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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

NCT01306019
Last update post date 2026-04-24 2026-04-29 api
Description = 2 years of age who have clinically significant defects of immunity despite prior haploidentical hematopoietic stem cell transplant, And who lack an hla-matched sibling donor. our current gene transfer treatment protocol can be regarded as a salvage/rescue protocol. prior successful retroviral gene transfer treatment instead of bone marrow transplant (bmt) in paris and london for 20 infants with xscid has provided proof of principle for efficacy. however, A major safety concern is the occurrence of 5 cases of leukemia at 3-5 years after treatment triggered in part by vector insertional mutagenesis activation of lmo2 and other dna regulatory genes by the strong enhancer present in the long-terminal repeat (ltr) of the moloney leukemia virus (mlv)- based vector. furthermore, Previous studies of gene transfer treatment of older xscid patients with mlv- based vectors demonstrated the additional problem of failure of adequate expansion of gene corrected t- lymphocytes to the very high levels seen in infants. to reduce or eliminate this leukemia risk, And possibly enhance performance sufficiently to achieve benefit in older xscid patients, We have generated a lentivector with improved safety and performance features. we have generated a self-inactivating (sin) lentiviral vector that is devoid of all viral transcription elements; that contains a short form of the human elongation factor 1a (ef1a) internal promoter to expres......">This is a phase i/ii non-randomized clinical trial... = 2 years of age who have clinically significant defects of immunity despite prior haploidentical hematopoietic stem cell transplant, And who lack an hla-matched sibling donor. our current gene transfer treatment protocol can be regarded as a salvage/rescue protocol. prior successful retroviral gene transfer treatment instead of bone marrow transplant (bmt) in paris and london for 20 infants with xscid has provided proof of principle for efficacy. however, A major safety concern is the occurrence of 5 cases of leukemia at 3-5 years after treatment triggered in part by vector insertional mutagenesis activation of lmo2 and other dna regulatory genes by the strong enhancer present in the long-terminal repeat (ltr) of the moloney leukemia virus (mlv)- based vector. furthermore, Previous studies of gene transfer treatment of older xscid patients with mlv- based vectors demonstrated the additional problem of failure of adequate expansion of gene corrected t- lymphocytes to the very high levels seen in infants. to reduce or eliminate this leukemia risk, And possibly enhance performance sufficiently to achieve benefit in older xscid patients, We have generated a lentivector with improved safety and performance features. we have generated a self-inactivating (sin) lentiviral vector that is devoid of all viral transcription elements; that contains a short form of the human elongation factor 1a (ef1a) internal promoter to expres...">This is a phase i/ii non-randomized clinical trial... api
Eligibility criteria null * inclusion criteria: * a proven mutation in the c... api
2026-04-29
X-linked Severe Combined Immunodeficiency (XSCID)
National Institute of Allergy and Infectious Diseases (NIAID) Recruiting
Developed in partnership with St. Jude, and licensed to Mustang Bio, Mustang Bio discontinued this program
NCT06817382
Last update post date 2026-04-20 2026-04-29 curator
Number of locations 9 10 curator
Eligibility criteria null 1:50 as determined by elisa within 14 days of day 1. note: other inclusion/exclusion criteria may apply.">Inclusion criteria * participant must be male at ... api
2026-04-29
Duchenne Muscular Dystrophy (DMD)
Insmed Gene Therapy LLC Recruiting
IND cleared in 4Q2024, Clinical trial initiation planned in 1H2025
NCT06451770
Last update post date 2025-11-28 2026-04-28 api
Eligibility criteria null Inclusion criteria: * refractory hypercholesterol... api
Protocol null LinkName:Clinical Trial Protocol;LinkUrl:https://w... curator
Clinical publications null LinkName:Phase 1 Trial of CRISPR-Cas9 Gene Editing... curator
2026-04-28
Hypercholesterolemia
Verve Therapeutics, Inc. Recruiting
Verve was acquired by Lilly in July 2025
NCT02362438
Last update post date 2026-04-21 2026-04-27 api
Eligibility criteria null * inclusion criteria: to participate in this stud... api
2026-04-28
Giant Axonal Neuropathy
National Institute of Neurological Disorders and Stroke (NINDS) Active not recruiting
Taysha discontinued development in September 2023, transferred development rights to NIH
NCT05788536
Development status Active Approved curator
Compound name DB-OTO OTARMENI curator
Recent updates Regeneron planning regulatory submission by year e... Otarmeni was approved 4/23/26 under the National P... curator
Alias type null proprietary name, proper name curator
Alias value null DB-OTO, lunsotogene parvec curator
Fda designation Fast Track, Orphan Drug Designation, Rare Pediatri... Accelerated Approval, Fast Track, Orphan Drug Desi... curator
News and press releases null LinkName:FDA Approval Documents;LinkUrl:https://ww... curator
News and press releases null LinkName:FDA Approves First-Ever Gene Therapy for ... curator
Eligibility criteria null Key inclusion criteria: 1. willingness of at leas... api
2026-04-28
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (...
