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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

NCT05456880
Protocol null LinkName:Clinical Trial Protocol;LinkUrl:https://w... curator
Clinical publications null LinkName:Base Editing of HBG1 and HBG2 Promoters f... curator
Recent updates Beam expects to dose 30 patients by mid-2025, data... clinical dose manufacturing is complete, BLA submi... curator
Clinical publications null LinkName:(Poster) Robust HbF induction and improve... curator
News and press releases null LinkName:Beam Therapeutics Reports Fourth Quarter ... curator
Eligibility criteria null Key inclusion criteria include: 1. age ≥12 years ... api
2026-04-08
Sickle Cell Disease
Beam Therapeutics Inc. Active not recruiting
Clinical dose manufacturing is complete, BLA submission expected as early as year-end 2026
NCT05073133
Related nctid LinkName:Phase 4: NCT05861986 (study subjects were... LinkName:Phase 4: NCT05861986 (study subjects were... curator
Related nctid LinkName:Phase 4: NCT05861999 (study subjects were... LinkName:Phase 4: NCT05861999 (study subjects were... curator
Related nctid null LinkName:Phase 4: NCT04488133 (study subjects were... curator
Related nctid null LinkName:Phase 3: NCT03505099;LinkUrl:https://www.... curator
Related nctid LinkName:Phase 3: NCT03505099;LinkUrl:https://www.... null curator
Related nctid LinkName:Phase 4: NCT05089656;LinkUrl:https://www.... null curator
Related nctid LinkName:Long Term Follow-Up: NCT04174157;LinkUrl:... LinkName:Patient Registry: NCT04174157;LinkUrl:htt... curator
Related nctid LinkName:Phase 1: NCT03381729;LinkUrl:https://www.... null curator
Related nctid null LinkName:Phase 3: NCT03461289;LinkUrl:https://clin... curator
Related nctid null LinkName:Long Term Follow-Up: NCT03421977;LinkUrl:... curator
Related nctid null LinkName:Phase 3: NCT03306277;LinkUrl:https://clin... curator
Related nctid null LinkName:Phase 3: NCT03837184;LinkUrl:https://clin... curator
Related nctid null LinkName:Long Term Follow-Up: NCT04042025;LinkUrl:... curator
Eligibility criteria null Inclusion criteria: 1. written informed consent/a... api
2026-04-08
Spinal Muscular Atrophy
Novartis Pharmaceuticals Completed
FDA approved 5/24/19, price/treatment $2.1M
NCT05903794
Last update post date 2025-04-17 2026-04-08 api
Estimated completion date 2026-02-28 2026-02-16 api
Overall status Active not recruiting Completed api
Enrollment type Estimated Actual api
Eligibility criteria null Inclusion criteria: 1. male or female, Age ≥ 50 y... api
2026-04-08
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Exegenesis Bio Completed
FDA cleared IND 1/18/23
NCT03199469
Last update post date 2026-01-14 2026-04-08 api
Preclinical publications null LinkName:AAV8 gene therapy and dietary insults tog... curator
Development status Active Inactive curator
Recent updates On clinical hold since 2021 Astellas has restarted their XLMTM program by crea... curator
Eligibility criteria null Inclusion criteria: * subject has a diagnosis of ... api
2026-04-08
X-Linked Myotubular Myopathy
Astellas Gene Therapies Active not recruiting
Astellas has restarted their XLMTM program by creating a new drug product ASP2957, which is based on this prior study, with substantial modifications ...
