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 141 results in ClinicalTrials
SCGE Platform Gene Therapy Clinical Trials downloaded on: 2025/12/16 03:07:31; 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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 | |||||
| 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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| 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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| 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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| 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 | ||||||
| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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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| 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 | |||||||
| 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 | ||||||
| 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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| 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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| 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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| 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 | ||||
| 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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| 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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| 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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| 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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| 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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| 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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