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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.

Showing 1-100 of 129 results in ClinicalTrials
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NCT05788536 Recruiting Phase2, Phase1
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Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Sponsor Regeneron Pharmaceuticals (Industry)
Compound OTARMENI
Therapeutic Modality 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: 2026-07-01 Enrollment: 36 Germany, Japan, Spain + 2 more
NCT07592273 Recruiting Phase3, Phase2
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Diabetic Retinopathy
Sponsor AbbVie (Industry)
Compound Surabgene lomparvovec
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2026-04-24 Last Modified Date: 2026-07-01 Enrollment: 576 Puerto Rico, United States
NCT05693142 Recruiting Phase3, Phase2
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Duchenne Muscular Dystrophy (DMD)
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-202
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-01-04 Last Modified Date: 2026-06-30 Enrollment: 65 Canada, United States
NCT07007065 Recruiting Phase3
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Neovascular Age-related Macular Degeneration
Sponsor AbbVie (Industry)
Compound ABBV-RGX-314
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2025-05-28 Last Modified Date: 2026-06-29 Enrollment: 561 Austria, Belgium, Bulgaria + 14 more
NCT06164730 Recruiting Phase1
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Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-102
Therapeutic Modality Gene editing
Target Gene PCSK9
Vector Type LDLR + GalNAc
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Fast Track
First Submitted: 2023-12-01 Last Modified Date: 2026-06-29 Enrollment: 85 Australia, Canada, Israel + 3 more
NCT01306019 Recruiting Phase2, Phase1
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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
Therapeutic Modality 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: 2026-06-29 Enrollment: 40 United States
NCT06285643 Recruiting Phase2
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Parkinson's Disease
Sponsor AskBio Inc (Industry)
Compound AB-1005
Therapeutic Modality Gene transfer
Target Gene GDNF
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2024-02-14 Last Modified Date: 2026-06-29 Enrollment: 127 Germany, Poland, United Kingdom + 1 more
NCT05419492 Recruiting Phase2, Phase1
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Dravet Syndrome
Sponsor Encoded Therapeutics (Industry)
Compound ETX101
Therapeutic Modality 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: 2026-06-29 Enrollment: 47 Australia, United Kingdom, United States
NCT05682144 Recruiting Phase1
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Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Sponsor Immusoft of CA, Inc. (Industry)
Compound ISP-001
Therapeutic Modality Gene transfer
Target Gene IDUA
Vector Type Sleeping Beauty transposon
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: 2026-06-26 Enrollment: 11 United States
NCT05606614 Recruiting Phase3
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Rett Syndrome
Sponsor Taysha Gene Therapies, Inc. (Industry)
Compound TSHA-102
Therapeutic Modality Gene transfer
Target Gene MiniMECP2
Vector Type scAAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-10-28 Last Modified Date: 2026-06-26 Enrollment: 15 Canada, United States
NCT05878860 Recruiting Phase3
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X-Linked Retinoschisis
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-201
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type AAV.SPR
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-12 Last Modified Date: 2026-06-26 Enrollment: 97 United States
NCT06063850 Recruiting Phase2, Phase1
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Mesial Temporal Lobe Epilepsy
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-260
Therapeutic Modality 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: 2026-06-26 Enrollment: 12 United States
NCT07226206 Recruiting Phase2, Phase1
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Hemophilia A
Sponsor Hoffmann-La Roche (Industry)
Compound SPK-8011QQ
Therapeutic Modality 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: 2026-06-23 Enrollment: 5 United States
