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Clinical Trials - Gene Therapy Trial Browser

The Gene Therapy Trial Browser represents a unique publicly accessible, free database for the benefit of users seeking information on gene therapy development. The information within integrates various sources, including clinicaltrials.gov, publications, sponsor press releases, patent applications, and more to give a comprehensive overview of the gene therapy clinical trial landscape.

Disclaimer: The information on this dashboard has been collected for the convenience of patients and researchers. The SCGE team are not medical doctors and cannot provide medical advice. Please discuss with your provider the risks/benefits of participating in a clinical trial, and do not send us your personal medical information. The information contained within this table does not make use of any confidential or privileged information-all data is collected from publicly available sources. The SCGE makes no comment as to the efficacy and safety of the items listed, as these are not known at the time of publication. For the most up to date information, or to inquire about enrollment, please refer to clinicaltrials.gov or the Sponsor's website for contact information.

Clinical Trials Daily Digest

Updates in the last 14 days

NCT07007065
Last update post date 2026-04-14 2026-05-12 api
Number of locations 100 111 api
Locations Puerto rico, Hungary, United states, United kingdo... Puerto rico, Hungary, United states, Czechia, Unit... api
Eligibility criteria null Inclusion criteria: * pseudophakic (at least 12 w... api
2026-05-19
Neovascular Age-related Macular Degeneration
AbbVie Recruiting
Another Phase 3 study (NCT05407636) is active, enrollment is completed
NCT04370054
Eligibility criteria null Main inclusion criteria * males who have been fol... api
2026-05-18
Hemophilia A
Pfizer Active not recruiting
Pfizer terminated this program due to marketing considerations, Development rights are being returned to Sangamo
NCT06817382
Last update post date 2026-04-20 2026-04-29 curator
Number of locations 9 10 curator
Eligibility criteria null 1:50 as determined by elisa within 14 days of day 1. note: other inclusion/exclusion criteria may apply.">Inclusion criteria * participant must be male at ... api
2026-05-18
Duchenne Muscular Dystrophy (DMD)
Insmed Gene Therapy LLC Recruiting
IND cleared in 4Q2024, Clinical trial initiation planned in 1H2025
NCT03588299
Last update post date 2026-03-19 2026-04-17 api
Eligibility criteria null Inclusion criteria: * males age 18 years or older... api
2026-05-18
Hemophilia A
Bayer Active not recruiting
License reverted to Ultragenyx in 2022, uncertain development status
NCT04747431
Eligibility criteria null 7 5. known presence of a structural brain lesion (eg, Tumor, Cortical infarct) that could reasonably explain symptoms in a symptomatic subject 6. known presence of an ad-causing mutation in psen1, Psen2 or app based on genetic testing history (if performed) 7. previous history of korsakoff encephalopathy, Severe alcohol or substance dependence (within 5 years of onset of dementia), Except where onset of increased alcohol consumption occurs at the time of ftd disease onset 8. history of untreated vitamin b12 deficiency 9. presence of untreated hypothyroidism (thyroid stimulating hormone \[tsh\] \> uln and free t4 \< lln) 10. egfr ≤ 30 ml/min (as calculated using the ckd-epi equation) 11. alanine aminotransferase \[alt\] or aspartate aminotransferase \[ast\] \> 2 × uln, Or total bilirubin \> uln) 12. respiratory failure that requires supplemental oxygen, Tracheostomy, Or reliance on non-invasive ventilation for \>2 hours during waking hours 13. inability to provide full consent or the lack of a legally authorized caregiver with adequate contact who can provide consent 14. any contraindication to mri or lumbar puncture (lp) (eg, Local infection, History of thrombocytopenia, Coagulopathy) 15. any contraindication to the icm administration procedure 16. medical conditions or laboratory or vital sign abnormalities that would increase risk of complications from icm injection, Anesthesia, Lp, And/or mri (e.g., Fever, Hypoxia, Tachycardia, Or evidence of active infection) 17. immunocompromised status 18. peripheral axonal sensory neuropathy 19. receipt of a vaccine within 14 days of dosing 20. a positive test result for human immunodeficiency virus (hiv), Human t cell leukemia virus (htlv) type 1 or type 2, Or hepatitis b or c; a mycobacterium tuberculosis positive test within 1 year of or determined at screening 21. malignant neoplasia (except localized skin cancer) or a documented history of hereditary cancer syndrome 22. any concurrent disease that, In the opinion of the investigator, May cause cognitive impairment unrelated to grn or c9orf72 mutations, Including other causes of dementia, Neurosyphilis, Hydrocephalus, Stroke, Small vessel ischemic disease, Uncontrolled hypothyroidism, Or vitamin deficiency 23. current or recent history of clinically significant