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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

NCT03861273
Last update post date 2026-04-20 2026-05-11 api
Number of locations 65 66 api
Eligibility criteria null Inclusion criteria * males who completed 6 months... api
2026-05-12
Hemophilia B
Pfizer Active not recruiting
FDA approved 4/25/24, Price/treatment $3.5M; Pfizer will no longer market Beqvez
NCT05353647
Last update post date 2025-04-18 2026-05-12 api
Estimated completion date 2027-07 2027-10 api
Eligibility criteria null Inclusion criteria: 1. a diagnosis of sickle cell... api
2026-05-12
Sickle Cell Disease
David Williams Active not recruiting
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
2026-05-12
Neovascular Age-related Macular Degeneration
AbbVie Recruiting
Another Phase 3 study (NCT05407636) is active, enrollment is completed
NCT03368742
Last update post date 2026-03-03 2026-05-08 api
Eligibility criteria null Inclusion criteria: * established clinical diagno... api
2026-05-08
Duchenne Muscular Dystrophy (DMD)
Solid Biosciences Inc. Active not recruiting
In September 2022, Solid Biosciences announced they would be pausing activities for SGT-001; "No longer developing" as of 2024 Annual Report
NCT05504837
Last update post date 2025-08-21 2026-05-07 curator
Estimated completion date 2026-01 2027-03 curator
Eligibility criteria Inclusion criteria: 1. the subject must have read... Inclusion criteria: 1. the subject must have read... curator
Number of locations 5 6 curator
Enrollment count 12 20 curator
Recent updates Interim data expected for cohort 3 by 2025 year en... Interim data for cohort 3 (highest dose) presented... curator
News and press releases null LinkName:Krystal Biotech Announces Positive Interi... curator
2026-05-08
Cystic Fibrosis
Krystal Biotech, Inc. Recruiting
Interim data for cohort 3 (highest dose) presented in January 2026
33 more update(s) recently — View All

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Showing 1-100 of 265 results in ClinicalTrials
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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
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
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: 2026-05-07 Enrollment: 47 Australia, United Kingdom, 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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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: 2026-04-29 Enrollment: 12 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
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
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
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: 2026-04-28 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 more
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
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: 2026-04-18 Enrollment: 11 Bulgaria, France, Germany + 3 more
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
NCT04411654 Active not recruiting Phase2, Phase1
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Gaucher Disease Type 2
Sponsor Prevail Therapeutics (Industry)
Compound LY3884961
Therapy Type Gene transfer
Target Gene GBA1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-05-11 Last Modified Date: 2026-04-01 Enrollment: 7 United Kingdom, United States
NCT04611503 Active not recruiting Phase2, Phase1
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Retinitis Pigmentosa
Sponsor STZ eyetrial (Other)
Compound RAAV.hPDE6A
Therapy Type Gene transfer
Target Gene PDE6A
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2020-05-20 Last Modified Date: 2026-04-01 Enrollment: 9 Germany
NCT06185673 Recruiting Phase2, Phase1
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Oculopharyngeal Muscular Dystrophy
Sponsor Benitec Biopharma, Inc. (Industry)
Compound BB-301
Therapy Type Gene transfer
Target Gene PABPN1
Vector Type AAV9-PL
Delivery System Viral transduction
Mechanism of Action Functional gene replacement/gene inactivation
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2023-12-15 Last Modified Date: 2026-04-01 Enrollment: 30 United States
NCT06164730 Recruiting Phase1
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Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-102
Therapy Type Gene editing
Target Gene PCSK9
Vector Type LDLR + GalNAc
Delivery System Lipid encapsulation
Mechanism of Action Gene inactivation
FDA Designations Fast Track
First Submitted: 2023-12-01 Last Modified Date: 2026-04-01 Enrollment: 85 Australia, Canada, Israel + 3 more
NCT06662188 Recruiting Phase2, Phase1
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SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Sponsor Jaguar Gene Therapy, LLC (Industry)
Compound JAG201
Therapy Type Gene transfer
Target Gene SHANK3
Vector Type AAV2/9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Rare Pediatric Disease Designation