NCT03217617 Recruiting Phase2, Phase1
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X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor Shenzhen Geno-Immune Medical Institute (Other)
Compound Ivlv-X1 lentiviral vector
Therapeutic Modality Gene transfer
Target Gene IL2RG
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-07-03 Last Modified Date: 2026-06-23 Enrollment: 10 China
NCT07287397 Recruiting Phase2, Phase1
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Amyotrophic Lateral Sclerosis
Sponsor Vector Y Therapeutics (Industry)
Compound VTx-002
Therapeutic Modality 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: 2026-06-23 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 more
NCT07223944 Recruiting Phase3
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Gaucher Disease Type 1
Sponsor Spur Therapeutics (Industry)
Compound FLT201
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type AAVS3
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-10-30 Last Modified Date: 2026-06-23 Enrollment: 45 Argentina, Australia, Brazil + 7 more
NCT03645460 Recruiting Na
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Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Sponsor Shenzhen Geno-Immune Medical Institute (Other)
Compound ADA lentiviral vector
Therapeutic Modality Gene transfer
Target Gene ADA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-07-17 Last Modified Date: 2026-06-23 Enrollment: 10 China
NCT07180355 Recruiting Phase1
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Friedreich's Ataxia
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-212
Therapeutic Modality 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: 2026-06-22 Enrollment: 10 United States
NCT05836259 Recruiting Phase2, Phase1
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Hypertrophic Cardiomyopathy
Sponsor Tenaya Therapeutics (Industry)
Compound TN-201
Therapeutic Modality 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: 2026-06-18 Enrollment: 30 United States
NCT06141460 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Shanghai Refreshgene Technology Co., Ltd. (Industry)
Compound RRG001
Therapeutic Modality 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: 2026-06-18 Enrollment: 48 China
NCT06255782 Recruiting Phase2, Phase1
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Ornithine Transcarbamylase (OTC) Deficiency
Sponsor iECURE, Inc. (Industry)
Compound ECUR-506
Therapeutic Modality 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: 2026-06-18 Enrollment: 20 Australia, Spain, United Kingdom + 1 more
NCT05641610 Recruiting Phase2, Phase1
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Hemophilia B
Sponsor Institute of Hematology & Blood Diseases Hospital, China (Other)
Compound ZS801
Therapeutic Modality Gene transfer
Target Gene F9
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-11-25 Last Modified Date: 2026-06-18 Enrollment: 21 China
NCT06332807 Recruiting Phase2, Phase1
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Phenylketonuria (PKU)
Sponsor NGGT INC. (Industry)
Compound NGGT002
Therapeutic Modality 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: 2026-06-18 Enrollment: 12 United States
NCT07064759 Recruiting Phase3
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Neovascular Age-Related Macular Degeneration (nAMD)
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-150
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-07-07 Last Modified Date: 2026-06-18 Enrollment: 480 Argentina, Australia, Bulgaria + 11 more
NCT06492863 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Frontera Therapeutics (Industry)
Compound FT-003
Therapeutic Modality 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: 2026-06-18 Enrollment: 78 China
NCT05757245 Recruiting Phase1
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Transfusion-dependent Alpha-Thalassemia
Sponsor First Affiliated Hospital of Guangxi Medical University (Other)
Compound GMCN-508A
Therapeutic Modality Gene transfer
Target Gene HBA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-02-24 Last Modified Date: 2026-06-18 Enrollment: 5 China
NCT06451770 Recruiting Phase1
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Hypercholesterolemia
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-201
Therapeutic Modality Gene editing
Target Gene ANGPTL3
Vector Type LDLR + GalNAc
