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Clinical Trials - Gene Therapy Trial Browser

The Gene Therapy Trial Browser represents a unique publicly accessible, free database for the benefit of users seeking information on gene therapy development. The information within integrates various sources, including clinicaltrials.gov, publications, sponsor press releases, patent applications, and more to give a comprehensive overview of the gene therapy clinical trial landscape.

Disclaimer: The information on this dashboard has been collected for the convenience of patients and researchers. The SCGE team are not medical doctors and cannot provide medical advice. Please discuss with your provider the risks/benefits of participating in a clinical trial, and do not send us your personal medical information. The information contained within this table does not make use of any confidential or privileged information-all data is collected from publicly available sources. The SCGE makes no comment as to the efficacy and safety of the items listed, as these are not known at the time of publication. For the most up to date information, or to inquire about enrollment, please refer to clinicaltrials.gov or the Sponsor's website for contact information.

Clinical Trials Daily Digest

Updates in the last 14 days

NCT07282847
Last update post date 2026-05-04 2026-05-06 api
Eligibility criteria null Inclusion criteria: 1. participant must be ≥18 ye... api
Record status Draft Active curator
Preclinical publications LinkName:Int J Mol Sci . 2022 Jun 4;23(11):6298. ... LinkName:Isogenic GAA-KO Murine Muscle Cell Lines ... curator
Indication doid null DOID:2752 curator
Development status null Active curator
Compound name null AB-1009 curator
Recent updates null FDA accepted IND in January 2026 curator
Dose 2 null 1.5E13 vg/kg curator
Dose 1 null 1E13 vg/kg curator
Vector type null AAV curator
Delivery system null viral transduction curator
Drug product type null viral vector curator
Route of administration null intravenous curator
Mechanism of action null functional gene replacement curator
Therapy route null in-vivo curator
Therapy type null gene transfer curator
Target gene null GAA curator
Fda designation Fast Track, Orphan Drug Designation curator
Preclinical publications null LinkName:Rescue of Pompe disease in mice by AAV-me... curator
Preclinical publications null LinkName:Low-Dose Liver-Targeted Gene Therapy for ... curator
News and press releases null LinkName:Genethon Signs Licensing Agreement with A... curator
News and press releases null LinkName:AskBio Announces FDA Acceptance of Invest... curator
2026-05-28
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
AskBio Inc Recruiting
FDA accepted IND in January 2026
NCT06461702
Delivery system LNP Endocytosis curator
Drug product type RNP Encapsulated RNP curator
Overall status Recruiting Unknown curator
Eligibility criteria null Inclusion criteria: 1. men or women aged ≥ 18 yea... api
Clinical publications null LinkName:In vivo base editing gene therapy for het... curator
2026-05-28
Familial Hypercholesterolemia
The First Affiliated Hospital of Bengbu Medical University Unknown
FDA cleared IND application in June 2025
NCT06856759
Eligibility criteria null Inclusion criteria: 1. 4-10 years old (at the tim... api
2026-05-28
Rett Syndrome
Guangzhou Women and Children's Medical Center Active not recruiting
NCT07052929
Last update post date 2026-03-13 2026-04-15 api
Number of locations 3 4 api
Locations United states Canada, United states api
Record status Draft Active curator
Target tissue muscle cells curator
Recent updates Astellas has restarted their XLMTM program by crea... Astellas has restarted their XLMTM program by crea... curator
Dose 2 2E12 vg/kg curator
Dose 1 1E12 vg/kg curator
Related nctid null LinkName:Phase 2/3: NCT03199469 (prior drug produc... curator
Related nctid null LinkName:Epidemiological study: NCT06581146;LinkUr... curator
News and press releases null LinkName:Astellas Takes Aim at XLMTM Again, Now wi... curator
News and press releases null LinkName:VALOR Clinical Trial Patient Information;... curator
Protocol null LinkName:(Abstract) VALOR study design: a phase 1/... curator
Protocol null LinkName:(Abstract 74P) Design of a phase 1/2 stud... curator
Preclinical publications null LinkName:(Abstract) Efficacy, Biodistribution, and... curator
Compound description MyoAAV3.8-MHCK7-hMTM1 curator
Indication doid DOID:0111225 curator
Development status Active curator
Compound name ASP2957 curator
Vector type AAV3.8 curator
Delivery system viral transduction curator
Drug product type viral vector curator
Route of administration intravenous curator
Mechanism of action functional gene replacement curator
Therapy route in-vivo curator
Therapy type gene transfer curator
Target gene MTM1 curator
Alias type null proprietary name curator
Alias value null KT430 curator
Clinical publications null LinkName:Intrahepatic Cholestasis Is a Clinically ... curator
Clinical publications null LinkName:Hepatobiliary disease in XLMTM: a common ... curator
News and press releases null LinkName:Astellas Sees Path Forward for Gene Thera... curator
Eligibility criteria null Inclusion criteria: * participant is projected to... api
2026-05-28
X-Linked Myotubular Myopathy
Astellas Gene Therapies Recruiting
Astellas has restarted their XLMTM program by creating a new drug product ASP2957, with substantial modifications to the capsid to detarget the liver,...
