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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.

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Showing 1-100 of 385 results in ClinicalTrials
NCT05614531 Enrolling by invitation Phase2, Phase1
View Report
Spinal Muscular Atrophy Type I
Sponsor Hangzhou Jiayin Biotech Ltd (Industry)
Compound EXG001-307
Therapy Type Gene transfer
Target Gene SMN1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-11-02 Last Modified Date: 2026-02-04 Enrollment: 12 China
NCT06185673 Recruiting Phase2, Phase1
View Report
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: 2026-02-04 Enrollment: 30 United States
NCT07190001 Not yet recruiting Early phase1
View Report
Hemoglobinopathies (Transfusion-dependent β-thalassemia and Sickle Cell Disease)
Sponsor Guangzhou Women and Children's Medical Center (Other)
Compound YOLT-204
Therapy Type Gene editing
Target Gene HBG1
Delivery System Lipid encapsulation
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2025-09-16 Last Modified Date: 2026-02-04 Enrollment: 18 China
NCT03727555 Recruiting Na
View Report
X-linked Adrenoleukodystrophy
Sponsor Shenzhen Geno-Immune Medical Institute (Other)
Compound (LV) TYF-ABCD1
Therapy Type Gene transfer
Target Gene ABCD1
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-09-25 Last Modified Date: 2026-02-04 Enrollment: 30 China
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: 2026-02-04 Enrollment: 10 United States
NCT04567550 Active not recruiting Phase2
View Report
Diabetic Retinopathy (DR), Center-Involved Diabetic Macular Edema (CI-DME)
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: 2020-09-15 Last Modified Date: 2026-02-04 Enrollment: 139 United States
NCT01422772 Completed Phase2, Phase1
View Report
Ischemic Heart Disease
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapy Type Gene transfer
Target Gene HGF
Vector Type none
Delivery System None (naked plasmid)
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2011-08-21 Last Modified Date: 2026-02-04 Enrollment: 9 South Korea
NCT01643330 Completed Phase2
View Report
Ischemic Cardiomyopathy, Non-ischemic Cardiomyopathy, Heart Failure, Cardiomyopathies
Sponsor Celladon Corporation (Industry)
Compound AAV1-CMV-Serca2a
Therapy Type Gene transfer
Target Gene SERCA2A
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2012-07-16 Last Modified Date: 2026-02-04 Enrollment: 250 Belgium, Denmark, Germany + 7 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: 2026-02-04 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 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: 2026-02-04 Enrollment: 27 Australia, Belgium, Canada + 6 more
NCT06300723 Enrolling by invitation Na
View Report
Sickle Cell Disease, Beta Thalassemia
Sponsor Bioray Laboratories (Industry)
Compound BRL-101
Therapy Type Gene editing
Target Gene BCL11A
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-03-03 Last Modified Date: 2026-02-04 Enrollment: 3 China
NCT06942572 Recruiting Phase2, Phase1
View Report
Stargardt Disease
Sponsor Splice Bio (Industry)
Compound SB-007
Therapy Type Gene transfer
Target Gene ABCA4
Vector Type dual AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2025-02-18 Last Modified Date: 2026-02-04 Enrollment: 57 United States
NCT07185256 Recruiting Phase2, Phase1
View Report
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: 2026-02-04 Enrollment: 10 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: 2026-02-04 Enrollment: 2 United States
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: 2026-02-04 Enrollment: 62 Canada, New Zealand
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: 2026-01-06 Enrollment: 30 United States
NCT03569891 Completed Phase3
View Report
Hemophilia B
Sponsor CSL Behring (Industry)
Compound HEMGENIX
Therapy Type Gene transfer
Target Gene F9-R338L
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Priority Review, Orphan Drug Designation
First Submitted: 2018-06-14 Last Modified Date: 2026-01-06 Enrollment: 67 Belgium, Denmark, Germany + 5 more
NCT05456880 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 Regenerative Medicine Advanced Therapy
First Submitted: 2022-06-23 Last Modified Date: 2026-01-06 Enrollment: 15 United States
NCT03837483 Active not recruiting Phase3
View Report
Wiskott-Aldrich Syndrome
Sponsor Fondazione Telethon (Other)
Compound WASKYRA
Therapy Type Gene transfer
