Years | 32 | 9 | China | WO2019241324A1; WO2022222869A1; WO2021180118A1 | Dosing completed of all Phase III subjects |
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| NCT06022744 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | LX109 | Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine | Other | Undisclosed | Undisclosed | In-vivo | Undisclosed | Intravitreal | Viral vector | Viral transduction | undisclosed | Dose 1: 7E9 vg/eye | Dose 2: 3.5E10 vg/eye | Na | Not yet recruiting | Active | 2023-08-28 | 2027-09-30 | 2023-09-05 | >= 50 Years | 12 | 0 |
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| NCT06641895 | Duchenne Muscular Dystrophy (DMD) | Orphan Drug Designation, Rare Pediatric Disease Designation | BBM-D101 | Shanghai Jiao Tong University School of Medicine | Other | Undisclosed | Gene transfer | In-vivo | Undisclosed | Intravenous | Viral vector | Undisclosed | Viral transduction | AAV | Undisclosed dose | Early phase1 | Recruiting | Active | 2024-10-08 | 2030-07-31 | 2025-03-25 | 4 Years - 8 Years | 6 | 1 | China | IND cleared January 2025 |
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| NCT05765981 | Aromatic L-amino Acid Decarboxylase (AADC) Deficiency | VGN-R09b (AADC + NTF) | Shanghai Jiao Tong University School of Medicine | Other | DDC | Gene transfer | In-vivo | Functional gene replacement | Intraparenchymal | Viral vector | Striatum | Viral transduction | AAV9 | Undisclosed single dose | Early phase1 | Recruiting | Active | 2023-01-18 | 2029-02-20 | 2023-03-13 | 24 Months - 7 Years | 6 | 1 | China | WO2023202637 | NMPA IND accepted 1/24/24; FDA IND accepted 7/26/24 | ||||
| NCT06141460 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | RRG001 | Shanghai Refreshgene Technology Co., Ltd. | Industry | Anti-VEGF | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Subretinal | Viral vector | Viral transduction | AAV | Dose 1: Undisclosed dose 1 | Dose 2: Undisclosed dose 2 | Dose 3: Undisclosed dose 3 | Dose 4: Undisclosed dose 4 | Dose 5: Dose range: 1E8 - 1E13 vg/eye | Phase2, Phase1 | Recruiting | Active | 2023-11-10 | 2030-12-31 | 2024-11-14 | >= 50 Years | 48 | 1 | China | WO2024002076A1 |
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| NCT06432140 | Aromatic L-amino Acid Decarboxylase (AADC) Deficiency | VGN-R09b | Shanghai Vitalgen BioPharma Co., Ltd. | Industry | DDC | Gene transfer | In-vivo | Functional gene replacement | Intraparenchymal | Viral vector | Putamen | Viral transduction | AAV9 | Dose 1: 6.0E11 vg | Dose 2: 1.28E12 vg | Phase1 | Not yet recruiting | Active | 2024-05-17 | 2030-09 | 2024-06-03 | 18 Months - 8 Years | 16 | 0 |
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| NCT06699108 | Bietti Crystalline Dystrophy | VGR-R01 | Shanghai Vitalgen BioPharma Co., Ltd. | Industry | CYP4V2 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/8 | Dose 1: 6.0E10 vg/eye | Dose 2: 1.2E11 vg/eye (expansion dose) | Dose 3: 2.0E11 vg/eye | Phase3 | Not yet recruiting | Active | 2024-11-19 | 2027-06-30 | 2024-11-21 | 18 Years - 69 Years | 45 | 1 | China | Phase I/II preliminary results presented at ASGCT 2024 |
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| NCT05441553 | Hemophilia B | Orphan Drug Designation | VGB-R04 | Shanghai Vitalgen BioPharma Co., Ltd. | Industry | F9 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Undisclosed dose (ranging from 4E11 - 2E12 vg/kg) | Phase2, Phase1 | Unknown | Active | 2022-06-15 | 2025-01 | 2022-07-01 | 18 Years - 65 Years | 26 | 1 | China | WO2023280323A1; WO2023072181A1 | Granted ODD in December 2021 |
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| NCT05694598 | Bietti Crystalline Dystrophy | VGR-R01 | Shanghai Vitalgen BioPharma Co., Ltd. | Industry | CYP4V2 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/8 | Dose 1: 6.0E10 vg/eye | Dose 2: 1.2E11 vg/eye (expansion dose) | Dose 3: 2.0E11 vg/eye | Phase1 | Not yet recruiting | Active | 2023-01-11 | 2025-09-01 | 2023-01-23 | 18 Years - 69 Years | 12 | 1 | China | WO2023284873A1; US20240018541A1 | Phase I/II preliminary results presented at ASGCT 2024 |
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| NCT06217861 | Glutaric Acidemia Type I | Orphan Drug Designation, Rare Pediatric Disease Designation | VGM-R02b | Shanghai Vitalgen BioPharma Co., Ltd. | Industry | GCDH | Gene transfer | In-vivo | Functional gene replacement | Intracerebroventricular | Viral vector | Viral transduction | AAV9 | Undisclosed dose | Phase1 | Recruiting | Active | 2023-12-12 | 2026-08 | 2024-05-17 | <= 6 Years | 12 | 1 | China | WO2023221942A1; WO2024017387A1 | Granted CTA approval by NMPA on 7/13/23 | ||||
| NCT06480461 | Parkinson's Disease | VGN-R09b | Shanghai Vitalgen BioPharma Co., Ltd. | Industry | DDC | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal | Viral vector | Striatum | Viral transduction | AAV9 | Dose 1: 8.0E11 vg | Dose 2: 1.6E12 vg | Dose 3: 3.2E12 vg | Phase2, Phase1 | Not yet recruiting | Active | 2024-06-14 | 2031-07-01 | 2024-06-28 | 40 Years - 75 Years | 39 | 0 | WO2024017387A1 |
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| NCT03217617 | X-linked Severe Combined Immunodeficiency (XSCID) | Ivlv-X1 lentiviral vector | Shenzhen Geno-Immune Medical Institute | Other | IL2RG | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | LV | 1E9 vg/kg | Phase2, Phase1 | Recruiting | Active | 2017-07-03 | 2027-12-31 | 2024-07-08 | 1 Month - 1 Year | 10 | 2 | China |
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| NCT03645460 | Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) | ADA lentiviral vector | Shenzhen Geno-Immune Medical Institute | Other | ADA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | LV | 1E9 vg/kg | Na | Recruiting | Active | 2018-07-17 | 2027-12-31 | 2024-07-17 | >= 1 Month | 10 | 2 | China |
