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.
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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 |
| PFU | Plaque forming units |
| RNP | Ribonucleoprotein |
| RV | Retrovirus |
| 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. |
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 |
| 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 |
| 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 |
| Route of Administration | How the drug product is introduced to the body |
| 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 |
| Status | Indicates the current recruitment status or the expanded access status |
| Study Status | Indicates the current recruitment status or the expanded access status |
| 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 | 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 | Status | Submit Date > | Completion Date | Last_Update | Eligibility Age | Enrollment Count | No. of Trial Sites | Locations | Has US IND | Patents | Recent Updates | Resources/Links |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Trial ID | Indication | 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 | Status | Submit Date > | Completion Date | Last_Update | Eligibility Age | Enrollment Count | No. of Trial Sites | Locations | Has US IND | Patents | Recent Updates | Resources/Links |
| NCT04517149 | X-Linked Retinitis Pigmentosa | 4D-125 | 4D Molecular Therapeutics | Industry | RPGR | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV | Dose 1: 3E11 vg/eye | Dose 2: 1E12 vg/eye | Phase2, Phase1 | Active not recruiting | 2020-08-14 | 2029-05 | 2024-01-12 | >= 12 Years | 21 | 7 | United States | US11613766B2; US20220290181A1 | January 2025: Sponsor announced they would terminate development of this program | ||||
| NCT05930561 | Diabetic Macular Edema, Diabetic Retinopathy | 4D-150 | 4D Molecular Therapeutics | Industry | MiVEGFC | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV | Dose 1: Dose range: 5E9 -3E10 vg/eye | Dose 2: Planned Phase 3 dose: 3E10 vg/eye | Phase2 | Recruiting | 2023-06-26 | 2028-07-01 | 2024-12-04 | >= 18 Years | 72 | 15 |
Locations:United States + 1 more
Puerto Rico, United States
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52-week interim data update expected at a scientific conference in mid-2025 |
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| NCT05248230 | Cystic Fibrosis Lung | 4D-710 (A101-CMV173-coCFTRΔR) | 4D Molecular Therapeutics | Industry | CFTR | Gene transfer | In-vivo | Functional gene replacement | Inhalational | Viral vector | Airway epithelial cells | Viral transduction | AAV A101 | Dose 1: 2.5E14 vg | Dose 2: 5E14 vg | Dose 3: 1E15 vg (maximum tolerated dose) | Dose 4: 2E15 vg (discontinued due to high transduction to interstitium) | Phase2, Phase1 | Recruiting | 2022-02-10 | 2030-01 | 2024-10-30 | >= 18 Years | 40 | 16 | United States | US11499166B2; WO2023172873A2 | Phase 1 AEROW enrollment completed in November 2024 (Cohorts 3 & 4 fully enrolled with n=3 each), follow-up ongoing; Interim data update expected to be presented in mid-2025 |
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| NCT04483440 | Choroideremia | 4D-110 | 4D Molecular Therapeutics | Industry | CHM | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV R100 | Dose 1: 3E11 vg/eye | Dose 2: 1E12 vg/eye | Phase1 | Active not recruiting | 2020-07-20 | 2027-06 | 2024-05-09 | >= 18 Years | 13 | 2 | United States | January 2025: Sponsor announced they would terminate development of this program |
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| NCT04519749 | Fabry Disease | 4D-310 | 4D Molecular Therapeutics | Industry | GLA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Cardiomyocyte | Viral transduction | AAV C102 | 1E13 vg/kg | Phase2, Phase1 | Active not recruiting | 2020-08-14 | 2030-06 | 2024-04-08 | >= 18 Years | 18 | 4 | United States | WO2021222094A1 | No further significant investment is expected on this program, pending additional financing or partnerships |
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| NCT05197270 | Neovascular (Wet) Age-Related Macular Degeneration | 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 | 2022-01-05 | 2026-11 | 2024-11-05 | >= 50 Years | 215 | 24 |
Locations:United States + 1 more
Puerto Rico, United States
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US11766489B2; US11613766B2; US20240226336A9 | Phase III trial to begin Q1 2025 |
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| NCT04676048 | Hemophilia A | ASC618 (HCB promotor) | ASC Therapeutics | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/8 | Undisclosed dose 1 | Phase2, Phase1 | Recruiting | 2020-12-15 | 2026-12 | 2023-02-01 | >= 18 Years | 12 | 1 | United States | First patient dosed January 2024 |
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| NCT04704921 | AMD, nAMD, Wet Age-related Macular Degeneration, wAMD, Wet AMD, CNV, Neovascular AMD, Neovascular Age-related Macular Degeneration, Choroidal Neovascularization | 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 | Phase3, Phase2 | Recruiting | 2021-01-08 | 2026-05 | 2025-01-29 | 50 Years - 89 Years | 540 | 91 | United States | US20230057519A1; US20200093939A1; | Pivotal data expected 2026 |
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| NCT02556736 | Advanced Retinitis Pigmentosa | RST-001 (AGN-151597) | AbbVie | Industry | ChR2 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Retinal ganglion | Viral transduction | AAV2.7m8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Completed | 2015-09-21 | 2024-10-21 | 2024-11-08 | >= 18 Years | 14 | 4 | United States |
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| NCT05725018 | Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, RDEB | Prademagene zamikeracel (EB-101) | Abeona Therapeutics, Inc | Industry | COL7A1 | Gene transfer | Ex-vivo | Functional gene replacement | Skin graft | Autologous cells | Keratinocytes | Viral transduction | LZRSE | Transduced 35cm^2 keratinocyte sheets, up to 6 wound sites | Phase3 | Recruiting | 2023-01-23 | 2025-06-30 | 2024-06-27 | >= 12 Months | 12 | 2 | United States | US20240067926A1; US20230045590A1 | BLA resubmission October 2024, new PDUFA date pending |
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| NCT04783181 | Congenital Adrenal Hyperplasia | BBP-631 (AAV5-CYP21A2) | Adrenas Therapeutics Inc | Industry | CYP21A2 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV5 | Dose 1: 1.5E13 vg/kg | Dose 2: 3.0E13 vg/kg | Dose 3: 6.0E13 vg/kg | Phase2, Phase1 | Active not recruiting | 2021-03-01 | 2029-02 | 2024-09-26 | >= 18 Years | 8 | 5 | United States | Bridge Bio is cutting this program, lack of sufficient efficacy |
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| NCT05536973 | Neovascular Age-related Macular Degeneration | Ixoberogene soroparvovec (ADVM-022) | Adverum Biotechnologies, Inc. | Industry | Codon optimized aflibercept | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Intravitreal | Viral vector | Viral transduction | AAV.7m8 | Dose 1: 6E10 vg/eye (selected as pivotal dose) | Dose 2: 2E11 vg/eye | Phase2 | Active not recruiting | 2022-09-08 | 2028-11 | 2024-01-08 | >= 50 Years | 69 | 39 |
Locations:United States + 2 more
France, United Kingdom, United States
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US20220265740A1; US20230322911A1 | H1 2025: Initiation of Phase III trial |
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| NCT05821959 | Sensorineural Hearing Loss, Bilateral | AK-OTOF (AAVAnc80-hOTOF) | 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 | 2023-02-08 | 2028-10 | 2025-02-06 | 14 | 6 |
Locations:United States + 2 more
Taiwan, United Kingdom, United States
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US20210017235A1; US11104885B2; US20240011039A1; EP4063510A1 |
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| NCT06467344 | Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration, Stargardt Disease 1 | 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 | 2024-05-31 | 2030-12-01 | 2025-01-22 | >= 18 Years | 13 | 10 | United States | US20240091381A1; WO2020214990A1 | Rare Pediatric Disease designation granted 4/25/24 |
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| NCT03533673 | Pompe Disease | ACTUS-101 (AAV2/8-LSPhGAA) | AskBio Inc | Industry | GAA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Hepatocyte | Viral transduction | AAV2/8 | Dose 1: 1.6E12 vg/kg | Dose 2: Undisclosed dose 2 | Dose 3: Undisclosed dose 3 | Phase2, Phase1 | Active not recruiting | 2018-04-13 | 2026-03 | 2023-06-28 | >= 18 Years | 7 | 1 | United States | US20180371440A1; US20220389399A1; US20230210884A1; | First patient dosed 1/22/19, most recent data presented at ASGCT in May 2022 |
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| NCT05230459 | Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein | AB-1003 (LION-101) | 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 | 2022-01-27 | 2028-12 | 2024-11-29 | 18 Years - 65 Years | 10 | 6 | United States | US20200061092A1; US20190008881A1 | First patient dosed (8/3/23) |
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| NCT06285643 | Parkinson Disease | AB-1005 (AAV2-GDNF) | AskBio Inc | Industry | GDNF | Gene transfer | In-vivo | Overexpression of protective allele/gene | CED | Viral vector | Viral transduction | AAV2 | Up to 1.8mL (3.3E12 vg/ml/gadoteridol 2mM co-infusion) | Phase2 | Recruiting | 2024-02-14 | 2027-11-30 | 2025-02-17 | 45 Years - 75 Years | 87 | 26 | United States | US20180140672A1; US20180344199A1; WO2023183594A2 | Received FDA Fast Track designation |
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| NCT05598333 | Congestive Heart Failure | AB-1002 (BNP116.sc-CMV.I1c) | AskBio Inc | Industry | PPP1R1A | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarterial | Viral vector | Cardiomyocyte | Viral transduction | AAV2i8 | Dose 1: 3.25E13 vg | Dose 2: 6.5E13 vg | Phase2 | Recruiting | 2022-10-25 | 2030-12-31 | 2024-10-10 | >= 18 Years | 150 | 28 | United States | US20230340528A1; AU2021320244A1 | Received Fast Track designation 4/18/24 |
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| NCT04998396 | Canavan Disease | BBP-812 (AAV9-ASPA) | Aspa Therapeutics | Industry | ASPA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Dose 3: Undisclosed expansion dose | Phase2, Phase1 | Recruiting | 2021-08-05 | 2030-10-08 | 2024-10-22 | <= 30 Months | 26 | 4 | United States | High dose enrollment in progress, completion date TBA |
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| NCT03964792 | Sickle Cell Disease | DREPAGLOBE | Assistance Publique - Hôpitaux de Paris | Other | HBB | Gene transfer | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells (range: 6 - 8.1E6 cells/kg) | Phase2, Phase1 | Active not recruiting | 2019-05-14 | 2024-01 | 2024-01-09 | 12 Years - 20 Years | 6 | 1 | France | US20200190536A1 | Uses the same βAS3-globin as NCT02247843; vector performed poorly |
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| NCT05071222 | Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency | EF1a-hArtemis LV (modified EF1a promotor) 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 | 2021-08-27 | 2041-07-19 | 2023-11-27 | <= 47 Months | 5 | 1 | France |
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| NCT03199469 | X-Linked Myotubular Myopathy | Resamirigene bilparvovec (AT132), rAAV8-des-hMTM1 | Astellas Gene Therapies | Industry | MTM1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: 1.3E14 vg/kg | Dose 2: 3.5E14 vg/kg | Phase3, Phase2 | Active not recruiting | 2017-06-21 | 2030-03-31 | 2024-12-03 | <= 5 Years | 27 | 6 |
Locations:United States + 3 more
Canada, France, Germany, United States
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US20220411819A1; WO2023196853A1; WO2022251208A1 | On clinical hold since 2021 |
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| NCT06483802 | Friedreich Ataxia, Cardiomyopathy | ASP2016 | Astellas Gene Therapies | Industry | FXN | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Undisclosed dose escalation | Phase1 | Suspended | 2024-06-26 | 2031-01-31 | 2025-01-08 | 18 Years - 40 Years | 14 | 5 | United States | WO2023044424A1; AR127081A1; EP4401756A1 | Sponsor announced termination of the program February 2025, no patients were enrolled in the study |
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| NCT04174105 | Pompe Disease (Late-onset) | Zocaglusagene nuzaparvovec (AT845) | Astellas Gene Therapies | Industry | GAA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: 3E13 vg/kg | Dose 2: 6E13 vg/kg | Dose 3: 1E14 vg/kg | Phase2, Phase1 | Recruiting | 2019-11-13 | 2029-11-30 | 2024-11-18 | 18 Years - 80 Years | 18 | 4 |
Locations:United States + 1 more
United Kingdom, United States