Target Gene WAS
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2019-02-08 Last Modified Date: 2026-01-06 Enrollment: 10 Italy, United States
NCT06983158 Suspended Phase2, Phase1
View Report
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: 2026-01-06 Enrollment: 12 United States
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, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-01-23 Last Modified Date: 2026-01-06 Enrollment: 12 United States
NCT05248230 Recruiting Phase2, Phase1
View Report
Cystic Fibrosis
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-710
Therapy Type Gene transfer
Target Gene CFTR
Vector Type AAV A101
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-02-10 Last Modified Date: 2026-01-06 Enrollment: 40 United States
NCT05477563 Recruiting Phase3
View Report
Beta-Thalassemia, Sickle Cell Disease
Sponsor Vertex Pharmaceuticals Incorporated (Industry)
Compound CASGEVY
Therapy Type Gene editing
Target Gene BCL11A
Vector Type RNP
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2022-07-26 Last Modified Date: 2026-01-06 Enrollment: 26 Germany, Italy, Saudi Arabia + 1 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: 2026-01-06 Enrollment: 39 France, Germany, Italy + 2 more
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: 2026-01-06 Enrollment: 33 Australia, Canada, Germany + 4 more
NCT05139316 Active not recruiting 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 Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2021-07-14 Last Modified Date: 2026-01-06 Enrollment: 49 Brazil, Canada, Denmark + 6 more
NCT06948019 Not yet recruiting Phase2, Phase1
View Report
Hereditary Spastic Paraplegia Type 47
Sponsor BlackfinBio Ltd (Industry)
Compound BFB-101
Therapy Type Gene transfer
Target Gene AP4B1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-04-22 Last Modified Date: 2025-12-18 Enrollment: 5 United States
NCT01301443 Completed Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration
Sponsor Oxford BioMedica (Industry)
Compound RetinoStat
Therapy Type Gene transfer
Target Gene COL18A1+PLG
Vector Type EIAV
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2011-02-21 Last Modified Date: 2025-12-18 Enrollment: 21 United States
NCT06461702 Recruiting 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: 2025-12-18 Enrollment: 13 China
NCT00891306 Completed Phase3, Phase2
View Report
Familial Lipoprotein Lipase Deficiency
Sponsor Amsterdam Molecular Therapeutics (Industry)
Compound GLYBERA
Therapy Type Gene transfer
Target Gene LPL
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2009-04-30 Last Modified Date: 2025-12-17 Enrollment: 5 Canada
NCT06818838 Recruiting Phase2, Phase1
View Report
Gaucher Disease Type 1
Sponsor Lingyi Biotech Co., Ltd. (Industry)
Compound LY-M001
Therapy Type Gene transfer
Target Gene GBA1
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-01-24 Last Modified Date: 2025-12-17 Enrollment: 18 China
NCT07223944 Not yet 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
First Submitted: 2025-10-30 Last Modified Date: 2025-12-17 Enrollment: 45
NCT07195825 Not yet recruiting Phase1
View Report
Parkinson's Disease
Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound BBM-P002
Therapy Type Undisclosed
Target Gene Undisclosed
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Undisclosed
First Submitted: 2025-09-10 Last Modified Date: 2025-12-17 Enrollment: 18 China
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
First Submitted: 2025-07-07 Last Modified Date: 2025-12-17 Enrollment: 400 United States
NCT04211714 Recruiting Phase1
View Report
Telomere Shortening, Bone Marrow Failure
Sponsor Elixirgen Therapeutics, Inc. (Industry)
Compound EXG34217
Therapy Type Gene transfer
Target Gene ZSCAN4
Vector Type SeV18/TS15ΔF
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2019-12-06 Last Modified Date: 2025-12-17 Enrollment: 12 United States
NCT01505062 Terminated Phase2, Phase1
View Report
Usher Syndrome, Retinitis Pigmentosa
Sponsor Sanofi (Industry)
Compound SAR421869
Therapy Type Gene transfer
Target Gene MYO7A
Vector Type EIAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2012-01-04 Last Modified Date: 2025-12-17 Enrollment: 9 France, United States
NCT06559176 Active not recruiting 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: 2025-12-16 Enrollment: 12 Canada, United Kingdom, United States
