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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 226 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
NCT04713475 Active not recruiting Phase2, Phase1
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GM1 Gangliosidosis, Beta-Galactosidase-1 (GLB1) Deficiency
Sponsor Gemma Biotherapeutics (Industry)
Compound PBGM01
Therapeutic Modality Gene transfer
Target Gene GLB1
Vector Type AAVhu68
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-01-04 Last Modified Date: 2026-06-30 Enrollment: 26 Brazil, Turkey (Türkiye), United Kingdom + 1 more
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
NCT06634420 Active not recruiting Phase3
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Hereditary Angioedema
Sponsor Intellia Therapeutics (Industry)
Compound NTLA-2002
Therapeutic Modality Gene editing
Target Gene KLKB1
Vector Type LDLR
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-10-07 Last Modified Date: 2026-06-26 Enrollment: 80 Australia, Canada, France + 6 more
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
NCT03566043 Active not recruiting Phase3, Phase2
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Mucopolysaccharidosis Type II (Hunter Syndrome)
Sponsor REGENXBIO Inc. (Industry)
Compound NAVSUNLI
Therapeutic Modality Gene transfer
Target Gene IDS
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-05-01 Last Modified Date: 2026-06-26 Enrollment: 48 Brazil, 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
NCT04537377 Active not recruiting Phase2, Phase1
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Wilson Disease
Sponsor Vivet Therapeutics SAS (Industry)
Compound VTX-801
Therapeutic Modality Gene transfer
Target Gene ATP7B-minigene
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-08-19 Last Modified Date: 2026-06-18 Enrollment: 4 Denmark, Germany, United Kingdom + 1 more
NCT06246513 Active not recruiting Phase3
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Limb-Girdle Muscular Dystrophy, Type 2E/R4
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-9003
Therapeutic Modality 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: 2026-06-18 Enrollment: 17 Belgium, Germany, Italy + 3 more
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
NCT06480461 Active not recruiting Phase2, Phase1
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Parkinson's Disease
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGN-R09b
Therapeutic Modality Gene transfer
Target Gene DDC
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2024-06-14 Last Modified Date: 2026-06-18 Enrollment: 39 China
NCT04127578 Active not recruiting Phase2, Phase1
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Parkinson's Disease
Sponsor Prevail Therapeutics (Industry)
Compound PR001
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2019-10-14 Last Modified Date: 2026-06-18 Enrollment: 32 Israel, United States
NCT04480567 Active not recruiting Phase2, Phase1
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Phenylketonuria (PKU)
Sponsor BioMarin Pharmaceutical (Industry)
Compound BMN 307
Therapeutic Modality Gene transfer
Target Gene PAH
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-07-08 Last Modified Date: 2026-06-18 Enrollment: 100 United Kingdom, United States
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
NCT03199469 Active not recruiting Phase3, Phase2
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X-Linked Myotubular Myopathy
Sponsor Astellas Gene Therapies (Industry)
Compound AT132
Therapeutic Modality Gene transfer
Target Gene MTM1
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-06-21 Last Modified Date: 2026-06-18 Enrollment: 27 Canada, France, Germany + 1 more
NCT05353647 Active not recruiting Phase2
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Sickle Cell Disease
Sponsor David Williams (Other)
Compound Autologous CD34+ HSC cells transduced with the lentiviral vector containing BCL11a-targeted shRNA
Therapeutic Modality Gene transfer
Target Gene BCL11A
Vector Type VSV-G
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2022-04-21 Last Modified Date: 2026-06-18 Enrollment: 25 United States
NCT06460844 Active not recruiting Phase1
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Retinitis Pigmentosa, Choroideremia
Sponsor Ray Therapeutics, Inc. (Industry)
Compound RTx-015
Therapeutic Modality 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: 2026-06-18 Enrollment: 10 United States
NCT04680065 Active not recruiting Phase1
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Multiple System Atrophy
