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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

NCT03533673
Last update post date 2025-12-19 2026-04-13 api
Estimated completion date 2026-03 2026-03-12 api
Overall status Active not recruiting Completed api
Eligibility criteria null Inclusion criteria: * diagnosis of pompe disease ... api
Development status Active Inactive curator
Recent updates First patient dosed 1/22/19, most recent data pres... trial will be completed with the patients already ... curator
News and press releases null LinkName:AskBio Announces FDA Acceptance of Invest... curator
2026-04-14
Pompe Disease
AskBio Inc Completed
Trial will be completed with the patients already enrolled, AskBio is shifting focus to new product AB-1009 for the same indication
NCT07007065
Last update post date 2026-04-06 2026-04-14 api
Number of locations 96 100 api
Locations Puerto rico, Hungary, United states, United kingdo... Puerto rico, Hungary, United states, Czechia, Unit... api
Eligibility criteria null Inclusion criteria: * pseudophakic (at least 12 w... api
2026-04-14
Neovascular Age-related Macular Degeneration
AbbVie Recruiting
Another Phase 3 study (NCT05407636) is active, enrollment is completed
NCT07287397
Last update post date 2026-03-17 2026-04-13 api
Eligibility criteria null Key inclusion criteria: 1. capable of, And willin... api
Record status Draft Active curator
Preclinical publications null LinkName:(Poster) AAV-mediated intrabody therapy t... curator
Preclinical publications null LinkName:(Poster) VecTabs® and advanced human in-v... curator
Preclinical publications null LinkName:(Poster) AAV-mediated intrabody therapy t... curator
Preclinical publications null LinkName:(Poster) Non-clinical development and Pha... curator
Indication doid null DOID:332 curator
Development status null Active curator
Compound name null VTx-002 curator
Recent updates null FDA cleared IND 12/25 curator
Dose 2 null undisclosed high dose curator
Dose 1 null undisclosed low dose curator
Vector type null AAV5.2 curator
Delivery system null viral transduction curator
Drug product type null viral vector curator
Route of administration null intracisternal curator
Mechanism of action null genetic delivery of therapeutic protein curator
Therapy route null in-vivo curator
Therapy type null gene transfer curator
Target gene null antibody to TDP-43 curator
Fda designation Fast Track curator
News and press releases null LinkName:VectorY Therapeutics Receives FDA Clearan... curator
News and press releases null LinkName:VectorY Therapeutics Receives FDA Fast Tr... curator
2026-04-14
Amyotrophic Lateral Sclerosis
Vector Y Therapeutics Recruiting
FDA cleared IND 12/25
NCT04046224
With has results False True api
Last update post date 2025-05-15 2026-04-14 api
Eligibility criteria Inclusion criteria: * ≥ 18 years of age * documen... Inclusion criteria: * ≥ 18 years of age * documen... api
Enrollment count 33 36 api
Description This is the first in human treatment with st-920, ... This is the first in human treatment with st-920, ... api
Related nctid null LinkName:Long Term Follow-Up: NCT05039866;LinkUrl:... curator
Recent updates Recent Type B meeting with FDA allowed ongoing Pha... Rolling BLA submission initiated December 2025, th... curator
2026-04-14
Fabry Disease
Sangamo Therapeutics Completed
Rolling BLA submission initiated December 2025, the company expects submission to be completed 2Q2026
NCT06064890
Last update post date 2026-02-27 2026-04-13 curator
Enrollment count 9 18 curator
Eligibility criteria null Inclusion criteria: * male or female, 30 to 75 ye... api
2026-04-14
Frontotemporal Dementia, FTD, FTD-GRN
AviadoBio Ltd Recruiting
Second dose cohort is complete, company intends to initiate a third dose cohort in Q3 2025, early biomarker data expected 2026
17 more update(s) recently — View All

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Showing 1-100 of 236 results in ClinicalTrials
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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: 2026-04-14 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 more
NCT04046224 Completed Phase2, Phase1
View Report
Fabry Disease
Sponsor Sangamo Therapeutics (Industry)
Compound ST-920
Therapy Type Gene transfer
Target Gene GLA
Vector Type AAV2/6
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2019-08-01 Last Modified Date: 2026-04-14 Enrollment: 36 Australia, Canada, Germany + 4 more
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: 2026-04-14 Enrollment: 18 Belgium, Canada, Italy + 6 more
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: 2026-04-10 Enrollment: 12 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 Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2017-06-21 Last Modified Date: 2026-04-08 Enrollment: 27 Canada, France, Germany + 1 more
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 Regenerative Medicine Advanced Therapy
First Submitted: 2022-06-23 Last Modified Date: 2026-04-08 Enrollment: 15 United States