NCT07592273
Last update post date 2026-05-18 2026-05-20 api
Estimated completion date 2036-02 2036-01 api
Overall status Not yet recruiting Recruiting api
2026-05-28
Diabetic Retinopathy
AbbVie Recruiting
This product is also under evaluation for wet AMD
38 more update(s) recently — View All

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Showing 1-100 of 228 results in ClinicalTrials
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NCT07282847 Recruiting Phase2, Phase1
View Report
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Sponsor AskBio Inc (Industry)
Compound AB-1009
Therapy Type Gene transfer
Target Gene GAA
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2025-11-26 Last Modified Date: 2026-05-28 Enrollment: 12 United States
NCT06856759 Active not recruiting Early phase1
View Report
Rett Syndrome
Sponsor Guangzhou Women and Children's Medical Center (Other)
Compound AAV-MECP2
Therapy Type Gene transfer
Target Gene MECP2
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-12-29 Last Modified Date: 2026-05-28 Enrollment: 8 China
NCT07052929 Recruiting Phase2, Phase1
View Report
X-Linked Myotubular Myopathy
Sponsor Astellas Gene Therapies (Industry)
Compound ASP2957
Therapy Type Gene transfer
Target Gene MTM1
Vector Type AAV3.8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-06-30 Last Modified Date: 2026-05-28 Enrollment: 9 Canada, United States
NCT07592273 Recruiting Phase3, Phase2
View Report
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: 2026-05-28 Enrollment: 576 United States
NCT05419492 Recruiting Phase2, Phase1
View Report
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: 2026-05-28 Enrollment: 47 Australia, United Kingdom, United States
NCT02599922 Active not recruiting Phase2, Phase1
View Report
Achromatopsia
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-401
Therapy Type 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: 2026-05-27 Enrollment: 32 United States
NCT06539208 Recruiting Phase2, Phase1
View Report
Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy
Sponsor YolTech Therapeutics Co., Ltd (Industry)
Compound YOLT-201
Therapy Type Gene editing
Target Gene TTR
Vector Type LNP
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2024-08-01 Last Modified Date: 2026-05-27 Enrollment: 31 China
NCT06389877 Recruiting Phase2, Phase1
View Report
Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-302
Therapy Type Gene editing
Target Gene SERPINA1 (p.Glu366Lys)
Vector Type LNP
Delivery System Endocytosis
Mechanism of Action Mutation correction
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-04-22 Last Modified Date: 2026-05-27 Enrollment: 106 Australia, Ireland, Netherlands + 3 more
NCT06164730 Recruiting Phase1
View Report
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: 2026-05-27 Enrollment: 85 Australia, Canada, Israel + 3 more
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: 2026-05-27 Enrollment: 36 Australia, Canada, South Africa + 1 more
NCT07007065 Recruiting Phase3
View Report
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: 2026-05-27 Enrollment: 561 Austria, Belgium, Bulgaria + 14 more
NCT06735755 Recruiting Phase2, Phase1
View Report
Glycogen Storage Disease Type Ia
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-301
Therapy Type Gene editing
Target Gene G6PC1 (p.R83C)
Vector Type LNP
Delivery System Endocytosis
Mechanism of Action Mutation correction
First Submitted: 2024-12-06 Last Modified Date: 2026-05-27 Enrollment: 36 United States
NCT06128629 Recruiting Phase3
View Report
Transthyretin Amyloidosis (ATTR) with Cardiomyopathy
Sponsor Intellia Therapeutics (Industry)
Compound NTLA-2001
Therapy Type Gene editing
Target Gene TTR
Vector Type LNP
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2023-11-08 Last Modified Date: 2026-05-27 Enrollment: 1200 Argentina, Australia, Austria + 23 more
NCT06680232 Recruiting Phase1
View Report
Chronic Hepatitis B
Sponsor Precision BioSciences, Inc. (Industry)
Compound PBGENE-HBV
Therapy Type Gene editing
Target Gene HBV DNA
Vector Type LNP
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Fast Track
First Submitted: 2024-10-24 Last Modified Date: 2026-05-27 Enrollment: 45 Hong Kong, Moldova, New Zealand + 1 more
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: 2026-05-27 Enrollment: 27 United States
NCT06839235 Recruiting Phase2, Phase1
View Report
Primary Hyperoxaluria Type 1 (PH1)
Sponsor Arbor Biotechnologies (Industry)