Regeneron Pharmaceuticals Recruiting
Otarmeni was approved 4/23/26 under the National Priority Voucher Program
16 more update(s) recently — View All

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Showing 1-100 of 388 results in ClinicalTrials
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: 2026-04-29 Enrollment: 40 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: 2026-04-29 Enrollment: 12 United States
NCT06451770 Recruiting Phase1
View Report
Hypercholesterolemia
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-201
Therapy Type Gene editing
Target Gene ANGPTL3
Vector Type LDLR + GalNAc
Delivery System Lipid encapsulation
Mechanism of Action Gene inactivation
First Submitted: 2024-06-04 Last Modified Date: 2026-04-28 Enrollment: 36 Australia, Canada, South Africa + 1 more
NCT02362438 Active not recruiting Phase1
View Report
Giant Axonal Neuropathy
Sponsor National Institute of Neurological Disorders and Stroke (NINDS) (NIH)
Compound ScAAV9/JeT-GAN
Therapy Type Gene transfer
Target Gene GAN
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2015-02-12 Last Modified Date: 2026-04-28 Enrollment: 14 United States
NCT05788536 Recruiting Phase2, Phase1
View Report
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Sponsor Regeneron Pharmaceuticals (Industry)
Compound OTARMENI
Therapy Type Gene transfer
Target Gene OTOF
Vector Type dual AAV1
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-03-15 Last Modified Date: 2026-04-28 Enrollment: 30 Germany, Spain, United Kingdom + 1 more
NCT06910813 Recruiting Phase2, Phase1
View Report
Nephropathic Cystinosis
Sponsor Novartis Pharmaceuticals (Industry)
Compound DFT383
Therapy Type Gene transfer
Target Gene CTNS
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-03-17 Last Modified Date: 2026-04-28 Enrollment: 30 United States
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: 2026-04-28 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 more
NCT07180355 Recruiting Phase1
View Report
Friedreich's Ataxia
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-212
Therapy Type Gene transfer
Target Gene FXN
Vector Type AAVhu68
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-08-22 Last Modified Date: 2026-04-28 Enrollment: 10 United States
NCT07226206 Recruiting Phase2, Phase1
View Report
Hemophilia A
Sponsor Hoffmann-La Roche (Industry)
Compound SPK-8011QQ
Therapy Type Gene transfer
Target Gene F8
Vector Type LK03
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-10-28 Last Modified Date: 2026-04-28 Enrollment: 5 United States
NCT04704921 Active not recruiting Phase3, Phase2
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor AbbVie (Industry)
Compound ABBV-RGX-314
Therapy Type Gene transfer
Target Gene Anti-VEGF
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2021-01-08 Last Modified Date: 2026-04-28 Enrollment: 671 United States
NCT06844214 Recruiting Phase2, Phase1
View Report
Myotonic Dystrophy
Sponsor Sanofi (Industry)
Compound SAR446268
Therapy Type Gene transfer
Target Gene DMPK
Vector Type AAV.SAN011
Delivery System Viral transduction
Mechanism of Action Gene inactivation
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2025-02-19 Last Modified Date: 2026-04-28 Enrollment: 32 Argentina, Australia, Canada + 3 more
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: 2026-04-25 Enrollment: 36 Australia, Spain, United States
NCT04903288 Active not recruiting Phase2
View Report
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor PTC Therapeutics (Industry)
Compound KEBILIDI, UPSTAZA
Therapy Type Gene transfer
Target Gene DDC
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Priority Review, Accelerated Approval, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-05-21 Last Modified Date: 2026-04-25 Enrollment: 13 Israel, Taiwan, United States
NCT06692712 Recruiting Phase3
View Report
Hereditary Spastic Paraplegia Type 50
Sponsor Elpida Therapeutics SPC (Industry)
Compound MELPIDA
Therapy Type Gene transfer