First Submitted: 2024-10-16 Last Modified Date: 2026-04-01 Enrollment: 6 United States
NCT06458595 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Chengdu Origen Biotechnology Co., Ltd. (Industry)
Compound KH658
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: 2024-06-09 Last Modified Date: 2026-04-01 Enrollment: 44 China
NCT06480461 Active not recruiting Phase2, Phase1
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Parkinson's Disease
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 Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2024-06-14 Last Modified Date: 2026-03-31 Enrollment: 39 China
NCT05454566 Not yet recruiting Phase2, Phase1
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Osteoarthritis, Knee
Sponsor ICM Co. Ltd. (Industry)
Compound ICM-203
Therapy Type Gene transfer
Target Gene NKX3-2
Vector Type AAV5.2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2022-07-04 Last Modified Date: 2026-03-27 Enrollment: 18
NCT05071222 Recruiting Phase2, Phase1
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Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency
Sponsor Assistance Publique - Hôpitaux de Paris (Other)
Compound ARTEGENE
Therapy Type Gene transfer
Target Gene DCLRE1C
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-08-27 Last Modified Date: 2026-03-27 Enrollment: 7 France
NCT05665166 Active not recruiting Phase2, Phase1
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Mucopolysaccharidosis II
Sponsor University of Manchester (Other)
Compound AVR-RD-05
Therapy Type Gene transfer
Target Gene IDS
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-12-07 Last Modified Date: 2026-03-27 Enrollment: 5 United Kingdom
NCT07185256 Recruiting Phase2, Phase1
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ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
Sponsor Opus Genetics, Inc (Industry)
Compound OPGx-BEST1
Therapy Type Gene transfer
Target Gene BEST1
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-07-17 Last Modified Date: 2026-03-24 Enrollment: 10 United States
NCT05836259 Recruiting Phase2, Phase1
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Hypertrophic Cardiomyopathy
Sponsor Tenaya Therapeutics (Industry)
Compound TN-201
Therapy Type Gene transfer
Target Gene MYBPC3
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-04-18 Last Modified Date: 2026-03-20 Enrollment: 30 United States
NCT04884815 Active not recruiting Phase2, Phase1
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Wilson Disease
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound UX701
Therapy Type Gene transfer
Target Gene ATP7B
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2021-05-07 Last Modified Date: 2026-03-18 Enrollment: 82 Canada, Portugal, Spain + 2 more
NCT06747273 Terminated Phase1
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Limb-Girdle Muscular Dystrophy, Type 2D/R3
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-9004
Therapy Type Gene transfer
Target Gene SGCA
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-12-18 Last Modified Date: 2026-03-18 Enrollment: 4 United States
NCT05973630 Active not recruiting Phase1
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Limb-Girdle Muscular Dystrophy, Type 2C/R5
Sponsor Atamyo Therapeutics (Industry)
Compound ATA-200
Therapy Type Gene transfer
Target Gene SGCG
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-07-24 Last Modified Date: 2026-03-18 Enrollment: 4 United States
NCT01519349 Completed Phase1
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Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis
Sponsor Nationwide Children's Hospital (Other)
Compound RAAV1.CMV.huFollistatin344
Therapy Type Gene transfer
Target Gene FST (FS344)
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2012-01-23 Last Modified Date: 2026-03-17 Enrollment: 15 United States
NCT02354781 Completed Phase2, Phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor Jerry R. Mendell (Other)
Compound RAAV1.CMV.huFollistatin344
Therapy Type Gene transfer
Target Gene FST (FS344)
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2015-01-26 Last Modified Date: 2026-03-17 Enrollment: 3 United States
NCT06983158 Suspended Phase2, Phase1
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Developmental and Epileptic Encephalopathy
Sponsor Capsida Biotherapeutics, Inc. (Industry)
Compound CAP-002
Therapy Type Gene transfer
Target Gene STXBP1
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2025-05-05 Last Modified Date: 2026-03-17 Enrollment: 12 United States
NCT05092685 Recruiting Phase2, Phase1
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Ornithine Transcarbamylase (OTC) Deficiency
Sponsor University College, London (Other)
Compound BGT-OTCD
Therapy Type Gene transfer
Target Gene OTC
Vector Type AAV-LK03
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2021-09-28 Last Modified Date: 2026-03-17 Enrollment: 12 United Kingdom