Compound ABO-101
Therapy Type Gene editing
Target Gene HAO1
Vector Type LNP
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-02-17 Last Modified Date: 2026-05-27 Enrollment: 23 France, Germany, Tunisia + 2 more
NCT06634420 Active not recruiting Phase3
View Report
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: 2026-05-27 Enrollment: 80 Australia, Canada, France + 6 more
NCT07226206 Recruiting Phase2, Phase1
View Report
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: 2026-05-26 Enrollment: 5 United States
NCT05881408 Active not recruiting Phase3
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound ELEVIDYS
Therapy Type Gene transfer
Target Gene Micro-dystrophin
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Priority Review, Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-19 Last Modified Date: 2026-05-22 Enrollment: 148 Australia, Belgium, Canada + 11 more
NCT01306019 Recruiting Phase2, Phase1
View Report
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: 2026-05-22 Enrollment: 40 United States
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: 2026-05-22 Enrollment: 311 United States
NCT04370054 Active not recruiting Phase3
View Report
Hemophilia A
Sponsor Pfizer (Industry)
Compound Giroctogogene fitelparvovec
Therapy Type Gene transfer
Target Gene F8
Vector Type AAV2/6
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2020-04-21 Last Modified Date: 2026-05-18 Enrollment: 77 Australia, Brazil, Canada + 13 more
NCT03588299 Active not recruiting Phase2, Phase1
View Report
Hemophilia A
Sponsor Bayer (Industry)
Compound DTX201
Therapy Type Gene transfer
Target Gene F8
Vector Type AAVhu37
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-07-06 Last Modified Date: 2026-05-18 Enrollment: 11 Bulgaria, France, Germany + 3 more
NCT06817382 Recruiting Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Insmed Gene Therapy LLC (Industry)
Compound INS1201
Therapy Type Gene transfer
Target Gene Micro-dystrophin
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-02-04 Last Modified Date: 2026-05-18 Enrollment: 12 United States
NCT04747431 Active not recruiting Phase2, Phase1
View Report
Frontotemporal Dementia, FTD, FTD-GRN, C9orf72
Sponsor Passage Bio, Inc. (Industry)
Compound PBFT02
Therapy Type Gene transfer
Target Gene GRN
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2021-02-02 Last Modified Date: 2026-05-15 Enrollment: 30 Australia, Brazil, Canada + 2 more
NCT05748873 Active not recruiting Phase2, Phase1
View Report
Retinitis Pigmentosa
Sponsor SparingVision (Industry)
Compound SPVN06
Therapy Type Gene transfer
Target Gene NXNL1
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-02-07 Last Modified Date: 2026-05-15 Enrollment: 33 France, United States
NCT06217861 Recruiting Phase1
View Report
Glutaric Acidemia Type I
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGM-R02b
Therapy Type Gene transfer
Target Gene GCDH
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-12-12 Last Modified Date: 2026-05-14 Enrollment: 12 China
NCT07287397 Recruiting Phase2, Phase1
View Report
Amyotrophic Lateral Sclerosis
Sponsor Vector Y Therapeutics (Industry)
Compound VTx-002
Therapy Type Gene transfer
Target Gene Antibody to TDP-43
Vector Type AAV5.2
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Fast Track
First Submitted: 2025-11-19 Last Modified Date: 2026-05-14 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 more
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: 2026-05-14 Enrollment: 5 France
NCT03861273 Active not recruiting Phase3
View Report
Hemophilia B
Sponsor Pfizer (Industry)
Compound BEQVEZ
Therapy Type Gene transfer
Target Gene F9-R338L
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2019-03-01 Last Modified Date: 2026-05-12 Enrollment: 51 Australia, Brazil, Canada + 13 more
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: 2026-05-12 Enrollment: 25 United States
NCT05504837 Recruiting Phase1
View Report
Cystic Fibrosis
Sponsor Krystal Biotech, Inc. (Industry)
Compound KB407
Therapy Type Gene transfer
Target Gene CFTR
Vector Type HSV-1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-08-15 Last Modified Date: 2026-05-08 Enrollment: 20 United States
NCT07160634 Recruiting Phase3
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-003
Therapy Type Gene transfer
Target Gene Micro-dystrophin
Vector Type AAV-SLB101
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Rare Pediatric Disease Designation