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US20210162073A1; US20220411819A1; US20220387562A1; WO2023150688A8 |
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| NCT05973630 | LGMD2C | 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 | 2023-07-24 | 2032-01-31 | 2025-02-14 | 6 Years - 13 Years | 6 | 3 |
Locations:United States + 2 more
France, Italy, United States
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EP4198134A1 | US IND cleared 11/12/24 |
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| NCT05224505 | LGMDR9 | ATA-100 (GNT0006) | Atamyo Therapeutics | Industry | FKRP | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/9 | Dose 1: 9.0E12 vg/kg | Dose 2: 2.7E13 vg/kg | Phase2, Phase1 | Recruiting | 2021-12-15 | 2030-10 | 2024-07-31 | 16 Years - 99 Years | 39 | 3 |
Locations:Denmark + 2 more
Denmark, France, United Kingdom
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US20230321277A1; EP4031673A1; EP4198047A1 | Dose-escalation (Phase 1b) recruitment completed September 2024 |
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| NCT05878860 | X-linked Retinoschisis | ATSN-201 (AAV.SPR-hGRK1-hRS1syn) | Atsena Therapeutics Inc. | Industry | RS1 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV.SPR | Dose 1: 1.5E10 vg/eye | Dose 2: 5.0E10 vg/eye | Dose 3: 1.7E11 vg/eye | Phase2, Phase1 | Recruiting | 2023-05-12 | 2029-10 | 2025-02-19 | >= 6 Years | 21 | 4 | United States | FDA granted Rare Pediatric Disease Designation |
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| NCT03920007 | Leber Congenital Amaurosis, LCA, LCA1 | ATSN-101 (AAV5-hGRK1-hGUCY2D) | Atsena Therapeutics Inc. | Industry | GUCY2D | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/5 | Dose 1: 1.0E10 vg/eye (300ul) | Dose 2: 3.0E10 vg/eye (300ul) | Dose 3: 1.0E11 vg/eye (300ul); expansion dose | Phase2, Phase1 | Active not recruiting | 2019-04-10 | 2027-05-19 | 2024-02-20 | >= 6 Years | 15 | 2 | United States | FDA granted Rare Pediatric Disease Designation, ODD, RMAT |
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| NCT06064890 | Frontotemporal Dementia, FTD, FTD-GRN, Dementia, Frontotemporal | AVB-101 (AAV.PGRN) | 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 | 2023-09-12 | 2030-10-31 | 2025-02-19 | 30 Years - 75 Years | 9 | 13 |
Locations:United States + 5 more
Netherlands, Poland, Spain, Sweden, United Kingdom, United States
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Partnership with Astellas for clinical development of this product announced October 2024 |
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| NCT05241444 | 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 | 2022-01-12 | 2037-02 | 2024-07-08 | 4 Months - 35 Years | 30 | 1 | United States | US20220273712A1; EP3672617A4; US20230183804A1 |
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| NCT03588299 | Hemophilia A | Peboctocogene camaparvovec (DTX201), liver-specific TTR promotor | Bayer | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVhu37 | Dose 1: 0.5E13 GC/kg | Dose 2: 1E13 GC/kg | Dose 3: 2E13 GC/kg | Dose 4: 4E13 GC/kg | Phase2, Phase1 | Active not recruiting | 2018-07-06 | 2026-11-03 | 2025-02-06 | >= 18 Years | 11 | 13 |
Locations:United States + 5 more
Bulgaria, France, Germany, Netherlands, United Kingdom, United States
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| NCT06611436 | Hemophilia B, Severe, Hemophilia B | BE-101 (Padua variant) | 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 | 2024-09-18 | 2027-07 | 2025-02-18 | >= 18 Years | 24 | 2 | United States |
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| NCT04850118 | X-Linked Retinitis Pigmentosa | Laruparetigene zovaparvovec (AGTC-501) | Beacon Therapeutics | Industry | RPGR | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2tYF | 6.8E11 vg/eye | Phase3, Phase2 | Recruiting | 2021-04-05 | 2029-10 | 2025-02-11 | 12 Years - 50 Years | 75 | 21 |
Locations:United States + 2 more
Australia, United Kingdom, United States
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First patient dosed in registrational trial 6/12/24 |
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| NCT05456880 | Sickle Cell Disease | BEAM-101 | Beam Therapeutics Inc. | Industry | HBG1/HBG2 | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Electroporation | none | ABE8 | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | 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 | 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: Undisclosed low dose | Dose 2: Undisclosed intermediate dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | 2024-04-22 | 2027-08 | 2025-01-10 | 18 Years - 70 Years | 106 | 5 |
Locations:Australia + 3 more
Australia, Netherlands, New Zealand, United Kingdom
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US20200399626A1; US20210371858A1; US20230080198A1 | First patient dosed 6/2024, initial clinical data expected 2H 2025 |
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| NCT04737460 | CLN7 | AAV9/CLN7 | Benjamin Greenberg | Other | MFSD8 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | Dose 1: 5E14 vg | Dose 2: 1E15 vg | Phase1 | Active not recruiting | 2021-01-15 | 2029-02-01 | 2024-12-13 | 1 Year - 18 Years | 4 | 1 | United States | WO2020033833A1; US11753655B2 |
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| NCT04323098 | Hemophilia A | ROCTAVIAN/valoctogene roxaparvovec | BioMarin Pharmaceutical | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Hepatocyte | Viral transduction | AAV5 | 6E13 vg/kg | Phase3 | Active not recruiting | 2020-03-24 | 2027-01 | 2024-04-04 | >= 18 Years | 22 | 12 |
Locations:United States + 3 more
Australia, Brazil, Taiwan, United States
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FDA approved 6/29/23, price/treatment $2.9M |
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| NCT04680065 | Multiple System Atrophy | AB-1005 (AAV2-GDNF) | Brain Neurotherapy Bio, Inc. | Industry | GDNF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal | Viral vector | Viral transduction | AAV2 | Undisclosed dose 1 | Phase1 | Recruiting | 2020-12-15 | 2028-12 | 2025-02-10 | 35 Years - 75 Years | 9 | 7 | United States | US20180140672A1; US20180344199A1; WO2023183594A2 | First patient randomized 11/17/23 | ||||
| NCT05541627 | Huntington Disease | AB-1001 (AAVrh10.CAG.hCYP46A1) | Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio) | Industry | CYP46A1 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal | Viral vector | Viral transduction | AAVrh10 | Dose 1: 4E8 vg/uL | Dose 2: 1.1E9 vg/uL | Phase2, Phase1 | Active not recruiting | 2022-09-13 | 2029-12-31 | 2024-08-06 | 18 Years - 65 Years | 5 | 1 | France | WO2012049314A1 | Bayer announced the discontinuation of this program November 2024 |
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| NCT03569891 | Hemophilia B | HEMGENIX/etranacogene dezaparvovec | CSL Behring | Industry | F9R338L | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAV5 | 2E13 vg/kg | Phase3 | Active not recruiting | 2018-06-14 | 2025-03 | 2025-01-07 | >= 18 Years | 67 | 33 |
Locations:United States + 7 more
Belgium, Denmark, Germany, Ireland, Netherlands, Sweden, United Kingdom, United States
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FDA approved 11/22/22; Price/treatment: $3.5M |
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| NCT06458595 | Age-related Macular Degeneration | 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 1 | Phase2, Phase1 | Recruiting | 2024-06-09 | 2026-12-28 | 2024-11-29 | 50 Years - 85 Years | 44 | 1 | China | WO2023236964A1 | |||||
| NCT05657301 | Age-Related Macular Degeneration | 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 | Dose 1: Undisclosed dose 1 | Dose 2: Undisclosed dose 2 | Dose 3: Undisclosed dose 3 | Dose 4: Undisclosed dose 4 | Dose 5: Undisclosed dose 5 | Phase1 | Recruiting | 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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| NCT05761899 | Hereditary Pulmonary Alveolar Proteinosis | Gene-Corrected Macrophages (SIN-LV/EFS/CSF2RA) | 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 | 2023-02-28 | 2038-10-01 | 2025-01-30 | >= 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 | 2023-12-18 | 2029-06 | 2024-07-09 | 7 Years - 18 Years | 6 | 1 | China | WO2022222869A1 |
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| NCT06364774 | Beta-Thalassemia | CHOP-ALS20 (βA-T87Q-globin) | 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 | 2024-03-26 | 2027-12-31 | 2024-12-31 | 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 | 2024-02-23 | 2025-07 | 2024-07-01 | 12 Years - 35 Years | 8 | 3 | China | US11384353B2; WO2020156575 |
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| NCT03311503 | Severe Combined Immunodeficiency, X Linked, Gene Therapy | G2SCID lentiviral vector transduced CD34+ cells (rHIV_IL2RGcoG2SCID) | 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 | 2017-10-12 | 2028-10-01 | 2025-01-06 | 0 Years - 5 Years | 12 | 4 | United States |
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| NCT05353647 | Sickle Cell Disease | Autologous CD34+ HSC cells transduced with the lentiviral vector containing BCL11a-targeted shRNA | David Williams | Other | BCL11A | Gene transfer | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | VSV-G | Transduced CD34+ cells | Phase2 | Active not recruiting | 2022-04-21 | 2026-05 | 2024-12-18 | 13 Years - 40 Years | 25 | 9 | United States |
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| NCT02247843 | Sickle Cell Disease | Lenti/G-βAS3-FB (contains anti-sickling mutations: T87Q, G16D, E22A) | Donald B. Kohn, M.D. | Other | HBB | Gene transfer | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase2, Phase1 | Active not recruiting | 2014-09-20 | 2025-12 | 2024-05-16 | >= 18 Years | 4 | 1 | United States | WO2020168004A1 | Uses the same βAS3-globin as NCT03964792 |
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| NCT05444894 | Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia | Renizgamglogene autogedtemcel (EDIT-301) | Editas Medicine, Inc. | Industry | HBG1/HBG2 | Gene editing | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | ASCas12a mRNA | Dose 1: Min: 5.7E6 CD34+ cells/kg | Dose 2: Max: 11.9E6 CD34+ cells/kg | Phase2, Phase1 | Recruiting | 2022-06-27 | 2025-12 | 2024-01-29 | 18 Years - 35 Years | 9 | 8 |
Locations:United States + 1 more
Canada, United States
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WO2017160890A1 | RMAT designation in 2023 | ||
| NCT04853576 | Sickle Cell Disease, Hemoglobinopathies | Renizgamglogene autogedtemcel (EDIT-301) | Editas Medicine, Inc. | Industry | HBG1/HBG2 | Gene editing | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | ASCas12a | Dose 1: Min: 2.9E6 CD34+ cells/kg | Dose 2: Max: 10.0E6 CD34+ cells/kg | Phase2, Phase1 | Active not recruiting | 2021-04-16 | 2025-08 | 2025-01-31 | 12 Years - 50 Years | 45 | 24 |
Locations:United States + 1 more
Canada, United States
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US12031132B2; US20200299661A1; | Paused development and initiated efforts to outlicense reni-cel; possible BLA submission by Q1 2025 |
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| NCT05518188 | Spasticity, Muscle, Microcephaly, Intellectual Deficiency, Growth Retardation, SPG50, Spastic Paraplegia | MELPIDA (scAAV9-AP4M1) | Elpida Therapeutics SPC | Industry | AP4M1 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | 1E15 vg | Phase2, Phase1 | Recruiting | 2022-08-24 | 2030-10-01 | 2024-10-08 | 4 Months - 10 Years | 4 | 1 | United States | WO2022226183A3 | Phase III Study approved, begins August 2024 |
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| NCT05419492 | Dravet Syndrome | ETX101 (rAAV9-reGABA-eTFSCN1A) | 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 | 2022-06-10 | 2031-04 | 2025-01-31 | 6 Months - 47 Months | 22 | 3 | United States | US20230203531A1; US20200397917A1; US20220136009A1; US20220168449A1; US20220170910A1 | Dosing and preliminary safety and efficacy data expected in 2H25 |
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| NCT06399107 | Sickle Cell Disease, Sickle-Cell Disease With Crisis | BAH243 | Essen Biotech | Other | HBB | Gene transfer | Ex-vivo | Overexpression of protective allele/gene | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | 2024-04-29 | 2025-12-28 | 2024-11-05 | 2 Years - 90 Years | 85 | 1 | China |
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| NCT05144386 | HIV-1-infection | EBT-101 (AAV9-SaCas9-GagD-LTR1) | Excision BioTherapeutics | Industry | HIV genes | Gene editing | In-vivo | Gene excision | Intravenous | Viral vector | Lymphoid tissues | Viral transduction | AAV9 | Cas9 | Dose 1: 0.9E12 vg/kg | Dose 2: 3.0E12 vg/kg | Phase1 | Completed | 2021-11-22 | 2024-11-14 | 2024-12-02 | 18 Years - 70 Years | 6 | 3 | United States | US20190367924A1; US20200392487A1; US20240139294A1 | Safe, but does not provide long-term viral suppression |