NCT03315182 Terminated Phase2, Phase1
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Mucopolysaccharidosis Type 3 B
Sponsor Abeona Therapeutics, Inc (Industry)
Compound ABO-101
Therapy Type Gene transfer
Target Gene NAGLU
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-10-10 Last Modified Date: 2026-03-17 Enrollment: 11 France, Germany, Spain + 1 more
NCT02618915 Terminated Phase2, Phase1
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Hemophilia B
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound DTX101
Therapy Type Gene transfer
Target Gene F9
Vector Type AAVrh10
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2015-11-23 Last Modified Date: 2026-03-17 Enrollment: 6 Bulgaria, United Kingdom, United States
NCT05672121 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Chengdu Origen Biotechnology Co., Ltd. (Industry)
Compound KH631
Therapy Type Gene transfer
Target Gene VEGFR1/R2 fusion protein
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2022-12-21 Last Modified Date: 2026-03-17 Enrollment: 42 China
NCT04273269 Terminated Phase2, Phase1
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GM1 Gangliosidosis
Sponsor LYSOGENE (Industry)
Compound LYS-GM101
Therapy Type Gene transfer
Target Gene GLB1
Vector Type AAVrh.10
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-01-21 Last Modified Date: 2026-03-17 Enrollment: 5 France, United Kingdom, United States
NCT04093349 Active not recruiting Phase2, Phase1
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Glycogen Storage Disease Type 2 (Pompe Disease)
Sponsor Spark Therapeutics, Inc. (Industry)
Compound SPK-3006
Therapy Type Gene transfer
Target Gene GAA
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2019-09-16 Last Modified Date: 2026-03-17 Enrollment: 4 Canada, Denmark, France + 5 more
NCT06641154 Recruiting Phase2, Phase1
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Crigler-Najjar Syndrome Type I
Sponsor Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare (Other gov)
Compound GT-UGT1A1-AAV8-02
Therapy Type Gene transfer
Target Gene UGT1A1
Vector Type rAAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-10-11 Last Modified Date: 2026-03-17 Enrollment: 5 Russia
NCT04443907 Terminated Phase1
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Sickle Cell Disease
Sponsor Novartis Pharmaceuticals (Industry)
Compound OTQ923
Therapy Type Gene editing
Target Gene HBG1/HBG2
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2020-04-29 Last Modified Date: 2026-03-17 Enrollment: 4 United States
NCT05906251 Terminated Phase1
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Limb Girdle Muscular Dystrophy, Type 2B/R2
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-6004
Therapy Type Gene transfer
Target Gene DYSF
Vector Type dual AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-06-07 Last Modified Date: 2026-03-17 Enrollment: 2 United States
NCT04091737 Terminated Phase1
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Sickle Cell Disease
Sponsor CSL Behring (Industry)
Compound CSL200
Therapy Type Gene transfer
Target Gene HBG1/HBG2
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2019-09-13 Last Modified Date: 2026-03-17 Enrollment: 1 United States
NCT06839235 Recruiting Phase2, Phase1
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Primary Hyperoxaluria Type 1 (PH1)
Sponsor Arbor Biotechnologies (Industry)
Compound ABO-101
Therapy Type Gene editing
Target Gene HAO1
Vector Type LNP
Delivery System Lipid encapsulation
Mechanism of Action Gene inactivation
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-02-17 Last Modified Date: 2026-03-17 Enrollment: 23 France, Germany, Tunisia + 2 more
NCT00028236 Completed Phase1
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X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound GALV MFGS-gc retrovirus
Therapy Type Gene transfer
Target Gene IL2RG
Vector Type MFGS/GALV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2001-12-17 Last Modified Date: 2026-03-17 Enrollment: 3 United States
NCT01344798 Completed Phase1
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Limb-Girdle Muscular Dystrophy, Type 2C/R5
Sponsor Genethon (Other)
Compound AAV1.DES.hSGCG
Therapy Type Gene transfer
Target Gene SGCG
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2011-03-17 Last Modified Date: 2026-03-17 Enrollment: 9 France
NCT04525352 Terminated Phase1
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Infantile Malignant Osteopetrosis
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-L401
Therapy Type Gene transfer
Target Gene TCIRG1
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track
First Submitted: 2020-08-11 Last Modified Date: 2026-03-17 Enrollment: 1 United States
NCT06332807 Recruiting Phase2, Phase1
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Phenylketonuria (PKU)
Sponsor NGGT INC. (Industry)
Compound NGGT002
