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.
Disclaimer: The information on this dashboard has been collected for the convenience of patients and researchers. The SCGE team are not medical doctors and cannot provide medical advice. Please discuss with your provider the risks/benefits of participating in a clinical trial, and do not send us your personal medical information. The information contained within this table does not make use of any confidential or privileged information-all data is collected from publicly available sources. 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, or to inquire about enrollment, please refer to clinicaltrials.gov or the Sponsor's website for contact information.
Showing all 230 results in ClinicalTrials
SCGE Platform Gene Therapy Clinical Trials downloaded on: 2025/12/16 03:17:09; 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 |
| 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 | 2025-07-17 | >= 50 Years | 400 | 99 |
Locations:United States + 1 more
Canada, United States
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Phase III trial to begin Q1 2025 |
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| NCT04517149 | X-Linked Retinitis Pigmentosa (XLRP) | 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 | ||||
| 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 | 2027-08-31 | 2025-05-22 | >= 18 Years | 13 | 2 | United States | January 2025: Sponsor announced they would terminate development of this program | |||||
| 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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| 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 (anticipated pivotal/commercial dose) | 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 | 2025-11-14 | >= 18 Years | 40 | 19 | United States | US11499166B2; WO2023172873A2 | Advancing to phase 2, pivotal dose selected; interim data and program update expected soon |
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| NCT05930561 | Diabetic Macular Edema, Diabetic Retinopathy | Regenerative Medicine Advanced Therapy | 4D-150 | 4D Molecular Therapeutics | Industry | VEGF Trap + VEGF-C RNAi | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV R100 | Dose 1: 5E9 vg/eye (n=1) | Dose 2: 1E10 vg/eye (n=12) | Dose 3: 3E10 vg/eye (n=9) | Dose 4: Planned Phase 3 dose: 3E10 vg/eye | Phase2 | Active not recruiting | Active | 2023-06-26 | 2029-02-28 | 2025-09-22 | >= 18 Years | 72 | 6 |
Locations:United States + 1 more
Puerto Rico, United States
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52-week interim data update presented 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 | 2031-01 | 2025-09-15 | >= 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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| 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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| 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 | 2027-11 | 2025-08-20 | 50 Years - 89 Years | 630 | 89 | United States | US20230057519A1; US20200093939A1; | Pivotal data expected 2026 |
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| NCT07007065 | Neovascular Age-related Macular Degeneration | 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 | 2025-05-28 | 2033-03 | 2025-12-04 | >= 50 Years | 561 | 11 | United States | Another Phase 3 study (NCT05407636) is active, enrollment is completed |
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| NCT05725018 | Recessive Dystrophic Epidermolysis Bullosa (RDEB) | Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | ZEVASKYN | Abeona Therapeutics, Inc | Industry | COL7A1 | Gene transfer | Ex-vivo | Functional gene replacement | Skin graft | Autologous cells | Keratinocytes | Viral transduction | LZRSE | Transduced 41.25cm^2 keratinocyte sheets, up to 6 wound sites | Phase3 | Active not recruiting | Approved | 2023-01-23 | 2025-09-30 | 2025-06-27 | >= 12 Months | 12 | 2 | United States | US20240067926A1; US20230045590A1 | FDA approved on 4/29/25, list price $3.1M USD |
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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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| NCT06856577 | 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 | Phase3 | Recruiting | Active | 2025-02-26 | 2030-11-23 | 2025-11-18 | >= 50 Years | 284 | 79 | United States | Phase 3 non-inferiority study will evaluate a broad patient population; initiated March 2025 |
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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 | 2025-10-14 | >= 18 Years | 27 | 4 | United States | Eli Lilly acquired this company in 2022 |
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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 | 2025-09-05 | 18 | 8 |
Locations:United States + 3 more
Canada, Taiwan, United Kingdom, United States
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US20210017235A1; US11104885B2; US20240011039A1; EP4063510A1 |
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| NCT03770572 | CLN3 Batten Disease, Juvenile Neuronal Ceroid Lipofuscinosis | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | CLN-301 | Alcyone Therapeutics, Inc | 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 | Active | 2018-12-07 | 2029-09-30 | 2025-08-07 | 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, when Alcyone's involvement began |
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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 | 2025-09-17 | 6 Years - 64 Years | 23 | 4 |
Locations:United States + 2 more
France, Germany, United States
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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 | 2025-12-02 | >= 12 Years | 15 | 10 | United States | US20240091381A1; WO2020214990A1 | Rare Pediatric Disease designation granted 4/25/24 |
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| NCT06285643 | Parkinson's 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 | 2025-11-19 | 45 Years - 75 Years | 87 | 39 |
Locations:United States + 3 more
Germany, Poland, United Kingdom, United States
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US20180140672A1; US20180344199A1; WO2023183594A2 | Received FDA Fast Track designation |
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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: Phase 1: 3.25E13 vg | Dose 2: Phase 1: 1.08E14 vg | Dose 3: Phase 2: 7.15E13 vg | Dose 4: Phase 2: 1.43E14 vg | Phase2 | Active not recruiting | Active | 2022-10-25 | 2030-12-31 | 2025-12-03 | >= 18 Years | 150 | 96 |
Locations:United States + 11 more
Austria, Belgium, Bulgaria, Canada, Germany, Hungary, Netherlands, Poland, Romania, Spain, United Kingdom, United States
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US20230340528A1; AU2021320244A1 | Received Fast Track designation 4/18/24 |