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| NCT02559830 | Metachromatic Leukodystrophy, Adrenoleukodystrophy | CD34+ hematopoietic stem cells transduced with ABCD1 or ARSA | Shenzhen Second People's Hospital | Other | ABCD1 or ARSA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | 2E6 transduced CD34+ cells/kg (maximum 20E6) | Phase2, Phase1 | Recruiting | Active | 2015-08-12 | 2025-10 | 2022-05-31 | 1 Year - 16 Years | 50 | 1 | China |
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| NCT03720418 | Parkinson Disease | OXB-102 | Sio Gene Therapies | Industry | TH-GCH1.DDC (tricistronic) | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal (putamen) | Viral vector | Striatal neurons | Viral transduction | EIAV | Dose 1: 1.9E7 TU | Dose 2: 4.0E7 TU | Dose 3: 1E8 TU | Phase2, Phase1 | Terminated | Inactive | 2018-10-16 | 2022-04-12 | 2022-05-03 | 30 Years - 70 Years | 6 | 3 |
Locations:France + 1 more
France, United Kingdom
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Axovant rebranded as Sio Gene Therapies, the company dissolved in 2023, returned rights to Oxford Biomedica which has shelved the program |
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| NCT05986864 | Neovascular (Wet) Age-Related Macular Degeneration (nAMD) | SKG0106 | Skyline Therapeutics (US) Inc. | Industry | Anti-VEGF | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV | Dose 1: 8E9 vg/eye | Dose 2: 2.4E10 vg/eye | Dose 3: 7.2E10 vg/eye | Phase2, Phase1 | Recruiting | Active | 2023-08-03 | 2026-01-30 | 2025-01-09 | >= 50 Years | 68 | 9 |
Locations:United States + 1 more
China, United States
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WO2023041015A1 | Therapy is also under development to treat diabetic macular edema |
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| NCT03368742 | Duchenne Muscular Dystrophy (DMD) | SGT-001 | Solid Biosciences Inc. | Industry | Micro-dystrophin | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 5E13 vg/kg | Dose 2: 2E14 vg/kg | Phase2, Phase1 | Active not recruiting | Inactive | 2017-12-05 | 2026-10-15 | 2025-04-16 | 4 Years - 17 Years | 12 | 2 | United States | In September 2022, Solid Biosciences announced they would be pausing activities for SGT-001; "No longer developing" as of 2024 Annual Report |
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| NCT06138639 | Duchenne Muscular Dystrophy (DMD) | Rare Pediatric Disease Designation | SGT-003 | Solid Biosciences Inc. | Industry | Micro-dystrophin | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravenous | Viral vector | Muscle cells | Viral transduction | AAV-SLB101 | 1E14 vg/kg | Phase2, Phase1 | Recruiting | Active | 2023-11-14 | 2031-05-06 | 2025-04-15 | 4 Years - 11 Years | 43 | 6 |
Locations:United States + 1 more
Canada, United States
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US20230183740A1; US20220031865A1; WO2021072197A1 | 20 subjects expected by Q4 2025 |
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| NCT05748873 | Retinitis Pigmentosa | SPVN06 | SparingVision | Industry | NXNL1 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2023-02-07 | 2029-03 | 2024-09-19 | >= 18 Years | 33 | 4 |
Locations:United States + 1 more
France, United States
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WO2024084075A1; EP3728610B1 | Enrollment of highest dose cohort is underway |
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| NCT06297486 | Hemophilia A | Dirloctogene samoparvovec | Spark Therapeutics, Inc. | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | LK03 | Dose 1: 5E11 vg/kg | Dose 2: 1E12 vg/kg | Dose 3: 1.5E12 vg/kg | Dose 4: 2E12 vg/kg | Phase3 | Withdrawn | Inactive | 2024-02-29 | 2035-09-04 | 2024-12-13 | >= 18 Years | 0 | 27 | United States | WO2019028192A1; US11168124B2; WO2019006390A1; US20220362408A1 | Product development discontinued, Study was withdrawn by Sponsor (no participants were enrolled) |
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| NCT06826612 | Huntington's Disease | SPK-10001 | Spark Therapeutics, Inc. | Industry | MiHTT | Gene transfer | In-vivo | Gene inactivation | Intraparenchymal (basal ganglia) | Viral vector | Viral transduction | AAV | Undisclosed dose escalation | Phase2, Phase1 | Recruiting | Active | 2025-02-03 | 2035-01-12 | 2025-03-20 | 25 Years - 65 Years | 53 | 1 | United States | |||||||
| NCT02341807 | Choroideremia | Breakthrough Therapy | SPK-7001 | Spark Therapeutics, Inc. | Industry | CHM | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: 5E10 vg/eye | Dose 2: 1E11 vg/eye | Phase2, Phase1 | Completed | Inactive | 2015-01-12 | 2022-10-12 | 2024-01-25 | >= 18 Years | 15 | 3 | United States | Roche acquired Spark in December 2019, announced they were halting this program in the 2021 annual report |
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| NCT00999609 | Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis | Breakthrough Therapy, Priority Review, Orphan Drug Designation, Rare Pediatric Disease Designation | LUXTURNA | Spark Therapeutics, Inc. | Industry | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | 1E11 vg/eye (0.3mL) | Phase3 | Active not recruiting | Approved | 2009-10-21 | 2030-01 | 2025-04-23 | >= 3 Years | 31 | 2 | United States | FDA approved 12/19/17, Price/treatment $850K/eye |
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| NCT03734588 | Hemophilia A | SPK-8016 | Spark Therapeutics, Inc. | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV | 5E11 vg/kg | Phase2, Phase1 | Completed | Inactive | 2018-11-06 | 2023-01-19 | 2024-02-23 | >= 18 Years | 4 | 11 | United States | Roche (maker of Hemlibra) acquired Spark in 2019, announced they would discontinue development of this program in Q2 2023 update | ||||||
| NCT04093349 | Glycogen Storage Disease Type 2 (Pompe Disease) | SPK-3006 | Spark Therapeutics, Inc. | Industry | GAA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV | Undisclosed dose escalation (unknown doses, unknown number of cohorts) | Phase2, Phase1 | Active not recruiting | Inactive | 2019-09-16 | 2032-04 | 2024-11-27 | >= 18 Years | 4 | 29 |
Locations:United States + 7 more