suicidal ideation within the past 6 months 24. for women of childbearing potential, A positive serum pregnancy test at the screening visit, A positive serum result on day 1 prior to administration of the investigational product, Or unwillingness to have additional pregnancy tests during the study. women of childbearing potential must use a highly effective method of birth control or engage in abstinence until 90 days postdose 25. women who are breastfeeding 26. for sexually active men, Unwillingness to use a medically accepted method of double-barrier contraception (such as a condom/diaphragm used with spermicide) or engage in abstinence from the date of screening until 90 days postdose 27. any condition (eg, History of any disease, Evidence of any current disease, Any finding upon physical examination, Or any laboratory abnormality) that, In the opinion of the investigator, Would put the subject at undue risk or would interfere with evaluation of the investigational product or interpretation of subject safety or study results 28. any acute illness requiring hospitalization within 30 days of enrollment 29. failure to meet the protocol-specified coagulation test criteria: * platelet count \> 100, 000 per ul * inr \< 1.5 * aptt \< 40 seconds 30. use of anticoagulants in the 2 weeks prior to screening 31. hypersensitivity or contraindications to corticosteroid use 32. known or suspected intolerance or hypersensitivity to pbft02 or any of its ingredients or to closely related compounds 33. any condition expected to increase risk of thrombosis 34. hypersensitivity or contraindications to apixaban additional criteria for ftd-c9orf72 (cohorts 4-5) only: presence of concurrent als is permitted provided the following criteria are not met: 35. alsfrs-r \< 35 at screening. 36. svc \< 75% of predicted normal adjusted for sex, Age, And height (from the sitting position). 37. bulbar-onset als. 38. current or anticipated need, In the opinion of the investigator for a diaphragm pacing system (dps) during the study period. 39. if taking riluzole or edaravone, Participant's dose has not been stable for ≥ 30 days prior to day 1 and/or dose adjustments are anticipated before the day 60 study visit.">Inclusion criteria: 1. documented to be a pathoge... api
2026-05-15
Frontotemporal Dementia, FTD, FTD-GRN, C9orf72
Passage Bio, Inc. Active not recruiting
12-month data from Dose 1 and interim safety and biomarker data from Dose 2 expected in 2H 2025; plan to seek regulatory feedback on FTD-GRN pivotal t...
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Showing 1-100 of 385 results in ClinicalTrials
NCT07007065 Recruiting Phase3
View Report
Neovascular Age-related Macular Degeneration
Sponsor AbbVie (Industry)
Compound ABBV-RGX-314
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2025-05-28 Last Modified Date: 2026-05-19 Enrollment: 561 Austria, Belgium, Bulgaria + 14 more
NCT04370054 Active not recruiting Phase3
View Report
Hemophilia A
Sponsor Pfizer (Industry)
Compound Giroctogogene fitelparvovec
Therapy Type Gene transfer
Target Gene F8
Vector Type AAV2/6
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2020-04-21 Last Modified Date: 2026-05-18 Enrollment: 77 Australia, Brazil, Canada + 13 more
NCT06817382 Recruiting Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Insmed Gene Therapy LLC (Industry)
Compound INS1201
Therapy Type Gene transfer
Target Gene Micro-dystrophin
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-02-04 Last Modified Date: 2026-05-18 Enrollment: 12 United States
NCT03588299 Active not recruiting Phase2, Phase1
View Report
Hemophilia A
Sponsor Bayer (Industry)
Compound DTX201
Therapy Type Gene transfer
Target Gene F8
Vector Type AAVhu37
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-07-06 Last Modified Date: 2026-05-18 Enrollment: 11 Bulgaria, France, Germany + 3 more
NCT04747431 Active not recruiting Phase2, Phase1
View Report
Frontotemporal Dementia, FTD, FTD-GRN, C9orf72
Sponsor Passage Bio, Inc. (Industry)
Compound PBFT02
Therapy Type Gene transfer
Target Gene GRN
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2021-02-02 Last Modified Date: 2026-05-15 Enrollment: 30 Australia, Brazil, Canada + 2 more
NCT05748873 Active not recruiting Phase2, Phase1
View Report
Retinitis Pigmentosa
Sponsor SparingVision (Industry)
Compound SPVN06
Therapy Type Gene transfer
Target Gene NXNL1
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-02-07 Last Modified Date: 2026-05-15 Enrollment: 33 France, United States
NCT05419492 Recruiting Phase2, Phase1
View Report
Dravet Syndrome
Sponsor Encoded Therapeutics (Industry)
Compound ETX101
Therapy Type Gene transfer
Target Gene SCN1A
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-06-10 Last Modified Date: 2026-05-15 Enrollment: 47 Australia, United Kingdom, United States
NCT06634420 Active not recruiting Phase3
View Report
Hereditary Angioedema
Sponsor Intellia Therapeutics (Industry)
Compound NTLA-2002
Therapy Type Gene editing
Target Gene KLKB1
Vector Type LDLR
Delivery System Lipid encapsulation