NCT06650319 Recruiting Early phase1
View Report
Wilson Disease
Sponsor Chaohui Yu (Other)
Compound LY-M003
Therapy Type Gene transfer
Target Gene ATP7B
Vector Type AAV8
Delivery System Viral transdution
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-10-15 Last Modified Date: 2025-12-16 Enrollment: 10 China
NCT06839235 Recruiting Phase2, Phase1
View Report
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: 2025-12-16 Enrollment: 23 France, Germany, United States
NCT06152237 Active not recruiting Phase2, Phase1
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, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-11-21 Last Modified Date: 2025-12-16 Enrollment: 6 Canada, United Kingdom, United States
NCT06526923 Recruiting Phase2, Phase1
View Report
Cystic Fibrosis
Sponsor Spirovant Sciences, Inc. (Industry)
Compound SP-101
Therapy Type Gene transfer
Target Gene CFTRΔR
Vector Type AAV2.5T
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-07-18 Last Modified Date: 2025-12-16 Enrollment: 15 United States
NCT06722170 Recruiting Na
View Report
DFNB9, Congenital Hearing Loss
Sponsor Yilai Shu (Other)
Compound EH002
Therapy Type Gene transfer
Target Gene OTOF
Vector Type dual AAV1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-12-05 Last Modified Date: 2025-12-16 Enrollment: 24 China
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: 2025-12-16 Enrollment: 32 Argentina, United States
NCT06736080 Not yet recruiting Phase2, Phase1
View Report
Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3)
Sponsor Assistance Publique - Hôpitaux de Paris (Other)
Compound Autologous Cells Transduced Ex Vivo with LV-UNC13D
Therapy Type Gene transfer
Target Gene UNC13D
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-12-04 Last Modified Date: 2025-12-16 Enrollment: 5 France
NCT06623279 Not yet recruiting Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)
Compound HG202
Therapy Type Gene editing
Target Gene VEGFA mRNA
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2024-09-30 Last Modified Date: 2025-12-16 Enrollment: 15
NCT06856759 Recruiting Early phase1
View Report
Rett Syndrome
Sponsor Guangzhou Women and Children's Medical Center (Other)
Compound AAV-MECP2
Therapy Type Gene transfer
Target Gene MECP2
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-12-29 Last Modified Date: 2025-12-16 Enrollment: 8 China
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 Fast Track, Regenerative Medicine Advanced Therapy
First Submitted: 2025-02-26 Last Modified Date: 2025-12-16 Enrollment: 284 United States
NCT05672121 Recruiting Phase2, Phase1
View Report
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: 2025-12-16 Enrollment: 42 China
NCT06662188 Recruiting Phase2, Phase1
View Report
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: 2025-12-16 Enrollment: 6 United States
NCT06432140 Not yet recruiting Phase1
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: 2025-12-16 Enrollment: 16
NCT07169175 Not yet recruiting Phase2, Phase1
View Report
Amyotrophic Lateral Sclerosis (ALS)
Sponsor SineuGene Therapeutics Co., Ltd. (Industry)
Compound SNUG01
Therapy Type Gene transfer
Target Gene TRIM72
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Orphan Drug Designation
First Submitted: 2025-08-08 Last Modified Date: 2025-12-16 Enrollment: 21 China, United States
NCT06761183 Not yet recruiting Early phase1
View Report
Ischemic Stroke
Sponsor Beijing Tiantan Hospital (Other)
Compound NXL-001
Therapy Type Gene transfer
Target Gene NeuroD1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-12-27 Last Modified Date: 2025-12-16 Enrollment: 9 China
NCT06539208 Recruiting Phase2, Phase1
View Report
Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy
Sponsor YolTech Therapeutics Co., Ltd (Industry)
Compound YOLT-201
Therapy Type Gene editing
Target Gene TTR
Vector Type LNP
Delivery System Lipid encapsulation
Mechanism of Action Gene inactivation
First Submitted: 2024-08-01 Last Modified Date: 2025-12-16 Enrollment: 31 China
NCT06706427 Active not recruiting Phase2, Phase1
View Report
Bietti Crystalline Dystrophy
Sponsor NGGT (Suzhou) Biotechnology Co., Ltd. (Industry)
Compound NGGT001
Therapy Type Gene transfer
Target Gene CYP4V2
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-11-14 Last Modified Date: 2025-12-16 Enrollment: 12 China
NCT06511349 Recruiting Early phase1
View Report
Type 1 Primary Hyperoxaluria