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| NCT05903794 | Neovascular (Wet) Age-related Macular Degeneration (nAMD) | EXG102-031 | Exegenesis Bio | Industry | ABD-VEGFR | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: Undisclosed dose 1 | Dose 2: Undisclosed dose 2 | Phase1 | Recruiting | 2023-06-06 | 2025-12-31 | 2024-09-19 | >= 50 Years | 12 | 2 | United States | US20240190943A1 | FDA cleared IND 1/18/23 |
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| NCT04418414 | Hemophilia A | CD68-ET3 | Expression Therapeutics, LLC | Industry | F8 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase1 | Not yet recruiting | 2020-05-28 | 2039-08 | 2024-02-20 | >= 18 Years | 7 | 0 | WO2022165390A1 |
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| NCT05762510 | Transfusion Dependent Beta-Thalassemia | 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 | 2022-01-06 | 2030-10-31 | 2023-05-09 | 5 Years - 35 Years | 5 | 1 | China |
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| NCT05757245 | Transfusion-dependent α-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 | 2023-02-24 | 2030-12-31 | 2023-04-18 | 5 Years - 35 Years | 5 | 1 | China |
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| NCT03173521 | Mucopolysaccharidosis Type VI | AAV2/8.TBG.hARSB | Fondazione Telethon | Other | ARSB | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/8 | Dose 1: 6E11 gc/kg | Dose 2: 2E12 gc/kg | Dose 3: 6E12 gc/kg | Phase2, Phase1 | Completed | 2017-04-12 | 2024-07-16 | 2024-11-22 | 4 Years - 65 Years | 9 | 2 |
Locations:Italy + 1 more
Italy, Turkey
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US20190343929A1 |
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| NCT03837483 | Wiskott-Aldrich Syndrome | Etuvetidigene autotemcel (formerly OTL-103) | Fondazione Telethon | Other | WAS | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells (4.4 - 14.5E6 cells/kg) | Phase3 | Active not recruiting | 2019-02-08 | 2027-09 | 2024-01-31 | <= 65 Years | 10 | 2 |
Locations:United States + 1 more
Italy, United States
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EP3973996A1 | Telethon intends to seek regulatory approval in Europe |
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| NCT05739643 | Krabbe Disease | FBX-101 | Forge Biologics, Inc | Industry | GALC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh10 | Dose 1: 1.6E13 gc/kg | Dose 2: Undisclosed dose 2 | Phase2, Phase1 | Active not recruiting | 2023-02-13 | 2026-11 | 2025-01-29 | <= 18 Years | 9 | 3 | United States | WO2018136710; WO2020132385A1 | Dose escalation to highest dose approved, will start 2Q24 |
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| NCT06492863 | Neovascular Age-related Macular Degeneration | 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 | 2024-07-01 | 2028-10-15 | 2024-07-09 | 50 Years - 80 Years | 78 | 1 | China | US20230295243A1 | FDA clearance for Phase 2 trial received 11/11/24 | ||||
| NCT05858983 | Biallelic RPE65 Mutation-associated Retinal Dystrophy | FT-001 (AAV2-hRPE65) | 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 | 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) | FT-002 (AAV5-GRK1-hRPGRORF15) | 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 | 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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| 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 | 2024-07-01 | 2028-11-15 | 2024-07-09 | 18 Years - 74 Years | 78 | 1 | China | US20230295243A1 | IND cleared 12/30/24 | ||||
| NCT03326336 | Non-syndromic Retinitis Pigmentosa | GS030 (rAAV2.7m8-CAG-ChrimsonR-tdTomato) | GenSight Biologics | Industry | ChR-tdT | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Retinal ganglion | Viral transduction | AAV2.7m8 | Dose 1: 5E10 vg/eye | Dose 2: 1.5E11 vg/eye | Dose 3: 5E11 vg/eye | Phase2, Phase1 | Recruiting | 2017-10-11 | 2025-12 | 2022-07-26 | 18 Years - 75 Years | 15 | 3 |
Locations:United States + 2 more
France, United Kingdom, United States
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US20240238613A1; US20240165198A1 |
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| NCT02652767 | Optic, Atrophy, Hereditary, Leber | LUMEVOQ (lenadogene nolparvovec) | GenSight Biologics | Industry | ND4G11778A | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV2 | 9E10 vg in 90uL | Phase3 | Completed | 2016-01-07 | 2019-07-04 | 2022-07-29 | >= 15 Years | 39 | 7 |
Locations:United States + 4 more
France, Germany, Italy, United Kingdom, United States
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WO2019241206A1 | EAP due to commence Q3 2024, France only; MAA in UK goal in 2026 |
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| NCT05835895 | Osteoarthritis, Knee, Osteo Arthritis Knee, Knee Osteoarthritis | GNSC-001 (scAAV2.5-CMV-IL1Ra) | Genascence Corporation | Industry | IL1RN | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarticular | Viral vector | Viral transduction | AAV2.5 | Dose 1: 1E11 vg/knee | Dose 2: 1E12 vg/knee | Dose 3: 1E13 vg/knee | Phase1 | Active not recruiting | 2023-04-19 | 2029-05 | 2024-12-27 | 40 Years - 75 Years | 67 | 9 | United States | US20220016213A1 | Initial data for Phase 1b trial expected Q4 2024 |
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| NCT06199531 | NGLY1 Deficiency | 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 | 2023-11-21 | 2028-01-31 | 2024-06-04 | 2 Years - 18 Years | 12 | 2 | United States | EP4329824A1 | Selected for START program (6/3/24) |
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| NCT05207657 | P47-Phox, Deficiency of | PCHIM-p47 | Great Ormond Street Hospital for Children NHS Foundation Trust | Other | NCF1 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | 2021-06-24 | 2029-04-01 | 2023-05-16 | >= 23 Months | 5 | 1 | United Kingdom |
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| NCT06178432 | Pompe Disease (Late-onset) | BBM-G102 | Huashan Hospital | Other | GAA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV | Undisclosed dose 1 | Early phase1 | Not yet recruiting | 2023-11-30 | 2028-12 | 2023-12-21 | >= 18 Years | 6 | 1 | China |
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| NCT06031727 | Neovascular Age-related Macular Degeneration(nAMD) | HG202 | HuidaGene Therapeutics Co., Ltd. | Industry | VEGFA mRNA | Gene editing | In-vivo | Gene inactivation | Subretinal | Viral vector | Viral transduction | AAV | hfCas13Y | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Early phase1 | Recruiting | 2023-09-01 | 2025-06-27 | 2024-11-26 | 50 Years - 80 Years | 12 | 2 | China | WO2023185878A1 | Preclinical/proof-of-concept data presented at ASGCT 2024 |
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| NCT05906953 | Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations | 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 | 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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FDA granted RPDD on 8/7/23 |
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| NCT05454566 | Osteoarthritis, Knee | ICM-203 (hNkx3.2-D2 CDS) | ICM Co. Ltd. | Industry | NKX3-2 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarticular | Viral vector | Viral transduction | AAV5.2 | Dose 1: 6E12 vg | Dose 2: 2E13 vg | Dose 3: 6E13 vg | Phase2, Phase1 | Not yet recruiting | 2022-07-04 | 2026-11 | 2024-10-15 | 50 Years - 80 Years | 18 | 0 | Update on Phase 1/2a study given at ASGCT 2024 |
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| NCT06289452 | Retinoschisis, Retinal Disease, Retinal Degeneration, Eye Diseases | IVB102 | InnoVec Biotherapeutics Inc. | Industry | RS1 | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Early phase1 | Recruiting | 2024-02-25 | 2029-12-31 | 2024-05-07 | >= 8 Years | 18 | 1 | China | WO2023143606A1 |
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| NCT06196840 | Neovascular Age-Related Macular Degeneration | LX102 | Innostellar Biotherapeutics Co.,Ltd | Industry | VEGF-trap | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: 2E10 vg | Dose 2: Undisclosed dose 2 | Phase2 | Recruiting | 2023-12-25 | 2029-05 | 2024-01-09 | 50 Years - 89 Years | 50 | 3 | China |
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| NCT06196827 | Inherited Retinal Dystrophy Associated With RPE65 Mutations | LX101 | Innostellar Biotherapeutics Co.,Ltd | Industry | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: Undisclosed low dose (0.3mL/eye) | Dose 2: Undisclosed high dose (0.3mL/eye) | Phase2, Phase1 | Active not recruiting | 2023-12-25 | 2027-12 | 2024-01-09 | >= 6 Years | 9 | 2 | China | US11970519B2 |
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| NCT04186650 | Epidermolysis Bullosa Dystrophica, Recessive | EF1a-COL7A1-SIN retroviral vector transduced autologous skin | Institut National de la Santé Et de la Recherche Médicale, France | Other gov | COL7A1 | Gene transfer | Ex-vivo | Functional gene replacement | Skin graft | Autologous cells | Keratinocytes | Viral transduction | RV | Up to 6 grafts of 50 cm^2 each | Phase2, Phase1 | Active not recruiting | 2019-11-25 | 2027-06-09 | 2025-01-29 | >= 18 Years | 3 | 1 | France | EP2766480B1; WO2022122900A1 |
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| 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 | Not yet recruiting | 2022-11-25 | 2028-12 | 2023-02-09 | >= 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 | Not yet recruiting | 2024-01-26 | 2030-01-31 | 2024-02-02 | >= 18 Years | 6 | 1 | China | CN116715752A |
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| NCT06622668 | Lung Disease, Pulmonary Disease, AATD, Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease | NTLA-3001 | Intellia Therapeutics | Industry | SERPINA1 | Gene editing | In-vivo | Functional gene replacement | Intravenous | MRNA, LNP | Liver | Lipid encapsulation | LNP | Cas9 mRNA | Dose 1: Undisclosed low dose | Dose 2: Undisclosed intermediate dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Withdrawn | 2024-09-30 | 2025-01-09 | 2025-01-17 | 18 Years - 75 Years | 0 | 1 | New Zealand |
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| NCT05120830 | Hereditary Angioedema | NTLA-2002 | Intellia Therapeutics | Industry | KLKB1 | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Hepatocyte | Lipid encapsulation | LDLR | Cas9 mRNA | Dose 1: 25 mg | Dose 2: 50 mg (pivotal dose) | Dose 3: 75 mg | Phase2, Phase1 | Active not recruiting | 2021-11-03 | 2026-03-31 | 2024-09-19 | >= 18 Years | 37 | 9 |
Locations:Australia + 5 more
Australia, France, Germany, Netherlands, New Zealand, United Kingdom
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US20230203480A1; US20240018496A1; WO2024011206A1 | BLA submission planned 2026; Phase II study met all primary and secondary endpoints, selected pivotal dose |
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| NCT06634420 | Hereditary Angioedema | NTLA-2002 | Intellia Therapeutics | Industry | KLKB1 | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Hepatocyte | Lipid encapsulation | LDLR | Cas9 mRNA | 50 mg | Phase3 | Recruiting | 2024-10-07 | 2027-09 | 2025-02-14 | >= 18 Years | 60 | 8 |
Locations:United States + 2 more
Canada, United Kingdom, United States
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US20230203480A1; US20240018496A1; WO2024011206A1 | BLA submission planned 2026; Phase II study met all primary and secondary endpoints, selected pivotal dose |
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| NCT06128629 | Transthyretin Amyloidosis (ATTR) with Cardiomyopathy | Nexiguran ziclumeran (NTLA-2001) | Intellia Therapeutics | Industry | TTR | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Hepatocyte | Lipid encapsulation | LNP | Cas9 mRNA | 55mg | Phase3 | Recruiting | 2023-11-08 | 2028-04 | 2025-02-05 | 18 Years - 90 Years | 765 | 84 |
Locations:United States + 19 more
Argentina, Australia, Canada, Denmark, France, Germany, Hungary, Israel, Italy, Korea, Netherlands, New Zealand, Portugal, Republic of, Singapore, Spain, Sweden, Taiwan, United Kingdom, United States
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US20230257747A1; WO2017173054A1; US20240076636A1; US20230287400A1; US20210087568A1; WO2020219876A1; US20220009878A1 | Initiate Phase III study for ATTRv-PN by year end |
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| NCT05811351 | Geographic Atrophy | JNJ-1887 (AAVCAGsCD59) | Janssen Research & Development, LLC | Industry | CD59 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Viral transduction | AAV2 | Dose 1: 3.56E11 vg/eye | Dose 2: 1.071E12 vg/mL | Phase2 | Active not recruiting | 2023-03-07 | 2026-02-26 | 2025-01-10 | >= 60 Years | 305 | 162 |
Locations:United States + 16 more