Mechanism of Action Gene inactivation
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-10-07 Last Modified Date: 2026-05-15 Enrollment: 80 Australia, Canada, France + 6 more
NCT07287397 Recruiting Phase2, Phase1
View Report
Amyotrophic Lateral Sclerosis
Sponsor Vector Y Therapeutics (Industry)
Compound VTx-002
Therapy Type Gene transfer
Target Gene Antibody to TDP-43
Vector Type AAV5.2
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Fast Track
First Submitted: 2025-11-19 Last Modified Date: 2026-05-14 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 more
NCT06217861 Recruiting Phase1
View Report
Glutaric Acidemia Type I
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGM-R02b
Therapy Type Gene transfer
Target Gene GCDH
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-12-12 Last Modified Date: 2026-05-14 Enrollment: 12 China
NCT05541627 Active not recruiting Phase2, Phase1
View Report
Huntington's Disease
Sponsor AskBio France, SAS, a subsidiary of AskBio Inc. (Industry)
Compound AB-1001
Therapy Type Gene transfer
Target Gene CYP46A1
Vector Type AAVrh10
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2022-09-13 Last Modified Date: 2026-05-14 Enrollment: 5 France
NCT03861273 Active not recruiting Phase3
View Report
Hemophilia B
Sponsor Pfizer (Industry)
Compound BEQVEZ
Therapy Type Gene transfer
Target Gene F9-R338L
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2019-03-01 Last Modified Date: 2026-05-12 Enrollment: 51 Australia, Brazil, Canada + 13 more
NCT05353647 Active not recruiting Phase2
View Report
Sickle Cell Disease
Sponsor David Williams (Other)
Compound Autologous CD34+ HSC cells transduced with the lentiviral vector containing BCL11a-targeted shRNA
Therapy Type Gene transfer
Target Gene BCL11A
Vector Type VSV-G
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2022-04-21 Last Modified Date: 2026-05-12 Enrollment: 25 United States
NCT03368742 Active not recruiting Phase2, Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-001
Therapy Type Gene transfer
Target Gene Micro-dystrophin
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-12-05 Last Modified Date: 2026-05-08 Enrollment: 12 United States
NCT07160634 Recruiting Phase3
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-003
Therapy Type Gene transfer
Target Gene Micro-dystrophin
Vector Type AAV-SLB101
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Rare Pediatric Disease Designation
First Submitted: 2025-08-29 Last Modified Date: 2026-05-08 Enrollment: 80 Australia, Canada
NCT05504837 Recruiting Phase1
View Report
Cystic Fibrosis
Sponsor Krystal Biotech, Inc. (Industry)
Compound KB407
Therapy Type Gene transfer
Target Gene CFTR
Vector Type HSV-1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-08-15 Last Modified Date: 2026-05-08 Enrollment: 20 United States
NCT05456880 Active not recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-101
Therapy Type Gene editing
Target Gene HBG1/HBG2
Vector Type none
Delivery System Electroporation
Mechanism of Action Mutation correction
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2022-06-23 Last Modified Date: 2026-05-07 Enrollment: 15 United States
NCT05878860 Recruiting Phase3
View Report
X-Linked Retinoschisis
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-201
Therapy Type Gene transfer
Target Gene RS1
Vector Type AAV.SPR
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-12 Last Modified Date: 2026-05-07 Enrollment: 97 United States
NCT07282847 Recruiting Phase2, Phase1
View Report
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Sponsor AskBio Inc (Industry)
Compound AB-1009
Therapy Type Gene transfer
Target Gene GAA
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2025-11-26 Last Modified Date: 2026-05-07 Enrollment: 12 United States
NCT05682144 Recruiting Phase1
View Report
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Sponsor Immusoft of CA, Inc. (Industry)
Compound ISP-001
Therapy Type Gene transfer
Target Gene IDUA
Vector Type Sleeping Beauty
Delivery System Electroporation
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-12-12 Last Modified Date: 2026-05-07 Enrollment: 11 United States
NCT06255782 Recruiting Phase2, Phase1
View Report
Ornithine Transcarbamylase (OTC) Deficiency
Sponsor iECURE, Inc. (Industry)
Compound ECUR-506
Therapy Type Gene transfer
Target Gene OTC/PCSK9 locus
Vector Type dual AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2023-12-19 Last Modified Date: 2026-05-07 Enrollment: 20 Australia, Spain, United Kingdom + 1 more
NCT06018558 Active not recruiting Phase2, Phase1
View Report
Geographic Atrophy
Sponsor Ocugen (Industry)
Compound OCU410
Therapy Type Gene transfer
Target Gene RORA
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-06-30 Last Modified Date: 2026-05-06 Enrollment: 60 United States
NCT06064890 Recruiting Phase2, Phase1
View Report
Frontotemporal Dementia, FTD, FTD-GRN
Sponsor AviadoBio Ltd (Industry)
Compound AVB-101
Therapy Type Gene transfer
Target Gene GRN