First Submitted: 2025-08-29 Last Modified Date: 2026-05-08 Enrollment: 80 Australia, Canada
NCT03368742 Active not recruiting Phase2, Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-001
Therapy Type Gene transfer
Target Gene Micro-dystrophin
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-12-05 Last Modified Date: 2026-05-08 Enrollment: 12 United States
NCT06255782 Recruiting Phase2, Phase1
View Report
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: 2026-05-07 Enrollment: 20 Australia, Spain, United Kingdom + 1 more
NCT05682144 Recruiting Phase1
View Report
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Sponsor Immusoft of CA, Inc. (Industry)
Compound ISP-001
Therapy Type Gene transfer
Target Gene IDUA
Vector Type Sleeping Beauty
Delivery System Electroporation
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-12-12 Last Modified Date: 2026-05-07 Enrollment: 11 United States
NCT05456880 Active not recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-101
Therapy Type Gene editing
Target Gene HBG1/HBG2
Vector Type none
Delivery System Electroporation
Mechanism of Action Mutation correction
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2022-06-23 Last Modified Date: 2026-05-07 Enrollment: 15 United States
NCT05878860 Recruiting Phase3
View Report
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: 2026-05-07 Enrollment: 97 United States
NCT06064890 Recruiting Phase2, Phase1
View Report
Frontotemporal Dementia, FTD, FTD-GRN
Sponsor AviadoBio Ltd (Industry)
Compound AVB-101
Therapy Type Gene transfer
Target Gene GRN
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2023-09-12 Last Modified Date: 2026-05-06 Enrollment: 18 Belgium, Canada, Italy + 6 more
NCT02716246 Recruiting Phase3, Phase2
View Report
Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound UX111
Therapy Type Gene transfer
Target Gene SGSH
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-03-17 Last Modified Date: 2026-05-06 Enrollment: 36 Australia, Spain, United States
NCT05444894 Active not recruiting Phase2, Phase1
View Report
Beta-Thalassemia Major
Sponsor Editas Medicine, Inc. (Industry)
Compound EDIT-301
Therapy Type Gene editing
Target Gene HBG1/HBG2
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-06-27 Last Modified Date: 2026-05-06 Enrollment: 9 Canada, United States
NCT05598333 Active not recruiting Phase2
View Report
Congestive Heart Failure
Sponsor AskBio Inc (Industry)
Compound AB-1002
Therapy Type Gene transfer
Target Gene PPP1R1A
Vector Type AAV2i8
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2022-10-25 Last Modified Date: 2026-05-06 Enrollment: 150 Austria, Belgium, Bulgaria + 9 more
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: 2026-05-06 Enrollment: 9 United States
NCT06199531 Active not recruiting Phase3
View Report
NGLY1 Deficiency
Sponsor Grace Science, LLC (Industry)
Compound GS-100
Therapy Type Gene transfer
Target Gene NGLY1
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, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-11-21 Last Modified Date: 2026-05-06 Enrollment: 10 United States
NCT04853576 Active not recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Editas Medicine, Inc. (Industry)
Compound EDIT-301
Therapy Type Gene editing
Target Gene HBG1/HBG2
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: 2026-05-06 Enrollment: 45 Canada, United States
NCT06285643 Recruiting Phase2
View Report
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: 2026-05-06 Enrollment: 127 Germany, Poland, United Kingdom + 1 more
NCT06018558 Active not recruiting Phase2, Phase1
View Report
Geographic Atrophy
Sponsor Ocugen (Industry)
Compound OCU410
Therapy Type Gene transfer
Target Gene RORA
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-06-30 Last Modified Date: 2026-05-06 Enrollment: 60 United States
NCT05926765 Recruiting Phase2
View Report
Grade 2 and 3 Radiation-Induced Late Xerostomia
Sponsor MeiraGTx, LLC (Industry)
Compound AAV-hAQP1
Therapy Type Gene transfer
Target Gene AQP1
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2023-06-22 Last Modified Date: 2026-05-06 Enrollment: 276 Canada, United Kingdom, United States
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
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: 2026-05-05 Enrollment: 30 Germany, Japan, Spain + 2 more
NCT06506461 Recruiting Phase1
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Sickle Cell Disease