NCT05386680
Related nctid null LinkName:Phase 4: NCT07448610;LinkUrl:https://clin... curator
Related nctid LinkName:Phase 3: NCT03505099;LinkUrl:https://clin... null curator
Eligibility criteria null Inclusion criteria * sma diagnosis * aged 2 to \<... api
Record status Draft Active curator
Recent updates Same drug product as Zolgensma (dosing and route o... curator
Protocol null LinkName:FDA Approval Documents;LinkUrl:https://ww... curator
Protocol null LinkName:Clinical Trial Protocol;LinkUrl:https://c... curator
Protocol null LinkName:Statistical Analysis Plan;LinkUrl:https:/... curator
Clinical publications null LinkName:Intrathecal onasemnogene abeparvovec for ... curator
Clinical publications null LinkName:Intrathecal onasemnogene abeparvovec in t... curator
Clinical publications null LinkName:Intrathecal Onasemnogene Abeparvovec for ... curator
Preclinical publications null LinkName:Single-Dose Intrathecal Dorsal Root Gangl... curator
Indication doid null DOID:13137 curator
Development status null Approved curator
Compound name null ITVISMA curator
Dose 3 null Approved Dose: 1.2E14 vg curator
Dose 2 null Phase 3: 1.2E14 vg curator
Dose 1 null Phase 1/2: 6.0E13 vg, 1.2E14 vg, 2.4E14 vg curator
Vector type null AAV9 curator
Delivery system null viral transduction curator
Drug product type null viral vector curator
Route of administration null intrathecal curator
Mechanism of action null functional gene replacement curator
Therapy route null in-vivo curator
Therapy type null gene transfer curator
Target gene null SMN1 curator
Alias type null proper name, proprietary names curator
Alias value null onasemnogene abeparvovec, OAV101, AVXS-101 curator
Fda designation Breakthrough Therapy, Orphan Drug Designation curator
Related nctid null LinkName:Patient Registry: NCT04174157;LinkUrl:htt... curator
Related nctid null LinkName:Long Term Follow Up: NCT05335876;LinkUrl:... curator
Related nctid null LinkName:Phase 3: NCT05089656;LinkUrl:https://clin... curator
Related nctid null LinkName:Phase 1: NCT03381729;LinkUrl:https://clin... curator
2026-04-07
Spinal Muscular Atrophy
Novartis Pharmaceuticals Completed
Same drug product as Zolgensma (dosing and route of administration differs), FDA approved 11/24/25 for list price $2.59M
50 more update(s) recently — View All

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Showing 1-100 of 139 results in ClinicalTrials
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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: 2026-04-07 Enrollment: 9 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, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2022-10-28 Last Modified Date: 2026-04-07 Enrollment: 15 Canada, 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
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
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 Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2022-06-10 Last Modified Date: 2026-04-06 Enrollment: 47 Australia, United Kingdom, United States
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: 2026-04-06 Enrollment: 561 Austria, Belgium, Bulgaria + 14 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: 2026-04-06 Enrollment: 27 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
NCT06856577 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 Fast Track, Regenerative Medicine Advanced Therapy
First Submitted: 2025-02-26 Last Modified Date: 2026-04-06 Enrollment: 284 United States
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 Lipid encapsulation
Mechanism of Action Gene inactivation
FDA Designations Fast Track
First Submitted: 2024-10-24 Last Modified Date: 2026-04-06 Enrollment: 45 Hong Kong, Moldova, New Zealand + 1 more
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
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 Lipid encapsulation
Mechanism of Action Gene inactivation
FDA Designations Fast Track
First Submitted: 2023-12-01 Last Modified Date: 2026-04-01 Enrollment: 85 Australia, Canada, Israel + 3 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 Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2016-03-17 Last Modified Date: 2026-04-01 Enrollment: 36 Australia, Spain, 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 Fast Track, Regenerative Medicine Advanced Therapy
First Submitted: 2023-12-19 Last Modified Date: 2026-04-01 Enrollment: 8 Australia, Spain, United Kingdom + 1 more
NCT06185673 Recruiting Phase2, Phase1
View Report
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
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: 2026-03-31 Enrollment: 12 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
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: 2026-03-30 Enrollment: 11 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 Lipid encapsulation
Mechanism of Action Gene inactivation
FDA Designations Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-11-08 Last Modified Date: 2026-03-27 Enrollment: 1200 Argentina, Australia, Austria + 23 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 Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2011-02-26 Last Modified Date: 2026-03-27 Enrollment: 40 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: 2026-03-27 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