Australia, Belgium, Canada, Czechia, Denmark, Germany, Hungary, Italy, Netherlands, Poland, Portugal, Spain, Sweden, Switzerland, Turkey, United Kingdom, United States
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WO2023089564A1 |
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| NCT04671433 | X-Linked Retinitis Pigmentosa | Botaretigene sparoparvovec (AAV2/5-RPGR) | Janssen Research & Development, LLC | Industry | RPGR | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/5 | Dose 1: 2.0E10 vg/mL | Dose 2: 2.0E11 vg/mL | Dose 3: 2.0E12 vg/mL | Phase3 | Completed | 2020-11-05 | 2024-09-30 | 2025-01-09 | >= 3 Years | 97 | 27 |
Locations:United States + 10 more
Belgium, Canada, Denmark, France, Israel, Italy, Netherlands, Spain, Switzerland, United Kingdom, United States
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WO2024079655A1; US11045558B2 | Phase 3 dosing complete, potential BLA filing in 2025 |
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| NCT04819841 | Sickle Cell Disease | KMAU-001 (nulabeglogene autogedtemcel) | 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 | 2021-03-24 | 2027-07-31 | 2024-10-09 | 12 Years - 40 Years | 15 | 3 | United States | US11851652B2 | Product was previously developed by Graphite Bio |
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| NCT06280378 | Transfusion-dependent Beta-Thalassemia | KL003 (βA-T87Q-globin) | 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 | Not yet recruiting | 2024-02-20 | 2027-05 | 2024-02-28 | 3 Years - 35 Years | 41 | 2 | China | Received NMPA approval on 1/3/24 |
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| NCT04917874 | Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa | VYJUVEK/beremagene geperpavec | Krystal Biotech, Inc. | Industry | COL7A1 | Gene transfer | In-vivo | Functional gene replacement | Topical | Viral vector | Viral transduction | HSV1 | Dose 1: Maximum weekly dose: 1.6E9 PFU (6 months to < 3 years) | Dose 2: Maximum weekly dose: 3.2E9 PFU (over 3 years old) | Phase3 | Completed | 2021-06-02 | 2023-07-31 | 2024-04-09 | >= 2 Months | 47 | 6 | United States | FDA approved 5/19/23, estimated price $600K/year | |||||
| NCT05504837 | Cystic Fibrosis | KB-407 | Krystal Biotech, Inc. | Industry | CFTR | Gene transfer | In-vivo | Functional gene replacement | Inhalational | Viral vector | Viral transduction | HSV-1 | Dose 1: Undisclosed dose (single administration) | Dose 2: Undisclosed dose (2 administrations) | Dose 3: Undisclosed dose (4 administrations) | Phase1 | Recruiting | 2022-08-15 | 2025-07 | 2025-02-04 | >= 18 Years | 12 | 3 | United States | WO2020163703A1; WO2021127524A1 | Completed dosing in first two cohorts and on track to start third in Q2 2024 |
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| NCT06049082 | Alpha 1-Antitrypsin Deficiency | 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 | 2023-09-15 | 2026-05 | 2024-12-16 | 18 Years - 70 Years | 12 | 2 | United States | Phase I Interim data expected 2H 2024 | ||||
| NCT05735158 | Autosomal Recessive Ichthyosis | KB-105 | Krystal Biotech, Inc. | Industry | TGM1 | Gene transfer | In-vivo | Functional gene replacement | Topical | Viral vector | Viral transduction | HSV-1 | Undisclosed single dose formulated as a topical gel, weekly application | Phase2 | Unknown | 2023-02-09 | 2024-04 | 2023-02-23 | >= 6 Months | 15 | 1 | United States | WO2019200163A1; EP3377637B1; US11717547B2; US20200101123A1 | Initiate Phase 2 cohort in 2024 |
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| NCT02852213 | 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 | 2016-07-20 | 2031-07 | 2024-12-20 | >= 24 Months | 42 | 3 | United States | US20230330267A1 |
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| NCT06109181 | Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC | LX2020 | Lexeo Therapeutics | Industry | PKP2 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh10 | Starting dose: 2.0E13 gc/kg | Phase2, Phase1 | Recruiting | 2023-10-25 | 2027-02 | 2025-01-23 | 18 Years - 65 Years | 10 | 5 | United States | FTD and ODD granted 12/2023; cohort 1 interim data expected 2H 2024 |
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| NCT05445323 | Friedreich Ataxia, Cardiomyopathy, Secondary | LX2006 | Lexeo Therapeutics | Industry | FXN | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh10 | Dose 1: 1.8E11 vg/kg | Dose 2: 5.6E11 vg/kg | Dose 3: 1.2E12 vg/kg | Phase2, Phase1 | Active not recruiting | 2022-06-23 | 2029-09 | 2024-11-15 | 18 Years - 50 Years | 8 | 3 | United States | WO2023150563A1 | Fast Track designation granted 4/16/24; update given at ASGCT 2024 |
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| NCT03634007 | Alzheimer Disease, Early Onset Alzheimer Disease | LX1001 (AAVrh.10hAPOE2-HA) | Lexeo Therapeutics | Industry | APOE2 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intrathecal | Viral vector | Viral transduction | AAVrh10 | Dose 1: 1.4E10 gc/mL CSF | Dose 2: 4.4E10 gc/mL CSF | Dose 3: 1.4E11 gc/mL CSF | Dose 4: 1.4E14 gc (fixed dose) | Phase2, Phase1 | Completed | 2018-08-03 | 2024-11-07 | 2024-11-22 | >= 50 Years | 15 | 4 | United States | WO2021108809A1; WO2024011237A1; WO2021076941A1; WO2021022208A1 | Interim Phase 1/2 data readout (all cohorts) 2H 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 | 2021-06-21 | 2027-10-01 | 2024-08-27 | 50 Years - 80 Years | 12 | 2 | United States | US6683058B1; US20030124095A1; WO2023196575A1 | Surgical treatment of the AD patients will be complete in December 2024, and the MCI cohort is expected be completed in 2025 |
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| NCT04774536 | Sickle Cell Disease | CRISPR_SCD001 | Mark Walters, MD | Other | HBB | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Electroporation | none | Cas9 mRNA | Transduced CD34+ cells (range: 3 - 20E6 cells/kg) | Phase2, Phase1 | Recruiting | 2021-02-17 | 2029-03-01 | 2024-09-20 | 12 Years - 35 Years | 9 | 2 | United States |
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| NCT03818763 | Hemophilia a | Pleightlet (Auto CD34+PBSCs -889ITGA2B-BDDFVIII-WPTS(MUT6)(VSVg)) | Medical College of Wisconsin | Other | F8 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | Megakaryocytes | Viral transduction | VSV-G | Transduced CD34+ cells (not to exceed 20ml/kg body weight) | Phase1 | Recruiting | 2019-01-15 | 2033-05-01 | 2025-02-07 | >= 18 Years | 5 | 1 | United States | WO2014066663A1 | First patient enrolled in March 2022, only 2 patients dosed so far 8/16/24 |
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| NCT03001310 | Achromatopsia | Entacingene turiparvovec (AAV8-hCARp.hCNGB3) | MeiraGTx UK II Ltd | Industry | CNGB3 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Cone cells | Viral transduction | AAV2/8 | Dose 1: 0.1E12 vg/mL; 0.5 mL | Dose 2: 0.3E12 vg/mL; 0.5mL | Dose 3: 0.6E12 vg/mL; 0.5mL | Dose 4: 1.0E12 vg/mL; 0.5mL | Phase2, Phase1 | Completed | 2016-11-22 | 2019-10-25 | 2023-03-08 | >= 3 Years | 23 | 2 |
Locations:United States + 1 more
United Kingdom, United States
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| NCT02781480 | Leber Congenital Amaurosis | Cevaretigene ritoparvovec (AAV2/5-RPE65P/OPTIRPRE65) | MeiraGTx UK II Ltd | Industry | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/5 | Dose 1: 1.0E11 vg/mL | Dose 2: 3.0E11 vg/mL | Dose 3: 1.0E12 vg/mL | Phase2, Phase1 | Completed | 2016-04-28 | 2018-12 | 2021-07-12 | >= 3 Years | 15 | 2 |
Locations:United States + 1 more
United Kingdom, United States
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WO2024079655A1 |
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| NCT03758404 | Achromatopsia | Aguracingene cadoparvovec/AAV-CNGA3 (AAV8-hG1.7p.co.CNGA3) | MeiraGTx UK II Ltd | Industry | CNGA3 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Cone cells | Viral transduction | AAV2/8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed intermediate dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Completed | 2018-11-27 | 2021-06-10 | 2022-12-01 | >= 3 Years | 11 | 2 |
Locations:United States + 1 more
United Kingdom, United States
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| NCT05603312 | Parkinson Disease | AAV-GAD | MeiraGTx, LLC | Industry | GAD1/2 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal | Viral vector | Viral transduction | AAV2 | Dose 1: 7.0E10 vg | Dose 2: 21E10 vg | Phase2, Phase1 | Completed | 2022-10-12 | 2024-09-06 | 2024-10-08 | 25 Years - 85 Years | 14 | 6 | United States | WO2024079655A1; WO2021119615A1; US8017385B2 | Initiation of Phase III design discussions in H2 2024 |
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| NCT05926765 | Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands | 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 | 2023-06-22 | 2025-07 | 2024-10-08 | >= 18 Years | 120 | 22 |
Locations:United States + 2 more
Canada, United Kingdom, United States
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WO2024079655A1; WO2024079657A1 | FDA in support of Phase II study as pivotal for BLA submission |
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| NCT04833907 | Canavan Disease | MYR101 (rAAV-Olig001-ASPA) | Myrtelle Inc. | Industry | ASPA | Gene transfer | In-vivo | Functional gene replacement | Intracerebroventricular | Viral vector | Oligodendrocytes | Viral transduction | AAVOlig001 | 3.7E13 vg | Phase2, Phase1 | Recruiting | 2021-03-24 | 2027-08-31 | 2025-01-09 | 3 Months - 60 Months | 24 | 1 | United States | US9636370B2; US10532110B2 | Selected for START program 7/2024 |
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| NCT06332807 | Phenylketonurias | 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 | 2024-02-07 | 2030-12-30 | 2025-01-08 | 18 Years - 55 Years | 12 | 2 | United States | WO2024094044A1 | Recieved Orphan Drug Designation from the FDA in Jan 2023 |
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| NCT04945772 | Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Degeneration | Sonpiretigene isteparvovec (MCO-010) | Nanoscope Therapeutics Inc. | Industry | MCO | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Bipolar cells | Viral transduction | AAV2 | Dose 1: 0.9E11 gc/eye | Dose 2: 1.2E11 gc/eye | Phase2 | Completed | 2021-06-15 | 2024-01-18 | 2024-03-22 | >= 18 Years | 27 | 6 |
Locations:United States + 1 more
Puerto Rico, United States
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Anticipated BLA filing Q1 2025 |
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| NCT05417126 | Stargardt Disease | Sonpiretigene isteparvovec (MCO-010) | Nanoscope Therapeutics Inc. | Industry | MCO | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravitreal | Viral vector | Bipolar cells | Viral transduction | AAV2 | 1.2E11 gc/eye | Phase2 | Completed | 2022-06-09 | 2023-09-28 | 2023-11-09 | >= 16 Years | 6 | 2 | United States | Phase III Study expected to begin 2025 |
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| NCT03952637 | Lysosomal Diseases, Gangliosidosis, GM1 | AXO-AAV-GM1 (AAV9-GLB1) | National Human Genome Research Institute (NHGRI) | NIH | GLB1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 1.5E13 vg/kg | Dose 2: 4.5E13 vg/kg | Phase2, Phase1 | Recruiting | 2019-05-15 | 2028-01-01 | 2024-11-27 | 6 Months - 12 Years | 45 | 1 | United States |
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| NCT06325709 | Chronic Granulomatous Disease (CGD), X-Linked Chronic Granulomatous Disease | Base-edited autologous CD34+ cells | National Institute of Allergy and Infectious Diseases (NIAID) | NIH | CYBB c.676 C>T | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Electroporation | none | ABE8e-SpRY | Transduced CD34+ cells | Phase2, Phase1 | Suspended | 2024-03-21 | 2032-12-31 | 2025-02-18 | 18 Years - 75 Years | 10 | 1 | United States | Trial stoppage rules triggered due to a serious adverse event |
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| NCT01306019 | X-linked Severe Combined Immunodeficiency (XSCID) | VSV-G pseudotyped CL20-i4-EF1α-hγc-OPT vector | National Institute of Allergy and Infectious Diseases (NIAID) | NIH | IL2RG | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | VSV-G | Transduced CD34+ cells (median dose: 6.73E6 cells/kg; range: 4.46-15.10E6 cells/kg) | Phase2, Phase1 | Recruiting | 2011-02-26 | 2032-12-31 | 2025-02-14 | 2 Years - 50 Years | 40 | 1 | United States | Developed in partnership with St. Jude, and licensed to Mustang Bio, development paused by Mustang Bio |
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| NCT05984927 | Age-Related Macular Degeneration | NG101 | Neuracle Genetics, Inc | Industry | Codon optimized aflibercept | Gene transfer | In-vivo | Genetic delivery of therapeutic protein | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: 1E9 vg | Dose 2: 3E9 vg | Dose 3: 8E9 vg | Phase2, Phase1 | Recruiting | 2023-07-24 | 2030-01 | 2025-02-03 | 50 Years - 89 Years | 18 | 4 |
Locations:United States + 1 more
Canada, United States