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2023-09-12 Last Modified Date: 2026-05-06 Enrollment: 18 Belgium, Canada, Italy + 6 more
NCT04581785 Terminated Phase2, Phase1
View Report
Methylmalonic Acidemia
Sponsor LogicBio Therapeutics, Inc (Industry)
Compound LB-001
Therapy Type Gene transfer
Target Gene MMUT
Vector Type AAV-LK03
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-10-02 Last Modified Date: 2026-05-06 Enrollment: 4 United States
NCT04853576 Active not recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Editas Medicine, Inc. (Industry)
Compound EDIT-301
Therapy Type Gene editing
Target Gene HBG1/HBG2
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-04-16 Last Modified Date: 2026-05-06 Enrollment: 45 Canada, United States
NCT06285643 Recruiting Phase2
View Report
Parkinson's Disease
Sponsor AskBio Inc (Industry)
Compound AB-1005
Therapy Type Gene transfer
Target Gene GDNF
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2024-02-14 Last Modified Date: 2026-05-06 Enrollment: 127 Germany, Poland, United Kingdom + 1 more
NCT07113743 Enrolling by invitation Phase2, Phase1
View Report
Chronic Granulomatous Disease (CGD)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound VSVG-PCCLChimGp91
Therapy Type Gene transfer
Target Gene CYBB
Vector Type VSV-G
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2025-08-08 Last Modified Date: 2026-05-06 Enrollment: 10 United States
NCT02716246 Recruiting Phase3, Phase2
View Report
Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound UX111
Therapy Type Gene transfer
Target Gene SGSH
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-03-17 Last Modified Date: 2026-05-06 Enrollment: 36 Australia, Spain, United States
NCT04680065 Active not recruiting Phase1
View Report
Multiple System Atrophy
Sponsor Brain Neurotherapy Bio, Inc. (Industry)
Compound AB-1005
Therapy Type Gene transfer
Target Gene GDNF
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2020-12-15 Last Modified Date: 2026-05-06 Enrollment: 9 United States
NCT06199531 Active not recruiting Phase3
View Report
NGLY1 Deficiency
Sponsor Grace Science, LLC (Industry)
Compound GS-100
Therapy Type Gene transfer
Target Gene NGLY1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-11-21 Last Modified Date: 2026-05-06 Enrollment: 10 United States
NCT05926765 Recruiting Phase2
View Report
Grade 2 and 3 Radiation-Induced Late Xerostomia
Sponsor MeiraGTx, LLC (Industry)
Compound AAV-hAQP1
Therapy Type Gene transfer
Target Gene AQP1
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2023-06-22 Last Modified Date: 2026-05-06 Enrollment: 276 Canada, United Kingdom, United States
NCT05598333 Active not recruiting Phase2
View Report
Congestive Heart Failure
Sponsor AskBio Inc (Industry)
Compound AB-1002
Therapy Type Gene transfer
Target Gene PPP1R1A
Vector Type AAV2i8
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2022-10-25 Last Modified Date: 2026-05-06 Enrollment: 150 Austria, Belgium, Bulgaria + 9 more
NCT05444894 Active not recruiting Phase2, Phase1
View Report
Beta-Thalassemia Major
Sponsor Editas Medicine, Inc. (Industry)
Compound EDIT-301
Therapy Type Gene editing
Target Gene HBG1/HBG2
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-06-27 Last Modified Date: 2026-05-06 Enrollment: 9 Canada, United States
NCT06291961 Completed Phase1
View Report
Beta-Thalassemia Major
Sponsor CorrectSequence Therapeutics Co., Ltd (Industry)
Compound CS-101
Therapy Type Gene editing
Target Gene BCL11A
Vector Type none
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-02-23 Last Modified Date: 2026-05-06 Enrollment: 9 China
NCT06389877 Recruiting Phase2, Phase1
View Report
Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-302
Therapy Type Gene editing
Target Gene SERPINA1 (p.Glu366Lys)
Vector Type LNP
Delivery System Lipid encapsulation
Mechanism of Action Mutation correction
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-04-22 Last Modified Date: 2026-05-06 Enrollment: 106 Australia, Ireland, Netherlands + 3 more
NCT06826612 Recruiting Phase2, Phase1
View Report
Huntington's Disease
Sponsor Hoffmann-La Roche (Industry)
Compound RG6662
Therapy Type Gene transfer
Target Gene MiHTT
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2025-02-03 Last Modified Date: 2026-05-05 Enrollment: 53 United States
NCT06506461 Recruiting Phase1
View Report
Sickle Cell Disease
Sponsor St. Jude Children's Research Hospital (Other)
Compound Gene-modified CD34+ cells
Therapy Type Gene editing
Target Gene BCL11A
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-06-14 Last Modified Date: 2026-05-05 Enrollment: 25 United States
NCT05788536 Recruiting Phase2, Phase1
View Report
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Sponsor Regeneron Pharmaceuticals (Industry)
Compound OTARMENI
Therapy Type Gene transfer
Target Gene OTOF
Vector Type dual AAV1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-03-15 Last Modified Date: 2026-05-05 Enrollment: 30 Germany, Japan, Spain + 2 more
NCT06092034 Recruiting Phase2
View Report
Danon Disease
