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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 389 results in ClinicalTrials
NCT05788536 Recruiting Phase2, Phase1
View Report
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Sponsor Regeneron Pharmaceuticals (Industry)
Compound OTARMENI
Therapy Type Gene transfer
Target Gene OTOF
Vector Type dual AAV1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-03-15 Last Modified Date: 2026-07-01 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
Therapy Type 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
NCT07592273 Recruiting Phase3, Phase2
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Diabetic Retinopathy
Sponsor AbbVie (Industry)
Compound Surabgene lomparvovec
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 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
Therapy Type 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
Therapy Type 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
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 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
Therapy Type 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
Therapy Type Gene transfer
Target Gene IL2RG
Vector Type VSV-G
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2011-02-26 Last Modified Date: 2026-06-29 Enrollment: 40 United States
NCT06285643 Recruiting Phase2
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Parkinson's Disease
Sponsor AskBio Inc (Industry)
Compound AB-1005
Therapy Type Gene transfer
Target Gene GDNF
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations 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
Therapy Type Gene transfer
Target Gene SCN1A
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-06-10 Last Modified Date: 2026-06-29 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
Therapy Type 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
Therapy Type 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
NCT01054339 Completed Phase2
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Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-0106
Therapy Type Gene transfer
Target Gene SERPINA1
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2010-01-21 Last Modified Date: 2026-06-26 Enrollment: 9 Ireland, United States
NCT04124042 Completed Phase2
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Osteoarthritis, Knee
Sponsor Xalud Therapeutics, Inc. (Industry)
Compound XT-150
Therapy Type Gene transfer
Target Gene Recombinant IL10
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2019-10-09 Last Modified Date: 2026-06-26 Enrollment: 289 Australia, United States
NCT05398029 Completed Phase1
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Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-101
Therapy Type 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
NCT06461702 Unknown Early phase1
View Report
Familial Hypercholesterolemia
Sponsor The First Affiliated Hospital of Bengbu Medical University (Other)
Compound YOLT-101
Therapy Type Gene editing
Target Gene PCSK9/LDLR
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2024-04-16 Last Modified Date: 2026-06-26 Enrollment: 13 China
NCT05968118 Unknown Phase3
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Peripheral Artery Disease
Sponsor Beijing Northland Biotech. Co., Ltd. (Industry)
Compound NL003
Therapy Type Gene transfer
Target Gene HGF
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-01-04 Last Modified Date: 2026-06-26 Enrollment: 36 China
NCT05878860 Recruiting Phase3
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X-Linked Retinoschisis
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-201
Therapy Type 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
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
Therapy Type 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
Therapy Type Gene editing
Target Gene NCF1 (c.75_76delGT)
Delivery System Electroporation
Mechanism of Action Mutation correction
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-07-11 Last Modified Date: 2026-06-26 Enrollment: 12 Canada, United Kingdom, United States
NCT06634420 Active not recruiting Phase3
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Hereditary Angioedema
Sponsor Intellia Therapeutics (Industry)
Compound NTLA-2002
Therapy Type Gene editing
Target Gene KLKB1
Vector Type LDLR
Delivery System 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
NCT03720418 Terminated Phase2, Phase1
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Parkinson's Disease
Sponsor Sio Gene Therapies (Industry)
Compound OXB-102
Therapy Type 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
Therapy Type Gene transfer
Target Gene MiGRIK2
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
First Submitted: 2023-09-01 Last Modified Date: 2026-06-26 Enrollment: 12 United States
NCT02144610 Terminated Phase3
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Critical Limb Ischemia
Sponsor AnGes USA, Inc. (Industry)
Compound COLLATEGENE
Therapy Type Gene transfer
Target Gene HGF
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2014-05-20 Last Modified Date: 2026-06-26 Enrollment: 46 Belgium, Canada, Denmark + 8 more
NCT03566043 Active not recruiting Phase3, Phase2
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Mucopolysaccharidosis Type II (Hunter Syndrome)