Sponsor St. Jude Children's Research Hospital (Other)
Compound Gene-modified CD34+ cells
Therapy Type Gene editing
Target Gene BCL11A
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-06-14 Last Modified Date: 2026-05-05 Enrollment: 25 United States
NCT06826612 Recruiting Phase2, Phase1
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Huntington's Disease
Sponsor Hoffmann-La Roche (Industry)
Compound RG6662
Therapy Type Gene transfer
Target Gene MiHTT
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2025-02-03 Last Modified Date: 2026-05-05 Enrollment: 53 United States
NCT06092034 Recruiting Phase2
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Danon Disease
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-A501
Therapy Type Gene transfer
Target Gene LAMP2B
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-10-11 Last Modified Date: 2026-05-05 Enrollment: 14 Germany, Italy, United Kingdom + 1 more
NCT06765980 Recruiting Phase2, Phase1
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Geographic Atrophy Secondary to Age-related Macular Degeneration
Sponsor Kriya Therapeutics, Inc. (Industry)
Compound KRIYA-825
Therapy Type Gene transfer
Target Gene CR2-CR1
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2024-12-19 Last Modified Date: 2026-05-01 Enrollment: 62 Canada, Israel, New Zealand
NCT04408625 Active not recruiting Phase2, Phase1
View Report
Frontotemporal Dementia
Sponsor Prevail Therapeutics (Industry)
Compound LY3884963
Therapy Type Gene transfer
Target Gene GRN
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-05-21 Last Modified Date: 2026-05-01 Enrollment: 35 Australia, Belgium, France + 3 more
NCT04704921 Active not recruiting Phase3, Phase2
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
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: 2021-01-08 Last Modified Date: 2026-04-28 Enrollment: 671 United States
NCT06844214 Recruiting Phase2, Phase1
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Myotonic Dystrophy
Sponsor Sanofi (Industry)
Compound SAR446268
Therapy Type Gene transfer
Target Gene DMPK
Vector Type AAV.SAN011
Delivery System Viral transduction
Mechanism of Action Gene inactivation
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2025-02-19 Last Modified Date: 2026-04-28 Enrollment: 32 Argentina, Australia, Canada + 3 more
NCT07180355 Recruiting Phase1
View Report
Friedreich's Ataxia
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-212
Therapy Type Gene transfer
Target Gene FXN
Vector Type AAVhu68
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-08-22 Last Modified Date: 2026-04-28 Enrollment: 10 United States
NCT06910813 Recruiting Phase2, Phase1
View Report
Nephropathic Cystinosis
Sponsor Novartis Pharmaceuticals (Industry)
Compound DFT383
Therapy Type Gene transfer
Target Gene CTNS
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-03-17 Last Modified Date: 2026-04-28 Enrollment: 30 United States
NCT04903288 Active not recruiting Phase2
View Report
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor PTC Therapeutics (Industry)
Compound KEBILIDI, UPSTAZA
Therapy Type Gene transfer
Target Gene DDC
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Priority Review, Accelerated Approval, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-05-21 Last Modified Date: 2026-04-25 Enrollment: 13 Israel, Taiwan, 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: 2026-04-21 Enrollment: 5 United States
NCT06692712 Recruiting Phase3
View Report
Hereditary Spastic Paraplegia Type 50
Sponsor Elpida Therapeutics SPC (Industry)
Compound MELPIDA
Therapy Type Gene transfer
Target Gene AP4M1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-11-14 Last Modified Date: 2026-04-21 Enrollment: 24 Spain, United States
NCT07223944 Recruiting Phase3
View Report
Gaucher Disease Type 1
Sponsor Spur Therapeutics (Industry)
Compound FLT201
Therapy Type Gene transfer
Target Gene GBA1
Vector Type AAVS3
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-10-30 Last Modified Date: 2026-04-21 Enrollment: 45 United States
NCT06432140 Active not recruiting Phase3, Phase2
View Report
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGN-R09b
Therapy Type Gene transfer
Target Gene DDC
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-05-17 Last Modified Date: 2026-04-21 Enrollment: 13 China
NCT04998396 Recruiting Phase2, Phase1
View Report
Canavan Disease
Sponsor Aspa Therapeutics (Industry)