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 Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2021-04-05 Last Modified Date: 2026-04-07 Enrollment: 85 Australia, Canada, United Kingdom + 1 more
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
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 Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2018-05-01 Last Modified Date: 2026-04-07 Enrollment: 48 Brazil, 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
NCT07226206 Not yet 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: 2026-04-07 Enrollment: 5 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
First Submitted: 2025-03-13 Last Modified Date: 2026-04-07 Enrollment: 135 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
NCT03580083 Suspended Phase2, Phase1
View Report
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-111
Therapy Type Gene transfer
Target Gene IDUA
Vector Type AAV2/9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-06-18 Last Modified Date: 2026-04-06 Enrollment: 21 Brazil, 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
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
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
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
NCT04145037 Terminated Phase2, Phase1
View Report
Gaucher Disease Type 1
Sponsor AVROBIO (Industry)
Compound AVR-RD-02
Therapy Type Gene transfer
Target Gene GBA1
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-08-27 Last Modified Date: 2026-04-06 Enrollment: 8 Canada, United States
NCT02651675 Terminated Phase2, Phase1
View Report
Homozygous Familial Hypercholesterolemia (HoFH)
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-501
Therapy Type Gene transfer
Target Gene LDLR
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2016-01-04 Last Modified Date: 2026-04-06 Enrollment: 9 Canada, Italy, Netherlands + 1 more
NCT06559176 Enrolling by invitation Phase2, Phase1
View Report
Chronic Granulomatous Disease
Sponsor Prime Medicine, Inc. (Industry)
Compound PM359
Therapy Type Gene editing
Target Gene NCF1 (c.75_76delGT)
Delivery System Electroporation
Mechanism of Action Mutation correction
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-07-11 Last Modified Date: 2026-04-06 Enrollment: 12 Canada, United Kingdom, 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
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
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
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
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
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
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
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
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
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
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
NCT05454566 Not yet recruiting Phase2, Phase1
View Report
Osteoarthritis, Knee
Sponsor ICM Co. Ltd. (Industry)
Compound ICM-203
Therapy Type Gene transfer
Target Gene NKX3-2
Vector Type AAV5.2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2022-07-04 Last Modified Date: 2026-03-27 Enrollment: 18
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
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
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
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
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
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
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: 2026-03-20 Enrollment: 11 Bulgaria, France, Germany + 3 more
NCT05176093 Completed Phase2
View Report
Amyotrophic Lateral Sclerosis (ALS)
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapy Type Gene transfer
Target Gene HGF
Delivery System None (naked plasmid)
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2021-11-05 Last Modified Date: 2026-03-19 Enrollment: 8 South Korea, United States
NCT04884815 Active not recruiting Phase2, Phase1
View Report
Wilson Disease
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound UX701
Therapy Type Gene transfer
Target Gene ATP7B
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2021-05-07 Last Modified Date: 2026-03-18 Enrollment: 82 Canada, Portugal, Spain + 2 more
NCT06492876 Recruiting Phase2, Phase1
View Report
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
NCT04519749 Active not recruiting Phase2, Phase1
View Report
Fabry Disease
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-310
Therapy Type Gene transfer
Target Gene GLA
Vector Type AAV C102
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-08-14 Last Modified Date: 2026-03-17 Enrollment: 18 United States
NCT04537377 Active not recruiting Phase2, Phase1
View Report
Wilson Disease
Sponsor Vivet Therapeutics SAS (Industry)
Compound VTX-801
Therapy Type Gene transfer
Target Gene ATP7B-minigene
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-08-19 Last Modified Date: 2026-03-17 Enrollment: 4 Denmark, Germany, United Kingdom + 1 more