Sponsor REGENXBIO Inc. (Industry)
Compound NAVSUNLI
Therapy Type 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
Therapy Type Gene transfer
Target Gene F8
Vector Type LK03
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-10-28 Last Modified Date: 2026-06-23 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
Therapy Type 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
Therapy Type Gene transfer
Target Gene Antibody to TDP-43
Vector Type AAV5.2
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Fast Track
First Submitted: 2025-11-19 Last Modified Date: 2026-06-23 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
Therapy Type Gene transfer
Target Gene GBA1
Vector Type AAVS3
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (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
Therapy Type 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
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
Therapy Type Gene transfer
Target Gene CYBB
Vector Type VSV-G
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2025-08-08 Last Modified Date: 2026-06-22 Enrollment: 10 United States
NCT07180355 Recruiting Phase1
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Friedreich's Ataxia
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-212
Therapy Type Gene transfer
Target Gene FXN
Vector Type AAVhu68
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-08-22 Last Modified Date: 2026-06-22 Enrollment: 10 United States
NCT05836259 Recruiting Phase2, Phase1
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Hypertrophic Cardiomyopathy
Sponsor Tenaya Therapeutics (Industry)
Compound TN-201
Therapy Type Gene transfer
Target Gene MYBPC3
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-04-18 Last Modified Date: 2026-06-18 Enrollment: 30 United States
NCT01496040 Completed Phase2, Phase1
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Leber Congenital Amaurosis
Sponsor Nantes University Hospital (Other)
Compound RAAV2/4.hRPE65
Therapy Type 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
NCT02652767 Completed Phase3
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Leber Hereditary Optic Neuropathy (LHON)
Sponsor GenSight Biologics (Industry)
Compound LUMEVOQ
Therapy Type Gene transfer
Target Gene ND4G11778A
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-01-07 Last Modified Date: 2026-06-18 Enrollment: 39 France, Germany, Italy + 2 more
NCT04537377 Active not recruiting Phase2, Phase1
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Wilson Disease
Sponsor Vivet Therapeutics SAS (Industry)
Compound VTX-801
Therapy Type 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
View Report
Limb-Girdle Muscular Dystrophy, Type 2E/R4
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-9003
Therapy Type Gene transfer
Target Gene SGCB
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-01-30 Last Modified Date: 2026-06-18 Enrollment: 17 Belgium, Germany, Italy + 3 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
Therapy Type 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
Therapy Type 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
Therapy Type 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
Therapy Type Gene transfer
Target Gene ABD-VEGFR
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2023-06-06 Last Modified Date: 2026-06-18 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
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2023-11-10 Last Modified Date: 2026-06-18 Enrollment: 48 China
NCT06255782 Recruiting Phase2, Phase1
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Ornithine Transcarbamylase (OTC) Deficiency
Sponsor iECURE, Inc. (Industry)
Compound ECUR-506
Therapy Type Gene transfer
Target Gene OTC/PCSK9 locus
Vector Type dual AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations 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
View Report
Parkinson's Disease
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGN-R09b
Therapy Type Gene transfer
Target Gene DDC
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2024-06-14 Last Modified Date: 2026-06-18 Enrollment: 39 China
NCT04127578 Active not recruiting Phase2, Phase1
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Parkinson's Disease
Sponsor Prevail Therapeutics (Industry)
Compound PR001
Therapy Type 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
Therapy Type 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
Therapy Type 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
View Report
X-Linked Myotubular Myopathy
Sponsor Astellas Gene Therapies (Industry)
Compound AT132
Therapy Type 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
NCT06611436 Terminated Phase2, Phase1
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Hemophilia B
Sponsor Be Biopharma (Industry)
Compound BE-101
Therapy Type Gene editing
Target Gene F9
Vector Type AAV6
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2024-09-18 Last Modified Date: 2026-06-18 Enrollment: 5 United States
NCT05144386 Completed Phase1
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HIV-1-infection
Sponsor Excision BioTherapeutics (Industry)
Compound EBT-101
Therapy Type 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
NCT00598481 Completed Phase2
View Report