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: 2026-03-23 Enrollment: 12 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 Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2022-07-26 Last Modified Date: 2026-03-23 Enrollment: 26 Germany, Italy, Saudi Arabia + 1 more
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 Lipid encapsulation
Mechanism of Action Mutation correction
FDA Designations Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2024-04-22 Last Modified Date: 2026-03-20 Enrollment: 106 Australia, Ireland, Netherlands + 3 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
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 Breakthrough Therapy, Support for Clinical Trials Advancing Rare Disease Therapeutics, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-06-01 Last Modified Date: 2026-03-18 Enrollment: 33 Australia, United Kingdom, United States
NCT06391736 Recruiting Phase2, Phase1
View Report
Pompe Disease (Late-onset)
Sponsor GeneCradle Inc (Industry)
Compound GC301
Therapy Type Gene transfer
Target Gene GAA
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-04-25 Last Modified Date: 2026-03-17 Enrollment: 33 China
NCT06511349 Recruiting Early phase1
View Report
Type 1 Primary Hyperoxaluria
Sponsor RenJi Hospital (Other)
Compound YOLT-203
Therapy Type Gene editing
Target Gene HAO1
Vector Type LNP
Delivery System Lipid encapsulation
Mechanism of Action Gene inactivation
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-07-04 Last Modified Date: 2026-03-17 Enrollment: 21 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 Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2021-08-05 Last Modified Date: 2026-03-17 Enrollment: 26 United States
NCT03466463 Recruiting Na
View Report
Crigler-Najjar Syndrome
Sponsor Genethon (Other)
Compound GNT0003
Therapy Type Gene transfer
Target Gene UGT1A1
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-02-01 Last Modified Date: 2026-03-17 Enrollment: 17 France, Italy, Netherlands
NCT05821959 Recruiting Phase2, Phase1
View Report
Sensorineural Hearing Loss, Bilateral
Sponsor Akouos, Inc. (Industry)
Compound AK-OTOF
Therapy Type Gene transfer
Target Gene OTOF
Vector Type dual Anc80L65
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-02-08 Last Modified Date: 2026-03-17 Enrollment: 22 Canada, Taiwan, United Kingdom + 1 more
NCT06615206 Recruiting Na
View Report
MECP2 Duplication Syndrome
Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)
Compound HG204
Therapy Type Gene editing
Target Gene MECP2
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Gene inactivation
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-09-04 Last Modified Date: 2026-03-17 Enrollment: 6 China
NCT06831825 Recruiting Phase1
View Report
Heart Failure With Reduced Ejection Fraction
Sponsor YAP Therapeutics, Inc. (Industry)
Compound YAP101
Therapy Type Gene transfer
Target Gene SAV1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2025-01-23 Last Modified Date: 2026-03-17 Enrollment: 24 United States
NCT06856759 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: 2026-03-17 Enrollment: 8 China
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
First Submitted: 2025-07-07 Last Modified Date: 2026-03-17 Enrollment: 480 Argentina, Bulgaria, Germany + 9 more
NCT06722170 Recruiting Na
View Report
DFNB9, Congenital Hearing Loss
Sponsor Yilai Shu (Other)
Compound EH002
Therapy Type Gene transfer
Target Gene OTOF
Vector Type dual AAV1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-12-05 Last Modified Date: 2026-03-17 Enrollment: 24 China
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 Lipid encapsulation
Mechanism of Action Gene inactivation
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-02-17 Last Modified Date: 2026-03-17 Enrollment: 23 France, Germany, Tunisia + 2 more
NCT06749639 Recruiting Early phase1
View Report
Inherited Retinal Diseases, RDH12 Retinopathy
Sponsor Peking Union Medical College Hospital (Other)
Compound PUMCH-E101
Therapy Type Gene transfer
Target Gene RDH12
Vector Type undisclosed
Delivery System Undisclosed
Mechanism of Action Functional gene replacement
First Submitted: 2024-12-19 Last Modified Date: 2026-03-17 Enrollment: 10 China
NCT06860672 Recruiting Early phase1
View Report
Snijders Blok-Campeau syndrome
Sponsor Yongguo Yu (Other)
Compound Dual Vector AAV-CHD3-R1025W Base Editor
Therapy Type Gene editing
Target Gene CDH3/R1025W
Vector Type dual AAV
Delivery System Viral transduction
Mechanism of Action Mutation correction
First Submitted: 2025-02-24 Last Modified Date: 2026-03-17 Enrollment: 1 China
NCT05641610 Recruiting Phase2, Phase1
View Report
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: 2026-03-17 Enrollment: 21 China
NCT05761899 Recruiting Phase2, Phase1
View Report
Hereditary Pulmonary Alveolar Proteinosis
Sponsor Children's Hospital Medical Center, Cincinnati (Other)
Compound Gene-Corrected Macrophages
Therapy Type Gene transfer
Target Gene CSF2RA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-02-28 Last Modified Date: 2026-03-17 Enrollment: 3 United States
NCT05791864 Recruiting Phase2, Phase1
View Report
Neuronal Ceroid Lipofuscinosis Type 2