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-A501
Therapy Type Gene transfer
Target Gene LAMP2B
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-10-11 Last Modified Date: 2026-05-05 Enrollment: 14 Germany, Italy, United Kingdom + 1 more
NCT05805007 Unknown Early phase1
View Report
Retinitis Pigmentosa
Sponsor Peking University Third Hospital (Other)
Compound ZVS203e
Therapy Type Gene editing
Target Gene RHO
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2023-03-14 Last Modified Date: 2026-05-02 Enrollment: 9 China
NCT01306019 Recruiting Phase2, Phase1
View Report
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound VSV-G pseudotyped CL20-i4-EF1α-hγc-OPT vector
Therapy Type Gene transfer
Target Gene IL2RG
Vector Type VSV-G
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2011-02-26 Last Modified Date: 2026-05-02 Enrollment: 40 United States
NCT06461702 Unknown Early phase1
View Report
Familial Hypercholesterolemia
Sponsor The First Affiliated Hospital of Bengbu Medical University (Other)
Compound YOLT-101
Therapy Type Gene editing
Target Gene PCSK9/LDLR
Vector Type none
Delivery System LNP
Mechanism of Action Gene inactivation
First Submitted: 2024-04-16 Last Modified Date: 2026-05-01 Enrollment: 13 China
NCT06765980 Recruiting Phase2, Phase1
View Report
Geographic Atrophy Secondary to Age-related Macular Degeneration
Sponsor Kriya Therapeutics, Inc. (Industry)
Compound KRIYA-825
Therapy Type Gene transfer
Target Gene CR2-CR1
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2024-12-19 Last Modified Date: 2026-05-01 Enrollment: 62 Canada, Israel, New Zealand
NCT04408625 Active not recruiting Phase2, Phase1
View Report
Frontotemporal Dementia
Sponsor Prevail Therapeutics (Industry)
Compound LY3884963
Therapy Type Gene transfer
Target Gene GRN
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-05-21 Last Modified Date: 2026-05-01 Enrollment: 35 Australia, Belgium, France + 3 more
NCT06237777 Unknown Phase1
View Report
Diabetic Macular Edema
Sponsor Wang Min (Other)
Compound SKG0106
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2024-01-11 Last Modified Date: 2026-05-01 Enrollment: 18 China
NCT02362438 Completed Phase1
View Report
Giant Axonal Neuropathy
Sponsor National Institute of Neurological Disorders and Stroke (NINDS) (NIH)
Compound ScAAV9/JeT-GAN
Therapy Type Gene transfer
Target Gene GAN
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2015-02-12 Last Modified Date: 2026-05-01 Enrollment: 14 United States
NCT07226206 Recruiting Phase2, Phase1
View Report
Hemophilia A
Sponsor Hoffmann-La Roche (Industry)
Compound SPK-8011QQ
Therapy Type Gene transfer
Target Gene F8
Vector Type LK03
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-10-28 Last Modified Date: 2026-04-28 Enrollment: 5 United States
NCT04704921 Active not recruiting Phase3, Phase2
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor AbbVie (Industry)
Compound ABBV-RGX-314
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2021-01-08 Last Modified Date: 2026-04-28 Enrollment: 671 United States
NCT06910813 Recruiting Phase2, Phase1
View Report
Nephropathic Cystinosis
Sponsor Novartis Pharmaceuticals (Industry)
Compound DFT383
Therapy Type Gene transfer
Target Gene CTNS
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-03-17 Last Modified Date: 2026-04-28 Enrollment: 30 United States
NCT06844214 Recruiting Phase2, Phase1
View Report
Myotonic Dystrophy
Sponsor Sanofi (Industry)
Compound SAR446268
Therapy Type Gene transfer
Target Gene DMPK
Vector Type AAV.SAN011
Delivery System Viral transduction
Mechanism of Action Gene inactivation
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2025-02-19 Last Modified Date: 2026-04-28 Enrollment: 32 Argentina, Australia, Canada + 3 more
NCT07180355 Recruiting Phase1
View Report
Friedreich's Ataxia
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-212
Therapy Type Gene transfer
Target Gene FXN
Vector Type AAVhu68
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-08-22 Last Modified Date: 2026-04-28 Enrollment: 10 United States
NCT06451770 Recruiting Phase1
View Report
Hypercholesterolemia
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-201
Therapy Type Gene editing
Target Gene ANGPTL3
Vector Type LDLR + GalNAc
Delivery System Lipid encapsulation
Mechanism of Action Gene inactivation
First Submitted: 2024-06-04 Last Modified Date: 2026-04-28 Enrollment: 36 Australia, Canada, South Africa + 1 more
NCT04903288 Active not recruiting Phase2
View Report
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor PTC Therapeutics (Industry)
Compound KEBILIDI, UPSTAZA
Therapy Type Gene transfer
Target Gene DDC
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Priority Review, Accelerated Approval, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-05-21 Last Modified Date: 2026-04-25 Enrollment: 13 Israel, Taiwan, United States
NCT07223944 Recruiting Phase3
View Report
Gaucher Disease Type 1
Sponsor Spur Therapeutics (Industry)
Compound FLT201
Therapy Type Gene transfer
Target Gene GBA1
Vector Type AAVS3