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WO2022010277A1; KR20230167312A; | Plans to expand trial into the USA |
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| NCT05228145 | Neuronal Ceroid Lipofuscinosis CLN5 | NGN-101 | Neurogene Inc. | Industry | CLN5 | Gene transfer | In-vivo | Functional gene replacement | Intracerebroventricular | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed intermediate dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Active not recruiting | 2021-12-17 | 2028-11 | 2024-08-12 | 3 Years - 9 Years | 6 | 2 |
Locations:United States + 1 more
United Kingdom, United States
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Preliminary data expected 2H 2024 |
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| NCT05898620 | Rett Syndrome | NGN-401 (scAAV9.P546.MECP2) | 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 | Phase2, Phase1 | Recruiting | 2023-06-01 | 2029-10 | 2024-10-28 | 4 Years - 10 Years | 16 | 8 |
Locations:United States + 2 more
Australia, United Kingdom, United States
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US9415121B2 | Selected for START program (6/5/24); Interim data expected 4Q:24 |
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| NCT05293626 | Leber Hereditary Optic Neuropathy (LHON) | OPVIKA (esonadogene imvoparvovec) | Neurophth Therapeutics Inc | Other | ND4G11778A | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV2 | Dose 1: Dose de-escalation: 0.5E9, 0.05mL | Dose 2: Starting dose: 1.5E9 vg, 0.05ml/eye | Dose 3: Intermediate dose: 3.0E9, 0.05mL | Dose 4: High dose: 4.5E9, 0.05mL | Phase2, Phase1 | Active not recruiting | 2021-12-09 | 2029-12 | 2024-09-04 | 18 Years - 75 Years | 12 | 3 | United States | WO2020038352A1; US11332741B1; EP4382601A1 | Granted ODD by FDA and EMA, completed US enrollment |
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| NCT05820152 | Leber Hereditary Optic Neuropathy (LHON) | NFS-02 | Neurophth Therapeutics Inc | Other | ND1G3460A | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV2 | Dose 1: 5.0E7 vg, 0.05mL/eye | Dose 2: Starting dose: 1.5E8 vg, 0.05mL/eye | Dose 3: 5.0E8 vg, 0.05mL/eye | Dose 4: 1.5E9 vg, 0.05mL/eye | Phase2, Phase1 | Terminated | 2023-04-06 | 2024-06-24 | 2024-09-20 | 18 Years - 75 Years | 11 | 4 |
Locations:United States + 1 more
China, United States
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WO2021115317A1 | First patient dosed 8/17/23 |
News and Press Releases :
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| NCT05073133 | Muscular Atrophy, Spinal | ZOLGENSMA (onasemnogene abeparvovec) | Novartis Pharmaceuticals | Industry | SMN1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | 1.1E14 vg/kg | Phase4 | Completed | 2021-09-20 | 2023-08-08 | 2024-10-09 | <= 24 Months | 16 | 5 |
Locations:Argentina + 1 more
Argentina, Brazil
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FDA approved 5/24/19, price/treatment $2.1M |
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| 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 | 2024-04-09 | 2026-10-30 | 2025-02-11 | >= 8 Years | 150 | 15 |
Locations:United States + 1 more
Canada, United States
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US20210123077A1 | First patient dosed 6/20/24; on track for 2026 BLA and MAA approval targets |
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| NCT05956626 | Stargardt Disease | OCU410ST | Ocugen | Industry | RORA | Gene transfer | In-vivo | Overexpression of protective allele/gene | Subretinal | Viral vector | Viral transduction | AAV5 | Dose 1: 3.75E10 vg/mL | Dose 2: 7.5E10 vg/mL | Dose 3: 2.25E11 vg/mL | Phase2, Phase1 | Recruiting | 2023-06-30 | 2025-10-28 | 2024-03-12 | 6 Years - 65 Years | 42 | 6 | United States | US20210123077A1 | FDA granted ODD, moving to Phase 2 with the medium and high doses |
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| NCT06018558 | Geographic Atrophy | OCU410 | Ocugen | Industry | RORA | Gene transfer | In-vivo | Overexpression of protective allele/gene | Subretinal | Viral vector | Viral transduction | AAV5 | Dose 1: 2.5E10 vg/mL | Dose 2: 5E10 vg/mL | Dose 3: 1.5E11 vg/mL | Phase2, Phase1 | Recruiting | 2023-06-30 | 2025-09-23 | 2024-10-01 | >= 50 Years | 63 | 9 | United States | US20210123077A1 | Dosing of 3rd cohort completed July 2024 |
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| NCT05616793 | LCA5 | OPGx-LCA5 (AAV8.hLCA5) | Opus Genetics, Inc | Industry | LCA5 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | 2022-11-07 | 2028-01-30 | 2024-07-01 | >= 13 Years | 15 | 1 | United States | Received Rare Pediatric Disease Designation from FDA |
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| NCT04283227 | Lysosomal Storage Diseases, Metachromatic Leukodystrophy | LENMELDY/atidarsagene autotemcel/OTL-200 | Orchard Therapeutics | Industry | ARSA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | VSV-G | Dose 1: Minimum dose: 4.2E6 CD34+ cells/kg | Dose 2: Maximum dose: 30E6 CD34+ cells/kg | Phase3 | Active not recruiting | 2020-02-13 | 2031-03-31 | 2024-01-18 | 6 | 1 | Italy | FDA approval granted 3/18/24 |
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| NCT06149403 | MPS-IH (Hurler Syndrome) | OTL-203 (Autologous CD34+ cells transduced with IDUA lentiviral vector) | Orchard Therapeutics | Industry | IDUA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Mean dose: 20.9E6 transduced CD34+ cells/kg | Phase3 | Recruiting | 2023-11-17 | 2031-03 | 2024-11-18 | 28 Days - 30 Months | 40 | 7 |
Locations:United States + 3 more
Italy, Netherlands, United Kingdom, United States
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First patient randomized 2/2024 |
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| NCT04903288 | AADC Deficiency | KEBILIDI/UPSTAZA (eladocagene exuparvovec) | PTC Therapeutics | Industry | DDC | Gene transfer | In-vivo | Functional gene replacement | Intraparenchymal | Viral vector | Putamen | Viral transduction | AAV2 | 1.8E11 vg/dose, 320 ul/dose | Phase2 | Active not recruiting | 2021-05-21 | 2028-04-30 | 2025-01-01 | 1 Year - 17 Years | 13 | 6 |
Locations:United States + 2 more
Israel, Taiwan, United States
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US-20190000991-A1; US-10898585-B2; US-20210236653-A1; US-11865188-B2; US-20240100186-A1 | FDA approval granted 11/13/24 |
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Clinical Publications :
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| NCT04119687 | Osteoarthritis, Knee | PCRX-201, FX-201 (AdV-NK-KB-IL1RA) enekinragene inzadenovec | Pacira Pharmaceuticals, Inc | Industry | IL1RA | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarticular | Viral vector | Viral transduction | Ad5 | Dose 1: 1.4E10 gc/knee | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase1 | Active not recruiting | 2019-10-03 | 2026-11-28 | 2024-02-02 | 30 Years - 80 Years | 72 | 7 | United States | US20190376080A1 | FDA granted RMAT designation March 2024 |
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| NCT04747431 | Frontotemporal Dementia, FTD, FTD-GRN, Dementia Frontotemporal, C9orf72 | PBFT02 | Passage Bio, Inc. | Industry | GRN | Gene transfer | In-vivo | Functional gene replacement | Intracisterna magna | Viral vector | Viral transduction | AAV1 | Dose 1: 3.3E10 GC/g brain weight | Dose 2: 1.1E11 GC/g brain weight | Dose 3: 2.2E11 GC/g brain weight | Phase2, Phase1 | Recruiting | 2021-02-02 | 2031-08 | 2025-01-24 | 35 Years - 75 Years | 25 | 7 |
Locations:United States + 3 more
Brazil, Canada, Portugal, United States
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WO2024081551A1 |
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| NCT04713475 | GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency | PBGM01 | Passage Bio, Inc. | Industry | GLB1 | Gene transfer | In-vivo | Functional gene replacement | Intracisterna magna | Viral vector | Viral transduction | AAVhu68 | Dose 1: 3.3E10 GC/g brain weight | Dose 2: 1.1E11 GC/g brain weight | Dose 3: 2.2E11 GC/g brain weight | Dose 4: Undisclosed dose 4 | Phase2, Phase1 | Active not recruiting | 2021-01-04 | 2029-02 | 2024-05-28 | 1 Month - 24 Months | 26 | 9 |
Locations:United States + 4 more
Brazil, Canada, Turkey, United Kingdom, United States
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WO2024081551A1 | Out-licensed development to Gemma Biotherapeutics, uncertain development status | ||||
| NCT06392724 | Duchenne Muscular Dystrophy (DMD) | GEN6050X | Peking Union Medical College Hospital | Other | DMD | Gene editing | In-vivo | Exon skipping/splice editor | Intravenous | Viral vector | Viral transduction | dual AAV9 | eTAM | 5E13 vg/kg body weight | Early phase1 | Recruiting | 2024-04-26 | 2027-12 | 2024-07-08 | 4 Years - 10 Years | 3 | 1 | China | CA3191505A1; WO2017215619A1 | Drug is being developed by GenAssist Ltd |
Preclinical Publications :
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| NCT05805007 | Retinitis Pigmentosa | ZVS203e | Peking University Third Hospital | Other | RHO | Gene editing | In-vivo | Gene inactivation | Subretinal | Viral vector | Viral transduction | AAV | Cas9 mRNA | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Early phase1 | Recruiting | 2023-03-14 | 2026-04 | 2023-10-24 | >= 18 Years | 9 | 1 | China |
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| NCT03861273 | Hemophilia B | BEQVEZ/fidanacogene elaparvovec | Pfizer | Industry | F9R338L | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAVrh74 | 5E11 vg/kg | Phase3 | Active not recruiting | 2019-03-01 | 2031-01-09 | 2024-12-03 | 18 Years - 65 Years | 51 | 64 |
Locations:United States + 17 more
Australia, Belgium, Brazil, Canada, France, Germany, Greece, Italy, Japan, Korea, Republic of, Saudi Arabia, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States
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FDA approved 4/25/24, Price/treatment $3.5M |
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| NCT04370054 | Hemophilia A | Giroctogogene fitelparvovec | Pfizer | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Liver | Viral transduction | AAV2/6 | 3E13 vg/kg | Phase3 | Active not recruiting | 2020-04-21 | 2028-10-25 | 2025-02-06 | 18 Years - 64 Years | 76 | 36 |
Locations:United States + 16 more
Australia, Brazil, Canada, France, Germany, Greece, Italy, Japan, Korea, Republic of, Saudi Arabia, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States
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WO2017074526 | Pfizer terminated this program due to marketing considerations |
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| NCT04408625 | Frontotemporal Dementia | LY3884963 (PR006) AAV9-CMVe-CBA-GRN | 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 | 2020-05-21 | 2029-08-31 | 2024-12-09 | 30 Years - 85 Years | 30 | 11 |
Locations:United States + 5 more
Australia, Belgium, France, Spain, United Kingdom, United States
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US20210261981A1; WO2022082017A2 |
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| NCT04411654 | Gaucher Disease, Type 2 | LY3884961 (PR001) AAV9-CMV-CBA-GBA1 | Prevail Therapeutics | Industry | GBA1 | Gene transfer | In-vivo | Functional gene replacement | Intracisterna magna | Viral vector | Viral transduction | AAV9 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Phase2, Phase1 | Active not recruiting | 2020-05-11 | 2028-05 | 2025-02-13 | 0 Months - 24 Months | 15 | 5 |
Locations:United States + 1 more
United Kingdom, United States
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WO2022082017A2; US20200318115A1; US11903985B2; US20220211871A1 |
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| NCT06559176 | Chronic Granulomatous Disease, Granulomatous Disease, Chronic | PM359 | Prime Medicine, Inc. | Industry | NCF1 (c.75_76delGT) | Gene editing | Ex-vivo | Mutation correction | Intravenous | Autologous cells | CD34+ cells | Electroporation | prime editor | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | 2024-07-11 | 2030-02 | 2025-02-17 | >= 6 Years | 12 | 5 |
Locations:United States + 2 more
Canada, United Kingdom, United States
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US20230357766A1; US11795452B2; US20230220374A1 |
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| NCT03566043 | Mucopolysaccharidosis Type II (MPS II) | RGX-121 (AAV9-IDS) | REGENXBIO Inc. | Industry | IDS | Gene transfer | In-vivo | Functional gene replacement | Intracisterna magna | Viral vector | Viral transduction | AAV9 | Dose 1: 1.3E10 GC/g brain mass | Dose 2: 6.5E10 GC/g brain mass | Dose 3: 2.9E11 GC/g brain mass | Phase3, Phase2 | Active not recruiting | 2018-05-01 | 2025-08 | 2025-01-28 | 4 Months - 5 Years | 48 | 5 |
Locations:United States + 1 more
Brazil, United States
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US20180036388A1; US20240108761A1 | BLA filing planned for Q3 2024 |