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2024-06-04 Last Modified Date: 2026-06-18 Enrollment: 36 Australia, Canada, South Africa + 1 more
NCT06942572 Recruiting Phase2, Phase1
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Stargardt Disease
Sponsor Splice Bio (Industry)
Compound SB-007
Therapeutic Modality 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: 2026-06-18 Enrollment: 57 United States
NCT06517888 Recruiting Phase2, Phase1
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Vestibular Schwannoma
Sponsor Akouos, Inc. (Industry)
Compound AAVAnc80-antiVEGF
Therapeutic Modality 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: 2026-06-18 Enrollment: 27 United States
NCT03538899 Recruiting Phase2, Phase1
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Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency
Sponsor University of California, San Francisco (Other)
Compound AProArt
Therapeutic Modality Gene transfer
Target Gene DCLRE1C
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-05-03 Last Modified Date: 2026-06-18 Enrollment: 24 United States
NCT05616793 Recruiting Phase2, Phase1
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Leber Congenital Amaurosis-Type 5
Sponsor Opus Genetics, Inc (Industry)
Compound OPGx-LCA5
Therapeutic Modality Gene transfer
Target Gene LCA5
Vector Type AAV8
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: 2022-11-07 Last Modified Date: 2026-06-18 Enrollment: 22 United States
NCT05071222 Recruiting Phase2, Phase1
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Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency
Sponsor Assistance Publique - Hôpitaux de Paris (Other)
Compound ARTEGENE
Therapeutic Modality 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: 2026-06-18 Enrollment: 7 France
NCT04774536 Recruiting Phase2, Phase1
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Sickle Cell Disease
Sponsor Mark Walters, MD (Other)
Compound CRISPR_SCD001
Therapeutic Modality Gene editing
Target Gene HBB
Delivery System Electroporation
Mechanism of Action Mutation correction
First Submitted: 2021-02-17 Last Modified Date: 2026-06-18 Enrollment: 9 United States
NCT02716246 Recruiting Phase3, Phase2
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Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound UX111
Therapeutic Modality Gene transfer
Target Gene SGSH
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-03-17 Last Modified Date: 2026-06-18 Enrollment: 36 Australia, Spain, United States
NCT06185673 Recruiting Phase2, Phase1
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Oculopharyngeal Muscular Dystrophy
Sponsor Benitec Biopharma, Inc. (Industry)
Compound BB-301
Therapeutic Modality 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: 2026-06-18 Enrollment: 30 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
Therapeutic Modality 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: 2026-06-18 Enrollment: 44 China
NCT06539208 Recruiting Phase2, Phase1
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Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy
Sponsor YolTech Therapeutics Co., Ltd (Industry)
Compound YOLT-201
Therapeutic Modality Gene editing
Target Gene TTR
Vector Type LNP
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2024-08-01 Last Modified Date: 2026-06-18 Enrollment: 31 China
NCT05477563 Recruiting Phase3
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Beta-Thalassemia, Sickle Cell Disease
Sponsor Vertex Pharmaceuticals Incorporated (Industry)
Compound CASGEVY
Therapeutic Modality Gene editing
Target Gene BCL11A
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-07-26 Last Modified Date: 2026-06-18 Enrollment: 26 Germany, Italy, Saudi Arabia + 1 more
NCT05230459 Recruiting Phase2, Phase1
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Limb-Girdle Muscular Dystrophy, Type 2I/R9
Sponsor AskBio Inc (Industry)
Compound AB-1003
Therapeutic Modality Gene transfer
Target Gene FKRP
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: 2022-01-27 Last Modified Date: 2026-06-18 Enrollment: 10 United States
NCT05858983 Recruiting Phase2, Phase1
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Biallelic RPE65 Mutation-associated Retinal Dystrophy
Sponsor Frontera Therapeutics (Industry)
Compound FT-001
Therapeutic Modality 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: 2026-06-18 Enrollment: 9 China
NCT05092685 Recruiting Phase2, Phase1