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Sponsor Fondazione Telethon (Other)
Compound STRIMVELIS
Therapy Type Gene transfer
Target Gene ADA
Vector Type MoMLV (RV)
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2008-01-10 Last Modified Date: 2026-06-18 Enrollment: 12 Israel, Italy
NCT02362438 Completed Phase1
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Giant Axonal Neuropathy
Sponsor National Institute of Neurological Disorders and Stroke (NINDS) (NIH)
Compound ScAAV9/JeT-GAN
Therapy Type Gene transfer
Target Gene GAN
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2015-02-12 Last Modified Date: 2026-06-18 Enrollment: 14 United States
NCT05353647 Active not recruiting Phase2
View Report
Sickle Cell Disease
Sponsor David Williams (Other)
Compound Autologous CD34+ HSC cells transduced with the lentiviral vector containing BCL11a-targeted shRNA
Therapy Type 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
View Report
Retinitis Pigmentosa, Choroideremia
Sponsor Ray Therapeutics, Inc. (Industry)
Compound RTx-015
Therapy Type Gene transfer
Target Gene Codon optimized ChR-3M
Vector Type AAV.7m8
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-06-10 Last Modified Date: 2026-06-18 Enrollment: 10 United States
NCT03580083 Suspended Phase2, Phase1
View Report
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-111
Therapy Type Gene transfer
Target Gene IDUA
Vector Type AAV2/9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-06-18 Last Modified Date: 2026-06-18 Enrollment: 21 Brazil, United States
NCT04581785 Terminated Phase2, Phase1
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Methylmalonic Acidemia
Sponsor LogicBio Therapeutics, Inc (Industry)
Compound LB-001
Therapy Type Gene transfer
Target Gene MMUT
Vector Type AAV-LK03
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-10-02 Last Modified Date: 2026-06-18 Enrollment: 4 United States
NCT05222178 Terminated Phase1
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Phenylketonuria (PKU)
Sponsor Homology Medicines, Inc (Industry)
Compound HMI-103
Therapy Type 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
View Report
Multiple System Atrophy
Sponsor Brain Neurotherapy Bio, Inc. (Industry)
Compound AB-1005
Therapy Type Gene transfer
Target Gene GDNF
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2020-12-15 Last Modified Date: 2026-06-18 Enrollment: 9 United States
NCT06422351 Suspended Phase2
View Report
Pyruvate Kinase Deficiency
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-L301
Therapy Type Gene transfer
Target Gene PKLR
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2024-05-15 Last Modified Date: 2026-06-18 Enrollment: 10 Spain, United States
NCT06332807 Recruiting Phase2, Phase1
View Report
Phenylketonuria (PKU)
Sponsor NGGT INC. (Industry)
Compound NGGT002
Therapy Type Gene transfer
Target Gene PAH
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-02-07 Last Modified Date: 2026-06-18 Enrollment: 12 United States
NCT07064759 Recruiting Phase3
View Report
Neovascular Age-Related Macular Degeneration (nAMD)
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-150
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (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
View Report
Krabbe Disease
Sponsor Forge Biologics, Inc (Industry)
Compound FBX-101
Therapy Type Gene transfer
Target Gene GALC
Vector Type AAVrh10
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-02-13 Last Modified Date: 2026-06-18 Enrollment: 9 United States
NCT06388200 Active not recruiting Phase3
View Report
Retinitis Pigmentosa
Sponsor Ocugen (Industry)
Compound OCU400
Therapy Type Gene transfer
Target Gene NR2E3
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations 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
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Frontera Therapeutics (Industry)
Compound FT-003
Therapy Type Gene transfer
Target Gene Aflibercept
Vector Type AAV2.7m8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2024-07-01 Last Modified Date: 2026-06-18 Enrollment: 78 China
NCT01482195 Completed Phase1
View Report
Retinal Disease, Retinitis Pigmentosa
Sponsor King Khaled Eye Specialist Hospital (Other gov)
Compound AAV2-VMD2-hMERTK
Therapy Type 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
View Report
Transfusion-dependent Alpha-Thalassemia
Sponsor First Affiliated Hospital of Guangxi Medical University (Other)
Compound GMCN-508A
Therapy Type 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
Therapy Type Gene transfer
Target Gene CYP46A1
Vector Type AAVrh10
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2022-09-13 Last Modified Date: 2026-06-18 Enrollment: 5 France
NCT04293185 Active not recruiting Phase3
View Report
Sickle Cell Disease
Sponsor Genetix Biotherapeutics Inc. (Industry)
Compound LYFGENIA
Therapy Type 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
NCT03612869 Unknown Phase3, Phase2
View Report
Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor LYSOGENE (Industry)
Compound LYS-SAF301
Therapy Type Gene transfer
Target Gene SGSH
Vector Type AAV2rh.10
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-07-04 Last Modified Date: 2026-06-18 Enrollment: 20 France, Germany, Netherlands + 2 more
NCT03315182 Terminated Phase2, Phase1
View Report
Mucopolysaccharidosis Type 3 B
Sponsor Abeona Therapeutics, Inc (Industry)