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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 | 2025-10-02 | 18 Years - 65 Years | 10 | 6 | United States | US20200061092A1; US20190008881A1 | First patient dosed (8/3/23) |
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| NCT03533673 | Pompe Disease | Fast Track | 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 (n=3) | 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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| 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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| 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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| 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 | 2025-10-07 | 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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| 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 (n=7) | Dose 2: 3.5E14 vg/kg (n=20) | Phase3, Phase2 | Active not recruiting | Active | 2017-06-21 | 2030-03-31 | 2025-10-14 | <= 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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| NCT05224505 | Limb-Girdle Muscular Dystrophy, Type 2I/R9 | Fast Track, Rare Pediatric Disease Designation | 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 | Phase1 | Active not recruiting | Active | 2021-12-15 | 2029-09-30 | 2025-10-06 | 16 Years - 99 Years | 6 | 3 |
Locations:Denmark + 2 more
Denmark, France, United Kingdom
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US20230321277A1; EP4031673A1; EP4198047A1 | Dose-finding study completed April 2025 |
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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 | 2031-01-31 | 2025-10-06 | 6 Years - 13 Years | 6 | 3 |
Locations:United States + 2 more
France, Italy, United States
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EP4198134A1 | First two patients dosed June 2025 |
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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: 3.0E10 vg/eye | Dose 3: 5.0E10 vg/eye | Dose 4: 1.5E10 vg/eye (150ul) | Dose 5: 2.3E10 vg/eye (225ul) | Phase2, Phase1 | Recruiting | Active | 2023-05-12 | 2029-10 | 2025-02-19 | >= 6 Years | 21 | 4 | United States | Pediatric dosing expected to begin Q4 2025; pivotal cohort expected to begin enrolling in Q1 2026 |
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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), n=3 | Dose 2: 3.0E10 vg/eye (300ul), n=3 | Dose 3: 1.0E11 vg/eye (300ul), n=9 | 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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| NCT06064890 | Frontotemporal Dementia, FTD, FTD-GRN | 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 | 2025-09-10 | 30 Years - 75 Years | 9 | 17 |
Locations:United States + 6 more
Canada, Netherlands, Poland, Spain, Sweden, United Kingdom, United States
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Second dose cohort is complete, company intends to initiate a third dose cohort in Q3 2025, early biomarker data expected 2026 |
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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 | 2025-05-22 | 4 Months - 35 Years | 30 | 1 | United States | US20220273712A1; EP3672617A4; US20230183804A1 |
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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 | 2025-11-21 | >= 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 | 2025-10-20 | >= 18 Years | 24 | 4 | United States |
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| NCT02599922 | Achromatopsia | Orphan Drug Designation | 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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| NCT04850118 | X-Linked Retinitis Pigmentosa (XLRP) | Fast Track, Orphan Drug Designation, 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 | Active not recruiting | Active | 2021-04-05 | 2029-10 | 2025-07-16 | 12 Years - 50 Years | 85 | 24 |
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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| NCT05456880 | Sickle Cell Disease | Regenerative Medicine Advanced Therapy | BEAM-101 | Beam Therapeutics Inc. | Industry | HBG1/HBG2 | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Electroporation | none | ABE8 | Dose 1: Transduced CD34+ cells (≥3.0E6 viable CD34+ cells/kg) | Dose 2: Range: 3.2-23.4E6 CD34+ cells/kg, N=17 | 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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| 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 | 2025-06-10 | 18 Years - 70 Years | 106 | 6 |
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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| 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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| NCT06185673 | Oculopharyngeal Muscular Dystrophy | Fast Track, 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 | 1st patient of cohort 2 was successfully treated in Q4 2025 |
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| NCT04737460 | CLN7 Batten Disease, Variant Late-Infantile Neuronal Ceroid Lipofuscinosis Type 7 | 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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| 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. | |||||
| 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 | ||||
| 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 | Active not recruiting | Active | 2020-02-13 | 2029-12-30 | 2025-08-27 | >= 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 | Active not recruiting | Active | 2024-07-18 | 2028-07-04 | 2025-11-24 | >= 18 Years | 36 | 10 |
Locations:France + 4 more
France, Italy, Netherlands, Spain, United Kingdom
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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 | Active not recruiting | Active | 2020-12-15 | 2028-08 | 2025-07-17 | 35 Years - 75 Years | 9 | 7 | United States | US20180140672A1; US20180344199A1; WO2023183594A2 | Phase 1 enrollment completed September 2025 |
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| 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 | 2028-04 | 2025-09-18 | 18 Years - 65 Years | 5 | 1 | France | WO2012049314A1 | Bayer announced the discontinuation of this program November 2024 |