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Ornithine Transcarbamylase (OTC) Deficiency
Sponsor University College, London (Other)
Compound BGT-OTCD
Therapeutic Modality 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: 2026-06-18 Enrollment: 12 United Kingdom
NCT06291935 Recruiting Phase1
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Retinitis Pigmentosa
Sponsor VeonGen Therapeutics GmbH (Industry)
Compound VG901
Therapeutic Modality Gene transfer
Target Gene CNGA1
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-02-01 Last Modified Date: 2026-06-18 Enrollment: 6 Germany
NCT04278131 Recruiting Phase2, Phase1
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Retinitis Pigmentosa
Sponsor Bionic Sight LLC (Industry)
Compound BS01
Therapeutic Modality Gene transfer
Target Gene ChronosFP
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2020-02-13 Last Modified Date: 2026-06-18 Enrollment: 20 United States
NCT06492850 Recruiting Phase2, Phase1
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X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Frontera Therapeutics (Industry)
Compound FT-002
Therapeutic Modality 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: 2026-06-18 Enrollment: 32 China
NCT05241444 Recruiting Phase1
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Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome (IPEX)
Sponsor Bacchetta, Rosa, MD (Other)
Compound CD4^LVFOXP3
Therapeutic Modality Gene transfer
Target Gene FOXP3
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-01-12 Last Modified Date: 2026-06-18 Enrollment: 30 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
Therapeutic Modality 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: 2026-06-18 Enrollment: 12 United States
NCT06467344 Recruiting Phase2, Phase1
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Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration
Sponsor Ascidian Therapeutics, Inc (Industry)
Compound ACDN-01
Therapeutic Modality 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: 2026-06-18 Enrollment: 15 United States
NCT06345898 Recruiting Early phase1
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X-Linked Retinoschisis
Sponsor West China Hospital (Other)
Compound JWK002
Therapeutic Modality 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: 2026-06-18 Enrollment: 12 China
NCT05432310 Recruiting Phase2, Phase1
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Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Sponsor University of California, Los Angeles (Other)
Compound Simoladagene autotemcel
Therapeutic Modality 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: 2026-06-18 Enrollment: 20 United States
NCT05860569 Recruiting Phase1
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Hypertriglyceridemia
Sponsor GeneCradle Inc (Industry)
Compound GC304
Therapeutic Modality Gene transfer
Target Gene GPIHBP1 or LPL
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement, Overexpression of protective allele/gene
First Submitted: 2023-04-23 Last Modified Date: 2026-06-18 Enrollment: 7 China
NCT06519552 Recruiting Phase1
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Mucopolysaccharidosis Type I (Hurler Syndrome)
Sponsor West China Hospital (Other)
Compound JWK-008
Therapeutic Modality Gene transfer
Target Gene IDUA
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-06-23 Last Modified Date: 2026-06-18 Enrollment: 6 China
NCT07195825 Recruiting Phase1
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Parkinson's Disease
Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound BBM-P002
Therapeutic Modality Gene transfer
Target Gene TH90del-AADC
Vector Type AAVT42
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2025-09-10 Last Modified Date: 2026-06-18 Enrollment: 18 China
NCT05926765 Recruiting Phase2
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Grade 2 and 3 Radiation-Induced Late Xerostomia
Sponsor MeiraGTx, LLC (Industry)
Compound AAV-hAQP1
Therapeutic Modality Gene transfer
Target Gene AQP1
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2023-06-22 Last Modified Date: 2026-06-18 Enrollment: 276 Canada, United Kingdom, United States
NCT06292650 Recruiting Early phase1
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Retinitis Pigmentosa
Sponsor Zhongmou Therapeutics (Industry)
Compound ZM-02
Therapeutic Modality 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: 2026-06-18 Enrollment: 12 China