Compound ABO-101
Therapy Type Gene transfer
Target Gene NAGLU
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-10-10 Last Modified Date: 2026-06-18 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
Therapy Type Gene editing
Target Gene VEGFA mRNA
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2024-09-30 Last Modified Date: 2026-06-18 Enrollment: 15
NCT01301443 Completed Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration
Sponsor Oxford BioMedica (Industry)
Compound RetinoStat
Therapy Type Gene transfer
Target Gene COL18A1+PLG
Vector Type EIAV
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2011-02-21 Last Modified Date: 2026-06-18 Enrollment: 21 United States
NCT06451770 Recruiting Phase1
View Report
Hypercholesterolemia
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-201
Therapy Type Gene editing
Target Gene ANGPTL3
Vector Type LDLR + GalNAc
Delivery System 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
Therapy Type Gene transfer
Target Gene IL2RG
Vector Type MFGS/GALV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2001-12-17 Last Modified Date: 2026-06-18 Enrollment: 3 United States
NCT06983158 Terminated Phase2, Phase1
View Report
Developmental and Epileptic Encephalopathy
Sponsor Capsida Biotherapeutics, Inc. (Industry)
Compound CAP-002
Therapy Type Gene transfer
Target Gene STXBP1
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2025-05-05 Last Modified Date: 2026-06-18 Enrollment: 1 United States
NCT02317887 Completed Phase2, Phase1
View Report
X-Linked Retinoschisis
Sponsor VegaVect, Inc. (Industry)
Compound RS1 AAV Vector
Therapy Type 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
NCT05898620 Active not recruiting Phase3
View Report
Rett Syndrome
Sponsor Neurogene Inc. (Industry)
Compound NGN-401
Therapy Type 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
View Report
Stargardt Disease
Sponsor Splice Bio (Industry)
Compound SB-007
Therapy Type Gene transfer
Target Gene ABCA4
Vector Type dual AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2025-02-18 Last Modified Date: 2026-06-18 Enrollment: 57 United States
NCT01505062 Terminated Phase2, Phase1
View Report
Usher Syndrome, Retinitis Pigmentosa
Sponsor Sanofi (Industry)
Compound SAR421869
Therapy Type Gene transfer
Target Gene MYO7A
Vector Type EIAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2012-01-04 Last Modified Date: 2026-06-18 Enrollment: 9 France, United States
NCT06517888 Recruiting Phase2, Phase1
View Report
Vestibular Schwannoma
Sponsor Akouos, Inc. (Industry)
Compound AAVAnc80-antiVEGF
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAVAnc80
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-07-19 Last Modified Date: 2026-06-18 Enrollment: 27 United States
NCT03538899 Recruiting Phase2, Phase1
View Report
Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency
Sponsor University of California, San Francisco (Other)
Compound AProArt
Therapy Type 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
View Report
Leber Congenital Amaurosis-Type 5
Sponsor Opus Genetics, Inc (Industry)
Compound OPGx-LCA5
Therapy Type 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
Therapy Type 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
View Report
Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency
Sponsor Assistance Publique - Hôpitaux de Paris (Other)
Compound ARTEGENE
Therapy Type Gene transfer
Target Gene DCLRE1C
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-08-27 Last Modified Date: 2026-06-18 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
Therapy Type Gene transfer
Target Gene Codon optimized aflibercept
Vector Type AAV.7m8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2025-02-26 Last Modified Date: 2026-06-18 Enrollment: 311 United States
NCT03328130 Terminated Phase2, Phase1
View Report
Retinitis Pigmentosa
Sponsor eyeDNA Therapeutics (Industry)
Compound CTX-PDE6B
Therapy Type 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
NCT03837483 Active not recruiting Phase3
View Report
Wiskott-Aldrich Syndrome
Sponsor Fondazione Telethon (Other)
Compound WASKYRA
Therapy Type Gene transfer
Target Gene WAS
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations 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
Therapy Type 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
Therapy Type 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
Therapy Type 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
NCT05345171 Active not recruiting Phase3
View Report
Ornithine Transcarbamylase (OTC) Deficiency
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound DTX301
Therapy Type 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
NCT00749957 Completed Phase2, Phase1
View Report
Leber Congenital Amaurosis-Type 2
Sponsor Beacon Therapeutics (Industry)
Compound RAAV2-CB-hRPE65
Therapy Type 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
NCT05805007 Unknown Early phase1
View Report
Retinitis Pigmentosa
Sponsor Peking University Third Hospital (Other)
Compound ZVS203e
Therapy Type Gene editing
Target Gene RHO
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2023-03-14 Last Modified Date: 2026-06-18 Enrollment: 9 China
NCT05121376 Active not recruiting Phase2, Phase1
View Report
Hereditary Angioedema
Sponsor BioMarin Pharmaceutical (Industry)