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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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| 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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| 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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| NCT06743646 | Bietti Crystalline Dystrophy | ZVS101e | Chigenovo Co., Ltd | Network | CYP4V2 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/8 | Dose 1: Phase 1/2: 7.5E10 vg/eye | Dose 2: Phase 1/2: 1.5E11 vg/eye | Dose 3: Phase 1/2: 2.2E11 vg/eye | Dose 4: Expansion/pivotal dose: 7.5E10 vg/eye | Phase3 | Recruiting | Active | 2024-12-17 | 2030-06-30 | 2025-01-03 | >= 18 Years | 62 | 8 | 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 | 2025-08-29 | >= 18 Years | 3 | 1 | United States | US20220315900A1; US20200199623A1 |
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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 | 2025-09-05 | 18 Years - 35 Years | 12 | 1 | United States | ||||||
| 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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| 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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| NCT02247843 | Sickle Cell Disease | LentiG-βAS3-FB (contains anti-sickling mutations: T87Q, G16D, E22A) | 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 | Inactive | 2014-09-20 | 2025-12 | 2024-05-16 | >= 18 Years | 4 | 1 | United States | WO2020168004A1 | Study was closed after FDA approvals of Casgevy and Lyfgenia |
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| NCT04798235 | Infantile GM2 Gangliosidosis | Orphan Drug Designation, Rare Pediatric Disease Designation | TSHA-101 | Dr. Anupam Sehgal | Other | 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 |
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| NCT05444894 | Beta-Thalassemia Major | Orphan Drug Designation, Rare Pediatric Disease Designation | 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 | Orphan Drug Designation, Rare Pediatric Disease Designation, 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 | 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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| NCT05419492 | Dravet Syndrome | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | 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 | 2025-06-12 | 6 Months - 47 Months | 22 | 3 | United States | US20230203531A1; US20200397917A1; US20220136009A1; US20220168449A1; US20220170910A1 | Enrollment in dose escalation studies expected to complete in 4Q25 |
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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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| 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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| 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 | Russia |
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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 | Phase3 | Recruiting | Approved | 2024-08-06 | 2030-12-31 | 2025-12-04 | >= 18 Years | 250 | 40 |
Locations:United States + 4 more
Canada, Czechia, Poland, Spain, United States
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FDA approved on 12/16/22 for the treatment of adult patients with high-risk Bacillus Calmette-GuÃÂÃÂÃÂérin (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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| NCT03837483 | Wiskott-Aldrich Syndrome | Orphan Drug Designation | Etuvetidigene autotemcel | Fondazione Telethon | Other | WAS | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells (Range: 2 - 15E6 cells/kg; Median: 7.05E6 cells/kg) | Phase3 | Active not recruiting | Active | 2019-02-08 | 2027-09 | 2025-09-29 | <= 65 Years | 10 | 2 |
Locations:United States + 1 more
Italy, United States
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EP3973996A1 | BLA submitted 3/11/25, PDUFA date anticipated late 2025 or early 2026 |
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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 | Inactive | 2023-02-13 | 2026-11 | 2025-11-20 | <= 18 Years | 9 | 3 | United States | WO2018136710; WO2020132385A1 | Program discontinuation was announced January 2025 |
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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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| 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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| 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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| 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 | |||||
| NCT04713475 | GM1 Gangliosidosis, Beta-Galactosidase-1 (GLB1) Deficiency | PBGM01 | Gemma Biotherapeutics | 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 | 2025-05-21 | 1 Month - 24 Months | 26 | 7 |
Locations:United States + 3 more
Brazil, Turkey (Türkiye), United Kingdom, United States
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WO2024081551A1 | Out-licensed development to Gemma Biotherapeutics, uncertain development status |
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| NCT03326336 | Non-syndromic Retinitis Pigmentosa | Fast Track, Orphan Drug Designation | 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 (n=3) | Dose 2: 1.5E11 vg/eye (n=3) | Dose 3: 5E11 vg/eye (n=4) | Phase2, Phase1 | Active not recruiting | Active | 2017-10-11 | 2027-10-26 | 2025-08-26 | 18 Years - 75 Years | 10 | 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, Regenerative Medicine Advanced Therapy | 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 | 2025-08-01 | 40 Years - 75 Years | 67 | 9 | United States | US20220016213A1 | Initial data for Phase 1b trial expected Q4 2024 |
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| NCT05824169 | Spinal Muscular Atrophy | Orphan Drug Designation | 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 | 2026-12 | 2025-07-03 | 0 Months - 6 Months | 18 | 4 | China | Phase 3 pivotal study for Type 2 SMA has completed enrollment |
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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 | 2025-07-03 | >= 6 Years | 33 | 1 | China | First subject dosed August 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 | 2025-07-03 | 18 Years - 60 Years | 7 | 1 | China | Recruitment started October 2024 |
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| 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 (n=2) | Dose 2: 5E12 vg/kg (n=3) | 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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Pivotal part of the trial has been initiated |
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| NCT04293185 | Sickle Cell Disease | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | LYFGENIA | Genetix Biotherapeutics Inc. | 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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| NCT06199531 | NGLY1 Deficiency | Support for Clinical Trials Advancing Rare Disease Therapeutics, Orphan Drug Designation, Rare Pediatric Disease Designation | 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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| 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 | 2025-09-12 | 18 Years - 65 Years | 50 | 13 | 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 | Early phase1 | Recruiting | Active | 2024-12-29 | 2029-10-23 | 2025-07-01 | 4 Years - 10 Years | 8 | 1 | China | |||||||