NCT06662188 Recruiting Phase2, Phase1
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SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Sponsor Jaguar Gene Therapy, LLC (Industry)
Compound JAG201
Therapeutic Modality 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: 2026-06-18 Enrollment: 6 United States
NCT05824169 Recruiting Phase2, Phase1
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Spinal Muscular Atrophy
Sponsor GeneCradle Inc (Industry)
Compound GC101
Therapeutic Modality 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: 2026-06-18 Enrollment: 18 China
NCT06692712 Recruiting Phase3
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Hereditary Spastic Paraplegia Type 50
Sponsor Elpida Therapeutics SPC (Industry)
Compound MELPIDA
Therapeutic Modality Gene transfer
Target Gene AP4M1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-11-14 Last Modified Date: 2026-06-18 Enrollment: 24 Spain, United States
NCT04797260 Recruiting Na
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Severe Combined Immunodeficiency Due to RAG1 Deficiency
Sponsor Leiden University Medical Center (Other)
Compound Autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector
Therapeutic Modality Gene transfer
Target Gene RAG1
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-03-11 Last Modified Date: 2026-06-18 Enrollment: 10 Australia, Italy, Netherlands + 4 more
NCT06399107 Recruiting Phase2, Phase1
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Sickle Cell Disease, Sickle-Cell Disease With Crisis
Sponsor Essen Biotech (Other)
Compound BAH243
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-04-29 Last Modified Date: 2026-06-18 Enrollment: 85 China
NCT05672121 Recruiting Phase2, Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Chengdu Origen Biotechnology Co., Ltd. (Industry)
Compound KH631
Therapeutic Modality 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: 2026-06-18 Enrollment: 42 China
NCT05986864 Recruiting Phase2, Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Skyline Therapeutics (US) Inc. (Industry)
Compound SKG0106
Therapeutic Modality 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: 2026-06-18 Enrollment: 68 China, United States
NCT06615206 Recruiting Na
View Report
MECP2 Duplication Syndrome
Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)
Compound HG204
Therapeutic Modality 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: 2026-06-18 Enrollment: 6 China
NCT05906953 Recruiting Phase2, Phase1
View Report
Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)
Compound HG004
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-09 Last Modified Date: 2026-06-18 Enrollment: 20 China, United States
NCT06833983 Recruiting Phase3
View Report
Hemophilia A
Sponsor Gritgen Therapeutics Co., Ltd. (Industry)
Compound GS1191-0445
Therapeutic Modality 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: 2026-06-17 Enrollment: 50 China
NCT06111638 Recruiting Phase3, Phase2
View Report
Hemophilia A
Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound BBM-H803
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2023-10-27 Last Modified Date: 2026-06-17 Enrollment: 55 China
NCT06831825 Recruiting Phase1
View Report
Heart Failure With Reduced Ejection Fraction
Sponsor YAP Therapeutics, Inc. (Industry)
Compound YAP101
Therapeutic Modality 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: 2026-06-17 Enrollment: 24 United States
NCT03952637 Recruiting Phase2, Phase1
View Report
GM1 Gangliosidosis
Sponsor National Human Genome Research Institute (NHGRI) (NIH)
Compound AXO-AAV-GM1
Therapeutic Modality Gene transfer
Target Gene GLB1
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: 2019-05-15 Last Modified Date: 2026-06-17 Enrollment: 54 United States
NCT06061549 Recruiting Phase1
View Report
Heart Failure With Preserved Ejection Fraction, Diastolic Heart Failure
Sponsor Sardocor Corp. (Industry)
Compound SRD-002
Therapeutic Modality Gene transfer
Target Gene SERCA2a
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2023-07-30 Last Modified Date: 2026-06-17 Enrollment: 10 United States
NCT07282847 Recruiting Phase2, Phase1
View Report
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Sponsor AskBio Inc (Industry)
Compound AB-1009
Therapeutic Modality 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: 2026-06-17 Enrollment: 12 United States
NCT04703842 Recruiting Phase2, Phase1
View Report