NCT05092685 Recruiting Phase2, Phase1
View Report
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
NCT03432364 Completed Phase2, Phase1
View Report
Beta-Thalassemia Major
Sponsor Sangamo Therapeutics (Industry)
Compound ST-400
Therapy Type Gene editing
Target Gene BCL11A
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2018-02-01 Last Modified Date: 2026-03-17 Enrollment: 5 United States
NCT02618915 Terminated Phase2, Phase1
View Report
Hemophilia B
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound DTX101
Therapy Type Gene transfer
Target Gene F9
Vector Type AAVrh10
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2015-11-23 Last Modified Date: 2026-03-17 Enrollment: 6 Bulgaria, United Kingdom, United States
NCT04186650 Active not recruiting Phase2, Phase1
View Report
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor Institut National de la Santé Et de la Recherche Médicale, France (Other gov)
Compound EF1a-COL7A1-SIN retroviral vector transduced autologous skin
Therapy Type Gene transfer
Target Gene COL7A1
Vector Type RV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2019-11-25 Last Modified Date: 2026-03-17 Enrollment: 3 France
NCT05164471 Terminated Phase2, Phase1
View Report
Hemophilia B
Sponsor Spur Therapeutics (Industry)
Compound FLT180a
Therapy Type Gene transfer
Target Gene F9
Vector Type AAVS3
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-11-22 Last Modified Date: 2026-03-17 Enrollment: 6 United Kingdom, United States
NCT02559830 Unknown Phase2, Phase1
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Metachromatic Leukodystrophy, Adrenoleukodystrophy
Sponsor Shenzhen Second People's Hospital (Other)
Compound CD34+ hematopoietic stem cells transduced with ABCD1 or ARSA
Therapy Type Gene transfer
Target Gene ABCD1 or ARSA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2015-08-12 Last Modified Date: 2026-03-17 Enrollment: 50 China
NCT06611436 Recruiting Phase2, Phase1
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Hemophilia B
Sponsor Be Biopharma (Industry)
Compound BE-101
Therapy Type Gene editing
Target Gene F9
Vector Type AAV6
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2024-09-18 Last Modified Date: 2026-03-17 Enrollment: 24 United States
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
NCT06819514 Not yet recruiting Phase2, Phase1
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Fabry Disease
Sponsor Guangzhou Jiayin Biotech Ltd (Industry)
Compound EXG110
Therapy Type Gene transfer
Target Gene GLA
Vector Type proprietary AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2025-01-22 Last Modified Date: 2026-03-17 Enrollment: 16 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
NCT05641610 Recruiting Phase2, Phase1
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Hemophilia B
Sponsor Institute of Hematology & Blood Diseases Hospital, China (Other)
Compound ZS801
Therapy Type Gene transfer
Target Gene F9
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-11-25 Last Modified Date: 2026-03-17 Enrollment: 21 China
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 Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-10-11 Last Modified Date: 2026-03-17 Enrollment: 14 Germany, Italy, United Kingdom + 1 more
NCT04819841 Recruiting Phase2, Phase1
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Sickle Cell Disease
Sponsor Kamau Therapeutics (Industry)
Compound KMAU-001
Therapy Type Gene editing
Target Gene HBB
Vector Type AAV6
Delivery System Viral transduction
Mechanism of Action Mutation correction
First Submitted: 2021-03-24 Last Modified Date: 2026-03-17 Enrollment: 15 United States
NCT05228145 Active not recruiting Phase2, Phase1
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Neuronal Ceroid Lipofuscinosis CLN5
Sponsor Neurogene Inc. (Industry)
Compound NGN-101
Therapy Type Gene transfer
Target Gene CLN5
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-12-17 Last Modified Date: 2026-03-17 Enrollment: 6 United Kingdom, United States
NCT05791864 Recruiting Phase2, Phase1
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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
NCT05811351 Completed Phase2
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Geographic Atrophy
Sponsor Janssen Research & Development, LLC (Industry)
Compound JNJ-1887
Therapy Type Gene transfer
Target Gene CD59
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-03-07 Last Modified Date: 2026-03-17 Enrollment: 305 Australia, Belgium, Canada + 14 more
NCT06474442 Recruiting Phase2
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Herpes Simplex Virus Type I Stromal Keratitis
Sponsor Shanghai BDgene Co., Ltd. (Industry)
Compound BD111
Therapy Type Gene editing
Target Gene HSV genes
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Gene excision
FDA Designations Orphan Drug Designation
First Submitted: 2024-06-13 Last Modified Date: 2026-03-17 Enrollment: 40 China
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
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