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| NCT05693142 | Duchenne Muscular Dystrophy | RGX-202 (AAV8-microdystrophin) | REGENXBIO Inc. | Industry | Micro-dystrophin | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravenous | Viral vector | Viral transduction | AAV8 | Dose 1: 1 x 10^14 GC/kg body weight | Dose 2: 2 x 10^14 GC/kg body weight | Phase3, Phase2 | Recruiting | 2023-01-04 | 2028-08 | 2025-01-20 | >= 1 Year | 65 | 5 | United States | US20230270886A1 | Pivotal trial initiation expected Q3-Q4 2024; EOP2 meeting with FDA in July 2024 |
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| NCT06460844 | Retinitis Pigmentosa | 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 | 2024-06-10 | 2026-05 | 2025-01-08 | >= 18 Years | 9 | 3 | United States |
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| NCT05788536 | Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF) | DB-OTO (AAV1-Myo15-OTOF exons 1-20 + AAV1-OTOF exons 21-45,47) | Regeneron Pharmaceuticals | Industry | OTOF | Gene transfer | In-vivo | Functional gene replacement | Intracochlear | Viral vector | Hair cell | Viral transduction | dual AAV1 | Dose 1: 3E13 vg/ml (unilateral) | Dose 2: Undisclosed high dose (unilateral) | Dose 3: Undisclosed expansion dose (bilateral) | Phase2, Phase1 | Recruiting | 2023-03-15 | 2031-04-19 | 2024-09-04 | <= 17 Years | 22 | 12 |
Locations:United States + 2 more
Spain, United Kingdom, United States
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Phase I/II interim data presented at ASGCT 2024 on 2 patients |
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| NCT06511349 | Type 1 Primary Hyperoxaluria | 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 | 2024-07-04 | 2026-01-31 | 2024-12-19 | >= 2 Years | 7 | 1 | China | First patient dosed (8/5/24), ODD and RPDD granted by FDA in September 2024 |
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| NCT04248439 | Fanconi Anemia Complementation Group A | Mozafancogene autotemcel (RP-L102) PGK-FANCA | Rocket Pharmaceuticals Inc. | Industry | FANCA | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Dose range: 2.0E5 - 4.1E6 transduced CD34+ cells/kg | Phase2 | Active not recruiting | 2020-01-24 | 2026-05 | 2024-04-10 | >= 1 Year | 5 | 2 | United States | CA3093708A1; CA3106241A1; WO2020037249A1 | BLA filing H1 2024; MAA accepted by EMA 4/2/24 |
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| NCT03812263 | Leukocyte Adhesion Defect - Type I | KRESLADI (marnetegragene autotemcel) RP-L201 | Rocket Pharmaceuticals Inc. | Industry | ITGB2 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | Chim.hCD18-LV | Dose 1: Dose range: 2.8-10E6 transduced CD34+ cells/kg | Dose 2: Min dose: 2.0E6 CD34+ cells/kg | Phase2, Phase1 | Completed | 2019-01-18 | 2023-09-12 | 2023-11-15 | >= 3 Months | 9 | 3 |
Locations:United States + 2 more
Spain, United Kingdom, United States
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EP3830248A1; CA3106241A1; WO2020037249A1 | BLA Submitted: Pending FDA (????) |
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| NCT05885412 | PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) | RP-A601 (AAVrh.74-PKP2a) | Rocket Pharmaceuticals Inc. | Industry | PKP2A | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh.74 | Starting dose: 8E13 GC/kg | Phase1 | Recruiting | 2023-05-22 | 2026-09 | 2024-10-04 | >= 18 Years | 9 | 3 | United States | 5/2024: Received Orphan Medicial Product designation from EMA |
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| NCT04105166 | Pyruvate Kinase Deficiency | RP-L301 | Rocket Pharmaceuticals Inc. | Industry | PKLR | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | PGK-coPRK-WPRE | Dose range: 2.3E6 - 6.5E6 transduced CD34+ cells/kg | Phase1 | Active not recruiting | 2019-09-24 | 2025-05 | 2024-02-05 | 8 Years - 50 Years | 5 | 3 |
Locations:United States + 1 more
Spain, United States
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CA3106241A1; WO2020037249A1 | Phase I data presented at ASGCT 2024; Global Phase II planned late 2024/early 2025 |
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| NCT06092034 | Danon Disease | RP-A501 | Rocket Pharmaceuticals Inc. | Industry | LAMP2B | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | 6.7E13 GC/kg | Phase2 | Active not recruiting | 2023-10-11 | 2029-09 | 2024-09-25 | >= 8 Years | 12 | 6 |
Locations:United States + 3 more
Germany, Italy, United Kingdom, United States
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US10703797B2; US20210379201A1 | Phase II enrollment completed 09/2024 |
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| NCT02610582 | Achromatopsia | RAAV.hCNGA3 (AAV8-hArr3-hCNGA3-WPREm) | STZ eyetrial | Other | CNGA3 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Cone cells | Viral transduction | AAV8 | 1E11 vg/dose | Phase2, Phase1 | Active not recruiting | 2015-09-18 | 2027-06 | 2024-04-18 | >= 6 Years | 13 | 1 | Germany | US20180353619; WO2017144080A1; WO2016146669A1; WO2018010965A1 |
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| NCT04611503 | Retinitis Pigmentosa | RAAV.hPDE6A | STZ eyetrial | Other | PDE6A | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV8 | Dose 1: 1E9 vg | Dose 2: 5E9 vg | Dose 3: 1E10 vg | Dose 4: 5E10 vg | Phase2, Phase1 | Active not recruiting | 2020-05-20 | 2027-07 | 2024-04-18 | >= 18 Years | 9 | 1 | Germany |
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| NCT04046224 | Fabry Disease | Isaralgagene civaparvovec (ST-920) | Sangamo Therapeutics | Industry | GLA | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/6 | Dose 1: 0.26E13 vg/kg | Dose 2: 0.53E13 vg/kg | Dose 3: 1.58E13 vg/kg | Dose 4: 2.63E13 vg/kg | Phase2, Phase1 | Active not recruiting | 2019-08-01 | 2025-09 | 2024-05-09 | >= 18 Years | 34 | 18 |
Locations:United States + 6 more
Australia, Canada, Germany, Italy, Taiwan, United Kingdom, United States
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US20200231989A1; US9877988B2; | Recent Type B meeting with FDA allowed ongoing Phase 1/2 study to serve as the primary basis for approval under Accelerated Approval Program, BLA submission H2 2025 |
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| NCT05972629 | Phenylketonuria | SAR444836 | Sanofi | Industry | PAH | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV SNY001 | Undisclosed dose 1 | Phase2, Phase1 | Recruiting | 2023-06-23 | 2027-07-31 | 2025-01-08 | 18 Years - 65 Years | 32 | 8 |
Locations:United States + 4 more
Argentina, Brazil, Israel, Turkey, United States
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Deprioritized by Sponsor | |||||
| NCT04703842 | Congestive Heart Failure, Heart Failure, Systolic, Heart Failure, HFrEF - Heart Failure With Reduced Ejection Fraction | SRD-001 (AAV1-CMV-SERCA2a) | Sardocor Corp. | Industry | SERCA2a | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarterial | Viral vector | Viral transduction | AAV1 | Dose 1: 3E13 vg | Dose 2: 4.5E13 vg | Phase2, Phase1 | Recruiting | 2021-01-07 | 2028-12 | 2024-03-26 | 18 Years - 80 Years | 57 | 5 | United States | WO2011130552A2; WO2020176732A1 | Anticipated Phase 2b in 4Q2024-1Q2025 |
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| NCT06061549 | Heart Failure, Diastolic, Heart Failure With Preserved Ejection Fraction | SRD-001 (AAV1-CMV-SERCA2a) | Sardocor Corp. | Industry | SERCA2a | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraarterial | Viral vector | Viral transduction | AAV1 | 3E13 vg | Phase1 | Recruiting | 2023-07-30 | 2029-08 | 2023-09-29 | >= 50 Years | 10 | 2 | United States | WO2011130552A2; WO2020176732A1 | First patients dosed Q1 2024, product/indication combo granted Fast Track designation |
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| NCT06224660 | DMD-Associated Dilated Cardiomyopathy | SRD-001 (AAV1-CMV-SERCA2a) | 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 | Not yet recruiting | 2024-01-11 | 2030-02 | 2024-01-25 | >= 18 Years | 12 | 0 | Anticipated Phase 2b in 2H2025 |
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| NCT05881408 | Duchenne Muscular Dystrophy | ELEVIDYS/delandistrogene moxeparvovec | Sarepta Therapeutics, Inc. | Industry | Micro-dystrophin | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravenous | Viral vector | Cardiac and skeletal muscle | Viral transduction | AAVrh74 | Dose 1: For patients < 70kg: 1.33E14 vg/kg | Dose 2: For patients >70kg: 9.3E15 vg | Phase3 | Recruiting | 2023-05-19 | 2028-06-30 | 2025-02-17 | 148 | 42 |
Locations:United States + 13 more
Australia, Belgium, Germany, Hong Kong, Israel, Italy, Japan, Korea, Republic of, Spain, Sweden, Taiwan, United Kingdom, United States
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US20230173101A1 | First approval was June 2023, expanded indication to all DMD patients regardless of ambulatory status or age on 6/20/24 |
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| NCT05906251 | Limb Girdle Muscular Dystrophy | SRP-6004 (rAAVrh74-MHCK7-DYSF) | Sarepta Therapeutics, Inc. | Industry | DYSF | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh74 | Dose 1: 2E12 vg | Dose 2: 6E12 vg | Phase1 | Active not recruiting | 2023-06-07 | 2028-08-31 | 2024-08-20 | 18 Years - 50 Years | 2 | 1 | United States | WO2021257595A1 |
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| NCT06246513 | Limb-girdle Muscular Dystrophy | Bidridistrogene xeboparvovec/SRP-9003 (AAVrh74-MHCK7-SGCB) | Sarepta Therapeutics, Inc. | Industry | SGCB | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVrh74 | 5E13 vg/kg | Phase3 | Recruiting | 2024-01-30 | 2029-11-30 | 2024-11-25 | >= 4 Years | 15 | 5 |
Locations:United States + 1 more
United Kingdom, United States
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US20230241252A1; WO2021257595A1; EP4219726A1 | Data from EMERGENE are expected in the first half of 2025; BLA submission anticipated in 2025 |
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| NCT01976091 | Limb-Girdle Muscular Dystrophy, Type 2D | Patidistrogene bexoparvovec (SRP-9004) rAAVrh74-tMCK-SGCA | Sarepta Therapeutics, Inc. | Industry | SGCA | Gene transfer | In-vivo | Functional gene replacement | Isolated limb infusion | Viral vector | Viral transduction | AAVrh74 | Dose 1: 1E12 vg/kg/limb | Dose 2: 3E12 vg/kg/limb | Phase2, Phase1 | Completed | 2013-07-24 | 2019-03-14 | 2023-06-15 | >= 7 Years | 6 | 0 | US20220370639A1; WO2021257595A1 |
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| NCT06370351 | OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders, Ear Diseases, Otorhinolaryngologic Diseases, Deafness, Hearing Loss, Sensorineural | SENS-501 | Sensorion | Industry | OTOF | Gene transfer | In-vivo | Functional gene replacement | Intracochlear | Viral vector | Viral transduction | AAV | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Dose 3: Undisclosed expansion dose | Phase2, Phase1 | Recruiting | 2024-04-09 | 2031-07 | 2024-09-26 | 6 Months - 31 Months | 12 | 2 |
Locations:Australia + 1 more
Australia, France
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First patient dosed Q3 2024 |
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| NCT06465550 | β-thalassemia | BD211 (βA-T87Q-globin) | 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 | 2024-06-12 | 2026-12 | 2024-06-24 | 3 Years - 35 Years | 9 | 3 | China | WO2023130911A1 | ||||
| NCT06474442 | Herpes Simplex Virus Type I Stromal Keratitis | BD111 | Shanghai BDgene Co., Ltd. | Industry | HSV genes | Gene editing | In-vivo | Gene excision | Intrastromal | Viral vector | Viral transduction | LV | SpCas9 mRNA | Dose 1: 1.25E6 TU/eye | Dose 2: 2.5E6 TU/eye | Dose 3: 5.0E6 TU/eye | Dose 4: 10E6 TU/eye | Phase2 | Not yet recruiting | 2024-06-13 | 2026-12 | 2024-06-25 | 18 Years - 70 Years | 40 | 1 | China | WO2024011980A1 |
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| NCT06111638 | Hemophilia A | BBM-H803 | Shanghai Belief-Delivery BioMed Co., Ltd | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV | Undisclosed dose 1 | Phase2, Phase1 | Recruiting | 2023-10-27 | 2030-06-30 | 2024-07-23 | >= 18 Years | 12 | 1 | China | WO2022222869A1; WO2021180118A1 | First patient dosed January 2024 | ||||
| NCT05203679 | Hemophilia B | BBM-H901 | Shanghai Belief-Delivery BioMed Co., Ltd | Industry | F9 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV843 | 1E13 vg/kg | Phase3 | Active not recruiting | 2021-12-29 | 2028-06-30 | 2024-07-24 | >= 18 Years | 32 | 9 | China | WO2019241324A1; WO2022222869A1; WO2021180118A1 | Dosing completed of all Phase III subjects |
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| NCT05765981 | Aromatic L-amino Acid Decarboxylase (AADC) Deficiency | VGN-R09b (AADC + NTF) | Shanghai Jiao Tong University School of Medicine | Other | DDC | Gene transfer | In-vivo | Functional gene replacement | Intraparenchymal | Viral vector | Striatum | Viral transduction | AAV9 | Undisclosed single dose | Early phase1 | Recruiting | 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 |