Canada, Denmark, France, Germany, Italy, Netherlands, United Kingdom, United States
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Roche acquired Spark in 2019, announced they were discontinuing this program mid 2024, only enrolled 4 patients |
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| NCT06526923 | Cystic Fibrosis | SP-101 | Spirovant Sciences, Inc. | Industry | CFTRΔR | Gene transfer | In-vivo | Functional gene replacement | Inhalational | Viral vector | Human airway epithelia | Viral transduction | AAV2.5T | Undisclosed dose | Phase2, Phase1 | Recruiting | Active | 2024-07-18 | 2026-12-31 | 2024-11-25 | 18 Years - 65 Years | 15 | 4 | United States | First patient dosed November 2024 |
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| NCT05164471 | Hemophilia B | FLT180a | Spur Therapeutics | Industry | F9 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAVS3 | Dose 1: 3.84E11 vg/kg | Dose 2: 6.40E11 vg/kg | Dose 3: 8.32E11 vg/kg | Dose 4: 1.28E12 vg/kg | Phase2, Phase1 | Terminated | Inactive | 2021-11-22 | 2023-05-31 | 2023-07-20 | 18 Years - 65 Years | 6 | 7 |
Locations:United States + 1 more
United Kingdom, United States
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Study was terminated early in Phase 1/2 trial, Freeline was acquired by Syncona |
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| NCT05324943 | Gaucher Disease, Type 1 | Regenerative Medicine Advanced Therapy | FLT201 | Spur Therapeutics | Industry | GBA1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVS3 | 4.5E11 vg/kg | Phase1 | Active not recruiting | Active | 2022-02-28 | 2025-01-31 | 2024-08-07 | >= 18 Years | 18 | 10 |
Locations:United States + 5 more
Brazil, Germany, Israel, Spain, United Kingdom, United States
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EP4118200A2; US20220387558A1; US20220162642A1; | Phase 3 trial planned in 2H2025 |
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| NCT04040049 | Fabry Disease | FLT190 | Spur Therapeutics | Industry | GLA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVS3 | 7.5E11 vg/kg | Phase2, Phase1 | Terminated | Inactive | 2019-07-26 | 2023-05-02 | 2023-06-05 | >= 18 Years | 3 | 13 |
Locations:United States + 6 more
Austria, Canada, Germany, Italy, Norway, United Kingdom, United States
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Development cancelled to prioritize other programs |
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| NCT06506461 | Sickle Cell Disease | Gene-modified CD34+ cells | St. Jude Children's Research Hospital | Other | BCL11A | Gene editing | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Electroporation | SpCas9 mRNA | Transduced CD34+ cells | Phase1 | Recruiting | Active | 2024-06-14 | 2032-12 | 2025-03-25 | 18 Years - 24 Years | 25 | 1 | United States | ||||||
| NCT00979238 | Hemophilia B | ScAAV2/8-LP1-hFIXco | St. Jude Children's Research Hospital | Other | F9 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/8 | Dose 1: 2E11 vg/kg (n=2) | Dose 2: 6E11 vg/kg (n=2) | Dose 3: 2E12 vg/kg (n=6) | Dose 4: Extension phase with new process: 2E12 vg/kg (n=2) | Dose 5: Extension phase with new process: 5E12 vg/kg (n=2) | Phase1 | Active not recruiting | Inactive | 2009-09-16 | 2032-12-31 | 2024-11-22 | >= 18 Years | 14 | 8 |
Locations:United States + 1 more
United Kingdom, United States
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Was the first Hemophilia B trial to show sustained efficacy |
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| NCT06293729 | Refractory Hypercholesterolemia, Familial Hypercholesterolemia | NGGT006 | Suzhou Municipal Hospital | Other | LDLR | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravenous | Viral vector | Viral transduction | AAV | Dose 1: 7.5E12 vg/kg | Dose 2: 1.5E13 vg/kg | Dose 3: 3E13 vg/kg | Early phase1 | Not yet recruiting | Active | 2024-02-27 | 2029-03-01 | 2024-04-17 | 18 Years - 55 Years | 9 | 0 | CN117887723A |
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| NCT05394064 | Adrenomyeloneuropathy (AMN) | SBT101 | SwanBio Therapeutics, Inc. | Industry | ABCD1 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | Dose 1: 1.0E14 vg | Dose 2: 3.0E14 vg | Phase2, Phase1 | Recruiting | Active | 2022-05-16 | 2029-03-30 | 2023-09-13 | 18 Years - 65 Years | 16 | 2 |
Locations:United States + 1 more
Netherlands, United States
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Sponsor expects to complete dosing in the Phase 1/2 PROPEL study of SBT101 early 2025 and report initial safety data from the high-dose cohort in the first half of 2025 |
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| NCT06152237 | Rett Syndrome | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | TSHA-102 | Taysha Gene Therapies, Inc. | Industry | MiniMECP2 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | scAAV9 | Dose 1: 5.7E14 vg (n=4) | Dose 2: 1E15 vg (n=6) | Phase2, Phase1 | Recruiting | Active | 2023-11-21 | 2031-11-02 | 2024-11-12 | 5 Years - 8 Years | 20 | 10 |
Locations:United States + 2 more
Canada, United Kingdom, United States
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Update on pivotal trial design expected in 1H2025 |
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| NCT05606614 | Rett Syndrome | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | TSHA-102 | Taysha Gene Therapies, Inc. | Industry | MiniMECP2 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | scAAV9 | Dose 1: 5.7E14 vg | Dose 2: 1E15 vg | Phase2, Phase1 | Recruiting | Active | 2022-10-28 | 2032-01-05 | 2024-11-13 | >= 12 Years | 18 | 6 |
Locations:United States + 1 more
Canada, United States
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US20240191254A1; US20240102050A1; US20190328804A1 | Clinical data expected H1 2025 |
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| NCT06228924 | Arrhythmogenic Right Ventricular Cardiomyopathy | TN-401 | Tenaya Therapeutics | Industry | PKP2 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 3E13 vg/kg | Dose 2: 6E13 vg/kg | Phase1 | Recruiting | Active | 2024-01-18 | 2029-10-01 | 2025-02-06 | 18 Years - 65 Years | 15 | 7 | United States | WO2022076648A1 | First patient dosed November 2024, initial data expected 2H2025 |