Compound BBP-812
Therapy Type Gene transfer
Target Gene ASPA
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-08-05 Last Modified Date: 2026-04-18 Enrollment: 26 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
Vector Type none
Delivery System Electroporation
Mechanism of Action Mutation correction
First Submitted: 2021-02-17 Last Modified Date: 2026-04-16 Enrollment: 9 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: 2026-04-16 Enrollment: 480 Argentina, Australia, Bulgaria + 11 more
NCT06364774 Recruiting Phase2, Phase1
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Beta-Thalassemia Major
Sponsor Children's Hospital of Philadelphia (Other)
Compound CHOP-ALS20
Therapy Type Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-03-26 Last Modified Date: 2026-04-16 Enrollment: 12 United States
NCT05885412 Recruiting Phase1
View Report
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-A601
Therapy Type Gene transfer
Target Gene PKP2
Vector Type AAVrh.74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2023-05-22 Last Modified Date: 2026-04-16 Enrollment: 9 United States
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: 2026-04-08 Enrollment: 27 Canada, France, Germany + 1 more
NCT04850118 Active not recruiting Phase3, Phase2
View Report
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-501
Therapy Type Gene transfer
Target Gene RPGR
Vector Type AAV2tYF
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2021-04-05 Last Modified Date: 2026-04-07 Enrollment: 85 Australia, Canada, United Kingdom + 1 more
NCT05835895 Active not recruiting Phase1
View Report
Osteoarthritis, Knee
Sponsor Genascence Corporation (Industry)
Compound GNSC-001
Therapy Type 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: 2026-04-07 Enrollment: 67 United States
NCT03566043 Active not recruiting Phase3, Phase2
View Report
Mucopolysaccharidosis Type II (Hunter Syndrome)
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-121
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: 2026-04-07 Enrollment: 48 Brazil, United States
NCT06884865 Active not recruiting Phase2
View Report
Osteoarthritis (OA) of the Knee
Sponsor Pacira Pharmaceuticals, Inc (Industry)
Compound PCRX-201
Therapy Type Gene transfer
Target Gene IL1RA
Vector Type Ad5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-03-13 Last Modified Date: 2026-04-07 Enrollment: 135 United States
NCT07048808 Recruiting Phase2
View Report
Refractory Angina, Coronary Artery Disease
Sponsor XyloCor Therapeutics, Inc. (Industry)
Compound XC001
Therapy Type Gene transfer
Target Gene VEGF
Vector Type Ad5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2025-06-24 Last Modified Date: 2026-04-07 Enrollment: 106 United States
NCT05432310 Recruiting Phase2, Phase1
View Report
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Sponsor University of California, Los Angeles (Other)
Compound Simoladagene autotemcel
Therapy Type Gene transfer
Target Gene ADA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-06-04 Last Modified Date: 2026-04-07 Enrollment: 20 United States
NCT05606614 Recruiting Phase3
View Report
Rett Syndrome
Sponsor Taysha Gene Therapies, Inc. (Industry)
Compound TSHA-102
Therapy Type Gene transfer
Target Gene MiniMECP2
Vector Type scAAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-10-28 Last Modified Date: 2026-04-07 Enrollment: 15 Canada, United States
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: 2026-04-06 Enrollment: 32 Argentina, Brazil, Canada + 7 more
NCT06325709 Recruiting Phase2, Phase1
View Report
X-Linked Chronic Granulomatous Disease
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound Base-edited autologous CD34+ cells
Therapy Type Gene editing
Target Gene CYBB c.676 C>T
Vector Type none
Delivery System Electroporation
Mechanism of Action Mutation correction
First Submitted: 2024-03-21 Last Modified Date: 2026-04-06 Enrollment: 10 United States
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: 2026-04-06 Enrollment: 35 United States
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: 2026-04-06 Enrollment: 9 United States
NCT05956626 Active not recruiting Phase3, Phase2
View Report
Stargardt Disease
Sponsor Ocugen (Industry)
Compound OCU410ST
Therapy Type Gene transfer
Target Gene RORA
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2023-06-30 Last Modified Date: 2026-04-06 Enrollment: 51 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: 2026-04-06 Enrollment: 140 Canada, United States