Congestive Heart Failure, Heart Failure With Reduced Ejection Fraction (HFrEF)
Sponsor Sardocor Corp. (Industry)
Compound SRD-001
Therapeutic Modality Gene transfer
Target Gene SERCA2a
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2021-01-07 Last Modified Date: 2026-06-17 Enrollment: 57 United States
NCT06765980 Recruiting Phase2, Phase1
View Report
Geographic Atrophy Secondary to Age-related Macular Degeneration
Sponsor Kriya Therapeutics, Inc. (Industry)
Compound KRIYA-825
Therapeutic Modality 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: 2026-06-17 Enrollment: 62 Canada, Israel, New Zealand
NCT07048808 Recruiting Phase2
View Report
Refractory Angina, Coronary Artery Disease
Sponsor XyloCor Therapeutics, Inc. (Industry)
Compound XC001
Therapeutic Modality Gene transfer
Target Gene VEGF
Vector Type Ad5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2025-06-24 Last Modified Date: 2026-06-17 Enrollment: 106 United States
NCT04676048 Recruiting Phase2, Phase1
View Report
Hemophilia A
Sponsor ASC Therapeutics (Industry)
Compound ASC618
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-12-15 Last Modified Date: 2026-06-17 Enrollment: 12 United States
NCT06207552 Recruiting Early phase1
View Report
Fabry Disease
Sponsor Children's Hospital of Fudan University (Other)
Compound BBM-F101
Therapeutic Modality Gene transfer
Target Gene GLA
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-12-18 Last Modified Date: 2026-06-17 Enrollment: 6 China
NCT06238908 Recruiting Early phase1
View Report
Hemophilia A
Sponsor Institute of Hematology & Blood Diseases Hospital, China (Other)
Compound NGGT003
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-01-26 Last Modified Date: 2026-06-17 Enrollment: 6 China
NCT06735755 Recruiting Phase2, Phase1
View Report
Glycogen Storage Disease Type Ia
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-301
Therapeutic Modality Gene editing
Target Gene G6PC1 (p.R83C)
Vector Type LNP
Delivery System Endocytosis
Mechanism of Action Mutation correction
First Submitted: 2024-12-06 Last Modified Date: 2026-06-17 Enrollment: 36 United States
NCT06064890 Recruiting Phase2, Phase1
View Report
Frontotemporal Dementia, FTD, FTD-GRN
Sponsor AviadoBio Ltd (Industry)
Compound AVB-101
Therapeutic Modality 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: 2026-06-17 Enrollment: 18 Belgium, Canada, Italy + 6 more
NCT06217861 Recruiting Phase1
View Report
Glutaric Acidemia Type I
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGM-R02b
Therapeutic Modality Gene transfer
Target Gene GCDH
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-12-12 Last Modified Date: 2026-06-17 Enrollment: 12 China
NCT06272149 Recruiting Early phase1
View Report
Type II Gaucher Disease
Sponsor Xinhua Hospital, Shanghai Jiao Tong University School of Medicine (Other)
Compound VGN-R08b
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Rare Pediatric Disease Designation
First Submitted: 2023-07-17 Last Modified Date: 2026-06-17 Enrollment: 6 China
NCT06270316 Recruiting Phase2, Phase1
View Report
Fabry Disease
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-191
Therapeutic Modality 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: 2026-06-17 Enrollment: 12 United States
NCT05761899 Recruiting Phase2, Phase1
View Report
Hereditary Pulmonary Alveolar Proteinosis
Sponsor Children's Hospital Medical Center, Cincinnati (Other)
Compound Gene-Corrected Macrophages
Therapeutic Modality Gene transfer
Target Gene CSF2RA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-02-28 Last Modified Date: 2026-06-17 Enrollment: 3 United States
NCT07218887 Recruiting Phase2, Phase1
View Report
BAG3 Mutation Associated Dilated Cardiomyopathy
Sponsor Alexion Pharmaceuticals, Inc. (Industry)
Compound ALXN2350
Therapeutic Modality Gene transfer
Target Gene BAG3
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-10-17 Last Modified Date: 2026-06-16 Enrollment: 6 Italy, Spain, United Kingdom + 1 more
NCT04998396 Recruiting Phase2, Phase1
View Report
Canavan Disease
Sponsor Aspa Therapeutics (Industry)
Compound BBP-812
Therapeutic Modality 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: 2026-06-12 Enrollment: 26 United States
NCT06364774 Recruiting Phase2, Phase1
View Report
Beta-Thalassemia Major
Sponsor Children's Hospital of Philadelphia (Other)
Compound CHOP-ALS20
Therapeutic Modality 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: 2026-06-12 Enrollment: 12 United States