Sponsor Brain Neurotherapy Bio, 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
First Submitted: 2020-12-15 Last Modified Date: 2026-06-18 Enrollment: 9 United States
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
NCT05739643 Active not recruiting Phase2, Phase1
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Krabbe Disease
Sponsor Forge Biologics, Inc (Industry)
Compound FBX-101
Therapeutic Modality Gene transfer
Target Gene GALC
Vector Type AAVrh10
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-02-13 Last Modified Date: 2026-06-18 Enrollment: 9 United States
NCT06388200 Active not recruiting Phase3
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Retinitis Pigmentosa
Sponsor Ocugen (Industry)
Compound OCU400
Therapeutic Modality Gene transfer
Target Gene NR2E3
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-04-09 Last Modified Date: 2026-06-18 Enrollment: 140 Canada, United States
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
NCT05541627 Active not recruiting Phase2, Phase1
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Huntington's Disease
Sponsor AskBio France, SAS, a subsidiary of AskBio Inc. (Industry)
Compound AB-1001
Therapeutic Modality Gene transfer
Target Gene CYP46A1
Vector Type AAVrh10
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2022-09-13 Last Modified Date: 2026-06-18 Enrollment: 5 France
NCT04293185 Active not recruiting Phase3
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Sickle Cell Disease
Sponsor Genetix Biotherapeutics Inc. (Industry)
Compound LYFGENIA
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type BB305 LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-02-12 Last Modified Date: 2026-06-18 Enrollment: 35 United States
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
NCT05898620 Active not recruiting Phase3
View Report
Rett Syndrome
Sponsor Neurogene Inc. (Industry)
Compound NGN-401
Therapeutic Modality Gene transfer
Target Gene MECP2
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-06-01 Last Modified Date: 2026-06-18 Enrollment: 33 Australia, United Kingdom, United States
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
View Report
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
NCT04248439 Active not recruiting Phase2
View Report
Fanconi Anemia Complementation Group A
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-L102
Therapeutic Modality Gene transfer
Target Gene FANCA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-01-24 Last Modified Date: 2026-06-18 Enrollment: 5 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
NCT06856577 Active not recruiting Phase3
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Adverum Biotechnologies, Inc. (Industry)
Compound ADVM-022
Therapeutic Modality Gene transfer
Target Gene Codon optimized aflibercept
Vector Type AAV.7m8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2025-02-26 Last Modified Date: 2026-06-18 Enrollment: 311 United States
NCT03837483 Active not recruiting Phase3
View Report
Wiskott-Aldrich Syndrome
Sponsor Fondazione Telethon (Other)
Compound WASKYRA
Therapeutic Modality Gene transfer
Target Gene WAS
Vector Type LV
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: 2019-02-08 Last Modified Date: 2026-06-18 Enrollment: 10 Italy, United States
NCT04774536 Recruiting Phase2, Phase1
View Report
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
NCT05345171 Active not recruiting Phase3
View Report
Ornithine Transcarbamylase (OTC) Deficiency
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound DTX301
Therapeutic Modality Gene transfer
Target Gene OTC
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2022-04-18 Last Modified Date: 2026-06-18 Enrollment: 37 Argentina, Brazil, Canada + 7 more
NCT05121376 Active not recruiting Phase2, Phase1
View Report
Hereditary Angioedema
Sponsor BioMarin Pharmaceutical (Industry)
Compound BMN 331
Therapeutic Modality Gene transfer
Target Gene SERPING1
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2021-10-28 Last Modified Date: 2026-06-18 Enrollment: 44 Australia, Spain, United States
NCT05973630 Active not recruiting Phase1
View Report
Limb-Girdle Muscular Dystrophy, Type 2C/R5
Sponsor Atamyo Therapeutics (Industry)
Compound ATA-200
Therapeutic Modality Gene transfer
Target Gene SGCG
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-07-24 Last Modified Date: 2026-06-18 Enrollment: 4 United States
NCT00999609 Active not recruiting Phase3
View Report
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Sponsor Spark Therapeutics, Inc. (Industry)
Compound LUXTURNA