| 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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| 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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| 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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| 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 | Active not recruiting | Active | 2024-02-25 | 2029-12-31 | 2025-04-30 | >= 8 Years | 18 | 1 | China | WO2023143606A1 |
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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: IIT Trial: 3E10 vg/eye or 1E11 vg/eye | Dose 2: 2E10 vg | Dose 3: 1.25E11 vg | Phase2 | Active not recruiting | Active | 2023-12-25 | 2029-10 | 2025-08-01 | 50 Years - 89 Years | 50 | 11 | China |
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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 | 2025-07-01 | >= 6 Years | 9 | 2 | China | US11970519B2 |
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| 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 | 2025-11-07 | 2 Years - 4 Years | 12 | 8 | United States | IND cleared in 4Q2024, Clinical trial initiation planned in 1H2025 |
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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 | 2025-08-01 | >= 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 | ||||||
| NCT06128629 | Transthyretin Amyloidosis (ATTR) with Cardiomyopathy | Orphan Drug Designation, Regenerative Medicine Advanced Therapy | NTLA-2001 | Intellia Therapeutics | Industry | TTR | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Hepatocyte | Lipid encapsulation | LNP | Cas9 mRNA | 55mg | Phase3 | Active not recruiting | Active | 2023-11-08 | 2028-04 | 2025-12-02 | 18 Years - 90 Years | 1200 | 126 |
Locations:United States + 24 more
Argentina, Australia, Austria, Belgium, Brazil, Canada, Czechia, Denmark, France, Germany, Hungary, Israel, Italy, Japan, Mexico, Netherlands, New Zealand, Norway, Portugal, 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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| 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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| 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 | Active not recruiting | Active | 2024-10-07 | 2027-09 | 2025-12-02 | >= 16 Years | 60 | 30 |
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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| 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 | 2025-10-03 | 2 Years - 9 Years | 6 | 3 | 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 | 2026-02-26 | 2025-11-07 | >= 60 Years | 305 | 162 |
Locations:United States + 16 more
Australia, Belgium, Canada, Czechia, Denmark, Germany, Hungary, Italy, Netherlands, Poland, Portugal, Spain, Sweden, Switzerland, Turkey (Türkiye), United Kingdom, United States
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WO2023089564A1 |
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| 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 | 2028-12-31 | 2025-11-21 | 12 Years - 40 Years | 15 | 4 | 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 | |||||
| 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 | 2026-12 | 2025-07-22 | 18 Years - 70 Years | 15 | 3 | United States | Interim data expected in 1H 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 | 2026-01 | 2025-08-21 | >= 18 Years | 12 | 5 | United States | WO2020163703A1; WO2021127524A1 | Interim data expected for cohort 3 by 2025 year end |
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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 | 2025-10-21 | >= 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 | Active not recruiting | Active | 2023-12-19 | 2025-12 | 2025-11-21 | <= 180 Days | 12 | 3 | China | |||||||
| 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 | 2025-07-22 | 20 | 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 | 2025-11-25 | <= 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 (Türkiye), United Kingdom
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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 | Dose 1: 2.0E13 gc/kg | Dose 2: 6E10 vg/kg | Phase2, Phase1 | Active not recruiting | Active | 2023-10-25 | 2027-03 | 2025-11-12 | 18 Years - 65 Years | 10 | 5 | United States | Completed enrollment of LX2020 Phase 1/2 trial, additional clinical data expected January 2026 |
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| NCT05445323 | Friedreich Ataxia, Cardiomyopathy, Secondary | Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | 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 (planned pivotal dose) | Phase2, Phase1 | Active not recruiting | Active | 2022-06-23 | 2029-09 | 2024-11-15 | 18 Years - 50 Years | 8 | 3 | United States | WO2023150563A1 | Plans to initiate registrational study in the first half of 2026 |
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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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| 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 | 2027-12-01 | 2025-10-21 | 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 | Active not recruiting | Active | 2019-01-15 | 2033-05-01 | 2025-11-13 | >= 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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| 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 | 2026-12 | 2025-12-02 | >= 18 Years | 276 | 24 |
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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| 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 | 2025-05-25 | >= 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 | 2025-12-02 | 18 Years - 55 Years | 12 | 5 | United States | WO2024094044A1 | Recieved Orphan Drug Designation from the FDA in Jan 2023 |
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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 (n=7) | Dose 2: 4.5E13 vg/kg (n=3) | Phase2, Phase1 | Recruiting | Active | 2019-05-15 | 2028-01-01 | 2025-11-28 | 6 Months - 12 Years | 54 | 1 | United States |
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| NCT01306019 | X-linked Severe Combined Immunodeficiency (XSCID) | Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | 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 | Dose 1: Transduced CD34+ cells (median dose: 6.73E6 cells/kg; range: 4.46-15.10E6 cells/kg, n=8) | Dose 2: Transduced CD34+ cells (median dose: 20.4E6 cells/kg; range: 16-25E6 cells/kg, n=5) | Phase2, Phase1 | Recruiting | Active | 2011-02-26 | 2032-12-31 | 2025-12-04 | 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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| 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 | Recruiting | Active | 2024-03-21 | 2032-12-31 | 2025-11-20 | 18 Years - 75 Years | 10 | 1 | United States | Trial stoppage rules triggered due to a serious adverse event |
