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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 267 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
NCT07667387 Not yet recruiting Phase2, Phase1
View Report
Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
Sponsor Rebecca Ahrens-Nicklas (Other)
Compound LNP.UCD.ABE
Therapeutic Modality Gene editing
Target Gene CPS1,OTC,ASS1,ASL,ARG,NAGS,SLC25A15
Delivery System Endocytosis
Mechanism of Action Mutation correction
First Submitted: 2026-06-18 Last Modified Date: 2026-07-01 Enrollment: 7 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
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
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
NCT05398029 Completed Phase1
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Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-101
Therapeutic Modality Gene editing
Target Gene PCSK9
Vector Type LDLR
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2022-05-19 Last Modified Date: 2026-06-26 Enrollment: 13 New Zealand, United Kingdom
NCT06851767 Enrolling by invitation Phase2, Phase1
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X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound BE-HSPC-IL2RG
Therapeutic Modality Gene editing
Target Gene IL2RG (p.Q144X)
Delivery System Undisclosed
Mechanism of Action Mutation correction
First Submitted: 2025-02-26 Last Modified Date: 2026-06-26 Enrollment: 18 United States
NCT06559176 Enrolling by invitation Phase2, Phase1
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Chronic Granulomatous Disease
Sponsor Prime Medicine, Inc. (Industry)
Compound PM359
Therapeutic Modality Gene editing
Target Gene NCF1 (c.75_76delGT)
Delivery System Electroporation
Mechanism of Action Mutation correction
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-07-11 Last Modified Date: 2026-06-26 Enrollment: 12 Canada, United Kingdom, United States
NCT03720418 Terminated Phase2, Phase1
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Parkinson's Disease
Sponsor Sio Gene Therapies (Industry)
Compound OXB-102
Therapeutic Modality Gene transfer
Target Gene TH-GCH1.DDC (tricistronic)
Vector Type EIAV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2018-10-16 Last Modified Date: 2026-06-26 Enrollment: 6 France, United Kingdom
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
NCT07113743 Enrolling by invitation Phase2, Phase1
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Chronic Granulomatous Disease (CGD)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound VSVG-PCCLChimGp91
Therapeutic Modality Gene transfer
Target Gene CYBB
Vector Type VSV-G
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2025-08-08 Last Modified Date: 2026-06-22 Enrollment: 10 United States
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
NCT01496040 Completed Phase2, Phase1
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Leber Congenital Amaurosis
Sponsor Nantes University Hospital (Other)
Compound RAAV2/4.hRPE65
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type AAV2/4
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2011-11-24 Last Modified Date: 2026-06-18 Enrollment: 9 France
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
NCT01801709 Completed Phase2, Phase1
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Metachromatic Leukodystrophy
Sponsor Institut National de la Santé Et de la Recherche Médicale, France (Other gov)
Compound AAVrh.10-CAG/cuARSA
Therapeutic Modality Gene transfer
Target Gene ARSA
Vector Type AAVrh.10
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2013-01-28 Last Modified Date: 2026-06-18 Enrollment: 5 France
NCT03041324 Terminated Phase2, Phase1
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Mucopolysaccharidosis Type II (Hunter Syndrome)
Sponsor Sangamo Therapeutics (Industry)
Compound SB-913
Therapeutic Modality Gene editing
Target Gene IDS
Vector Type AAV2/6
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-01-13 Last Modified Date: 2026-06-18 Enrollment: 9 United States
NCT03300453 Completed Phase2, Phase1
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Sanfilippo Syndrome B
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-110
Therapeutic Modality Gene transfer
Target Gene NAGLU
Vector Type AAV2/5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-06-15 Last Modified Date: 2026-06-18 Enrollment: 4 France
NCT05903794 Completed Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Exegenesis Bio (Industry)
Compound EXG102-031
Therapeutic Modality Gene transfer
Target Gene ABD-VEGFR
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2023-06-06 Last Modified Date: 2026-06-18 Enrollment: 12 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
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
NCT06611436 Terminated Phase2, Phase1
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Hemophilia B
Sponsor Be Biopharma (Industry)
Compound BE-101
Therapeutic Modality Gene editing
Target Gene F9
Vector Type AAV6
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2024-09-18 Last Modified Date: 2026-06-18 Enrollment: 5 United States
NCT05144386 Completed Phase1