Sponsor Tern Therapeutics, LLC (Industry)
Compound TTX-381
Therapy Type Gene transfer
Target Gene TPP1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-03-17 Last Modified Date: 2026-03-17 Enrollment: 16 Germany, United Kingdom
NCT05885412 Recruiting Phase1
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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 Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-05-22 Last Modified Date: 2026-03-17 Enrollment: 9 United States
NCT06465550 Recruiting Phase1
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Beta-Thalassemia Major
Sponsor Shanghai BDgene Co., Ltd. (Industry)
Compound BD211
Therapy Type Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-06-12 Last Modified Date: 2026-03-17 Enrollment: 9 China
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: 2026-03-17 Enrollment: 62 Canada, New Zealand
NCT06049082 Recruiting Phase1
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Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Krystal Biotech, Inc. (Industry)
Compound KB-408
Therapy Type Gene transfer
Target Gene SERPINA1
Vector Type HSV-1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2023-09-15 Last Modified Date: 2026-03-17 Enrollment: 15 United States
NCT07160634 Recruiting Phase3
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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: 2026-03-17 Enrollment: 80 Australia, Canada
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: 2026-03-17 Enrollment: 65 Canada, 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: 2026-03-17 Enrollment: 10 United States
NCT04703842 Recruiting Phase2, Phase1
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Congestive Heart Failure, Heart Failure With Reduced Ejection Fraction (HFrEF)
Sponsor Sardocor Corp. (Industry)
Compound SRD-001
Therapy Type Gene transfer
Target Gene SERCA2a
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2021-01-07 Last Modified Date: 2026-03-17 Enrollment: 57 United States
NCT02852213 Recruiting Phase1
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Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor Krzysztof Bankiewicz (Other)
Compound AAV2-hAADC
Therapy Type Gene transfer
Target Gene DDC
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-07-20 Last Modified Date: 2026-03-17 Enrollment: 42 United States
NCT03727555 Recruiting Na
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X-linked Adrenoleukodystrophy
Sponsor Shenzhen Geno-Immune Medical Institute (Other)
Compound (LV) TYF-ABCD1
Therapy Type Gene transfer
Target Gene ABCD1
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-09-25 Last Modified Date: 2026-03-17 Enrollment: 30 China
NCT06650319 Recruiting Early phase1
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Wilson Disease
Sponsor Chaohui Yu (Other)
Compound LY-M003
Therapy Type Gene transfer
Target Gene ATP7B
Vector Type AAV8
Delivery System Viral transdution
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-10-15 Last Modified Date: 2026-03-17 Enrollment: 18 China
NCT04774536 Recruiting Phase2, Phase1
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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: 2026-03-17 Enrollment: 9 United States
NCT05504837 Recruiting Phase1
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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: 2026-03-17 Enrollment: 12 United States
NCT05986864 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Skyline Therapeutics (US) Inc. (Industry)
Compound SKG0106
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: 2023-08-03 Last Modified Date: 2026-03-17 Enrollment: 68 China, United States
NCT06207552 Recruiting Early phase1
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Fabry Disease
Sponsor Children's Hospital of Fudan University (Other)
Compound BBM-F101
Therapy Type Gene transfer
Target Gene GLA
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-12-18 Last Modified Date: 2026-03-17 Enrollment: 6 China
NCT04676048 Recruiting Phase2, Phase1
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Hemophilia A
Sponsor ASC Therapeutics (Industry)
Compound ASC618
Therapy Type Gene transfer
Target Gene F8
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-12-15 Last Modified Date: 2026-03-17 Enrollment: 12 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: 2026-03-17 Enrollment: 32 Argentina, Australia, Canada + 3 more
NCT06141460 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Shanghai Refreshgene Technology Co., Ltd. (Industry)
Compound RRG001
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: 2026-03-17 Enrollment: 48 China
NCT05248230 Recruiting Phase2
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Cystic Fibrosis
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-710
Therapy Type Gene transfer
Target Gene CFTR
Vector Type AAV A101
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-02-10 Last Modified Date: 2026-03-17 Enrollment: 30 United States
NCT03952637 Recruiting Phase2, Phase1
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GM1 Gangliosidosis
Sponsor National Human Genome Research Institute (NHGRI) (NIH)
Compound AXO-AAV-GM1
Therapy Type Gene transfer