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
NCT02247843 Completed Phase2, Phase1
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Sickle Cell Disease
Sponsor Donald B. Kohn, M.D. (Other)
Compound LentiG-βAS3-FB (contains anti-sickling mutations: T87Q, G16D, E22A)
Therapy Type Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2014-09-20 Last Modified Date: 2026-03-17 Enrollment: 4 United States
NCT06851767 Enrolling by invitation Phase2, Phase1
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X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound BE-HSPC-IL2RG
Therapy Type Gene editing
Target Gene IL2RG (p.Q144X)
Delivery System Undisclosed
Mechanism of Action Mutation correction
First Submitted: 2025-02-26 Last Modified Date: 2026-03-17 Enrollment: 18 United States
NCT05820152 Terminated Phase2, Phase1
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Leber Hereditary Optic Neuropathy (LHON)
Sponsor Neurophth Therapeutics Inc (Other)
Compound NFS-02
Therapy Type Gene transfer
Target Gene ND1G3460A
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-04-06 Last Modified Date: 2026-03-17 Enrollment: 11 China, United States
NCT05230459 Recruiting Phase2, Phase1
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Limb-Girdle Muscular Dystrophy, Type 2I/R9
Sponsor AskBio Inc (Industry)
Compound AB-1003
Therapy Type Gene transfer
Target Gene FKRP
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-01-27 Last Modified Date: 2026-03-17 Enrollment: 10 United States
NCT04517149 Active not recruiting Phase2, Phase1
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X-Linked Retinitis Pigmentosa (XLRP)
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-125
Therapy Type Gene transfer
Target Gene RPGR
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-08-14 Last Modified Date: 2026-03-17 Enrollment: 21 United States
NCT04713475 Active not recruiting Phase2, Phase1
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GM1 Gangliosidosis, Beta-Galactosidase-1 (GLB1) Deficiency
Sponsor Gemma Biotherapeutics (Industry)
Compound PBGM01
Therapy Type Gene transfer
Target Gene GLB1
Vector Type AAVhu68
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-01-04 Last Modified Date: 2026-03-17 Enrollment: 26 Brazil, Turkey (Türkiye), United Kingdom + 1 more
NCT01496040 Completed Phase2, Phase1
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Leber Congenital Amaurosis
Sponsor Nantes University Hospital (Other)
Compound RAAV2/4.hRPE65
Therapy Type Gene transfer
Target Gene RPE65
Vector Type AAV2/4
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2011-11-24 Last Modified Date: 2026-03-17 Enrollment: 9 France
NCT02354781 Completed Phase2, Phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor Jerry R. Mendell (Other)
Compound RAAV1.CMV.huFollistatin344
Therapy Type Gene transfer
Target Gene FST (FS344)
Vector Type AAV1
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2015-01-26 Last Modified Date: 2026-03-17 Enrollment: 3 United States
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
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
NCT02186418 Terminated Phase2, Phase1
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Sickle Cell Disease
Sponsor Children's Hospital Medical Center, Cincinnati (Other)
Compound ARU-1801
Therapy Type Gene transfer
Target Gene HBG(G16D)
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2014-06-30 Last Modified Date: 2026-03-17 Enrollment: 7 United States
NCT05152823 Enrolling by invitation Phase2, Phase1
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Spinal Muscular Atrophy with Respiratory Distress
Sponsor Megan Waldrop (Other)
Compound ACTX-401
Therapy Type Gene transfer
Target Gene IGHMBP2
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-11-25 Last Modified Date: 2026-03-17 Enrollment: 10 United States
NCT04480567 Active not recruiting Phase2, Phase1
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Phenylketonuria (PKU)
Sponsor BioMarin Pharmaceutical (Industry)
Compound BMN 307
Therapy Type Gene transfer
Target Gene PAH
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-07-08 Last Modified Date: 2026-03-17 Enrollment: 100 United Kingdom, United States
NCT05878860 Recruiting Phase2, Phase1
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X-Linked Retinoschisis
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-201
Therapy Type Gene transfer
Target Gene RS1
Vector Type AAV.SPR
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-05-12 Last Modified Date: 2026-03-17 Enrollment: 21 United States
NCT02453477 Unknown Phase2, Phase1
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Beta-Thalassemia Major
Sponsor IRCCS San Raffaele (Other)
Compound OTL-300
Therapy Type Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2015-05-15 Last Modified Date: 2026-03-17 Enrollment: 10 Italy
NCT06550011 Active not recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Avirmax Biopharma Inc (Industry)
Compound ABI-110
Therapy Type Gene transfer
Target Gene VEGFtrap