Compound BMN 331
Therapy Type 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
Therapy Type Gene transfer
Target Gene SGCG
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-07-24 Last Modified Date: 2026-06-18 Enrollment: 4 United States
NCT01422772 Completed Phase2, Phase1
View Report
Ischemic Heart Disease
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapy Type Gene transfer
Target Gene HGF
Vector Type none
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
Therapy Type 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
NCT00999609 Active not recruiting Phase3
View Report
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Sponsor Spark Therapeutics, Inc. (Industry)
Compound LUXTURNA
Therapy Type 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
Therapy Type 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
Therapy Type 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

Clinical Trials Daily Digest

Updates in the last 14 days

NCT05788536
Browse condition terms Previous: Db-oto, Gene therapy, Congenital hearing loss, Sen... Current: Db-oto, Gene therapy, Congenital hearing loss, Sen... api
Last update post date Previous: 2026-05-05 Current: 2026-07-01 api
Estimated completion date Previous: 2032-02-28 Current: 2032-02-25 api
Brief title Previous: A study of db-oto, An adeno-associated virus (aav)... Current: A study of db-oto, An adeno-associated virus (aav)... api
Eligibility std age Previous: Child Current: Child, Adult, Older adult api
Eligibility max age Previous: 17 years Current: null api
Eligibility criteria Previous: Key inclusion criteria: 1. willingness of at leas... Current: Key inclusion criteria: 1. willingness to provide... api
Enrollment count Previous: 30 Current: 36 api
Intervention description Previous: Db-oto will be administered as a single intracochl... Current: Administered per the protocol api
Intervention name Previous: Db-oto, Db-oto Current: Db-oto api
Description Previous: Regeneron is conducting a study of an investigatio... Current: Regeneron is conducting a study of an investigatio... api
Editor type Previous: none Current: curator
Recent updates Previous: Otarmeni was approved 4/23/26 under the National P... Current: Otarmeni was approved 4/23/26 under the National P... curator
News and press releases Previous: null Current: LinkName:Otarmeni™ (lunsotogene parvec) Receives E... curator
Number of locations Previous: 15 Current: 16 api
Locations Previous: United states, United kingdom, Germany, Spain Current: United states, Japan, United kingdom, Germany, Spa... api
Development status Previous: Active Current: Approved curator
Compound name Previous: DB-OTO Current: OTARMENI curator
Alias type Previous: null Current: proprietary name, proper name curator
Alias value Previous: null Current: DB-OTO, lunsotogene parvec curator
Fda designation Previous: Fast Track, Orphan Drug Designation, Rare Pediatri... Current: Accelerated Approval, Fast Track, Orphan Drug Desi... curator
News and press releases Previous: null Current: LinkName:FDA Approval Documents;LinkUrl:https://ww... curator
News and press releases Previous: null Current: LinkName:FDA Approves First-Ever Gene Therapy for ... curator
2026-07-01
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (...
Regeneron Pharmaceuticals Recruiting
Otarmeni was approved 4/23/26 under the National Priority Voucher Program, Marketing Authorization Application was accepted for review by the EMA
NCT07667387
Record status Previous: Draft Current: Active curator
Compound description Previous: Current: personalized variant-specific LNP.UCD.ABE curator
Indication doid Previous: Current: DOID:9267 curator
Development status Previous: Current: Active curator
Compound name Previous: Current: LNP.UCD.ABE curator
Recent updates Previous: Current: This clinical trial protocol is a master protocol ... curator
Dose 1 Previous: Current: undisclosed dose based on body weight with potenti... curator
Editor type Previous: Current: ABE mRNA curator
Delivery system Previous: Current: Endocytosis curator
Drug product type Previous: Current: Encapsulated mRNA curator
Route of administration Previous: Current: Intravenous curator
Mechanism of action Previous: Current: Mutation correction curator
Therapy route Previous: Current: in vivo curator
Therapy type Previous: Current: Gene editing curator
Target gene Previous: Current: CPS1,OTC,ASS1,ASL,ARG,NAGS,SLC25A15 curator
Preclinical publications Previous: null Current: LinkName:Pre-IND Official Responses/Meeting Summar... curator
Preclinical publications Previous: null Current: LinkName:Pre-IND Briefing Book;LinkUrl:https://scg... curator
Preclinical publications Previous: null Current: LinkName:Patient-Specific In Vivo Gene Editing to ... curator
Preclinical publications Previous: null Current: LinkName:How to create personalized gene editing p... curator
Last update post date Previous: 2026-06-25 Current: 2026-06-29 api
Eligibility criteria Previous: Inclusion criteria: 1. diagnosis of a severe urea... Current: Inclusion criteria: 1. diagnosis of a severe urea... api
2026-07-01
Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
Rebecca Ahrens-Nicklas Not yet recruiting
This clinical trial protocol is a master protocol for participants with a mutation in one of the 7 genes that cause a urea cycle disorder. Participant...