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| NCT05836259 | Hypertrophic Cardiomyopathy | TN-201 | Tenaya Therapeutics | Industry | MYBPC3 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 3E13 vg/kg | Dose 2: 6E13 vg/kg | Phase2, Phase1 | Recruiting | Active | 2023-04-18 | 2032-08 | 2024-11-15 | 18 Years - 75 Years | 30 | 10 | United States | WO2021163357A2; US20240084327A1 | Expects to Complete Enrollment of Cohort 2 in 1H25 and to Report Initial Data in 2H25 |
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| NCT04669535 | Tay-Sachs Disease, Sandhoff Disease | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | AXO-AAV-GM2 | Terence Flotte | Other | HEXA/HEXB | Gene transfer | In-vivo | Functional gene replacement | Intracisternal/intrathecal | Viral vector | Viral transduction | dual AAVrh.8 | Dose 1: 1E14 vg | Dose 2: Undisclosed 3-part dose escalation | Phase1 | Completed | Active | 2020-11-23 | 2024-12-16 | 2025-01-09 | 6 Months - 12 Years | 9 | 2 | United States | Sio Gene Therapies licensed this program from the University of Massachusetts Medical School in 2018, Sio terminated this agreement in 2022, Sio dissolved in 2023 |
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| NCT05791864 | Neuronal Ceroid Lipofuscinosis Type 2 | TTX-381 | Tern Therapeutics, LLC | Industry | TPP1 | Gene transfer | In-vivo | Functional gene replacement | Subretinal, intracisternal (RGX-181) | Viral vector | Viral transduction | AAV9 | Dose 1: 2E10 gc/eye | Dose 2: 6E10 gc/eye | Phase2, Phase1 | Recruiting | Active | 2023-03-17 | 2030-07-30 | 2025-02-13 | 12 Months - 84 Months | 16 | 1 | United Kingdom | Tern Therapeutics acquired RGX-381 and RGX-181 from REGENXBIO August 2024; Completed enrollment of cohort 2, selected expansion dose |
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| NCT06107400 | Alpha Thalassemia Hemoglobin H Constant Spring | RM-004 | The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army | Other | (HBA2):c.427T>C (p.Ter143Gln) | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Lipid encapsulation | LNP | SpRY-CBE | Transduced CD34+ cells | Early phase1 | Recruiting | Active | 2023-10-25 | 2026-10-31 | 2024-06-03 | 12 Years - 35 Years | 5 | 1 | China | WO2023193616A1 | First patient dosed (5/27/24) | |||
| NCT02716246 | Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome) | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | UX111 | Ultragenyx Pharmaceutical Inc | Industry | SGSH | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 0.5E13 vg/kg | Dose 2: 1.0E13 vg/kg | Dose 3: 3.0E13 vg/kg | Phase3, Phase2 | Recruiting | Active | 2016-03-17 | 2027-07 | 2025-04-15 | 36 | 5 |
Locations:United States + 2 more
Australia, Spain, United States
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US20220347298A1 | BLA filed under Accelerated Approval, PDUFA date 8/18/25 |
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| NCT04884815 | Wilson Disease | UX701 | Ultragenyx Pharmaceutical Inc | Industry | ATP7B | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 5.0E12 GC/kg | Dose 2: 1.0E13 GC/kg | Dose 3: 2.0E13 GC/kg | Dose 4: Undisclosed dose 4 | Phase2, Phase1 | Active not recruiting | Active | 2021-05-07 | 2031-11 | 2024-11-27 | >= 18 Years | 78 | 16 |
Locations:United States + 4 more
Canada, Portugal, Spain, United Kingdom, United States
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EP3906066B1 | Cohort 4 (moderately increased dose) enrollment expected to complete H2 2025 |
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| NCT05345171 | Ornithine Transcarbamylase (OTC) Deficiency | DTX301 | Ultragenyx Pharmaceutical Inc | Industry | OTC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | 1.0E13 GC/kg | Phase3 | Active not recruiting | Active | 2022-04-18 | 2028-12 | 2025-03-11 | >= 12 Years | 50 | 25 |
Locations:United States + 11 more
Argentina, Brazil, Canada, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, United Kingdom, United States
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EP4038194A1 | Enrollment expected to be completed early 2025 |
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| NCT05139316 | Glycogen Storage Disease Type Ia | DTX401 | Ultragenyx Pharmaceutical Inc | Industry | G6PC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | 1.0E13 GC/kg | Phase3 | Active not recruiting | Active | 2021-07-14 | 2026-02 | 2025-02-21 | >= 8 Years | 49 | 20 |
Locations:United States + 8 more
Brazil, Canada, Denmark, Germany, Italy, Japan, Netherlands, Spain, United States
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US20220017922A1 | BLA filing expected mid 2025 |
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| NCT02618915 | Hemophilia B | Fast Track, Orphan Drug Designation | DTX101 | Ultragenyx Pharmaceutical Inc | Industry | F9 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh10 | Dose 1: 1.6E12 GC/kg | Dose 2: 5.0E12 GC/kg | Phase2, Phase1 | Terminated | Inactive | 2015-11-23 | 2017-10-18 | 2018-11-14 | >= 18 Years | 6 | 9 |
Locations:United States + 2 more
Bulgaria, United Kingdom, United States
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DTX101 did not demonstrate sufficient efficacy, program was terminated in 2017 |
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| NCT06270316 | Fabry Disease | Fast Track, Orphan Drug Designation | AMT-191 | UniQure Biopharma B.V. | Industry | GLA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV5 | Dose 1: 6.0E13 gc/kg | Dose 2: 3.0E14 gc/kg | Phase2, Phase1 | Recruiting | Active | 2023-12-19 | 2027-04-29 | 2025-04-11 | 18 Years - 50 Years | 12 | 8 | United States | WO2015060722A1; WO2020104424A1 | First patient dosed 8/15/24; IDMC recommended proceeding with enrollment of 2nd cohort | ||||