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| NCT06141460 | Neovascular Age-related Macular Degeneration | 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 | 2023-11-10 | 2030-12-31 | 2024-11-14 | >= 50 Years | 48 | 1 | China | WO2024002076A1 |
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| NCT06217861 | Glutaric Acidemia Type I, Glutaric Aciduria Type I | 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 | 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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| NCT06480461 | Parkinson's Disease | VGN-R09b (AADC + NTF) | Shanghai Vitalgen BioPharma Co., Ltd. | Industry | DDC | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intraparenchymal | Viral vector | Striatum | Viral transduction | AAV9 | Dose 1: 8.0E11 vg | Dose 2: 1.6E12 vg | Dose 3: 3.2E12 vg | Phase2, Phase1 | Not yet recruiting | 2024-06-14 | 2031-07-01 | 2024-06-28 | 40 Years - 75 Years | 39 | 0 | WO2024017387A1 |
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| NCT05694598 | Bietti Crystalline Dystrophy | VGR-R01 (rAAV2/8-hCYP4V2) | Shanghai Vitalgen BioPharma Co., Ltd. | Industry | CYP4V2 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/8 | Dose 1: 6.0E10 vg/eye | Dose 2: 1.2E11 vg/eye (expansion dose) | Dose 3: 2.0E11 vg/eye | Phase1 | Not yet recruiting | 2023-01-11 | 2025-09-01 | 2023-01-23 | 18 Years - 69 Years | 12 | 1 | China | WO2023284873A1; US20240018541A1 | Phase I/II preliminary results presented at ASGCT 2024 |
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| NCT05441553 | Hemophilia B | VGB-R04 | Shanghai Vitalgen BioPharma Co., Ltd. | Industry | F9 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | Undisclosed dose (ranging from 4E11 - 2E12 vg/kg) | Phase2, Phase1 | Unknown | 2022-06-15 | 2025-01 | 2022-07-01 | 18 Years - 65 Years | 26 | 1 | China | WO2023280323A1; WO2023072181A1 | Granted ODD in December 2021 |
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| NCT02559830 | Metachromatic Leukodystrophy, Adrenoleukodystrophy | CD34+ hematopoietic stem cells transduced with ABCD1 | Shenzhen Second People's Hospital | Other | ABCD1 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | 2E6 transduced CD34+ cells/kg (maximum 20E6) | Phase2, Phase1 | Recruiting | 2015-08-12 | 2025-10 | 2022-05-31 | 1 Year - 16 Years | 50 | 1 | China |
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| NCT05986864 | Neovascular Age-related Macular Degeneration | 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 | 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 |
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| NCT06138639 | Duchenne Muscular Dystrophy | SGT-003 (AAV-SLB101-CK8-microdystrophin containing R16-R17 nNOS binding domain) | 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 | 2023-11-14 | 2031-05-06 | 2025-02-06 | 4 Years - 11 Years | 43 | 6 |
Locations:United States + 1 more
Canada, United States
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US20230183740A1; US20220031865A1; WO2021072197A1 | FDA granted Rare Pediatric Disease Designation 4/1/24; topline data expected Q4 2024 |
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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 | 2023-02-07 | 2029-03 | 2024-09-19 | >= 18 Years | 33 | 4 |
Locations:United States + 1 more
France, United States
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WO2024084075A1; EP3728610B1 | Enrollment of highest dose cohort is underway |
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| NCT00999609 | Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis | LUXTURNA/voretigene neparvovec (AAV2-hRPE65v2) | Spark Therapeutics, Inc. | Industry | RPE65 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | 1E11 vg/eye (0.3mL) | Phase3 | Active not recruiting | 2009-10-21 | 2029-07 | 2024-04-15 | >= 3 Years | 31 | 2 | United States | FDA approved 12/19/17, Price/treatment $850K/eye |
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| NCT06297486 | Hemophilia A | Dirloctogene samoparvovec/SPK-8011 (AAV LK03 capsid + TTRmut-hFVIII-X07) | Spark Therapeutics, Inc. | Industry | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | LK03 | Dose 1: 5E11 vg/kg | Dose 2: 1E12 vg/kg | Dose 3: 1.5E12 vg/kg | Dose 4: 2E12 vg/kg | Phase3 | Withdrawn | 2024-02-29 | 2035-09-04 | 2024-12-13 | >= 18 Years | 0 | 27 | United States | WO2019028192A1; US11168124B2; WO2019006390A1; US20220362408A1 | Product development discontinued, Study was withdrawn by Sponsor (no participants were enrolled) |
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| NCT05324943 | Gaucher Disease, Type 1 | FLT201 (AAVS3-LSP-GBA1-85) | Spur Therapeutics | Industry | GBA1 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAVS3 | 4.5E11 vg/kg | Phase1 | Active not recruiting | 2022-02-28 | 2025-01-31 | 2024-08-07 | >= 18 Years | 18 | 10 |
Locations:United States + 5 more
Brazil, Germany, Israel, Spain, United Kingdom, United States
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EP4118200A2; US20220387558A1; US20220162642A1; | Phase 3 trial planned in 2H2025 |
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| NCT06293729 | Refractory Hypercholesterolemia, Familial Hypercholesterolemia | NGGT006 | Suzhou Municipal Hospital | Other | LDLR | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravenous | Viral vector | Viral transduction | AAV | Dose 1: 7.5E12 vg/kg | Dose 2: 1.5E13 vg/kg | Dose 3: 3E13 vg/kg | Early phase1 | Not yet recruiting | 2024-02-27 | 2029-03-01 | 2024-04-17 | 18 Years - 55 Years | 9 | 0 | CN117887723A |
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| NCT05394064 | AMN, AMN Gene Mutation, X-ALD | SBT101 | SwanBio Therapeutics, Inc. | Industry | ABCD1 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | Dose 1: 1.0E14 vg | Dose 2: 3.0E14 vg | Phase2, Phase1 | Recruiting | 2022-05-16 | 2029-03-30 | 2023-09-13 | 18 Years - 65 Years | 16 | 2 |
Locations:United States + 1 more
Netherlands, United States
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| NCT05606614 | Rett Syndrome | TSHA-102 | Taysha Gene Therapies, Inc. | Industry | MiniMECP2 | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | scAAV9 | Dose 1: 5.7E14 vg | Dose 2: 1E15 vg | Phase2, Phase1 | Recruiting | 2022-10-28 | 2032-01-05 | 2024-11-13 | >= 12 Years | 18 | 6 |
Locations:United States + 1 more
Canada, United States
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US20240191254A1; US20240102050A1; US20190328804A1 | Recieved RMAT designation in May 2024 |
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| NCT02362438 | Giant Axonal Neuropathy, Gene Transfer | ScAAV9/JeT-GAN | Taysha Gene Therapies, Inc. | Industry | GAN | Gene transfer | In-vivo | Functional gene replacement | Intrathecal | Viral vector | Viral transduction | AAV9 | Dose 1: 3.5E13 vg | Dose 2: 1.2E14 vg | Dose 3: 1.8E14 vg | Dose 4: 3.5E14 vg | Phase1 | Active not recruiting | 2015-02-12 | 2035-04-01 | 2023-11-21 | 3 Years - 99 Years | 21 | 1 | United States | US20240102050A1 |
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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 | 2024-01-18 | 2029-10-01 | 2025-02-06 | 18 Years - 65 Years | 15 | 7 | United States | WO2022076648A1 | Dosing expected to begin H2 2024 |
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| NCT05836259 | Hypertrophic Cardiomyopathy | TN-201 | Tenaya Therapeutics | Industry | MYBPC3 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 3E13 vg/kg | Dose 2: 6E13 vg/kg | Phase2, Phase1 | Recruiting | 2023-04-18 | 2032-08 | 2024-11-15 | 18 Years - 75 Years | 30 | 10 | United States | WO2021163357A2; US20240084327A1 | Initial data from cohort 1 available H2 2024; enrolling patients for high dose (Cohort 2) |
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| NCT06107400 | Alpha Thalassemia Hemoglobin H Constant Spring, Hemoglobinopathies, Hereditary Diseases | 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 | 2023-10-25 | 2026-10-31 | 2024-06-03 | 12 Years - 35 Years | 5 | 1 | China | WO2023193616A1 | First patient dosed (5/27/24) |
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| NCT04884815 | Wilson Disease | Rivunatpagene miziparvovec (UX701) | Ultragenyx Pharmaceutical Inc | Industry | ATP7B | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 5.0E12 GC/kg | Dose 2: 1.0E13 GC/kg | Dose 3: 2.0E13 GC/kg | Dose 4: Undisclosed dose 4 | Phase2, Phase1 | Active not recruiting | 2021-05-07 | 2031-11 | 2024-11-27 | >= 18 Years | 78 | 16 |
Locations:United States + 4 more
Canada, Portugal, Spain, United Kingdom, United States
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EP3906066B1 | All patients in Phase I have been dosed, data expected H2 2024, expansion dose selection to follow |
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| NCT02716246 | MPS IIIA, Sanfilippo Syndrome, Sanfilippo a, Mucopolysaccharidosis III | Rebisufligene etisparvovec (scAAV9.U1a.hSGSH) | Ultragenyx Pharmaceutical Inc | Industry | SGSH | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV9 | Dose 1: 0.5E13 vg/kg | Dose 2: 1.0E13 vg/kg | Dose 3: 3.0E13 vg/kg | Phase3, Phase2 | Recruiting | 2016-03-17 | 2027-07 | 2025-02-19 | 36 | 5 |
Locations:United States + 2 more
Australia, Spain, United States
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US20220347298A1 | Accelerated Approval BLA filing planned for 2024/early 2025 |
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| NCT05139316 | Glycogen Storage Disease Type IA | Pariglasgene brecaparvovec (DTX401) | Ultragenyx Pharmaceutical Inc | Industry | G6PC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | 1.0E13 GC/kg | Phase3 | Active not recruiting | 2021-07-14 | 2026-02 | 2025-01-28 | >= 8 Years | 49 | 20 |
Locations:United States + 8 more
Brazil, Canada, Denmark, Germany, Italy, Japan, Netherlands, Spain, United States
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US20220017922A1 | Marketing application expected 2025 |
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| NCT05345171 | OTC Deficiency | Avalotcagene ontaparvovec/DTX301 | Ultragenyx Pharmaceutical Inc | Industry | OTC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV8 | 1.0E13 GC/kg | Phase3 | Recruiting | 2022-04-18 | 2028-12 | 2025-02-04 | >= 12 Years | 50 | 25 |
Locations:United States + 11 more
Argentina, Brazil, Canada, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, United Kingdom, United States
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EP4038194A1 | Enrollment expected to be completed early 2025 |
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| NCT05243017 | Huntington Disease | AMT-130 (rAAV5-miHTT) | UniQure Biopharma B.V. | Industry | MiHTT | Gene transfer | In-vivo | MiRNA knockdown of mutant/aberrant gene | Intraparenchymal | Viral vector | Viral transduction | AAV5 | Dose 1: 6E12 gc/subject | Dose 2: 6E13 gc/subject | Phase2, Phase1 | Active not recruiting | 2021-11-01 | 2029-10-07 | 2024-11-15 | 25 Years - 65 Years | 14 | 4 |
Locations:Poland + 1 more
Poland, United Kingdom
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US20230119344A1; US20220213482A1; US20210371862A1 | FDA granted RMAT designation Q2 2024 |
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| NCT06100276 | Amyotrophic Lateral Sclerosis | AMT-162 | UniQure Biopharma B.V. | Industry | MiSOD1 | Gene transfer | In-vivo | MiRNA knockdown of mutant/aberrant gene | Intrathecal | Viral vector | Viral transduction | AAVrh10 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed intermediate dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Recruiting | 2023-10-20 | 2031-03-30 | 2024-10-15 | >= 18 Years | 20 | 10 | United States | First patient dosed October 2024; IDMC recommended proceeding with 2nd cohort |
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| NCT06270316 | Fabry Disease | 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 | 2023-12-19 | 2027-04-29 | 2024-10-31 | 18 Years - 50 Years | 12 | 2 | United States | WO2015060722A1; WO2020104424A1 | First patient dosed 8/15/24; IDMC recommended proceeding with enrollment of 2nd cohort | ||||
| 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 | 2023-09-01 | 2027-06-30 | 2024-12-03 | 18 Years - 65 Years | 12 | 12 | United States | First patient dosed 11/21/24 | |||||
| NCT05092685 | Ornithine Transcarbamylase Deficiency | BGT-OTCD (AAVLK03hOTC) | University College, London | Other | OTC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV-LK03 | Dose 1: 6E11 vg/kg | Dose 2: 2E12 vg/kg | Dose 3: 6E12 vg/kg | Phase2, Phase1 | Recruiting | 2021-09-28 | 2027-06-30 | 2023-11-07 | 0 Days - 16 Years | 12 | 1 | United Kingdom | US20220372512A1; US20230093183A1 | Granted ODD by FDA and EMA |