NCT06641154 Recruiting Phase2, Phase1
View Report
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
Therapeutic Modality 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: 2026-06-12 Enrollment: 5 Russia
NCT06526923 Recruiting Phase2, Phase1
View Report
Cystic Fibrosis
Sponsor Spirovant Sciences, Inc. (Industry)
Compound SP-101
Therapeutic Modality 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: 2026-06-12 Enrollment: 15 United States
NCT05248230 Recruiting Phase2
View Report
Cystic Fibrosis
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-710
Therapeutic Modality 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: 2026-06-12 Enrollment: 30 United States
NCT03466463 Recruiting Na
View Report
Crigler-Najjar Syndrome
Sponsor Genethon (Other)
Compound GNT0003
Therapeutic Modality Gene transfer
Target Gene UGT1A1
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-02-01 Last Modified Date: 2026-06-12 Enrollment: 17 France, Italy, Netherlands
NCT05821959 Recruiting Phase2, Phase1
View Report
Sensorineural Hearing Loss, Bilateral
Sponsor Akouos, Inc. (Industry)
Compound AK-OTOF
Therapeutic Modality Gene transfer
Target Gene OTOF
Vector Type dual Anc80L65
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-02-08 Last Modified Date: 2026-06-12 Enrollment: 22 Canada, Taiwan, United Kingdom + 1 more
NCT02852213 Recruiting Phase1
View Report
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor Krzysztof Bankiewicz (Other)
Compound AAV2-hAADC
Therapeutic Modality Gene transfer
Target Gene DDC
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-07-20 Last Modified Date: 2026-06-12 Enrollment: 42 United States
NCT06224660 Recruiting Phase1
View Report
DMD-Associated Dilated Cardiomyopathy
Sponsor Sardocor Corp. (Industry)
Compound SRD-001
Therapeutic Modality Gene transfer
Target Gene SERCA2a
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2024-01-11 Last Modified Date: 2026-06-12 Enrollment: 12 United States
NCT03727555 Recruiting Na
View Report
X-linked Adrenoleukodystrophy
Sponsor Shenzhen Geno-Immune Medical Institute (Other)
Compound (LV) TYF-ABCD1
Therapeutic Modality 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: 2026-06-12 Enrollment: 30 China
NCT05040217 Recruiting Phase1
View Report
Alzheimer's Disease, Mild Cognitive Impairment
Sponsor Mark Tuszynski (Other)
Compound AAV2-BDNF
Therapeutic Modality Gene transfer
Target Gene BDNF
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2021-06-21 Last Modified Date: 2026-06-12 Enrollment: 12 United States
NCT06049082 Recruiting Phase1
View Report
Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Krystal Biotech, Inc. (Industry)
Compound KB-408
Therapeutic Modality Gene transfer
Target Gene SERPINA1
Vector Type HSV-1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2023-09-15 Last Modified Date: 2026-06-12 Enrollment: 15 United States
NCT06910813 Recruiting Phase2, Phase1
View Report
Nephropathic Cystinosis
Sponsor Novartis Pharmaceuticals (Industry)
Compound DFT383
Therapeutic Modality 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: 2026-06-12 Enrollment: 30 United States
NCT05504837 Recruiting Phase1
View Report
Cystic Fibrosis
Sponsor Krystal Biotech, Inc. (Industry)
Compound KB407
Therapeutic Modality 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: 2026-06-12 Enrollment: 20 United States
NCT05762510 Recruiting Early phase1
View Report
Beta-Thalassemia Major
Sponsor First Affiliated Hospital of Guangxi Medical University (Other)
Compound GMCN-508B
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-01-06 Last Modified Date: 2026-06-12 Enrollment: 5 China
NCT06465550 Recruiting Phase1
View Report
Beta-Thalassemia Major
Sponsor Shanghai BDgene Co., Ltd. (Industry)
Compound BD211
Therapeutic Modality 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: 2026-06-12 Enrollment: 9 China
NCT06092034 Recruiting Phase2
View Report
Danon Disease
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-A501
Therapeutic Modality Gene transfer
Target Gene LAMP2B
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-10-11 Last Modified Date: 2026-06-12 Enrollment: 14 Germany, Italy, United Kingdom + 1 more
NCT07058662 Recruiting Phase2, Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Belief BioMed (Beijing) Co., Ltd (Industry)
Compound BBM-D101
Therapeutic Modality 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: 2026-06-12 Enrollment: 9 China
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.