| NCT03300453 | Sanfilippo Syndrome B | AMT-110 | UniQure Biopharma B.V. | Industry | NAGLU | Gene transfer | In-vivo | Functional gene replacement | Intraparenchymal | Viral vector | Viral transduction | AAV2/5 | Total: 4E12 vg (960uL) ; 16 sites: 2.4E11 vg/site | Phase2, Phase1 | Completed | Inactive | 2016-06-15 | 2019-11-27 | 2019-12-02 | 18 Months - 60 Months | 4 | 1 | France | UniQure announced discontinuation in November 2016 |
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| NCT05243017 | Huntington's Disease | Regenerative Medicine Advanced Therapy | AMT-130 | UniQure Biopharma B.V. | Industry | MiHTT | Gene transfer | In-vivo | MiRNA knockdown of mutant/aberrant gene | Intraparenchymal | Viral vector | Viral transduction | AAV5 | Dose 1: 6E12 gc/subject | Dose 2: 6E13 gc/subject | Phase2, Phase1 | Active not recruiting | Active | 2021-11-01 | 2029-10-07 | 2025-03-10 | 25 Years - 65 Years | 14 | 4 |
Locations:Poland + 1 more
Poland, United Kingdom
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US20230119344A1; US20220213482A1; US20210371862A1 | FDA granted RMAT designation Q2 2024, FDA allowing Sponsor to seek Accelerated Approval |
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| NCT06063850 | Mesial Temporal Lobe Epilepsy | AMT-260 | UniQure Biopharma B.V. | Industry | MiGRIK2 | Gene transfer | In-vivo | MiRNA knockdown of mutant/aberrant gene | Intraparenchymal | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2023-09-01 | 2027-06-30 | 2024-12-03 | 18 Years - 65 Years | 12 | 12 | United States | First patient dosed 11/21/24 |
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| NCT06100276 | Amyotrophic Lateral Sclerosis (ALS) | AMT-162 | UniQure Biopharma B.V. | Industry | MiSOD1 | Gene transfer | In-vivo | MiRNA knockdown of mutant/aberrant gene | Intrathecal | Viral vector | Viral transduction | AAVrh10 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed intermediate dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2023-10-20 | 2031-03-30 | 2024-10-15 | >= 18 Years | 20 | 10 | United States | First patient dosed October 2024; IDMC recommended proceeding with 2nd cohort |
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| NCT05092685 | Ornithine Transcarbamylase (OTC) Deficiency | Orphan Drug Designation | BGT-OTCD | University College, London | Other | OTC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV-LK03 | Dose 1: 6E11 vg/kg | Dose 2: 2E12 vg/kg | Dose 3: 6E12 vg/kg | Phase2, Phase1 | Recruiting | Active | 2021-09-28 | 2027-06-30 | 2023-11-07 | 0 Days - 16 Years | 12 | 1 | United Kingdom | US20220372512A1; US20230093183A1 | Granted ODD by FDA and EMA |
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| NCT00643747 | Leber Congenital Amaurosis-Type 2 | TgAAG76 | University College, London | Other | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: 1E11 vg | Dose 2: 1E12 vg | Phase2, Phase1 | Completed | Inactive | 2008-03-20 | 2014-12 | 2015-12-07 | 5 Years - 30 Years | 12 | 1 | United Kingdom | Vector was originally produced by Targeted Genetics Corporation, clinical trial showed a lack of sustained efficacy |
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| NCT03001830 | Hemophilia A | AAV2/8-HLP-FVIII-V3 | University College, London | Other | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/8 | Dose 1: 6E11 vg/kg | Dose 2: 2E12 vg/kg | Dose 3: 4E12 vg/kg | Dose 4: 6E12 vg/kg | Phase2, Phase1 | Active not recruiting | Active | 2016-12-09 | 2029-12 | 2025-03-05 | >= 18 Years | 14 | 4 |
Locations:United States + 1 more
United Kingdom, United States
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| NCT04601974 | Drug Resistant Epilepsy | Lenti-CAMK2A-KCNA1 | University College, London | Other | KCNA1 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intracranial | Viral vector | Viral transduction | LV | Undisclosed dose (single administration) | Phase2, Phase1 | Not yet recruiting | Active | 2020-10-06 | 2032-09 | 2023-05-15 | >= 18 Years | 10 | 0 | ||||||||
| NCT05432310 | Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) | Simoladagene autotemcel | University of California, Los Angeles | Other | ADA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells, minimum dose: Fresh (1E6 cells/kg); Cryopreserved (2E6 cells/kg) | Phase2, Phase1 | Recruiting | Active | 2022-06-04 | 2026-12-31 | 2025-04-03 | >= 1 Month | 20 | 1 | United States | Orchard terminated this program, returned the program to UCLA which administers the therapy under Compassionate Use |
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| NCT02234934 | X-Linked Chronic Granulomatous Disease | VSVG-PCCLChimGp91 | University of California, Los Angeles | Other | CYBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | VSV-G | Transduced CD34+ cells (6.5-32.6E6 cells/kg) | Phase2, Phase1 | Completed | Inactive | 2014-09-04 | 2024-12-01 | 2024-12-20 | >= 23 Months | 10 | 3 | United States | US20220378937A1 | Commercial rights are owned by Orchard, which has deprioritized the program |
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| NCT03897361 | Cystinosis | Orphan Drug Designation, Rare Pediatric Disease Designation | CTNS-RD-04 or CTNS-RD-04-LB | University of California, San Diego | Other | CTNS | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Min dose: 3E6 CD34+ cells/kg | Phase2, Phase1 | Completed | Active | 2019-02-06 | 2024-09-18 | 2025-03-20 | >= 18 Years | 6 | 1 | United States | US20240009247A1 | AVROBIO sold product to Novartis in May 2023, Novartis will sponsor any subsequent trials |
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| NCT03538899 | Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency | AProArt | University of California, San Francisco | Other | DCLRE1C | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | Active | 2018-05-03 | 2038-06 | 2025-01-13 | >= 2 Months | 25 | 1 | United States |