NCT06063850 Recruiting Phase2, Phase1
View Report
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: 2026-04-06 Enrollment: 12 United States
NCT05725018 Active not recruiting Phase3
View Report
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor Abeona Therapeutics, Inc (Industry)
Compound ZEVASKYN
Therapy Type Gene transfer
Target Gene COL7A1
Vector Type LZRSE
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-01-23 Last Modified Date: 2026-04-06 Enrollment: 12 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: 2026-04-03 Enrollment: 31 United States
NCT06458595 Recruiting Phase2, Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Chengdu Origen Biotechnology Co., Ltd. (Industry)
Compound KH658
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2024-06-09 Last Modified Date: 2026-04-01 Enrollment: 44 China
NCT06662188 Recruiting Phase2, Phase1
View Report
SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Sponsor Jaguar Gene Therapy, LLC (Industry)
Compound JAG201
Therapy Type Gene transfer
Target Gene SHANK3
Vector Type AAV2/9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Rare Pediatric Disease Designation
First Submitted: 2024-10-16 Last Modified Date: 2026-04-01 Enrollment: 6 United States
NCT04411654 Active not recruiting Phase2, Phase1
View Report
Gaucher Disease Type 2
Sponsor Prevail Therapeutics (Industry)
Compound LY3884961
Therapy Type Gene transfer
Target Gene GBA1
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: 2020-05-11 Last Modified Date: 2026-04-01 Enrollment: 7 United Kingdom, United States
NCT05203679 Active not recruiting Phase3, Phase2
View Report
Hemophilia B
Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound BBM-H901
Therapy Type Gene transfer
Target Gene F9
Vector Type AAV843
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-12-29 Last Modified Date: 2026-04-01 Enrollment: 32 China
NCT04611503 Active not recruiting Phase2, Phase1
View Report
Retinitis Pigmentosa
Sponsor STZ eyetrial (Other)
Compound RAAV.hPDE6A
Therapy Type 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: 2026-04-01 Enrollment: 9 Germany
NCT06185673 Recruiting Phase2, Phase1
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Oculopharyngeal Muscular Dystrophy
Sponsor Benitec Biopharma, Inc. (Industry)
Compound BB-301
Therapy Type Gene transfer
Target Gene PABPN1
Vector Type AAV9-PL
Delivery System Viral transduction
Mechanism of Action Functional gene replacement, Gene inactivation
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2023-12-15 Last Modified Date: 2026-04-01 Enrollment: 30 United States
NCT06292650 Recruiting Early phase1
View Report
Retinitis Pigmentosa
Sponsor Zhongmou Therapeutics (Industry)
Compound ZM-02
Therapy Type Gene transfer
Target Gene Ch2.0
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2024-02-27 Last Modified Date: 2026-03-31 Enrollment: 12 China
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: 2026-03-31 Enrollment: 39 China
NCT05665166 Active not recruiting Phase2, Phase1
View Report
Mucopolysaccharidosis II
Sponsor University of Manchester (Other)
Compound AVR-RD-05
Therapy Type Gene transfer
Target Gene IDS
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-12-07 Last Modified Date: 2026-03-27 Enrollment: 5 United Kingdom
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: 2026-03-27 Enrollment: 7 France
NCT07185256 Recruiting Phase2, Phase1
View Report
ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
Sponsor Opus Genetics, Inc (Industry)
Compound OPGx-BEST1
Therapy Type Gene transfer
Target Gene BEST1
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-07-17 Last Modified Date: 2026-03-24 Enrollment: 10 United States
NCT05477563 Recruiting Phase3
View Report
Beta-Thalassemia, Sickle Cell Disease
Sponsor Vertex Pharmaceuticals Incorporated (Industry)
Compound CASGEVY
Therapy Type Gene editing
Target Gene BCL11A
Vector Type RNP
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-07-26 Last Modified Date: 2026-03-23 Enrollment: 26 Germany, Italy, Saudi Arabia + 1 more
NCT05836259 Recruiting Phase2, Phase1
View Report
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: 2026-03-20 Enrollment: 30 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: 2026-03-18 Enrollment: 4 United States
NCT05898620 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: 2026-03-18 Enrollment: 33 Australia, United Kingdom, United States