Sponsor RenJi Hospital (Other)
Compound YOLT-203
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: 2024-07-04 Last Modified Date: 2025-12-16 Enrollment: 21 China
NCT06826612 Recruiting Phase2, Phase1
View Report
Huntington's Disease
Sponsor Spark Therapeutics, Inc. (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: 2025-12-16 Enrollment: 53 United States
NCT06735755 Recruiting Phase2, Phase1
View Report
Glycogen Storage Disease Type Ia
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-301
Therapy Type Gene editing
Target Gene G6PC1 (p.R83C)
Vector Type LNP
Delivery System Lipid encapsulation
Mechanism of Action Mutation correction
First Submitted: 2024-12-06 Last Modified Date: 2025-12-16 Enrollment: 36 United States
NCT06615206 Recruiting Na
View Report
MECP2 Duplication Syndrome
Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)
Compound HG204
Therapy Type Gene editing
Target Gene MECP2
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Gene inactivation
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-09-04 Last Modified Date: 2025-12-16 Enrollment: 6 China
NCT07058662 Recruiting Phase2, Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Belief BioMed (Beijing) Co., Ltd (Industry)
Compound BBM-D101
Therapy Type Gene transfer
Target Gene Undisclosed
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Undisclosed
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-07-01 Last Modified Date: 2025-12-16 Enrollment: 9 China
NCT06699108 Not yet recruiting Phase3
View Report
Bietti Crystalline Dystrophy
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGR-R01
Therapy Type Gene transfer
Target Gene CYP4V2
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-11-19 Last Modified Date: 2025-12-16 Enrollment: 45 China
NCT06831825 Recruiting Phase1
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Heart Failure With Reduced Ejection Fraction
Sponsor YAP Therapeutics, Inc. (Industry)
Compound YAP101
Therapy Type Gene transfer
Target Gene SAV1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2025-01-23 Last Modified Date: 2025-12-16 Enrollment: 24 United States
NCT06692712 Not yet recruiting Phase3
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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: 2025-12-16 Enrollment: 24
NCT06634420 Active not recruiting Phase3
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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 Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2024-10-07 Last Modified Date: 2025-12-16 Enrollment: 60 Australia, Canada, France + 6 more
NCT06611436 Recruiting Phase2, Phase1
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Hemophilia B
Sponsor Be Biopharma (Industry)
Compound BE-101
Therapy Type Gene editing
Target Gene F9
Vector Type AAV6
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2024-09-18 Last Modified Date: 2025-12-16 Enrollment: 24 United States
NCT06680232 Recruiting Phase1
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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: 2025-12-16 Enrollment: 45 Hong Kong, Moldova, New Zealand + 1 more
NCT06833983 Recruiting Phase3
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Hemophilia A
Sponsor Gritgen Therapeutics Co., Ltd. (Industry)
Compound GS1191-0445
Therapy Type Gene transfer
Target Gene F8
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-01-28 Last Modified Date: 2025-12-16 Enrollment: 50 China
NCT06515002 Active not recruiting Phase2, Phase1
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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: 2025-12-16 Enrollment: 36 France, Italy, Netherlands + 2 more
NCT06389877 Recruiting Phase2, Phase1
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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 Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2024-04-22 Last Modified Date: 2025-12-15 Enrollment: 106 Australia, Netherlands, New Zealand + 1 more
NCT06391736 Recruiting Phase2, Phase1
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Pompe Disease (Late-onset)
Sponsor GeneCradle Inc (Industry)
Compound GC301
Therapy Type Gene transfer
Target Gene GAA
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-04-25 Last Modified Date: 2025-12-15 Enrollment: 33 China
NCT06467344 Recruiting Phase2, Phase1
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Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration
Sponsor Ascidian Therapeutics, Inc (Industry)
Compound ACDN-01
Therapy Type Gene editing
Target Gene ABCA4 pre-mRNA
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Exon skipping/splice editor
FDA Designations Fast Track, Rare Pediatric Disease Designation
First Submitted: 2024-05-31 Last Modified Date: 2025-12-15 Enrollment: 15 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: 2025-12-15 Enrollment: 12 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: 2025-12-15 Enrollment: 44 China
NCT06345898 Recruiting Early phase1
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X-Linked Retinoschisis
Sponsor West China Hospital (Other)
Compound JWK002
Therapy Type Gene transfer
Target Gene RS1
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-03-28 Last Modified Date: 2025-12-15 Enrollment: 12 China
NCT06483802 Withdrawn Phase1
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Friedreich Ataxia, Cardiomyopathy
Sponsor Astellas Gene Therapies (Industry)
Compound ASP2016
Therapy Type Gene transfer
Target Gene FXN
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track
First Submitted: 2024-06-26 Last Modified Date: 2025-12-15 Enrollment: 0
NCT06480461 Not yet 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: 2025-12-15 Enrollment: 39
NCT06325709 Recruiting Phase2, Phase1
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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: 2025-12-15 Enrollment: 10 United States
NCT06474442 Recruiting Phase2
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Herpes Simplex Virus Type I Stromal Keratitis
Sponsor Shanghai BDgene Co., Ltd. (Industry)
Compound BD111
Therapy Type Gene editing
Target Gene HSV genes
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Gene excision
FDA Designations Orphan Drug Designation
First Submitted: 2024-06-13 Last Modified Date: 2025-12-15 Enrollment: 40 China
NCT06388200 Recruiting Phase3
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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 Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2024-04-09 Last Modified Date: 2025-12-15 Enrollment: 150 Canada, United States
NCT06460844 Recruiting Phase1
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Retinitis Pigmentosa, Choroideremia
Sponsor Ray Therapeutics, Inc. (Industry)
Compound RTx-015
Therapy Type Gene transfer
Target Gene Codon optimized ChR-3M
Vector Type AAV.7m8
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-06-10 Last Modified Date: 2025-12-15 Enrollment: 18 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: 2025-12-15 Enrollment: 12 Australia, France
NCT06451770 Recruiting Phase1
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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: 2025-12-15 Enrollment: 36 Australia, Canada, South Africa + 1 more
NCT06465550 Recruiting Phase1
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Beta-Thalassemia Major
Sponsor Shanghai BDgene Co., Ltd. (Industry)
Compound BD211
Therapy Type Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-06-12 Last Modified Date: 2025-12-15 Enrollment: 9 China
NCT06492850 Recruiting Phase2, Phase1
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X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Frontera Therapeutics (Industry)
Compound FT-002
Therapy Type Gene transfer
Target Gene RPGRORF15
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2024-07-01 Last Modified Date: 2025-12-15 Enrollment: 32 China
NCT06422351 Not yet recruiting Phase2
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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 Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2024-05-15 Last Modified Date: 2025-12-15 Enrollment: 10 Spain, United States
NCT06492863 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Frontera Therapeutics (Industry)
Compound FT-003
Therapy Type Gene transfer
Target Gene Aflibercept
Vector Type AAV2.7m8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2024-07-01 Last Modified Date: 2025-12-15 Enrollment: 78 China
NCT06392724 Active not recruiting Early phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor Peking Union Medical College Hospital (Other)
Compound GEN6050X
Therapy Type Gene editing
Target Gene DMD
Vector Type dual AAV9
Delivery System Viral transduction
Mechanism of Action Exon skipping/splice editor