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| NCT02240407 | Glycogen Storage Disease Type 2 (Pompe Disease) | RAAV9-DES-hGAA | University of Florida | Other | GAA | Gene transfer | In-vivo | Functional gene replacement | Intramuscular (tibialis anterior) | Viral vector | Viral transduction | AAV2/9 | 4.6E13 vg/TA muscle | Phase1 | Completed | Active | 2014-09-11 | 2021-08-26 | 2022-04-05 | 18 Years - 50 Years | 2 | 1 | United States | US20220347297A1 |
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| NCT00976352 | Glycogen Storage Disease Type 2 (Pompe Disease) | AAV1-CMV-hGAA | University of Florida | Other | GAA | Gene transfer | In-vivo | Functional gene replacement | Intramuscular (diaphragm) | Viral vector | Viral transduction | AAV2/1 | Dose 1: 1E12 vg (n=3) | Dose 2: 5E12 vg (n=6) | Phase2, Phase1 | Completed | Inactive | 2009-07-13 | 2015-12 | 2018-09-14 | 2 Years - 18 Years | 9 | 1 | United States | Sponsors of this trial redesigned the drug product by changing the capsid (AAV2/1 -> AAV2/9) and promoter (CMV->DES), and site of administration (diaphragm -> tibialis anterior), updated trial record is NCT02240407 |
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| NCT05665166 | Mucopolysaccharidosis II | Orphan Drug Designation, Rare Pediatric Disease Designation | AVR-RD-05 | University of Manchester | Other | IDS | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | Inactive | 2022-12-07 | 2027-09 | 2024-10-02 | 3 Months - 22 Months | 5 | 1 | United Kingdom | AVROBIO supported this IIT, Program development was halted in July 2023 |
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| NCT04201405 | Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome) | OTL-201 | University of Manchester | Other | SGSH | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Dose range: 4.37 - 22.7E6 CD34+ cells/kg | Phase2, Phase1 | Active not recruiting | Active | 2019-12-05 | 2026-10-30 | 2025-03-30 | 3 Months - 24 Months | 5 | 1 | United Kingdom |
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| NCT00927134 | X-Linked Chronic Granulomatous Disease | Retroviral SF71-gp91phox transduced CD34+ cells | University of Zurich | Other | CYBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | SFFV | Transduced CD34+ cells (dose range: 3.6-5.1E6 cells/kg) | Phase2, Phase1 | Completed | Inactive | 2009-06-22 | 2011-09 | 2011-09-27 | 1 Year - 18 Years | 2 | 1 | Switzerland | Enrolled 2 patients, 2 patients enrolled under NCT00564759, same vector used in both |
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| NCT02317887 | X-Linked Retinoschisis | RS1 AAV Vector | VegaVect, Inc. | Industry | RS1 | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV8 | Dose 1: 1E9 vg/eye | Dose 2: 1E10 vg/eye | Dose 3: 1E11 vg/eye | Dose 4: < 3E11 vg/eye | Dose 5: < 6E11 vg/eye | Phase2, Phase1 | Completed | Active | 2014-12-16 | 2024-10-16 | 2025-02-14 | >= 18 Years | 12 | 1 | United States | US9873893B2; US20210290433A1; WO2019144077A1; US20180214577A1 |
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| NCT05477563 | Beta-Thalassemia, Sickle Cell Disease | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | CASGEVY | Vertex Pharmaceuticals Incorporated | Industry | BCL11A | Gene editing | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Electroporation | RNP | Cas9 mRNA | Dose 1: Minimum dose: 3E6 CD34+ cells/kg | Dose 2: Maximum dose: 20E6 CD34+ cells/kg | Phase3 | Recruiting | Approved | 2022-07-26 | 2027-06-09 | 2025-04-03 | 12 Years - 35 Years | 26 | 6 |
Locations:United States + 3 more
Germany, Italy, Saudi Arabia, United States
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FDA approved 12/8/23, price/treatment $2.2M, expanded indication to beta thalassemia 1/16/24 |
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| NCT05398029 | Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease | VERVE-101 | Verve Therapeutics, Inc. | Industry | PCSK9 | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Hepatocyte | Lipid encapsulation | LDLR | ABE8.8m | Dose 1: 0.1 mg/kg | Dose 2: 0.3 mg/kg | Dose 3: 0.45 mg/kg | Dose 4: 0.6 mg/kg | Phase1 | Active not recruiting | Active | 2022-05-19 | 2025-06 | 2024-12-30 | 18 Years - 75 Years | 44 | 3 |
Locations:New Zealand + 1 more
New Zealand, United Kingdom
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US12029795B2; US20240010609A1; US20240131166A1; US20240011023A1 | This program is deprioritized in favor of VERVE-102 |
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| NCT06164730 | Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease | VERVE-102 | Verve Therapeutics, Inc. | Industry | PCSK9 | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Lipid encapsulation | LDLR + GalNAc | base editor | Dose 1: 0.3mg/kg | Dose 2: 0.45mg/kg | Dose 3: 0.6mg/kg | Dose 4: Undisclosed dose 4 | Phase1 | Recruiting | Active | 2023-12-01 | 2026-08 | 2024-11-12 | 18 Years - 70 Years | 36 | 8 |
Locations:Australia + 3 more
Australia, Canada, New Zealand, United Kingdom
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US12029795B2; US20240010609A1; US20240131166A1; US20240011023A1 | Initial data for the Heart-2 Phase 1b clinical trial of VERVE-102 targeting PCSK9 expected in second quarter of 2025; Dosing in the Heart-2 trial has moved to the 0.6 mg/kg cohort; IND cleared by FDA in March 2025 |
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| NCT06451770 | Hypercholesterolemia | VERVE-201 | Verve Therapeutics, Inc. | Industry | ANGPTL3 | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Hepatocyte | Lipid encapsulation | LDLR + GalNAc | ABE | Dose 1: Undisclosed dose 1 | Dose 2: Undisclosed dose 2 | Dose 3: Undisclosed dose 3 | Dose 4: Undisclosed dose 4 | Phase1 | Recruiting | Active | 2024-06-04 | 2027-03 | 2025-02-13 | 18 Years - 70 Years | 36 | 3 |
Locations:Canada + 1 more
Canada, United Kingdom
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US20240010609A1; US20230340435A1; WO2023049299A2 | First patient dosed November 2024, expected update 2H 2025 |