Target Gene AP4M1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-11-14 Last Modified Date: 2026-04-21 Enrollment: 24 Spain, United States
NCT03861273 Active not recruiting Phase3
View Report
Hemophilia B
Sponsor Pfizer (Industry)
Compound BEQVEZ
Therapy Type Gene transfer
Target Gene F9-R338L
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2019-03-01 Last Modified Date: 2026-04-21 Enrollment: 51 Australia, Brazil, Canada + 13 more
NCT07223944 Recruiting Phase3
View Report
Gaucher Disease Type 1
Sponsor Spur Therapeutics (Industry)
Compound FLT201
Therapy Type Gene transfer
Target Gene GBA1
Vector Type AAVS3
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy
First Submitted: 2025-10-30 Last Modified Date: 2026-04-21 Enrollment: 45 United States
NCT06432140 Active not recruiting Phase3, Phase2
View Report
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGN-R09b
Therapy Type Gene transfer
Target Gene DDC
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-05-17 Last Modified Date: 2026-04-21 Enrollment: 13 China
NCT04248439 Active not recruiting Phase2
View Report
Fanconi Anemia Complementation Group A
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-L102
Therapy Type Gene transfer
Target Gene FANCA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2020-01-24 Last Modified Date: 2026-04-21 Enrollment: 5 United States
NCT06826612 Recruiting Phase2, Phase1
View Report
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: 2026-04-18 Enrollment: 53 United States
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: 2026-04-18 Enrollment: 11 Bulgaria, France, Germany + 3 more
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: 2026-04-18 Enrollment: 26 United States
NCT06422351 Suspended Phase2
View Report
Pyruvate Kinase Deficiency
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-L301
Therapy Type Gene transfer
Target Gene PKLR
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2024-05-15 Last Modified Date: 2026-04-16 Enrollment: 10 Spain, United States
NCT04774536 Recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Mark Walters, MD (Other)
Compound CRISPR_SCD001
Therapy Type Gene editing
Target Gene HBB
Vector Type none
Delivery System Electroporation
Mechanism of Action Mutation correction
First Submitted: 2021-02-17 Last Modified Date: 2026-04-16 Enrollment: 9 United States
NCT06515002 Terminated Phase2, Phase1
View Report
Cystic Fibrosis
Sponsor Boehringer Ingelheim (Industry)
Compound BI 3720931
Therapy Type Gene transfer
Target Gene CFTR
Vector Type rSIV.F/HN
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-07-18 Last Modified Date: 2026-04-16 Enrollment: 5 France, Italy, Netherlands + 2 more
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: 2026-04-16 Enrollment: 480 Argentina, Australia, Bulgaria + 11 more
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: 2026-04-16 Enrollment: 47 Australia, 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: 2026-04-16 Enrollment: 20 Australia, Spain, 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: 2026-04-16 Enrollment: 9 Canada, United States
NCT06364774 Recruiting Phase2, Phase1
View Report
Beta-Thalassemia Major
Sponsor Children's Hospital of Philadelphia (Other)
Compound CHOP-ALS20
Therapy Type Gene transfer
Target Gene HBB
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-03-26 Last Modified Date: 2026-04-16 Enrollment: 12 United States
NCT05885412 Recruiting Phase1
View Report
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-A601
Therapy Type Gene transfer
Target Gene PKP2
Vector Type AAVrh.74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-05-22 Last Modified Date: 2026-04-16 Enrollment: 9 United States
NCT03533673 Completed Phase1
View Report
Pompe Disease
Sponsor AskBio Inc (Industry)
Compound ACTUS-101
Therapy Type Gene transfer
Target Gene GAA
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track
First Submitted: 2018-04-13 Last Modified Date: 2026-04-14 Enrollment: 7 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: 2026-04-14 Enrollment: 561 Austria, Belgium, Bulgaria + 14 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