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-10-30 Last Modified Date: 2026-04-21 Enrollment: 45 United States
NCT06692712 Recruiting Phase3
View Report
Hereditary Spastic Paraplegia Type 50
Sponsor Elpida Therapeutics SPC (Industry)
Compound MELPIDA
Therapy Type Gene transfer
Target Gene AP4M1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-11-14 Last Modified Date: 2026-04-21 Enrollment: 24 Spain, United States
NCT04248439 Active not recruiting Phase2
View Report
Fanconi Anemia Complementation Group A
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-L102
Therapy Type Gene transfer
Target Gene FANCA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-01-24 Last Modified Date: 2026-04-21 Enrollment: 5 United States
NCT06432140 Active not recruiting Phase3, Phase2
View Report
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGN-R09b
Therapy Type Gene transfer
Target Gene DDC
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-05-17 Last Modified Date: 2026-04-21 Enrollment: 13 China
NCT04998396 Recruiting Phase2, Phase1
View Report
Canavan Disease
Sponsor Aspa Therapeutics (Industry)
Compound BBP-812
Therapy Type Gene transfer
Target Gene ASPA
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-08-05 Last Modified Date: 2026-04-18 Enrollment: 26 United States
NCT06422351 Suspended Phase2
View Report
Pyruvate Kinase Deficiency
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-L301
Therapy Type Gene transfer
Target Gene PKLR
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2024-05-15 Last Modified Date: 2026-04-16 Enrollment: 10 Spain, United States
NCT06364774 Recruiting Phase2, Phase1
View Report
Beta-Thalassemia Major
Sponsor Children's Hospital of Philadelphia (Other)
Compound CHOP-ALS20
Therapy Type Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-03-26 Last Modified Date: 2026-04-16 Enrollment: 12 United States
NCT04774536 Recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Mark Walters, MD (Other)
Compound CRISPR_SCD001
Therapy Type Gene editing
Target Gene HBB
Vector Type none
Delivery System Electroporation
Mechanism of Action Mutation correction
First Submitted: 2021-02-17 Last Modified Date: 2026-04-16 Enrollment: 9 United States
NCT07052929 Recruiting Phase2, Phase1
View Report
X-Linked Myotubular Myopathy
Sponsor Astellas Gene Therapies (Industry)
Compound ASP2957
Therapy Type Gene transfer
Target Gene MTM1
Vector Type AAV3.8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-06-30 Last Modified Date: 2026-04-16 Enrollment: 9 Canada, United States
NCT06515002 Terminated Phase2, Phase1
View Report
Cystic Fibrosis
Sponsor Boehringer Ingelheim (Industry)
Compound BI 3720931
Therapy Type Gene transfer
Target Gene CFTR
Vector Type rSIV.F/HN
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-07-18 Last Modified Date: 2026-04-16 Enrollment: 5 France, Italy, Netherlands + 2 more
NCT05885412 Recruiting Phase1
View Report
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-A601
Therapy Type Gene transfer
Target Gene PKP2
Vector Type AAVrh.74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2023-05-22 Last Modified Date: 2026-04-16 Enrollment: 9 United States
NCT07064759 Recruiting Phase3
View Report
Neovascular Age-Related Macular Degeneration (nAMD)
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-150
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-07-07 Last Modified Date: 2026-04-16 Enrollment: 480 Argentina, Australia, Bulgaria + 11 more
NCT03533673 Completed Phase1
View Report
Pompe Disease
Sponsor AskBio Inc (Industry)
Compound ACTUS-101
Therapy Type Gene transfer
Target Gene GAA
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track
First Submitted: 2018-04-13 Last Modified Date: 2026-04-14 Enrollment: 7 United States
NCT04046224 Completed Phase2, Phase1
View Report
Fabry Disease
Sponsor Sangamo Therapeutics (Industry)
Compound ST-920
Therapy Type Gene transfer
Target Gene GLA
Vector Type AAV2/6
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2019-08-01 Last Modified Date: 2026-04-14 Enrollment: 36 Australia, Canada, Germany + 4 more
NCT05073133 Completed Phase4
View Report
Spinal Muscular Atrophy
Sponsor Novartis Pharmaceuticals (Industry)
Compound ZOLGENSMA
Therapy Type Gene transfer
Target Gene SMN1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-09-20 Last Modified Date: 2026-04-08 Enrollment: 16 Argentina, Brazil
NCT03199469 Active not recruiting Phase3, Phase2
View Report
X-Linked Myotubular Myopathy
Sponsor Astellas Gene Therapies (Industry)
Compound AT132
Therapy Type Gene transfer
Target Gene MTM1
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-06-21 Last Modified Date: 2026-04-08 Enrollment: 27 Canada, France, Germany + 1 more
NCT05903794 Completed Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Exegenesis Bio (Industry)
Compound EXG102-031
Therapy Type Gene transfer
Target Gene ABD-VEGFR
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2023-06-06 Last Modified Date: 2026-04-08 Enrollment: 12 United States
NCT04850118 Active not recruiting Phase3, Phase2
View Report