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| NCT06291935 | Retinitis Pigmentosa | Orphan Drug Designation, Rare Pediatric Disease Designation | VG901 | ViGeneron GmbH | Industry | CNGA1 | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV2 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Phase1 | Recruiting | Active | 2024-02-01 | 2026-04 | 2025-03-28 | >= 18 Years | 6 | 1 | Germany | US12043848B2; US20220409744A1 | First patient dosed 4/10/24 |
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| NCT04537377 | Wilson Disease | Orphan Drug Designation | VTX-801 | Vivet Therapeutics SAS | Industry | ATP7B-minigene | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAV8 | Undisclosed dose escalation, 3 levels | Phase2, Phase1 | Active not recruiting | Inactive | 2020-08-19 | 2029-06-18 | 2025-03-25 | 18 Years - 65 Years | 4 | 10 |
Locations:United States + 3 more
Denmark, Germany, United Kingdom, United States
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Sponsor terminated the trial due to efficacy concerns at the two doses tested, insufficient funding for further dose escalation |
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| NCT06237777 | Diabetic Macular Edema | SKG0106 | Wang Min | Other | Anti-VEGF | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV | Undisclosed dose escalation, 3 levels | Phase1 | Recruiting | Active | 2024-01-11 | 2026-01 | 2024-04-30 | >= 18 Years | 18 | 2 | China | IND for nAMD (2nd indication) was approved in June 2023 |
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| NCT01161576 | CLN2 Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis | Orphan Drug Designation, Rare Pediatric Disease Designation | LX1004 | Weill Medical College of Cornell University | Other | TPP1 | Gene transfer | In-vivo | Functional gene replacement | Intracisternal | Viral vector | Viral transduction | AAVrh10 | Dose 1: 2.85E11 gc | Dose 2: 9.0E11 gc | Dose 3: 3E12 particle units (AAV2 vector) | Phase1 | Completed | Inactive | 2010-03-22 | 2020-12-31 | 2021-02-02 | 2 Years - 18 Years | 12 | 1 | United States | Product was licensed to Lexeo Therapeutics, renamed LX1004, product development was discontinued |
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| NCT06300476 | Stargardt Disease | JWK006 | West China Hospital | Other | ABCA4 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | dual AAV8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Active not recruiting | Active | 2024-03-03 | 2029-12-30 | 2024-03-08 | 10 Years - 18 Years | 9 | 1 | China | CN115074369A | Investigator-initiated trial conducted at West China Hospital |
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| NCT06114056 | Duchenne Muscular Dystrophy (DMD) | JWK007 | West China Hospital | Other | Micro-dystrophin | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravenous | Viral vector | Viral transduction | AAVrh74 | Dose 1: 1.0E14 vg/kg | Dose 2: 2.0E14 vg/kg | Phase1 | Recruiting | Active | 2023-10-29 | 2028-12-31 | 2024-01-17 | 5 Years - 10 Years | 6 | 1 | China | Investigator-initiated trial conducted at West China Hospital |
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| NCT06519552 | Mucopolysaccharidosis Type I (Hurler Syndrome) | JWK-008 | West China Hospital | Other | IDUA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV5 | Dose 1: 5.0E12 vg/kg | Dose 2: 2.0E13 vg/kg | Phase1 | Recruiting | Active | 2024-06-23 | 2029-06-22 | 2024-07-25 | >= 18 Years | 6 | 1 | China |
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| NCT06345898 | X-Linked Retinoschisis | JWK002 | West China Hospital | Other | RS1 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Early phase1 | Recruiting | Active | 2024-03-28 | 2033-11-30 | 2025-02-07 | 5 Years - 18 Years | 12 | 1 | China | Investigator-initiated trial conducted at West China Hospital |
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| NCT04124042 | Osteoarthritis, Knee | Fast Track | XT-150 | Xalud Therapeutics, Inc. | Industry | Recombinant IL10 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarticular | Plasmid | None (naked plasmid) | Dose 1: 150ug | Dose 2: 450ug (extension study) | Phase2 | Completed | Active | 2019-10-09 | 2022-04-26 | 2025-03-27 | 45 Years - 85 Years | 289 | 6 |
Locations:United States + 1 more
Australia, United States
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| NCT06302608 | Bietti Crystalline Dystrophy | Orphan Drug Designation | NGGT001 | Xiamen Ophthalmology Center Affiliated to Xiamen University | Other | CYP4V2 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: 1.5E11 vg/eye | Dose 2: 3.0E11 vg/eye | Early phase1 | Active not recruiting | Active | 2024-02-18 | 2028-05-29 | 2024-03-12 | >= 18 Years | 6 | 1 | China | WO2023116745A1 |
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| NCT05822739 | Parkinson's Disease | BBM-P002 | Xiangya Hospital of Central South University | Other | Undisclosed | Undisclosed | In-vivo | Undisclosed | Intraparenchymal | Viral vector | Viral transduction | AAV | Undisclosed | Early phase1 | Not yet recruiting | Active | 2023-03-01 | 2028-12-30 | 2023-04-21 | 40 Years - 70 Years | 6 | 1 | China |
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| NCT06663878 | Wilson Disease | MWAV201 | Xinhua Hospital, Shanghai Jiao Tong University School of Medicine | Other | Undisclosed | Undisclosed | Undisclosed | Undisclosed | Intravenous | Undisclosed | Undisclosed | undisclosed | undisclosed | Undisclosed | Na | Not yet recruiting | Active | 2024-10-10 | 2031-05 | 2024-10-29 | 18 Years - 65 Years | 9 | 1 | China |
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| NCT06272149 | Type II Gaucher Disease | Rare Pediatric Disease Designation | VGN-R08b | Xinhua Hospital, Shanghai Jiao Tong University School of Medicine | Other | GBA1 | Gene transfer | In-vivo | Functional gene replacement | Intracerebroventricular | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Early phase1 | Recruiting | Active | 2023-07-17 | 2029-02-28 | 2024-02-22 | 0 Months - 24 Months | 6 | 1 | China | WO2024017387A1 | FIH interim data presented at ASGCT 2024 |