Target Gene GLB1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-05-15 Last Modified Date: 2026-03-17 Enrollment: 54 United States
NCT06270316 Recruiting Phase2, Phase1
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Fabry Disease
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-191
Therapy Type Gene transfer
Target Gene GLA
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2023-12-19 Last Modified Date: 2026-03-17 Enrollment: 12 United States
NCT04747431 Recruiting Phase2, Phase1
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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: 2026-03-17 Enrollment: 30 Australia, Brazil, Canada + 2 more
NCT06451770 Recruiting Phase1
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Hypercholesterolemia
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-201
Therapy Type Gene editing
Target Gene ANGPTL3
Vector Type LDLR + GalNAc
Delivery System Lipid encapsulation
Mechanism of Action Gene inactivation
First Submitted: 2024-06-04 Last Modified Date: 2026-03-17 Enrollment: 36 Australia, Canada, South Africa + 1 more
NCT06743646 Recruiting Phase3
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Bietti Crystalline Dystrophy
Sponsor Chigenovo Co., Ltd (Network)
Compound ZVS101e
Therapy Type Gene transfer
Target Gene CYP4V2
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-12-17 Last Modified Date: 2026-03-17 Enrollment: 62 China
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: 2026-03-17 Enrollment: 25 United States
NCT06224660 Recruiting Phase1
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DMD-Associated Dilated Cardiomyopathy
Sponsor Sardocor Corp. (Industry)
Compound SRD-001
Therapy Type Gene transfer
Target Gene SERCA2a
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2024-01-11 Last Modified Date: 2026-03-17 Enrollment: 12 United States
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: 2026-03-17 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 more
NCT05092685 Recruiting Phase2, Phase1
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Ornithine Transcarbamylase (OTC) Deficiency
Sponsor University College, London (Other)
Compound BGT-OTCD
Therapy Type Gene transfer
Target Gene OTC
Vector Type AAV-LK03
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2021-09-28 Last Modified Date: 2026-03-17 Enrollment: 12 United Kingdom
NCT06526923 Recruiting Phase2, Phase1
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Cystic Fibrosis
Sponsor Spirovant Sciences, Inc. (Industry)
Compound SP-101
Therapy Type Gene transfer
Target Gene CFTRΔR
Vector Type AAV2.5T
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-07-18 Last Modified Date: 2026-03-17 Enrollment: 15 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 DB-OTO
Therapy Type Gene transfer
Target Gene OTOF
Vector Type dual AAV1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-03-15 Last Modified Date: 2026-03-17 Enrollment: 30 Germany, Spain, United Kingdom + 1 more
NCT05672121 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Chengdu Origen Biotechnology Co., Ltd. (Industry)
Compound KH631
Therapy Type Gene transfer
Target Gene VEGFR1/R2 fusion protein
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2022-12-21 Last Modified Date: 2026-03-17 Enrollment: 42 China
NCT04704921 Recruiting Phase3, Phase2
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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: 2026-03-17 Enrollment: 630 United States
NCT06641154 Recruiting Phase2, Phase1
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Crigler-Najjar Syndrome Type I
Sponsor Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare (Other gov)
Compound GT-UGT1A1-AAV8-02
Therapy Type Gene transfer
Target Gene UGT1A1
Vector Type rAAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-10-11 Last Modified Date: 2026-03-17 Enrollment: 5 Russia
NCT06217861 Recruiting Phase1
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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: 2026-03-17 Enrollment: 12 China
NCT05762510 Recruiting Early phase1
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Beta-Thalassemia Major
Sponsor First Affiliated Hospital of Guangxi Medical University (Other)
Compound GMCN-508B
Therapy Type Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-01-06 Last Modified Date: 2026-03-17 Enrollment: 5 China
NCT06332807 Recruiting Phase2, Phase1
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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: 2026-03-17 Enrollment: 12 United States
NCT06111638 Recruiting Phase3, Phase2
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Hemophilia A
Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound BBM-H803
Therapy Type Gene transfer
Target Gene F8
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2023-10-27 Last Modified Date: 2026-03-17 Enrollment: 55 China
NCT06539208 Recruiting Phase2, Phase1
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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 Lipid encapsulation
Mechanism of Action Gene inactivation
First Submitted: 2024-08-01 Last Modified Date: 2026-03-17 Enrollment: 31 China
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: 2026-03-17 Enrollment: 53 United States