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-501
Therapy Type Gene transfer
Target Gene RPGR
Vector Type AAV2tYF
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2021-04-05 Last Modified Date: 2026-04-07 Enrollment: 85 Australia, Canada, United Kingdom + 1 more
NCT07048808 Recruiting Phase2
View Report
Refractory Angina, Coronary Artery Disease
Sponsor XyloCor Therapeutics, Inc. (Industry)
Compound XC001
Therapy Type Gene transfer
Target Gene VEGF
Vector Type Ad5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2025-06-24 Last Modified Date: 2026-04-07 Enrollment: 106 United States
NCT05881408 Active not recruiting Phase3
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound ELEVIDYS
Therapy Type Gene transfer
Target Gene Micro-dystrophin
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Priority Review, Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-19 Last Modified Date: 2026-04-07 Enrollment: 148 Australia, Belgium, Canada + 11 more
NCT05386680 Completed Phase3
View Report
Spinal Muscular Atrophy
Sponsor Novartis Pharmaceuticals (Industry)
Compound ITVISMA
Therapy Type Gene transfer
Target Gene SMN1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Orphan Drug Designation
First Submitted: 2022-05-18 Last Modified Date: 2026-04-07 Enrollment: 27 Australia, Belgium, Canada + 6 more
NCT05606614 Recruiting Phase3
View Report
Rett Syndrome
Sponsor Taysha Gene Therapies, Inc. (Industry)
Compound TSHA-102
Therapy Type Gene transfer
Target Gene MiniMECP2
Vector Type scAAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-10-28 Last Modified Date: 2026-04-07 Enrollment: 15 Canada, United States
NCT05432310 Recruiting Phase2, Phase1
View Report
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Sponsor University of California, Los Angeles (Other)
Compound Simoladagene autotemcel
Therapy Type Gene transfer
Target Gene ADA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-06-04 Last Modified Date: 2026-04-07 Enrollment: 20 United States
NCT05835895 Active not recruiting Phase1
View Report
Osteoarthritis, Knee
Sponsor Genascence Corporation (Industry)
Compound GNSC-001
Therapy Type Gene transfer
Target Gene IL1RN
Vector Type AAV2.5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2023-04-19 Last Modified Date: 2026-04-07 Enrollment: 67 United States
NCT03566043 Active not recruiting Phase3, Phase2
View Report
Mucopolysaccharidosis Type II (Hunter Syndrome)
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-121
Therapy Type Gene transfer
Target Gene IDS
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-05-01 Last Modified Date: 2026-04-07 Enrollment: 48 Brazil, United States
NCT06884865 Active not recruiting Phase2
View Report
Osteoarthritis (OA) of the Knee
Sponsor Pacira Pharmaceuticals, Inc (Industry)
Compound PCRX-201
Therapy Type Gene transfer
Target Gene IL1RA
Vector Type Ad5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-03-13 Last Modified Date: 2026-04-07 Enrollment: 135 United States
NCT06517888 Recruiting Phase2, Phase1
View Report
Vestibular Schwannoma
Sponsor Akouos, Inc. (Industry)
Compound AAVAnc80-antiVEGF
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAVAnc80
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-07-19 Last Modified Date: 2026-04-06 Enrollment: 27 United States
NCT05345171 Active not recruiting Phase3
View Report
Ornithine Transcarbamylase (OTC) Deficiency
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound DTX301
Therapy Type Gene transfer
Target Gene OTC
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2022-04-18 Last Modified Date: 2026-04-06 Enrollment: 32 Argentina, Brazil, Canada + 7 more
NCT06325709 Recruiting Phase2, Phase1
View Report
X-Linked Chronic Granulomatous Disease
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound Base-edited autologous CD34+ cells
Therapy Type Gene editing
Target Gene CYBB c.676 C>T
Vector Type none
Delivery System Electroporation
Mechanism of Action Mutation correction
First Submitted: 2024-03-21 Last Modified Date: 2026-04-06 Enrollment: 10 United States
NCT06063850 Recruiting Phase2, Phase1
View Report
Mesial Temporal Lobe Epilepsy
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-260
Therapy Type Gene transfer
Target Gene MiGRIK2
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
First Submitted: 2023-09-01 Last Modified Date: 2026-04-06 Enrollment: 12 United States
NCT04145037 Terminated Phase2, Phase1
View Report
Gaucher Disease Type 1
Sponsor AVROBIO (Industry)
Compound AVR-RD-02
Therapy Type Gene transfer
Target Gene GBA1
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-08-27 Last Modified Date: 2026-04-06 Enrollment: 8 Canada, United States
NCT05139316 Completed Phase3
View Report
Glycogen Storage Disease Type Ia
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound DTX401
Therapy Type Gene transfer
Target Gene G6PC
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-07-14 Last Modified Date: 2026-04-06 Enrollment: 49 Brazil, Canada, Denmark + 6 more
NCT05725018 Active not recruiting Phase3
View Report