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| NCT04125732 | Coronary Artery Disease, Ischemia, Angina Refractory, Cardiovascular Diseases, Heart Diseases | XC001 | XyloCor Therapeutics, Inc. | Industry | VEGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramyocardial | Viral vector | Viral transduction | Ad5 | Dose 1: 1E9 vp | Dose 2: 1E10 vp | Dose 3: 4E10 vp | Dose 4: 1E11 vp (Expansion Dose) | Phase2, Phase1 | Completed | Active | 2019-10-04 | 2023-05-30 | 2024-01-30 | 18 Years - 80 Years | 41 | 16 | United States | US20240115732A1 |
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| NCT06831825 | Heart Failure With Reduced Ejection Fraction | YAP101 | YAP Therapeutics, Inc. | Industry | SAV1 | Gene transfer | In-vivo | Gene inactivation | Transendocardial | Viral vector | Cardiomyocyte | Viral transduction | AAV9 | Dose 1: 5.0E12 vg | Dose 2: 1.0E13 vg | Dose 3: 5.0E13 vg | Phase1 | Not yet recruiting | Active | 2025-01-23 | 2027-06 | 2025-02-18 | 18 Years - 79 Years | 24 | 0 |
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| NCT06722170 | DFNB9, Congenital Hearing Loss | EH002 | Yilai Shu | Other | OTOF | Gene transfer | In-vivo | Functional gene replacement | Intracochlear | Viral vector | Hair cell | Viral transduction | dual AAV1 | Dose 1: 9E11 vg/ear (unilateral) | Dose 2: 1.5E12 vg/ear (unilateral) | Dose 3: 1.5E12 vg/ear (bilateral) | Na | Recruiting | Active | 2024-12-05 | 2029-11 | 2025-03-30 | >= 6 Months | 18 | 2 | China |
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| NCT06539208 | Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy | YOLT-201 | YolTech Therapeutics Co., Ltd | Industry | TTR | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Liver | Lipid encapsulation | LNP | Cas9 mRNA | Undisclosed dose ascension | Phase2, Phase1 | Recruiting | Active | 2024-08-01 | 2026-06-30 | 2024-08-06 | 18 Years - 80 Years | 31 | 3 | China | Dose escalation completed December 2024 |
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| NCT06292650 | Retinitis Pigmentosa | Orphan Drug Designation | ZM-02 | Zhongmou Therapeutics | Industry | Ch2.0 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Viral transduction | AAV | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Early phase1 | Recruiting | Active | 2024-02-27 | 2028-12-25 | 2024-12-13 | 18 Years - 65 Years | 12 | 1 | China | Two patients treated early 2024 |
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| NCT06066008 | X-linked Retinoschisis | ZM-01 | Zhongmou Therapeutics | Industry | RS1 | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV8 | Dose 1: 2.07E11 vg/eye | Dose 2: Undisclosed high dose | Early phase1 | Recruiting | Active | 2023-09-25 | 2027-10 | 2024-02-21 | 3 Years - 18 Years | 9 | 1 | China | |||||||
| NCT03207009 | Beta-Thalassemia Major | Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | ZYNTEGLO | bluebird bio | Industry | HBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | BB305 LV | Minimum dose: 5.0E6 CD34+ cells/kg | Phase3 | Completed | Approved | 2017-06-29 | 2022-11-15 | 2024-03-07 | 0 Years - 50 Years | 19 | 9 |
Locations:United States + 5 more
France, Germany, Greece, Italy, United Kingdom, United States
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FDA approval granted 8/17/22, $2.8M estimated cost/treatment |
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| NCT03852498 | Cerebral Adrenoleukodystrophy (CALD) | Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation | SKYSONA | bluebird bio | Industry | ABCD1 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | At least 5.0E6 CD34+ cells/kg; MOI = 40 | Phase3 | Completed | Approved | 2019-02-13 | 2023-07-24 | 2024-05-24 | <= 17 Years | 35 | 8 |
Locations:United States + 5 more
France, Germany, Italy, Netherlands, United Kingdom, United States
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US9061031B2 | FDA approval granted 9/16/22; Cost/treatment $3M |
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| NCT04293185 | Sickle Cell Disease | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | LYFGENIA | bluebird bio | Industry | HBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | BB305 LV | Dose 1: Minimum dose: 3E6 CD34+ cells/kg | Dose 2: Maximum dose: 14E6 CD34+ cells/kg | Dose 3: Median dose: 6.4E6 CD34+ cells/kg | Phase3 | Active not recruiting | Approved | 2020-02-12 | 2027-11 | 2024-12-10 | 2 Years - 50 Years | 35 | 9 | United States | FDA approved 12/8/23, price/treatment $3.1M |
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| NCT03328130 | Retinitis Pigmentosa | CTX-PDE6B | eyeDNA Therapeutics | Industry | PDE6B | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/5 | Dose 1: 3.4E11 vg/eye | Dose 2: 6.4E11 vg/eye | Phase2, Phase1 | Recruiting | Active | 2017-10-05 | 2029-12 | 2024-03-07 | >= 13 Years | 23 | 1 | France | |||||||
| NCT06255782 | Ornithine Transcarbamylase (OTC) Deficiency | Fast Track | ECUR-506 | iECURE, Inc. | Industry | OTC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Hepatocyte | Viral transduction | dual AAV8 | ARCUS | Dose 1: 1.3E13 GC/kg | Dose 2: Undisclosed high dose | Phase2, Phase1 | Recruiting | Active | 2023-12-19 | 2026-09 | 2025-03-28 | 24 Hours - 7 Months | 13 | 7 |
Locations:United States + 3 more
Australia, Spain, United Kingdom, United States
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US20240101986A1; EP3288594B1; US9493788B2 | Sponsor reports complete clinical response in first infant dosed |
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References
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The SCGE makes no comment as to the efficacy and safety of the items listed, as these are not known at the time of publication.
For the most up to date information, please refer to clinicaltrials.gov or the Sponsor's website.
The information contained within this table does not make use of any confidential or privileged information-all data is collected from publicly available sources.