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HIV-1-infection
Sponsor Excision BioTherapeutics (Industry)
Compound EBT-101
Therapeutic Modality Gene editing
Target Gene HIV genes
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Gene excision
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2021-11-22 Last Modified Date: 2026-06-18 Enrollment: 6 United States
NCT02362438 Completed Phase1
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Giant Axonal Neuropathy
Sponsor National Institute of Neurological Disorders and Stroke (NINDS) (NIH)
Compound ScAAV9/JeT-GAN
Therapeutic Modality Gene transfer
Target Gene GAN
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2015-02-12 Last Modified Date: 2026-06-18 Enrollment: 14 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
NCT03580083 Suspended Phase2, Phase1
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Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-111
Therapeutic Modality Gene transfer
Target Gene IDUA
Vector Type AAV2/9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-06-18 Last Modified Date: 2026-06-18 Enrollment: 21 Brazil, United States
NCT04581785 Terminated Phase2, Phase1
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Methylmalonic Acidemia
Sponsor LogicBio Therapeutics, Inc (Industry)
Compound LB-001
Therapeutic Modality Gene transfer
Target Gene MMUT
Vector Type AAV-LK03
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-10-02 Last Modified Date: 2026-06-18 Enrollment: 4 United States
NCT05222178 Terminated Phase1
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Phenylketonuria (PKU)
Sponsor Homology Medicines, Inc (Industry)
Compound HMI-103
Therapeutic Modality Gene transfer
Target Gene PAH
Vector Type AAVHSC15
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track
First Submitted: 2021-12-21 Last Modified Date: 2026-06-18 Enrollment: 3 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
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
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
NCT01482195 Completed Phase1
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Retinal Disease, Retinitis Pigmentosa
Sponsor King Khaled Eye Specialist Hospital (Other gov)
Compound AAV2-VMD2-hMERTK
Therapeutic Modality Gene transfer
Target Gene MERTK
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2011-09-28 Last Modified Date: 2026-06-18 Enrollment: 6 Saudi Arabia
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
View Report
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
NCT03315182 Terminated Phase2, Phase1
View Report
Mucopolysaccharidosis Type 3 B
Sponsor Abeona Therapeutics, Inc (Industry)
Compound ABO-101
Therapeutic Modality Gene transfer
Target Gene NAGLU
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-10-10 Last Modified Date: 2026-06-18 Enrollment: 11 France, Germany, Spain + 1 more
NCT06623279 Not yet recruiting Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)
Compound HG202
Therapeutic Modality Gene editing
Target Gene VEGFA mRNA
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2024-09-30 Last Modified Date: 2026-06-18 Enrollment: 15
NCT01301443 Completed Phase1
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Neovascular (Wet) Age-Related Macular Degeneration
Sponsor Oxford BioMedica (Industry)
Compound RetinoStat
Therapeutic Modality Gene transfer
Target Gene COL18A1+PLG
Vector Type EIAV
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2011-02-21 Last Modified Date: 2026-06-18 Enrollment: 21 United States
NCT06451770 Recruiting Phase1
View Report
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
NCT00028236 Completed Phase1
View Report
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound GALV MFGS-gc retrovirus
Therapeutic Modality Gene transfer
Target Gene IL2RG
Vector Type MFGS/GALV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2001-12-17 Last Modified Date: 2026-06-18 Enrollment: 3 United States
NCT06983158 Terminated Phase2, Phase1
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Developmental and Epileptic Encephalopathy
Sponsor Capsida Biotherapeutics, Inc. (Industry)
Compound CAP-002
Therapeutic Modality Gene transfer
Target Gene STXBP1
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2025-05-05 Last Modified Date: 2026-06-18 Enrollment: 1 United States
NCT02317887 Completed Phase2, Phase1
View Report
X-Linked Retinoschisis
Sponsor VegaVect, Inc. (Industry)
Compound RS1 AAV Vector
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2014-12-16 Last Modified Date: 2026-06-18 Enrollment: 12 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
NCT01505062 Terminated Phase2, Phase1
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Usher Syndrome, Retinitis Pigmentosa
Sponsor Sanofi (Industry)
Compound SAR421869
Therapeutic Modality Gene transfer
Target Gene MYO7A
Vector Type EIAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2012-01-04 Last Modified Date: 2026-06-18 Enrollment: 9 France, 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
NCT03328130 Terminated Phase2, Phase1
View Report
Retinitis Pigmentosa
Sponsor eyeDNA Therapeutics (Industry)
Compound CTX-PDE6B