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| NCT02362438 | Giant Axonal Neuropathy | Orphan Drug Designation, Rare Pediatric Disease Designation | 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 (n=2) | Dose 2: 1.2E14 vg (n=4) | Dose 3: 1.8E14 vg (n=5) | Dose 4: 3.5E14 vg (n=3) | Phase1 | Active not recruiting | Active | 2015-02-12 | 2035-04-01 | 2025-09-30 | 3 Years - 99 Years | 14 | 1 | United States | US20240102050A1 | Taysha discontinued development in September 2023, transferred development rights to NIH |
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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/eye | Dose 2: 3E9 vg/eye | Dose 3: 8E9 vg/eye | Phase2, Phase1 | Recruiting | Active | 2023-07-24 | 2030-01 | 2025-10-30 | 50 Years - 89 Years | 18 | 5 |
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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| NCT05898620 | Rett Syndrome | Support for Clinical Trials Advancing Rare Disease Therapeutics, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | 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 due to HLH) | Phase3 | Recruiting | Active | 2023-06-01 | 2029-12 | 2025-11-07 | >= 3 Years | 33 | 15 |
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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| 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 (selected for Phase 3 dose) | 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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| NCT06910813 | Nephropathic Cystinosis | Orphan Drug Designation, Rare Pediatric Disease Designation | DFT383 | Novartis Pharmaceuticals | Industry | CTNS | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Min dose: 3E6 CD34+ cells/kg | Phase2, Phase1 | Recruiting | Active | 2025-03-17 | 2044-03-14 | 2025-11-07 | 2 Years - 5 Years | 30 | 4 | United States | AVROBIO sold product to Novartis in May 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 1: 5E9 vg/eye (n=3) | Dose 2: 1E10 vg/eye (n=3) | Dose 3: 3E10 vg/eye (n=3) | Dose 4: 1E11 vg/eye (n=3) | 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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| 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 | Active not recruiting | Active | 2023-06-30 | 2026-05-29 | 2025-06-17 | >= 50 Years | 60 | 12 | United States | US20210123077A1 | Dosing complete in the Phase 2 clinical trial, full data expected 1Q 2026 |
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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 | 2026-12-31 | 2025-10-16 | >= 5 Years | 150 | 17 |
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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| 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: 1.5E11 vg/ml (pivotal dose) | Dose 4: 2.25E11 vg/ml (Maximum Tolerated Dose) | Phase3, Phase2 | Recruiting | Active | 2023-06-30 | 2026-09-28 | 2025-10-16 | >= 5 Years | 51 | 14 | United States | US20210123077A1 | First patient dosed in Phase 2/3 pivotal trial, BLA submission planned in 2027 |
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| NCT05616793 | Leber Congenital Amaurosis-Type 5 | Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | OPGx-LCA5 | Opus Genetics, Inc | Industry | LCA5 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: 1E10 vg/eye (up to 300ul) | Dose 2: 3E10 vg/eye | Dose 3: 1E11 vg/eye | Phase2, Phase1 | Recruiting | Active | 2022-11-07 | 2028-06-15 | 2025-06-12 | >= 13 Years | 15 | 1 | United States | First participant enrolled in run-in period for planned adaptive Phase 3 trial |
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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 | 2025-06-15 | 28 Days - 30 Months | 41 | 5 |
Locations:United States + 3 more
Italy, Netherlands, United Kingdom, United States
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Last patient randomized in registrational trial July 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 | 2025-09-05 | 6 | 1 | Italy | FDA approval granted 3/18/24 |
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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 | 2025-11-05 | 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 | Pacira announced Phase 2 enrollment was complete in November 2025 |
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| NCT04747431 | Frontotemporal Dementia, FTD, FTD-GRN, C9orf72 | Fast Track, Orphan Drug Designation | PBFT02 | Passage Bio, Inc. | Industry | GRN | Gene transfer | In-vivo | Functional gene replacement | Intracisterna magna | Viral vector | Viral transduction | AAV1 | Dose 1: 4.5E13 GC | Dose 2: 2.2E13 GC | Phase2, Phase1 | Recruiting | Active | 2021-02-02 | 2031-08 | 2025-10-24 | 35 Years - 75 Years | 30 | 8 |
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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| 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 | Active not recruiting | Active | 2024-04-26 | 2027-12 | 2025-07-29 | 4 Years - 10 Years | 3 | 1 | China | CA3191505A1; WO2017215619A1 | Drug is being developed by GenAssist Ltd |
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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 | ||||||
| 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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| NCT04281485 | Duchenne Muscular Dystrophy (DMD) | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | PF-06939926 | Pfizer | Industry | Mini-dystrophin | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: Phase 1: 1E14 vg/kg (n=3) | Dose 2: Phase 1: 3E14 vg/kg (n=19) | Dose 3: Phase 3: 2E14 vg/kg | Phase3 | Active not recruiting | Inactive | 2020-02-11 | 2039-04-15 | 2025-10-20 | 4 Years - 7 Years | 114 | 51 |
Locations:United States + 14 more
Australia, Belgium, Canada, France, Germany, Israel, Italy, Japan, Russia, South Korea, 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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| NCT04370054 | Hemophilia A | Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy | Giroctogogene fitelparvovec | Pfizer | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAV2/6 | Dose 1: Phase 1/2: 9E11 vg/kg (n=2) | Dose 2: Phase 1/2: 2E12 vg/kg (n=2) | Dose 3: Phase 1/2: 1E13 vg/kg (n=2) | Dose 4: Phase 1/2: 3E13 vg/kg (n=5) | Dose 5: Phase 3: 3E13 vg/kg (n=75) | Phase3 | Active not recruiting | Active | 2020-04-21 | 2028-10-25 | 2025-08-17 | 18 Years - 64 Years | 77 | 36 |
Locations:United States + 15 more