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor Abeona Therapeutics, Inc (Industry)
Compound ZEVASKYN
Therapy Type Gene transfer
Target Gene COL7A1
Vector Type LZRSE
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-01-23 Last Modified Date: 2026-04-06 Enrollment: 12 United States
NCT06388200 Active not recruiting Phase3
View Report
Retinitis Pigmentosa
Sponsor Ocugen (Industry)
Compound OCU400
Therapy Type Gene transfer
Target Gene NR2E3
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-04-09 Last Modified Date: 2026-04-06 Enrollment: 140 Canada, United States
NCT02651675 Terminated Phase2, Phase1
View Report
Homozygous Familial Hypercholesterolemia (HoFH)
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-501
Therapy Type Gene transfer
Target Gene LDLR
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2016-01-04 Last Modified Date: 2026-04-06 Enrollment: 9 Canada, Italy, Netherlands + 1 more
NCT05956626 Active not recruiting Phase3, Phase2
View Report
Stargardt Disease
Sponsor Ocugen (Industry)
Compound OCU410ST
Therapy Type Gene transfer
Target Gene RORA
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2023-06-30 Last Modified Date: 2026-04-06 Enrollment: 51 United States
NCT03580083 Suspended Phase2, Phase1
View Report
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-111
Therapy Type Gene transfer
Target Gene IDUA
Vector Type AAV2/9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-06-18 Last Modified Date: 2026-04-06 Enrollment: 21 Brazil, United States
NCT05739643 Active not recruiting Phase2, Phase1
View Report
Krabbe Disease
Sponsor Forge Biologics, Inc (Industry)
Compound FBX-101
Therapy Type Gene transfer
Target Gene GALC
Vector Type AAVrh10
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-02-13 Last Modified Date: 2026-04-06 Enrollment: 9 United States
NCT04293185 Active not recruiting Phase3
View Report
Sickle Cell Disease
Sponsor Genetix Biotherapeutics Inc. (Industry)
Compound LYFGENIA
Therapy Type Gene transfer
Target Gene HBB
Vector Type BB305 LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-02-12 Last Modified Date: 2026-04-06 Enrollment: 35 United States
NCT06680232 Recruiting Phase1
View Report
Chronic Hepatitis B
Sponsor Precision BioSciences, Inc. (Industry)
Compound PBGENE-HBV
Therapy Type Gene editing
Target Gene HBV DNA
Vector Type LNP
Delivery System Lipid encapsulation
Mechanism of Action Gene inactivation
FDA Designations Fast Track
First Submitted: 2024-10-24 Last Modified Date: 2026-04-06 Enrollment: 45 Hong Kong, Moldova, New Zealand + 1 more
NCT06856577 Recruiting Phase3
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Adverum Biotechnologies, Inc. (Industry)
Compound ADVM-022
Therapy Type Gene transfer
Target Gene Codon optimized aflibercept
Vector Type AAV.7m8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2025-02-26 Last Modified Date: 2026-04-06 Enrollment: 284 United States
NCT04671433 Completed Phase3
View Report
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Janssen Research & Development, LLC (Industry)
Compound Botaretigene sparoparvovec
Therapy Type Gene transfer
Target Gene RPGR
Vector Type AAV2/5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-11-05 Last Modified Date: 2026-04-06 Enrollment: 105 Belgium, Canada, Denmark + 8 more
NCT06559176 Enrolling by invitation Phase2, Phase1
View Report
Chronic Granulomatous Disease
Sponsor Prime Medicine, Inc. (Industry)
Compound PM359
Therapy Type Gene editing
Target Gene NCF1 (c.75_76delGT)
Delivery System Electroporation
Mechanism of Action Mutation correction
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-07-11 Last Modified Date: 2026-04-06 Enrollment: 12 Canada, United Kingdom, United States
NCT03496012 Completed Phase3
View Report
Choroideremia
Sponsor Biogen (Industry)
Compound BIIB111
Therapy Type Gene transfer
Target Gene CHM
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2018-03-07 Last Modified Date: 2026-04-03 Enrollment: 169 Canada, Denmark, Finland + 5 more
NCT02652767 Completed Phase3
View Report
Leber Hereditary Optic Neuropathy (LHON)
Sponsor GenSight Biologics (Industry)
Compound LUMEVOQ
Therapy Type Gene transfer
Target Gene ND4G11778A
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-01-07 Last Modified Date: 2026-04-03 Enrollment: 39 France, Germany, Italy + 2 more
NCT00999609 Active not recruiting Phase3
View Report
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Sponsor Spark Therapeutics, Inc. (Industry)
Compound LUXTURNA
Therapy Type Gene transfer
Target Gene RPE65
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Priority Review, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2009-10-21 Last Modified Date: 2026-04-03 Enrollment: 31 United States
NCT00979238 Completed Phase1
View Report
Hemophilia B
Sponsor St. Jude Children's Research Hospital (Other)
Compound ScAAV2/8-LP1-hFIXco
Therapy Type Gene transfer
Target Gene F9
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2009-09-16 Last Modified Date: 2026-04-02 Enrollment: 14 United Kingdom, United States