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
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
NCT05073133 Completed Phase4
View Report
Spinal Muscular Atrophy
Sponsor Novartis Pharmaceuticals (Industry)
Compound ZOLGENSMA
Therapy Type Gene transfer
Target Gene SMN1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-09-20 Last Modified Date: 2026-04-08 Enrollment: 16 Argentina, Brazil
NCT05903794 Completed Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Exegenesis Bio (Industry)
Compound EXG102-031
Therapy Type Gene transfer
Target Gene ABD-VEGFR
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2023-06-06 Last Modified Date: 2026-04-08 Enrollment: 12 United States
NCT05456880 Active not recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-101
Therapy Type Gene editing
Target Gene HBG1/HBG2
Vector Type none
Delivery System Electroporation
Mechanism of Action Mutation correction
FDA Designations Regenerative Medicine Advanced Therapy
First Submitted: 2022-06-23 Last Modified Date: 2026-04-08 Enrollment: 15 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
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
NCT05386680 Completed Phase3
View Report
Spinal Muscular Atrophy
Sponsor Novartis Pharmaceuticals (Industry)
Compound ITVISMA
Therapy Type Gene transfer
Target Gene SMN1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Orphan Drug Designation
First Submitted: 2022-05-18 Last Modified Date: 2026-04-07 Enrollment: 27 Australia, Belgium, Canada + 6 more
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
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
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
NCT05835895 Active not recruiting Phase1
View Report
Osteoarthritis, Knee
Sponsor Genascence Corporation (Industry)
Compound GNSC-001
Therapy Type Gene transfer
Target Gene IL1RN
Vector Type AAV2.5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Regenerative Medicine Advanced Therapy
First Submitted: 2023-04-19 Last Modified Date: 2026-04-07 Enrollment: 67 United States
NCT05881408 Active not recruiting Phase3
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound ELEVIDYS
Therapy Type Gene transfer
Target Gene Micro-dystrophin
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Priority Review, Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-19 Last Modified Date: 2026-04-07 Enrollment: 148 Australia, Belgium, Canada + 11 more
NCT04850118 Active not recruiting Phase3, Phase2
View Report
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-501
Therapy Type Gene transfer
Target Gene RPGR
Vector Type AAV2tYF
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations 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
NCT04680065 Active not recruiting Phase1
View Report
Multiple System Atrophy
Sponsor Brain Neurotherapy Bio, Inc. (Industry)
Compound AB-1005
Therapy Type Gene transfer
Target Gene GDNF
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2020-12-15 Last Modified Date: 2026-04-06 Enrollment: 9 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
NCT05725018 Active not recruiting Phase3
View Report
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor Abeona Therapeutics, Inc (Industry)
Compound ZEVASKYN
Therapy Type Gene transfer
Target Gene COL7A1
Vector Type LZRSE
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-01-23 Last Modified Date: 2026-04-06 Enrollment: 12 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
NCT05345171 Active not recruiting Phase3
View Report
Ornithine Transcarbamylase (OTC) Deficiency
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound DTX301
Therapy Type Gene transfer
Target Gene OTC
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2022-04-18 Last Modified Date: 2026-04-06 Enrollment: 32 Argentina, Brazil, Canada + 7 more
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
NCT05139316 Completed Phase3
View Report
Glycogen Storage Disease Type Ia
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound DTX401
Therapy Type Gene transfer
Target Gene G6PC