Vector Type AAV.N54
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-07-25 Last Modified Date: 2026-03-17 Enrollment: 18 United States
NCT00985517 Completed Phase2, Phase1
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Idiopathic Parkinson's Disease
Sponsor Sangamo Therapeutics (Industry)
Compound CERE-120
Therapy Type Gene transfer
Target Gene NRTN
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2009-09-25 Last Modified Date: 2026-03-17 Enrollment: 57 United States
NCT06622668 Withdrawn Phase2, Phase1
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Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Intellia Therapeutics (Industry)
Compound NTLA-3001
Therapy Type Gene editing
Target Gene SERPINA1
Vector Type LNP
Delivery System Lipid encapsulation
Mechanism of Action Functional gene replacement
First Submitted: 2024-09-30 Last Modified Date: 2026-03-17 Enrollment: 0 New Zealand
NCT05735158 Unknown Phase2
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Autosomal Recessive Congenital Ichthyosis
Sponsor Krystal Biotech, Inc. (Industry)
Compound KB105
Therapy Type Gene transfer
Target Gene TGM1
Vector Type HSV-1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-02-09 Last Modified Date: 2026-03-17 Enrollment: 15 United States
NCT04174105 Active not recruiting Phase2, Phase1
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Pompe Disease (Late-onset)
Sponsor Astellas Gene Therapies (Industry)
Compound AT845
Therapy Type Gene transfer
Target Gene GAA
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2019-11-13 Last Modified Date: 2026-03-17 Enrollment: 11 United Kingdom, 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
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: 2026-03-17 Enrollment: 13 Israel, Taiwan, United States
NCT06280378 Recruiting Phase2, Phase1
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Beta-Thalassemia Major
Sponsor Kanglin Biotechnology (Hangzhou) Co., Ltd. (Industry)
Compound KL003
Therapy Type Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-02-20 Last Modified Date: 2026-03-17 Enrollment: 41 China
NCT03001830 Active not recruiting Phase2, Phase1
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Hemophilia A
Sponsor University College, London (Other)
Compound AAV2/8-HLP-FVIII-V3
Therapy Type Gene transfer
Target Gene F8
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-12-09 Last Modified Date: 2026-03-17 Enrollment: 14 United Kingdom, United States
NCT04240314 Completed Phase2, Phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor Megan Waldrop (Other)
Compound AT702
Therapy Type Gene transfer
Target Gene DMD (exon 2)
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Exon skipping/splice editor
First Submitted: 2020-01-22 Last Modified Date: 2026-03-17 Enrollment: 3 United States
NCT05761899 Recruiting Phase2, Phase1
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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
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
NCT05121376 Active not recruiting Phase2, Phase1
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Hereditary Angioedema
Sponsor BioMarin Pharmaceutical (Industry)
Compound BMN 331
Therapy Type Gene transfer
Target Gene SERPING1
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2021-10-28 Last Modified Date: 2026-03-17 Enrollment: 44 Australia, Spain, United States
NCT05972629 Active not recruiting Phase2, Phase1
View Report
Phenylketonuria (PKU)
Sponsor Sanofi (Industry)
Compound SAR444836
Therapy Type Gene transfer
Target Gene PAH
Vector Type AAV SNY001
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-06-23 Last Modified Date: 2026-03-17 Enrollment: 32 Argentina, Brazil, Israel + 2 more
NCT02610582 Active not recruiting Phase2, Phase1
View Report
Achromatopsia
Sponsor STZ eyetrial (Other)
Compound RAAV.hCNGA3
Therapy Type Gene transfer
Target Gene CNGA3
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2015-09-18 Last Modified Date: 2026-03-17 Enrollment: 13 Germany
NCT05824169 Recruiting Phase2, Phase1
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Spinal Muscular Atrophy
Sponsor GeneCradle Inc (Industry)
Compound GC101
Therapy Type Gene transfer
Target Gene SMN1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2023-04-10 Last Modified Date: 2026-03-17 Enrollment: 18 China
NCT00076557 Terminated Phase2, Phase1
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Hemophilia B
Sponsor Avigen (Industry)
Compound AAV2-hFIX16
Therapy Type Gene transfer
Target Gene F9
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2004-01-26 Last Modified Date: 2026-03-17 Enrollment: 15 United States
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
NCT03920007 Active not recruiting Phase2, Phase1
View Report
Leber Congenital Amaurosis, LCA, LCA1
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-101
Therapy Type Gene transfer
Target Gene GUCY2D
Vector Type AAV2/5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2019-04-10 Last Modified Date: 2026-03-17 Enrollment: 15 United States