NCT07592273
Last update post date Previous: 2026-06-26 Current: 2026-07-01 api
Number of locations Previous: 8 Current: 11 api
Locations Previous: United states Current: Puerto rico, United states api
Indication doid Previous: DOID:9191, DOID:8947 Current: DOID:8947 curator
Record status Previous: Draft Current: Active curator
Recent updates Previous: Current: this product is also under evaluation for wet AMD curator
Dose 3 Previous: 1.0E12 GC/eye Current: curator
Dose 2 Previous: 5.0E11 GC/eye Current: Phase 3 dose: 1.0E12 GC/eye curator
Dose 1 Previous: 2.5E11 GC/eye Current: Phase 2 dose: 2.5E11 GC/eye, 5.0E11 GC/eye,1.0E12 ... curator
News and press releases Previous: null Current: LinkName:REGENXBIO Announces Pivotal Program for S... curator
Development status Previous: Current: Active curator
Compound name Previous: Current: Surabgene lomparvovec curator
Vector type Previous: Current: AAV8 curator
Delivery system Previous: Current: Viral transduction curator
Drug product type Previous: Current: Viral vector curator
Route of administration Previous: Current: Suprachoroidal curator
Mechanism of action Previous: Current: Genetic delivery of therapeutic protein curator
Therapy route Previous: Current: In-vivo curator
Therapy type Previous: Current: Gene transfer curator
Target gene Previous: Current: Anti-VEGF curator
Alias type Previous: null Current: proprietary name curator
Alias value Previous: null Current: ABBV-RGX-314 curator
Related nctid Previous: null Current: LinkName:Long Term Follow-Up: NCT05296447;LinkUrl:... curator
Related nctid Previous: null Current: LinkName:Phase 2: NCT04567550;LinkUrl:https://clin... curator
Related nctid Previous: null Current: LinkName:Phase 2: NCT06942520 (uses subretinal inj... curator
News and press releases Previous: null Current: LinkName:REGENXBIO Reports Third Quarter 2025 Fina... curator
Clinical publications Previous: null Current: LinkName:(Presentation) Suprachoroidal Surabgene L... curator
Estimated completion date Previous: 2036-02 Current: 2036-01 api
Overall status Previous: Not yet recruiting Current: Recruiting api
2026-07-01
Diabetic Retinopathy
AbbVie Recruiting
This product is also under evaluation for wet AMD
NCT04713475
Last update post date Previous: 2025-05-21 Current: 2026-06-30 api
Editor type Previous: none Current: curator
Route of administration Previous: Intracisterna magna Current: Intracisternal curator
Eligibility criteria Previous: null Current: Inclusion criteria: * all patients: documented gm... api
2026-06-30
GM1 Gangliosidosis, Beta-Galactosidase-1 (GLB1) Deficiency
Gemma Biotherapeutics Active not recruiting
Out-licensed development to Gemma Biotherapeutics, uncertain development status
NCT05693142
Last update post date Previous: 2025-12-17 Current: 2026-06-30 api
Recent updates Previous: Pivotal topline data announced May 2026, Sponsor p... Current: Pivotal topline data announced May 2026, dosing co... curator
News and press releases Previous: null Current: LinkName:REGENXBIO Completes Dosing in Confirmator... curator
Editor type Previous: none Current: curator
Clinical publications Previous: null Current: LinkName:(Corporate Presentation) AFFINITY DUCHENN... curator
News and press releases Previous: null Current: LinkName:REGENXBIO Announces Positive Topline Resu... curator
Eligibility criteria Previous: null Current: Part 1 - key inclusion criteria: * the participan... api
News and press releases Previous: null Current: LinkName:REGENXBIO Highlights Key 2026 Catalysts a... curator
2026-06-30
Duchenne Muscular Dystrophy (DMD)
REGENXBIO Inc. Recruiting
Pivotal topline data announced May 2026, dosing completed in confirmatory study. Sponsor plans to pursue accelerated approval and submit a BLA in Q3 2...
27 more update(s) recently — View All
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.