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2021-07-14 Last Modified Date: 2026-04-06 Enrollment: 49 Brazil, Canada, Denmark + 6 more
NCT06388200 Active not recruiting Phase3
View Report
Retinitis Pigmentosa
Sponsor Ocugen (Industry)
Compound OCU400
Therapy Type Gene transfer
Target Gene NR2E3
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2024-04-09 Last Modified Date: 2026-04-06 Enrollment: 140 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
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
NCT04671433 Completed Phase3
View Report
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Janssen Research & Development, LLC (Industry)
Compound Botaretigene sparoparvovec
Therapy Type Gene transfer
Target Gene RPGR
Vector Type AAV2/5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-11-05 Last Modified Date: 2026-04-06 Enrollment: 105 Belgium, Canada, Denmark + 8 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
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
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
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
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
NCT04293185 Active not recruiting Phase3
View Report
Sickle Cell Disease
Sponsor Genetix Biotherapeutics Inc. (Industry)
Compound LYFGENIA
Therapy Type Gene transfer
Target Gene HBB
Vector Type BB305 LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2020-02-12 Last Modified Date: 2026-04-06 Enrollment: 35 United States
NCT02652767 Completed Phase3
View Report
Leber Hereditary Optic Neuropathy (LHON)
Sponsor GenSight Biologics (Industry)
Compound LUMEVOQ
Therapy Type Gene transfer
Target Gene ND4G11778A
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-01-07 Last Modified Date: 2026-04-03 Enrollment: 39 France, Germany, Italy + 2 more
NCT00999609 Active not recruiting Phase3
View Report
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Sponsor Spark Therapeutics, Inc. (Industry)
Compound LUXTURNA
Therapy Type Gene transfer
Target Gene RPE65
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Priority Review, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2009-10-21 Last Modified Date: 2026-04-03 Enrollment: 31 United States
NCT03496012 Completed Phase3
View Report
Choroideremia
Sponsor Biogen (Industry)
Compound BIIB111
Therapy Type Gene transfer
Target Gene CHM
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy
First Submitted: 2018-03-07 Last Modified Date: 2026-04-03 Enrollment: 169 Canada, Denmark, Finland + 5 more
NCT00979238 Completed Phase1
View Report
Hemophilia B
Sponsor St. Jude Children's Research Hospital (Other)
Compound ScAAV2/8-LP1-hFIXco
Therapy Type Gene transfer
Target Gene F9
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2009-09-16 Last Modified Date: 2026-04-02 Enrollment: 14 United Kingdom, 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
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
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
NCT04411654 Active not recruiting Phase2, Phase1
View Report
Gaucher Disease Type 2
Sponsor Prevail Therapeutics (Industry)
Compound LY3884961
Therapy Type Gene transfer
Target Gene GBA1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-05-11 Last Modified Date: 2026-04-01 Enrollment: 7 United Kingdom, United States
NCT05203679 Active not recruiting Phase3, Phase2
View Report
Hemophilia B
Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound BBM-H901
Therapy Type Gene transfer
Target Gene F9
Vector Type AAV843
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-12-29 Last Modified Date: 2026-04-01 Enrollment: 32 China
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
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
NCT06066008 Completed Early phase1
View Report
X-Linked Retinoschisis
Sponsor Zhongmou Therapeutics (Industry)
Compound ZM-01
Therapy Type Gene transfer
Target Gene RS1
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-09-25 Last Modified Date: 2026-03-31 Enrollment: 2 China
NCT06480461 Active not recruiting Phase2, Phase1