NCT03311503 Recruiting Phase2, Phase1
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X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor David Williams (Other)
Compound G2SCID lentiviral vector transduced CD34+ cells
Therapy Type Gene transfer
Target Gene IL2RG
Vector Type HIV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-10-12 Last Modified Date: 2026-03-17 Enrollment: 12 United States
NCT06064890 Recruiting Phase2, Phase1
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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: 2026-03-17 Enrollment: 9 Belgium, Canada, Italy + 6 more
NCT06818838 Recruiting Phase2, Phase1
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Gaucher Disease Type 1
Sponsor Lingyi Biotech Co., Ltd. (Industry)
Compound LY-M001
Therapy Type Gene transfer
Target Gene GBA1
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-01-24 Last Modified Date: 2026-03-17 Enrollment: 12 China
NCT06291935 Recruiting Phase1
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Retinitis Pigmentosa
Sponsor VeonGen Therapeutics GmbH (Industry)
Compound VG901
Therapy Type Gene transfer
Target Gene CNGA1
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-02-01 Last Modified Date: 2026-03-17 Enrollment: 6 Germany
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: 2026-03-17 Enrollment: 10 China
NCT06370351 Recruiting Phase2, Phase1
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OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders
Sponsor Sensorion (Industry)
Compound SENS-501
Therapy Type Gene transfer
Target Gene OTOF
Vector Type dual AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-04-09 Last Modified Date: 2026-03-17 Enrollment: 12 Australia, France
NCT06492850 Recruiting Phase2, Phase1
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X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Frontera Therapeutics (Industry)
Compound FT-002
Therapy Type Gene transfer
Target Gene RPGRORF15
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2024-07-01 Last Modified Date: 2026-03-17 Enrollment: 32 China
NCT06238908 Recruiting Early phase1
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Hemophilia A
Sponsor Institute of Hematology & Blood Diseases Hospital, China (Other)
Compound NGGT003
Therapy Type Gene transfer
Target Gene F8
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-01-26 Last Modified Date: 2026-03-17 Enrollment: 6 China
NCT06467344 Recruiting Phase2, Phase1
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Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration
Sponsor Ascidian Therapeutics, Inc (Industry)
Compound ACDN-01
Therapy Type Gene editing
Target Gene ABCA4 pre-mRNA
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Exon skipping/splice editor
FDA Designations Fast Track, Rare Pediatric Disease Designation
First Submitted: 2024-05-31 Last Modified Date: 2026-03-17 Enrollment: 15 United States
NCT06735755 Recruiting Phase2, Phase1
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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 Lipid encapsulation
Mechanism of Action Mutation correction
First Submitted: 2024-12-06 Last Modified Date: 2026-03-17 Enrollment: 36 United States
NCT07218887 Recruiting Phase2, Phase1
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BAG3 Mutation Associated Dilated Cardiomyopathy
Sponsor Alexion Pharmaceuticals, Inc. (Industry)
Compound ALXN2350
Therapy Type Gene transfer
Target Gene BAG3
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-10-17 Last Modified Date: 2026-03-17 Enrollment: 6 Spain, United States
NCT06492876 Recruiting Phase2, Phase1
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Diabetic Macular Edema
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: 2026-03-17 Enrollment: 78 China
NCT06461702 Recruiting Early phase1
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Familial Hypercholesterolemia
Sponsor The First Affiliated Hospital of Bengbu Medical University (Other)
Compound YOLT-101
Therapy Type Gene editing
Target Gene PCSK9/LDLR
Vector Type none
Delivery System LNP
Mechanism of Action Gene inactivation
First Submitted: 2024-04-16 Last Modified Date: 2026-03-17 Enrollment: 13 China
NCT07058662 Recruiting Phase2, Phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor Belief BioMed (Beijing) Co., Ltd (Industry)
Compound BBM-D101
Therapy Type Gene transfer
Target Gene Undisclosed
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Undisclosed
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-07-01 Last Modified Date: 2026-03-17 Enrollment: 9 China
NCT05926765 Recruiting Phase2
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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 Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-06-22 Last Modified Date: 2026-03-17 Enrollment: 276 Canada, United Kingdom, United States
NCT06519552 Recruiting Phase1
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Mucopolysaccharidosis Type I (Hurler Syndrome)
Sponsor West China Hospital (Other)
Compound JWK-008
Therapy Type Gene transfer
Target Gene IDUA
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-06-23 Last Modified Date: 2026-03-17 Enrollment: 6 China