Australia, Brazil, Canada, France, Germany, Greece, Italy, Japan, Saudi Arabia, South Korea, Spain, Sweden, Taiwan, Turkey (Türkiye), 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 | F9-R338L | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAVrh74 | Dose 1: Phase 2: 5E11 vg/kg (n=15) | Dose 2: Phase 3: 5E11 vg/kg (n=45) | Dose 3: Approved dose: 5E11 vg/kg | Phase3 | Active not recruiting | Marketing Discontinued | 2019-03-01 | 2031-02-25 | 2025-09-30 | 18 Years - 65 Years | 51 | 60 |
Locations:United States + 15 more
Australia, Brazil, Canada, France, Germany, Greece, Italy, Japan, Saudi Arabia, South Korea, Spain, Sweden, Taiwan, Turkey (Türkiye), 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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| 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 | 2025-09-26 | 18 Years - 70 Years | 45 | 4 |
Locations:United States + 3 more
Hong Kong, Moldova, New Zealand, United States
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IND cleared by FDA in March 2025 |
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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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| NCT04127578 | Parkinson's 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 | 2025-11-20 | 35 Years - 80 Years | 32 | 14 |
Locations:United States + 1 more
Israel, United States
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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 | 2025-06-11 | 0 Months - 24 Months | 7 | 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 | Active not recruiting | Active | 2024-07-11 | 2030-02 | 2025-05-25 | >= 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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| 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 (n=3) | Dose 2: 6.5E10 GC/g brain mass (n=7) | Dose 3: Pivotal dose: 2.9E11 GC/g brain mass (n=15) | 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 | BLA Submitted, PDUFA date extended from 11/9/25 to 2/8/26 |
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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: Single Patient IND: 1E10 GC/g brain mass (n=1) | Dose 2: 1E10 GC/g brain mass (n=2) | Dose 3: 5E10 GC/g brain mass (n=6) | 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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| 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 | 2025-11-10 | >= 1 Year | 65 | 16 |
Locations:United States + 1 more
Canada, United States
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US20230270886A1 | Pivotal trial underway; BLA expected 2026 |
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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 | 2030-10 | 2025-06-13 | >= 18 Years | 18 | 4 | United States |
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| NCT05788536 | Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF) | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | DB-OTO | Regeneron Pharmaceuticals | Industry | OTOF | Gene transfer | In-vivo | Functional gene replacement | Intracochlear | Viral vector | Hair cell | Viral transduction | dual AAV1 | 7.2E12 vg/ear (one or both ears); concentration: 3E13 vg/ml | Phase2, Phase1 | Recruiting | Active | 2023-03-15 | 2031-04-19 | 2025-10-10 | <= 17 Years | 30 | 15 |
Locations:United States + 3 more
Germany, Spain, United Kingdom, United States
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Regeneron planning regulatory submission by year end 2025 |
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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 | 2026-12-31 | 2025-05-16 | >= 2 Years | 21 | 1 | China | First patient dosed (8/5/24) |
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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 | Inactive | 2020-01-24 | 2026-05 | 2024-04-10 | >= 1 Year | 5 | 2 | United States | CA3093708A1; CA3106241A1; WO2020037249A1 | Rocket withdrew BLA in October 2025 due to changes in corporate priorities |
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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: 3.8E13 GC/kg | Dose 2: 6.7E13 GC/kg | Dose 3: 1.1E14 GC/kg (dose discontinued) | Phase2 | Recruiting | Active | 2023-10-11 | 2032-04 | 2025-10-07 | >= 8 Years | 14 | 6 |
Locations:United States + 3 more
Germany, Italy, United Kingdom, United States
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US10703797B2; US20210379201A1 | Clinical hold lifted August 2025 |
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| NCT05885412 | PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) | Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy | 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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| NCT02610582 | Achromatopsia | RAAV.hCNGA3 | STZ eyetrial | Other | CNGA3 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Cone cells | Viral transduction | AAV8 | Dose 1: 1E10 vg/dose (n=3) | Dose 2: 5E10 vg/dose (n=3) | Dose 3: 1E11 vg/dose (n=3) | 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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| 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 | Recruiting | Active | 2025-02-19 | 2030-02-28 | 2025-11-21 | 10 Years - 50 Years | 32 | 4 |
Locations:United States + 1 more
Argentina, United States
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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 | Active not recruiting | Inactive | 2023-06-23 | 2030-04-02 | 2025-09-23 | 18 Years - 65 Years | 32 | 11 |
Locations:United States + 4 more
Argentina, Brazil, Israel, Turkey (Türkiye), United States
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Deprioritized by Sponsor |
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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: Phase 1: 3E13 vg (n=6) | Dose 2: Phase 1: 4.5E13 vg (n=3) | Dose 3: Phase 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 | First patient dosed in Phase 2 portion of trial in September 2025 |
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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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| NCT06061549 | Heart Failure With Preserved Ejection Fraction, Diastolic Heart Failure | Fast Track | SRD-002 | 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 | Phase1 | Recruiting | Active | 2023-07-30 | 2029-08 | 2023-09-29 | >= 50 Years | 10 | 2 | United States | WO2011130552A2; WO2020176732A1 | First patients dosed in Phase 2 portion of trial |
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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 | Active not recruiting | Approved | 2023-05-19 | 2028-06-30 | 2025-06-22 | 148 | 46 |
Locations:United States + 13 more
Australia, Belgium, Canada, Germany, Hong Kong, Israel, Italy, Japan, South Korea, 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; new black box warning on risk of liver injury and liver failure added November 2025 |