View Report
Parkinson's Disease
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGN-R09b
Therapy Type Gene transfer
Target Gene DDC
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2024-06-14 Last Modified Date: 2026-03-31 Enrollment: 39 China
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
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
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
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
NCT03569891 Completed Phase3
View Report
Hemophilia B
Sponsor CSL Behring (Industry)
Compound HEMGENIX
Therapy Type Gene transfer
Target Gene F9-R338L
Vector Type AAV5
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Priority Review, Orphan Drug Designation
First Submitted: 2018-06-14 Last Modified Date: 2026-03-27 Enrollment: 67 Belgium, Denmark, Germany + 5 more
NCT07185256 Recruiting Phase2, Phase1
View Report
ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
Sponsor Opus Genetics, Inc (Industry)
Compound OPGx-BEST1
Therapy Type Gene transfer
Target Gene BEST1
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-07-17 Last Modified Date: 2026-03-24 Enrollment: 10 United States
NCT05477563 Recruiting Phase3
View Report
Beta-Thalassemia, Sickle Cell Disease
Sponsor Vertex Pharmaceuticals Incorporated (Industry)
Compound CASGEVY
Therapy Type Gene editing
Target Gene BCL11A
Vector Type RNP
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
FDA Designations 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
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
NCT06747273 Terminated Phase1
View Report
Limb-Girdle Muscular Dystrophy, Type 2D/R3
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-9004
Therapy Type Gene transfer
Target Gene SGCA
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-12-18 Last Modified Date: 2026-03-18 Enrollment: 4 United States
NCT05973630 Active not recruiting Phase1
View Report
Limb-Girdle Muscular Dystrophy, Type 2C/R5
Sponsor Atamyo Therapeutics (Industry)
Compound ATA-200
Therapy Type Gene transfer
Target Gene SGCG
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-07-24 Last Modified Date: 2026-03-18 Enrollment: 4 United States
NCT05898620 Recruiting Phase3
View Report
Rett Syndrome
Sponsor Neurogene Inc. (Industry)
Compound NGN-401
Therapy Type Gene transfer
Target Gene MECP2
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations 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
NCT02599922 Active not recruiting Phase2, Phase1
View Report
Achromatopsia
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-401
Therapy Type Gene transfer
Target Gene CNGB3
Vector Type AAV2tYF
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2015-11-05 Last Modified Date: 2026-03-17 Enrollment: 32 United States
NCT02725580 Completed Phase2, Phase1
View Report
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease)
Sponsor Emily de los Reyes (Other)
Compound AT-GTX-501
Therapy Type Gene transfer
Target Gene CLN6
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-03-16 Last Modified Date: 2026-03-17 Enrollment: 13 United States
NCT02341807 Completed Phase2, Phase1
View Report
Choroideremia
Sponsor Spark Therapeutics, Inc. (Industry)
Compound SPK-7001
Therapy Type Gene transfer
Target Gene CHM
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2015-01-12 Last Modified Date: 2026-03-17 Enrollment: 15 United States
NCT05614531 Enrolling by invitation Phase2, Phase1
View Report
Spinal Muscular Atrophy Type I
Sponsor Guangzhou Jiayin Biotech Ltd (Industry)
Compound EXG001-307
Therapy Type Gene transfer
Target Gene SMN1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-11-02 Last Modified Date: 2026-03-17 Enrollment: 12 China
NCT06246513 Active not recruiting Phase3
View Report
Limb-Girdle Muscular Dystrophy, Type 2E/R4
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-9003
Therapy Type Gene transfer
Target Gene SGCB
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-01-30 Last Modified Date: 2026-03-17 Enrollment: 17 Belgium, Germany, Italy + 3 more