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Priority Review, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2009-10-21 Last Modified Date: 2026-06-18 Enrollment: 31 United States
NCT03920007 Active not recruiting Phase2, Phase1
View Report
Leber Congenital Amaurosis, LCA, LCA1
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-101
Therapeutic Modality Gene transfer
Target Gene GUCY2D
Vector Type AAV2/5
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: 2019-04-10 Last Modified Date: 2026-06-18 Enrollment: 15 United States
NCT06196827 Active not recruiting Phase1
View Report
Inherited Retinal Dystrophy Associated With RPE65 Mutations
Sponsor Innostellar Biotherapeutics Co.,Ltd (Industry)
Compound LX101
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-12-25 Last Modified Date: 2026-06-18 Enrollment: 9 China
NCT05445323 Active not recruiting Phase2, Phase1
View Report
Friedreich Ataxia, Cardiomyopathy, Secondary
Sponsor Lexeo Therapeutics (Industry)
Compound LX2006
Therapeutic Modality Gene transfer
Target Gene FXN
Vector Type AAVrh10
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: 2022-06-23 Last Modified Date: 2026-06-18 Enrollment: 8 United States
NCT02716246 Recruiting Phase3, Phase2
View Report
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
NCT06196840 Active not recruiting Phase2
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Innostellar Biotherapeutics Co.,Ltd (Industry)
Compound LX102
Therapeutic Modality Gene transfer
Target Gene VEGF-trap
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2023-12-25 Last Modified Date: 2026-06-18 Enrollment: 50 China
NCT06185673 Recruiting Phase2, Phase1
View Report
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
NCT05835895 Active not recruiting Phase1
View Report
Osteoarthritis, Knee
Sponsor Genascence Corporation (Industry)
Compound GNSC-001
Therapeutic Modality Gene transfer
Target Gene IL1RN
Vector Type AAV2.5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2023-04-19 Last Modified Date: 2026-06-18 Enrollment: 67 United States
NCT03326336 Active not recruiting Phase2, Phase1
View Report
Non-syndromic Retinitis Pigmentosa
Sponsor GenSight Biologics (Industry)
Compound GS030
Therapeutic Modality Gene transfer
Target Gene ChR-tdT
Vector Type AAV2.7m8
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2017-10-11 Last Modified Date: 2026-06-18 Enrollment: 10 France, United Kingdom, United States
NCT04850118 Active not recruiting Phase3, Phase2
View Report
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-501
Therapeutic Modality Gene transfer
Target Gene RPGR
Vector Type AAV2tYF
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2021-04-05 Last Modified Date: 2026-06-18 Enrollment: 85 Australia, Canada, United Kingdom + 1 more
NCT06289452 Active not recruiting Early phase1
View Report
Retinoschisis, Retinal Disease, Retinal Degeneration, Eye Diseases
Sponsor InnoVec Biotherapeutics Inc. (Industry)
Compound IVB102
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Rare Pediatric Disease Designation
First Submitted: 2024-02-25 Last Modified Date: 2026-06-18 Enrollment: 18 China
NCT05956626 Active not recruiting Phase3, Phase2
View Report
Stargardt Disease
Sponsor Ocugen (Industry)
Compound OCU410ST
Therapeutic Modality Gene transfer
Target Gene RORA
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2023-06-30 Last Modified Date: 2026-06-18 Enrollment: 51 United States
NCT06458595 Recruiting Phase2, Phase1
View Report
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
NCT04201405 Active not recruiting Phase2, Phase1
View Report
Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor University of Manchester (Other)
Compound OTL-201
Therapeutic Modality Gene transfer
Target Gene SGSH
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2019-12-05 Last Modified Date: 2026-06-18 Enrollment: 5 United Kingdom
NCT05243017 Active not recruiting Phase2, Phase1
View Report
Huntington's Disease
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-130
Therapeutic Modality Gene transfer
Target Gene MiHTT
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2021-11-01 Last Modified Date: 2026-06-18 Enrollment: 14 Poland, United Kingdom
NCT02599922 Active not recruiting Phase2, Phase1
View Report
Achromatopsia
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-401
Therapeutic Modality Gene transfer
Target Gene CNGB3
Vector Type AAV2tYF
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2015-11-05 Last Modified Date: 2026-06-18 Enrollment: 32 United States
NCT04853576 Active not recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Editas Medicine, Inc. (Industry)
Compound EDIT-301
Therapeutic Modality Gene editing