Therapy Type Gene transfer
Target Gene PAH
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-02-07 Last Modified Date: 2026-03-17 Enrollment: 12 United States
NCT05858983 Recruiting Phase2, Phase1
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Biallelic RPE65 Mutation-associated Retinal Dystrophy
Sponsor Frontera Therapeutics (Industry)
Compound FT-001
Therapy Type Gene transfer
Target Gene RPE65
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-04-29 Last Modified Date: 2026-03-17 Enrollment: 9 China
NCT05986864 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Skyline Therapeutics (US) Inc. (Industry)
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: 2023-08-03 Last Modified Date: 2026-03-17 Enrollment: 68 China, United States
NCT04240314 Completed Phase2, Phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor Megan Waldrop (Other)
Compound AT702
Therapy Type Gene transfer
Target Gene DMD (exon 2)
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Exon skipping/splice editor
First Submitted: 2020-01-22 Last Modified Date: 2026-03-17 Enrollment: 3 United States
NCT06647979 Recruiting Phase1
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Sickle Cell Disease, Beta-Thalassemia
Sponsor Daniel Bauer (Other)
Compound Autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein
Therapy Type Gene editing
Target Gene BCL11A
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-10-11 Last Modified Date: 2026-03-17 Enrollment: 10 United States
NCT04783181 Active not recruiting Phase2, Phase1
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Congenital Adrenal Hyperplasia
Sponsor Adrenas Therapeutics Inc (Industry)
Compound BBP-631
Therapy Type Gene transfer
Target Gene CYP21A2
Vector Type AAV5
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: 2021-03-01 Last Modified Date: 2026-03-17 Enrollment: 8 United States
NCT03311503 Recruiting Phase2, Phase1
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X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor David Williams (Other)
Compound G2SCID lentiviral vector transduced CD34+ cells
Therapy Type Gene transfer
Target Gene IL2RG
Vector Type HIV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-10-12 Last Modified Date: 2026-03-17 Enrollment: 12 United States
NCT02416622 Completed Phase2, Phase1
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X-Linked Retinoschisis
Sponsor Beacon Therapeutics (Industry)
Compound RAAV2tYF-CB-hRS1
Therapy Type Gene transfer
Target Gene RS1
Vector Type AAV2tYF
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2015-04-02 Last Modified Date: 2026-03-17 Enrollment: 27 United States
NCT05824169 Recruiting Phase2, Phase1
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Spinal Muscular Atrophy
Sponsor GeneCradle Inc (Industry)
Compound GC101
Therapy Type Gene transfer
Target Gene SMN1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2023-04-10 Last Modified Date: 2026-03-17 Enrollment: 18 China
NCT00076557 Terminated Phase2, Phase1
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Hemophilia B
Sponsor Avigen (Industry)
Compound AAV2-hFIX16
Therapy Type Gene transfer
Target Gene F9
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2004-01-26 Last Modified Date: 2026-03-17 Enrollment: 15 United States
NCT04798235 Active not recruiting Phase2, Phase1
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Infantile GM2 Gangliosidosis
Sponsor Dr. Anupam Sehgal (Other)
Compound TSHA-101
Therapy Type Gene transfer
Target Gene HEXA/HEXB
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-02-22 Last Modified Date: 2026-03-17 Enrollment: 3 Canada
NCT01161576 Completed Phase1
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CLN2 Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis
Sponsor Weill Medical College of Cornell University (Other)
Compound LX1004
Therapy Type Gene transfer
Target Gene TPP1
Vector Type AAVrh10
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2010-03-22 Last Modified Date: 2026-03-17 Enrollment: 12 United States
NCT06370351 Recruiting Phase2, Phase1
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OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders
Sponsor Sensorion (Industry)
Compound SENS-501
Therapy Type Gene transfer
Target Gene OTOF
Vector Type dual AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-04-09 Last Modified Date: 2026-03-17 Enrollment: 12 Australia, France
NCT06141460 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Shanghai Refreshgene Technology Co., Ltd. (Industry)
Compound RRG001
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: 2023-11-10 Last Modified Date: 2026-03-17 Enrollment: 48 China
NCT05541627 Active not recruiting Phase2, Phase1
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Huntington's Disease
Sponsor Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio) (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: 2026-03-17 Enrollment: 5 France