Therapeutic Modality Gene transfer
Target Gene PDE6B
Vector Type AAV2/5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Rare Pediatric Disease Designation
First Submitted: 2017-10-05 Last Modified Date: 2026-06-18 Enrollment: 19 France
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
NCT03964792 Completed Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Assistance Publique - Hôpitaux de Paris (Other)
Compound DREPAGLOBE
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2019-05-14 Last Modified Date: 2026-06-18 Enrollment: 6 France
NCT02559830 Unknown Phase2, Phase1
View Report
Metachromatic Leukodystrophy, Adrenoleukodystrophy
Sponsor Shenzhen Second People's Hospital (Other)
Compound CD34+ hematopoietic stem cells transduced with ABCD1 or ARSA
Therapeutic Modality Gene transfer
Target Gene ABCD1 or ARSA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2015-08-12 Last Modified Date: 2026-06-18 Enrollment: 50 China
NCT00749957 Completed Phase2, Phase1
View Report
Leber Congenital Amaurosis-Type 2
Sponsor Beacon Therapeutics (Industry)
Compound RAAV2-CB-hRPE65
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2008-09-08 Last Modified Date: 2026-06-18 Enrollment: 12 United States
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
NCT01422772 Completed Phase2, Phase1
View Report
Ischemic Heart Disease
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Vector Type none
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2011-08-21 Last Modified Date: 2026-06-18 Enrollment: 9 South Korea
NCT05164471 Terminated Phase2, Phase1
View Report
Hemophilia B
Sponsor Spur Therapeutics (Industry)
Compound FLT180a
Therapeutic Modality Gene transfer
Target Gene F9
Vector Type AAVS3
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-11-22 Last Modified Date: 2026-06-18 Enrollment: 6 United Kingdom, 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
NCT00643747 Completed Phase2, Phase1
View Report
Leber Congenital Amaurosis-Type 2
Sponsor University College, London (Other)
Compound TgAAG76
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2008-03-20 Last Modified Date: 2026-06-18 Enrollment: 12 United Kingdom
NCT02781480 Completed Phase2, Phase1
View Report
Leber Congenital Amaurosis
Sponsor MeiraGTx UK II Ltd (Industry)
Compound Cevaretigene ritoparvovec
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type AAV2/5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-04-28 Last Modified Date: 2026-06-18 Enrollment: 15 United Kingdom, United States
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
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
NCT02725580 Completed Phase2, Phase1
View Report
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease)
Sponsor Emily de los Reyes (Other)
Compound AT-GTX-501
Therapeutic Modality Gene transfer
Target Gene CLN6
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-03-16 Last Modified Date: 2026-06-18 Enrollment: 13 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
NCT05906251 Terminated Phase1
View Report
Limb Girdle Muscular Dystrophy, Type 2B/R2
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-6004
Therapeutic Modality Gene transfer
Target Gene DYSF
Vector Type dual AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-06-07 Last Modified Date: 2026-06-18 Enrollment: 2 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
NCT06948019 Not yet recruiting Phase2, Phase1
View Report
Hereditary Spastic Paraplegia Type 47
Sponsor BlackfinBio Ltd (Industry)
Compound BFB-101
Therapeutic Modality Gene transfer
Target Gene AP4B1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-04-22 Last Modified Date: 2026-06-18 Enrollment: 5 United States
NCT05820152 Terminated Phase2, Phase1
View Report
Leber Hereditary Optic Neuropathy (LHON)
Sponsor Neurophth Therapeutics Inc (Other)
Compound NFS-02
Therapeutic Modality Gene transfer
Target Gene ND1G3460A
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-04-06 Last Modified Date: 2026-06-18 Enrollment: 11 China, United States
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
NCT06747273 Terminated Phase1
View Report
Limb-Girdle Muscular Dystrophy, Type 2D/R3
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-9004
Therapeutic Modality Gene transfer
Target Gene SGCA
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-12-18 Last Modified Date: 2026-06-18 Enrollment: 4 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
NCT03173521 Completed Phase2, Phase1
View Report
Mucopolysaccharidosis Type VI
Sponsor Fondazione Telethon (Other)
Compound AAV2/8.TBG.hARSB
Therapeutic Modality Gene transfer
Target Gene ARSB
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-04-12 Last Modified Date: 2026-06-18 Enrollment: 9 Italy, Turkey (Türkiye)
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
NCT04669535 Terminated Phase1
View Report
Tay-Sachs Disease, Sandhoff Disease
Sponsor Terence Flotte (Other)
Compound AXO-AAV-GM2
Therapeutic Modality Gene transfer
Target Gene HEXA/HEXB
Vector Type dual AAVrh.8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-11-23 Last Modified Date: 2026-06-18 Enrollment: 9 United States
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.