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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 | Dose 2: 5E13 vg/kg | Dose 3: 7.41E13 vg/kg (Phase 3 dose) | Phase3 | Active not recruiting | Active | 2024-01-30 | 2029-11-30 | 2025-07-10 | >= 4 Years | 17 | 10 |
Locations:United States + 5 more
Belgium, Germany, Italy, Spain, United Kingdom, United States
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US20230241252A1; WO2021257595A1; EP4219726A1 | FDA placed clinical hold in July 2025 due to safety concerns with the AAV after the death of a patient who had received SRP-9004, which uses the same AAV vector, the Company plans to meet with FDA regarding BLA submission in 2025 |
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| NCT06370351 | OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders | 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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| 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 | Recruiting | Active | 2024-06-13 | 2027-03 | 2025-05-18 | 18 Years - 70 Years | 40 | 1 | China | WO2024011980A1 |
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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 | |||||
| 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 | ||||
| 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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| 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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| 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 |
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| NCT06111638 | Hemophilia A | Orphan Drug Designation | BBM-H803 | Shanghai Xinzhi 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 | 2025-07-17 | >= 18 Years | 12 | 8 | 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 Xinzhi 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, Phase2 | Active not recruiting | Approved (NMPA) | 2021-12-29 | 2028-06-30 | 2025-07-01 | >= 18 Years | 32 | 9 | China | WO2019241324A1; WO2022222869A1; WO2021180118A1 | In April 2025, Product was approved by National Medical Products Administration (NMPA) in 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 | 2025-09-09 | >= 1 Month | 10 | 2 | China |
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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 | 2025-09-09 | 1 Month - 1 Year | 10 | 2 | China |
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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 (n=3) | Dose 2: 2E14 vg/kg (n=9) | Phase2, Phase1 | Active not recruiting | Inactive | 2017-12-05 | 2026-10-15 | 2025-12-03 | 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) | Fast Track, Orphan Drug Designation, 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 | 2025-10-14 | 0 Years - 17 Years | 40 | 12 |
Locations:United States + 3 more
Canada, Italy, United Kingdom, 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 | 2030-09 | 2025-09-16 | >= 18 Years | 33 | 6 |
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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| 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 | Dose 1: 1.5E10 vg/eye (Phase 1: n=3) | Dose 2: 4.8E10 vg/eye (Phase 1: n=6) | Dose 3: 1.5E11 vg/eye (Phase 1: n=3; Phase 3: n=29) | Dose 4: APPROVED DOSE: 1.5E11 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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| 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 | 2025-10-24 | 25 Years - 65 Years | 53 | 3 | United States | |||||||
| 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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| 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 | 2025-11-14 | 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 | 2025-12 | 2025-09-19 | >= 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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| 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 | Phase3 | Recruiting | Active | 2022-10-28 | 2031-06 | 2025-11-21 | 6 Years - 21 Years | 15 | 5 |
Locations:United States + 1 more
Canada, United States
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US20240191254A1; US20240102050A1; US20190328804A1 | Clinical data expected H1 2025 |
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| NCT06152237 | Rett Syndrome | Breakthrough Therapy, 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 | Active not recruiting | Active | 2023-11-21 | 2031-11-02 | 2025-10-15 | 5 Years - 8 Years | 6 | 5 |
Locations:United States + 2 more
Canada, United Kingdom, United States
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Dosing of the first patient in the pivotal trial is expected 4Q25 |
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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 announced December 2025 |
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| NCT05836259 | Hypertrophic Cardiomyopathy | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation | 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 | 2025-07-15 | 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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| NCT05791864 | Neuronal Ceroid Lipofuscinosis Type 2 | Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy | 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; Dose escalation cohort is fully enrolled, enrollment in expansion cohort is ongoing |
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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) |
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| NCT04884815 | Wilson Disease | Fast Track, Orphan Drug Designation | 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 | 2034-03 | 2025-09-19 | >= 18 Years | 82 | 17 |
Locations:United States + 4 more
Canada, Portugal, Spain, United Kingdom, United States
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EP3906066B1 | Cohort 4 (moderately increased dose) enrollment completed, data expected 1H2026 |
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| NCT05345171 | Ornithine Transcarbamylase (OTC) Deficiency | Fast Track, Orphan Drug Designation | DTX301 | Ultragenyx Pharmaceutical Inc | Industry | OTC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: Phase 1/2: 3.4E12 GC/kg | Dose 2: Phase 1/2: 1.0E13 GC/kg | Dose 3: Phase 1/2: 1.7E13 GC/kg | Dose 4: Phase 3: 1.0E13 GC/kg | Phase3 | Active not recruiting | Active | 2022-04-18 | 2031-03 | 2025-10-09 | >= 12 Years | 32 | 16 |
Locations:United States + 9 more
Argentina, Brazil, Canada, France, Germany, Japan, Netherlands, Portugal, Spain, United States
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EP4038194A1 | Enrollment of Phase 3 study complete in February 2025 |