Target Gene HBG1/HBG2
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-04-16 Last Modified Date: 2026-06-18 Enrollment: 45 Canada, United States
NCT06539208 Recruiting Phase2, Phase1
View Report
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
NCT06300476 Active not recruiting Phase2, Phase1
View Report
Stargardt Disease
Sponsor West China Hospital (Other)
Compound JWK006
Therapeutic Modality Gene transfer
Target Gene ABCA4
Vector Type dual AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-03-03 Last Modified Date: 2026-06-18 Enrollment: 9 China
NCT05477563 Recruiting Phase3
View Report
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
View Report
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
View Report
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
NCT04611503 Active not recruiting Phase2, Phase1
View Report
Retinitis Pigmentosa
Sponsor STZ eyetrial (Other)
Compound RAAV.hPDE6A
Therapeutic Modality Gene transfer
Target Gene PDE6A
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2020-05-20 Last Modified Date: 2026-06-18 Enrollment: 9 Germany
NCT05293626 Active not recruiting Phase2, Phase1
View Report
Leber Hereditary Optic Neuropathy (LHON)
Sponsor Neurophth Therapeutics Inc (Other)
Compound OPVIKA
Therapeutic Modality Gene transfer
Target Gene ND4G11778A
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2021-12-09 Last Modified Date: 2026-06-18 Enrollment: 12 United States
NCT05092685 Recruiting Phase2, Phase1
View Report
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
View Report
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
View Report
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
NCT04517149 Active not recruiting Phase2, Phase1
View Report
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-125
Therapeutic Modality Gene transfer
Target Gene RPGR
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-08-14 Last Modified Date: 2026-06-18 Enrollment: 21 United States
NCT05224505 Active not recruiting Phase1
View Report
Limb-Girdle Muscular Dystrophy, Type 2I/R9
Sponsor Atamyo Therapeutics (Industry)
Compound GNT0006
Therapeutic Modality Gene transfer
Target Gene FKRP
Vector Type AAV2/9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Rare Pediatric Disease Designation
First Submitted: 2021-12-15 Last Modified Date: 2026-06-18 Enrollment: 6 Denmark, France, United Kingdom
NCT06492850 Recruiting Phase2, Phase1
View Report
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
View Report
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
View Report
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
NCT06149403 Active not recruiting Phase3
View Report
Mucopolysaccharidosis Type I (Hurler Syndrome)
Sponsor Orchard Therapeutics (Industry)
Compound OTL-203
Therapeutic Modality Gene transfer
Target Gene IDUA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-11-17 Last Modified Date: 2026-06-18 Enrollment: 41 Italy, Netherlands, United Kingdom + 1 more
NCT05456880 Active not recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-101
Therapeutic Modality Gene editing
Target Gene HBG1/HBG2
Delivery System Electroporation
Mechanism of Action Mutation correction
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2022-06-23 Last Modified Date: 2026-06-18 Enrollment: 15 United States
NCT06550011 Active not recruiting Phase2, Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Avirmax Biopharma Inc (Industry)
Compound ABI-110
Therapeutic Modality Gene transfer
Target Gene VEGFtrap
Vector Type AAV.N54
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-07-25 Last Modified Date: 2026-06-18 Enrollment: 18 United States
NCT06467344 Recruiting Phase2, Phase1
View Report
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
View Report
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
View Report
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
View Report
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
View Report
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
NCT06884865 Active not recruiting Phase2
View Report
Osteoarthritis (OA) of the Knee
Sponsor Pacira Pharmaceuticals, Inc (Industry)
Compound PCRX-201
Therapeutic Modality Gene transfer
Target Gene IL1RA
Vector Type Ad5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-03-13 Last Modified Date: 2026-06-18 Enrollment: 135 United States
NCT07195825 Recruiting Phase1
View Report
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
View Report
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
View Report
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
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.