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| NCT04601974 | Drug Resistant Epilepsy | Lenti-CAMK2A-KCNA1 | University College, London | Other | KCNA1 | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intracranial | Viral vector | Viral transduction | LV | Undisclosed dose (single administration) | Phase2, Phase1 | Not yet recruiting | 2020-10-06 | 2032-09 | 2023-05-15 | >= 18 Years | 10 | 0 | |||||||
| NCT03001830 | Hemophilia A | AAV2/8-HLP-FVIII-V3 | University College, London | Other | F8 | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Viral transduction | AAV2/8 | Dose 1: 6E11 vg/kg | Dose 2: 2E12 vg/kg | Dose 3: 4E12 vg/kg | Dose 4: 6E12 vg/kg | Phase2, Phase1 | Recruiting | 2016-12-09 | 2029-12 | 2024-12-06 | >= 18 Years | 18 | 4 |
Locations:United States + 1 more
United Kingdom, United States
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| NCT05432310 | Adenosine Deaminase Severe Combined Immune Deficiency | Simoladagene autotemcel (formerly OTL-101) | 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 | 2022-06-04 | 2026-12-31 | 2024-05-16 | >= 1 Month | 20 | 1 | United States | Administered Under Compassionate Use |
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| NCT02234934 | Granulomatous Disease, Chronic, X-linked | PCCLChimGp91/VSVg lentiviral vector (formerly OTL-102) | University of California, Los Angeles | Other | CYBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | VSV-G | Transduced CD34+ cells (6.5-32.6E6 cells/kg) | Phase2, Phase1 | Completed | 2014-09-04 | 2024-12-01 | 2024-12-20 | >= 23 Months | 10 | 3 | United States | US20220378937A1 | Commercial rights are owned by Orchard, which has deprioritized the program |
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| NCT03897361 | Lysosomal Storage Diseases, Cystinosis | CTNS-RD-04 or CTNS-RD-04-LB | University of California, San Diego | Other | CTNS | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Min dose: 3E6 CD34+ cells/kg | Phase2, Phase1 | Active not recruiting | 2019-02-06 | 2024-11 | 2024-06-12 | >= 18 Years | 6 | 1 | United States | US20240009247A1 | AVROBIO sold product to Novartis in May 2023, Novartis will sponsor any subsequent trials |
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| NCT03538899 | Severe Combined Immunodeficiency | AProArt (endogenous Artemis promotor) | University of California, San Francisco | Other | DCLRE1C | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Transduced CD34+ cells | Phase2, Phase1 | Recruiting | 2018-05-03 | 2038-06 | 2025-01-13 | >= 2 Months | 25 | 1 | United States |
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| NCT02240407 | Pompe Disease | RAAV9-DES-hGAA | University of Florida | Other | GAA | Gene transfer | In-vivo | Functional gene replacement | Intramuscular | Viral vector | Viral transduction | AAV9 | 4.6E13 vg/TA muscle | Phase1 | Completed | 2014-09-11 | 2021-08-26 | 2022-04-05 | 18 Years - 50 Years | 2 | 1 | United States | US20220347297A1 |
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| NCT04201405 | Mucopolysaccharidosis Type IIIA | OTL-201 (Autologous CD34+ cells transduced with LV-CD11b-hSGSH) | University of Manchester | Other | SGSH | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | Dose range: 4.37 - 22.7E6 CD34+ cells/kg | Phase2, Phase1 | Active not recruiting | 2019-12-05 | 2024-10-30 | 2024-02-13 | 3 Months - 24 Months | 5 | 1 | United Kingdom |
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| NCT02317887 | Retinoschisis, X-Linked | RS1 AAV Vector (AAV8-scRS/IRBPhRS) | VegaVect, Inc. | Industry | RS1 | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV8 | Dose 1: 1E9 vg/eye | Dose 2: 1E10 vg/eye | Dose 3: 1E11 vg/eye | Dose 4: < 3E11 vg/eye | Dose 5: < 6E11 vg/eye | Phase2, Phase1 | Completed | 2014-12-16 | 2024-10-16 | 2025-02-14 | >= 18 Years | 12 | 1 | United States | US9873893B2; US20210290433A1; WO2019144077A1; US20180214577A1 |
Preclinical Publications :
Clinical Publications :
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| NCT05477563 | Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Anemia, Sickle Cell Disease | CASGEVY/exagamglogene autotemcel | 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 | 2022-07-26 | 2025-02 | 2024-05-16 | 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 |
Preclinical Publications :
Clinical Publications :
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| NCT06164730 | Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease | VERVE-102 | Verve Therapeutics, Inc. | Industry | PCSK9 | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Lipid encapsulation | LDLR + GalNAc | base editor | Dose 1: Undisclosed dose 1 | Dose 2: Undisclosed dose 2 | Dose 3: Undisclosed dose 3 | Dose 4: Undisclosed dose 4 | Phase1 | Recruiting | 2023-12-01 | 2026-08 | 2024-11-12 | 18 Years - 70 Years | 36 | 8 |
Locations:Australia + 3 more
Australia, Canada, New Zealand, United Kingdom
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US12029795B2; US20240010609A1; US20240131166A1; US20240011023A1 | First patient dosed in Phase 1b trial May 2024 |
Preclinical Publications :
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| NCT05398029 | Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, Hypercholesterolemia | VERVE-101 | Verve Therapeutics, Inc. | Industry | PCSK9 | Gene editing | In-vivo | Gene inactivation | Intravenous | MRNA, LNP | Hepatocyte | Lipid encapsulation | LDLR | ABE8.8m | Dose 1: 0.1 mg/kg | Dose 2: 0.3 mg/kg | Dose 3: 0.45 mg/kg | Dose 4: 0.6 mg/kg | Phase1 | Active not recruiting | 2022-05-19 | 2025-06 | 2024-12-30 | 18 Years - 75 Years | 44 | 3 |
Locations:New Zealand + 1 more
New Zealand, United Kingdom
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US12029795B2; US20240010609A1; US20240131166A1; US20240011023A1 | Update given during ASGCT 2024 on the off-target assessments done by VERVE |
Preclinical Publications :
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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 | 2024-06-04 | 2027-03 | 2025-02-13 | 18 Years - 70 Years | 36 | 3 |
Locations:Canada + 1 more
Canada, United Kingdom
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US20240010609A1; US20230340435A1; WO2023049299A2 |
Preclinical Publications :
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| NCT06291935 | Retinitis Pigmentosa | VG901 (AAV2.NN-CNGA1) | ViGeneron GmbH | Industry | CNGA1 | Gene transfer | In-vivo | Functional gene replacement | Intravitreal | Viral vector | Viral transduction | AAV2 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed high dose | Phase1 | Recruiting | 2024-02-01 | 2025-12 | 2024-03-04 | >= 18 Years | 6 | 1 | Germany | US12043848B2; US20220409744A1 | First patient dosed 4/10/24 |
Preclinical Publications :
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| NCT06300476 | Stargardt Disease 1 | JWK006 | West China Hospital | Other | ABCA4 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | dual AAV8 | Dose 1: Undisclosed low dose | Dose 2: Undisclosed medium dose | Dose 3: Undisclosed high dose | Phase2, Phase1 | Active not recruiting | 2024-03-03 | 2029-12-30 | 2024-03-08 | 10 Years - 18 Years | 9 | 1 | China | CN115074369A | Investigator-initiated trial conducted at West China Hospital |
Preclinical Publications :
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| NCT06114056 | Duchenne Muscular Dystrophy | JWK007 | West China Hospital | Other | Micro-dystrophin | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intravenous | Viral vector | Viral transduction | AAVrh74 | Dose 1: 1.0E14 vg/kg | Dose 2: 2.0E14 vg/kg | Phase1 | Recruiting | 2023-10-29 | 2028-12-31 | 2024-01-17 | 5 Years - 10 Years | 6 | 1 | China | Investigator-initiated trial conducted at West China Hospital |
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| 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 | 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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| NCT06302608 | Bietti's Crystalline Dystrophy | NGGT001 | Xiamen Ophthalmology Center Affiliated to Xiamen University | Other | CYP4V2 | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2 | Dose 1: 1.5E11 vg/eye | Dose 2: 3.0E11 vg/eye | Early phase1 | Active not recruiting | 2024-02-18 | 2028-05-29 | 2024-03-12 | >= 18 Years | 6 | 1 | China | WO2023116745A1 |
News and Press Releases :
Clinical Publications :
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| NCT06272149 | Type II Gaucher Disease | 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 | 2023-07-17 | 2029-02-28 | 2024-02-22 | 0 Months - 24 Months | 6 | 1 | China | WO2024017387A1 | FIH interim data presented at ASGCT 2024 |
News and Press Releases :
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| NCT04125732 | Coronary Artery Disease, Ischemia, Angina Refractory, Cardiovascular Diseases, Heart Diseases | Encoberminogene rezmadenovec/XC001 | XyloCor Therapeutics, Inc. | Industry | VEGF | Gene transfer | In-vivo | Overexpression of protective allele/gene | Intramyocardial | Viral vector | Viral transduction | Ad5 | Dose 1: 1E9 vp | Dose 2: 1E10 vp | Dose 3: 4E10 vp | Dose 4: 1E11 vp (Expansion Dose) | Phase2, Phase1 | Completed | 2019-10-04 | 2023-05-30 | 2024-01-30 | 18 Years - 80 Years | 41 | 16 | United States | US20240115732A1 |
Preclinical Publications :
News and Press Releases :
Clinical Publications :
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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 | 2024-08-01 | 2026-06-30 | 2024-08-06 | 18 Years - 80 Years | 31 | 3 | China | First patient dosed (6/28/24) |
Preclinical Publications :
News and Press Releases :
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| NCT04293185 | Sickle Cell Disease | LYFGENIA/lovotibeglogene autotemcel | bluebird bio | Industry | HBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | BB305 LV | Dose 1: Minimum dose: 3E6 CD34+ cells/kg | Dose 2: Maximum dose: 14E6 CD34+ cells/kg | Dose 3: Median dose: 6.4E6 CD34+ cells/kg | Phase3 | Active not recruiting | 2020-02-12 | 2027-11 | 2024-12-10 | 2 Years - 50 Years | 35 | 9 | United States | FDA approved 12/8/23, price/treatment $3.1M |
Preclinical Publications :
Clinical Publications :
Protocol :
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| NCT03852498 | Cerebral Adrenoleukodystrophy (CALD) | SKYSONA/elivaldogene autotemcel | bluebird bio | Industry | ABCD1 | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | LV | At least 5.0E6 CD34+ cells/kg; MOI = 40 | Phase3 | Completed | 2019-02-13 | 2023-07-24 | 2024-05-24 | <= 17 Years | 35 | 8 |
Locations:United States + 5 more
France, Germany, Italy, Netherlands, United Kingdom, United States
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US9061031B2 | FDA approval granted 9/16/22; Cost/treatment $3M |
Clinical Publications :
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| NCT03207009 | Beta-Thalassemia | ZYNTEGLO/betibeglogene autotemcel | bluebird bio | Industry | HBB | Gene transfer | Ex-vivo | Functional gene replacement | Intravenous | Autologous cells | CD34+ cells | Viral transduction | BB305 LV | Minimum dose: 5.0E6 CD34+ cells/kg | Phase3 | Completed | 2017-06-29 | 2022-11-15 | 2024-03-07 | 0 Years - 50 Years | 19 | 9 |
Locations:United States + 5 more
France, Germany, Greece, Italy, United Kingdom, United States
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FDA approval granted 8/17/22, $2.8M estimated cost/treatment |
Clinical Publications :
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| NCT03328130 | Retinitis Pigmentosa | CTX-PDE6B (HORA-PDE6B) | eyeDNA Therapeutics | Industry | PDE6B | Gene transfer | In-vivo | Functional gene replacement | Subretinal | Viral vector | Viral transduction | AAV2/5 | Dose 1: 3.4E11 vg/eye | Dose 2: 6.4E11 vg/eye | Phase2, Phase1 | Recruiting | 2017-10-05 | 2029-12 | 2024-03-07 | >= 13 Years | 23 | 1 | France | ||||||
| NCT06255782 | Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn | ECUR-506 | iECURE, Inc. | Industry | OTC | Gene transfer | In-vivo | Functional gene replacement | Intravenous | Viral vector | Hepatocyte | Viral transduction | AAV8 | ARCUS | Dose 1: 1.3E13 GC/kg | Dose 2: Undisclosed high dose | Phase2, Phase1 | Recruiting | 2023-12-19 | 2026-09 | 2025-02-03 | 24 Hours - 7 Months | 13 | 5 |
Locations:United States + 2 more
Australia, United Kingdom, United States
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US20240101986A1; EP3288594B1; US9493788B2 | Sponsor reports complete clinical response in first infant dosed |
Preclinical Publications :
News and Press Releases :
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References
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The SCGE makes no comment as to the efficacy and safety of the items listed, as these are not known at the time of publication.
For the most up to date information, please refer to clinicaltrials.gov or the Sponsor's website.
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