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-04-26 Last Modified Date: 2025-12-15 Enrollment: 3 China
NCT06270316 Recruiting Phase2, Phase1
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Fabry Disease
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-191
Therapy Type Gene transfer
Target Gene GLA
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2023-12-19 Last Modified Date: 2025-12-12 Enrollment: 12 United States
NCT06246513 Active not recruiting Phase3
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Limb-Girdle Muscular Dystrophy, Type 2E/R4
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-9003
Therapy Type Gene transfer
Target Gene SGCB
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-01-30 Last Modified Date: 2025-12-12 Enrollment: 17 Belgium, Germany, Italy + 3 more
NCT06228924 Recruiting Phase1
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Arrhythmogenic Right Ventricular Cardiomyopathy
Sponsor Tenaya Therapeutics (Industry)
Compound TN-401
Therapy Type Gene transfer
Target Gene PKP2
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-01-18 Last Modified Date: 2025-12-12 Enrollment: 15 United States
NCT06253507 Enrolling by invitation Phase2, Phase1
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Autosomal Recessive Chronic Granulomatous Disease (CGD)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound PCHIM-p47
Therapy Type Gene transfer
Target Gene NCF1
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-02-09 Last Modified Date: 2025-12-12 Enrollment: 10 United States
NCT06280378 Recruiting Phase2, Phase1
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Beta-Thalassemia Major
Sponsor Kanglin Biotechnology (Hangzhou) Co., Ltd. (Industry)
Compound KL003
Therapy Type Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-02-20 Last Modified Date: 2025-12-12 Enrollment: 41 China
NCT06288230 Recruiting Phase2, Phase1
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Spinal Muscular Atrophy
Sponsor Lantu Biopharma (Industry)
Compound Vesemnogene lantuparvovec
Therapy Type Gene transfer
Target Gene SMN1
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-02-18 Last Modified Date: 2025-12-12 Enrollment: 20 China
NCT06217861 Recruiting Phase1
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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: 2025-12-12 Enrollment: 12 China
NCT06292650 Recruiting Early phase1
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Retinitis Pigmentosa
Sponsor Zhongmou Therapeutics (Industry)
Compound ZM-02
Therapy Type Gene transfer
Target Gene Ch2.0
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2024-02-27 Last Modified Date: 2025-12-12 Enrollment: 12 China
NCT06285643 Recruiting Phase2
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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 Fast Track, Regenerative Medicine Advanced Therapy
First Submitted: 2024-02-14 Last Modified Date: 2025-12-12 Enrollment: 87 Germany, Poland, United Kingdom + 1 more
NCT06293729 Not yet recruiting Early phase1
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Refractory Hypercholesterolemia, Familial Hypercholesterolemia
Sponsor Suzhou Municipal Hospital (Other)
Compound NGGT006
Therapy Type Gene transfer
Target Gene LDLR
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-02-27 Last Modified Date: 2025-12-12 Enrollment: 9
NCT06255782 Recruiting Phase2, Phase1
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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 Fast Track
First Submitted: 2023-12-19 Last Modified Date: 2025-12-12 Enrollment: 8 Australia, Spain, United Kingdom + 1 more
NCT06297486 Withdrawn Phase3
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Hemophilia A
Sponsor Spark Therapeutics, Inc. (Industry)
Compound Dirloctogene samoparvovec
Therapy Type Gene transfer
Target Gene F8
Vector Type LK03
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-02-29 Last Modified Date: 2025-12-12 Enrollment: 0 United States
NCT05984927 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Neuracle Genetics, Inc (Industry)
Compound NG101
Therapy Type Gene transfer
Target Gene Codon optimized aflibercept
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Fast Track
First Submitted: 2023-07-24 Last Modified Date: 2025-12-09 Enrollment: 18 Canada, 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: 2025-12-09 Enrollment: 36 Australia, Canada, Israel + 2 more