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| 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 (n=22) | Phase3, Phase2 | Recruiting | Active | 2016-03-17 | 2027-07 | 2025-11-06 | 36 | 5 |
Locations:United States + 2 more
Australia, Spain, United States
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US20220347298A1 | BLA filed under Accelerated Approval, CRL issued 7/11/25 requesting additional information and improvements related to CMC and manufacturing inspections |
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| NCT05139316 | Glycogen Storage Disease Type Ia | Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy | DTX401 | Ultragenyx Pharmaceutical Inc | Industry | G6PC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: Phase 1/2: 2E12 GC/kg | Dose 2: Phase 1/2: 6E12 GC/kg | Dose 3: Phase 3: 1.0E13 GC/kg | Phase3 | Active not recruiting | Active | 2021-07-14 | 2026-02 | 2025-11-19 | >= 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 rolling submission initiated, complete submission expected in 4Q25 |
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| NCT05243017 | Huntington's Disease | Breakthrough Therapy, Fast Track, Orphan Drug Designation, 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 | 2031-12 | 2025-11-20 | 18 Years - 75 Years | 12 | 18 | United States | First 3 patients in first cohort enrolled September 2025 |
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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 | 2031-04-30 | 2025-10-23 | 18 Years - 50 Years | 12 | 8 | United States | WO2015060722A1; WO2020104424A1 | First patient dosed 8/15/24; IDMC recommended proceeding with enrollment of 2nd cohort | ||||
| NCT06100276 | Amyotrophic Lateral Sclerosis (ALS) | Fast Track, Orphan Drug Designation | 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 | Active not recruiting | Active | 2023-10-20 | 2031-06-30 | 2025-10-22 | >= 18 Years | 20 | 12 |
Locations:United States + 1 more
Sweden, United States
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Dose-limiting toxicity resulted in an SAE during 3Q 2025 |
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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 | Liver | 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 | First patient treated early 2024 |
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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 (n=1) | Dose 2: 2E12 vg/kg (n=2) | 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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| NCT05432310 | Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) | Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation | 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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| 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 | Dose 1: Median dose: 7.7E6 CD34+ cells/kg | Dose 2: Dose range: 2.2-12.1E6 CD34+ cells/kg | Phase2, Phase1 | Recruiting | Active | 2018-05-03 | 2038-06 | 2025-01-13 | >= 2 Months | 25 | 1 | United States |
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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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| 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 | Active | 2022-12-07 | 2027-09 | 2024-10-02 | 3 Months - 22 Months | 5 | 1 | United Kingdom | AVROBIO supported this IIT until July 2023, license was returned to University of Manchester |
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| NCT06291935 | Retinitis Pigmentosa | Orphan Drug Designation, Rare Pediatric Disease Designation | VG901 | VeonGen Therapeutics 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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| 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 | 2025-11-06 | 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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| NCT06164730 | Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease | Fast Track | 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 | 2025-11-17 | 18 Years - 70 Years | 36 | 14 |
Locations:Australia + 4 more
Australia, Canada, Israel, New Zealand, United Kingdom
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US12029795B2; US20240010609A1; US20240131166A1; US20240011023A1 | Verve was acquired by Lilly in June 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 | 2027-12 | 2025-11-28 | 18 Years - 70 Years | 36 | 10 |
Locations:Australia + 3 more
Australia, Canada, South Africa, United Kingdom
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US20240010609A1; US20230340435A1; WO2023049299A2 | First patient dosed November 2024, expected update 2H 2025 |
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| NCT04537377 | Wilson Disease | Fast Track, 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 | 2025-05-29 | 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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| 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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| 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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| 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 | Active not recruiting | Active | 2023-10-29 | 2029-11-13 | 2025-06-18 | 5 Years - 10 Years | 3 | 1 | China | Investigator-initiated trial conducted at West China Hospital |
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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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| 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 | Recruiting | Active | 2025-01-23 | 2027-06 | 2025-04-27 | 18 Years - 79 Years | 24 | 1 | United States |
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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 | 2025-07-25 | >= 6 Months | 24 | 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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| NCT06066008 | X-Linked Retinoschisis | ZM-01 | Zhongmou Therapeutics | Industry | RS1 | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV2/8 | 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 | |||||||
| 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 | 2025-12-04 | 18 Years - 65 Years | 12 | 1 | China | Two patients treated early 2024 |
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| NCT03328130 | Retinitis Pigmentosa | Rare Pediatric Disease Designation | 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 (n=7) | Dose 2: 6.4E11 vg/eye (n=10) | Phase2, Phase1 | Recruiting | Active | 2017-10-05 | 2029-12 | 2024-03-07 | >= 13 Years | 23 | 1 | France |
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| 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 | 2025-11-14 | 24 Hours - 7 Months | 8 | 10 |
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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