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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, meeting abstracts, and more to give a comprehensive overview of the gene therapy clinical trial landscape.

Showing 1-59 of 59 results in ClinicalTrials
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NCT06255782 Active not recruiting Phase3
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Ornithine Transcarbamylase (OTC) Deficiency
Sponsor iECURE, Inc. (Industry)
Compound ECUR-506
Therapeutic Modality Gene transfer
Target Gene OTC/PCSK9 locus
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2023-12-19 Last Modified Date: 1790744400000 Enrollment: 20 Australia, Spain, United Kingdom + 1 more
NCT05878860 Recruiting Phase3
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X-Linked Retinoschisis
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-201
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-12 Last Modified Date: 1790658000000 Enrollment: 97 Canada, United Kingdom, United States
NCT05821959 Recruiting Phase3
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Bilateral Sensorineural Hearing Loss
Sponsor Eli Lilly and Company (Industry)
Compound AK-OTOF
Therapeutic Modality Gene transfer
Target Gene OTOF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-02-08 Last Modified Date: 1790658000000 Enrollment: 48 Canada, Taiwan, United Kingdom + 1 more
NCT07160634 Recruiting Phase3
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Duchenne Muscular Dystrophy (DMD)
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-003
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-08-29 Last Modified Date: 1790571600000 Enrollment: 80 Australia, Canada, United States
NCT07592273 Recruiting Phase3, Phase2
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Diabetic Retinopathy
Sponsor AbbVie (Industry)
Compound Surabgene lomparvovec
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2026-04-24 Last Modified Date: 1790226000000 Enrollment: 576 Puerto Rico, United States
NCT04671433 Completed Phase3
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X-Linked Retinitis Pigmentosa (XLRP)
Sponsor MeiraGTx Ocular UK Ltd (Industry)
Compound Botaretigene sparoparvovec
Therapeutic Modality Gene transfer
Target Gene RPGR
Vector Type Adeno-associated virus
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: 1790226000000 Enrollment: 105 Belgium, Canada, Denmark + 8 more
NCT02716246 Recruiting Phase3, Phase2
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Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound UX111
Therapeutic Modality Gene transfer
Target Gene SGSH
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-03-17 Last Modified Date: 1790053200000 Enrollment: 36 Australia, Spain, United States
NCT07007065 Recruiting Phase3
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Neovascular Age-related Macular Degeneration
Sponsor AbbVie (Industry)
Compound ABBV-RGX-314
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2025-05-28 Last Modified Date: 1789448400000 Enrollment: 561 Austria, Belgium, Bulgaria + 14 more
NCT02144610 Terminated Phase3
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Critical Limb Ischemia
Sponsor AnGes USA, Inc. (Industry)
Compound COLLATEGENE
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2014-05-20 Last Modified Date: 1789016400000 Enrollment: 46 Belgium, Canada, Denmark + 8 more
NCT05345171 Active not recruiting Phase3
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Ornithine Transcarbamylase (OTC) Deficiency
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound DTX301
Therapeutic Modality Gene transfer
Target Gene OTC
Vector Type Adeno-associated virus
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: 1788930000000 Enrollment: 37 Argentina, Brazil, Canada + 7 more
NCT07770828 Recruiting Phase3
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Dry Age-related Macular Degeneration
Sponsor Ocugen (Industry)
Compound OCU410
Therapeutic Modality Gene transfer
Target Gene RORA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2026-08-13 Last Modified Date: 1788930000000 Enrollment: 237 United States
NCT05139316 Completed Phase3
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Glycogen Storage Disease Type Ia
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound GENGLYCOS
Therapeutic Modality Gene transfer
Target Gene G6PC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-07-14 Last Modified Date: 1788843600000 Enrollment: 49 Brazil, Canada, Denmark + 6 more
NCT03199469 Active not recruiting Phase3, Phase2
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X-Linked Myotubular Myopathy
Sponsor Astellas Gene Therapies (Industry)
Compound AT132
Therapeutic Modality Gene transfer
Target Gene MTM1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-06-21 Last Modified Date: 1788411600000 Enrollment: 27 Canada, France, Germany + 1 more
NCT07223944 Recruiting Phase3
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Gaucher Disease Type 1
Sponsor Spur Therapeutics (Industry)
Compound FLT201
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-10-30 Last Modified Date: 1788411600000 Enrollment: 45 Argentina, Australia, Brazil + 7 more
NCT07064759 Recruiting Phase3
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Neovascular Age-Related Macular Degeneration (nAMD)
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-150
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-07-07 Last Modified Date: 1788238800000 Enrollment: 480 Argentina, Australia, Bulgaria + 11 more
NCT03612869 Unknown Phase3, Phase2
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Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor LYSOGENE (Industry)
Compound LYS-SAF301
Therapeutic Modality Gene transfer
Target Gene SGSH
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-07-04 Last Modified Date: 1788238800000 Enrollment: 20 France, Germany, Netherlands + 2 more
NCT03566043 Active not recruiting Phase3, Phase2
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Mucopolysaccharidosis Type II (Hunter Syndrome)
Sponsor REGENXBIO Inc. (Industry)
Compound NAVSUNLI
Therapeutic Modality Gene transfer
Target Gene IDS
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-05-01 Last Modified Date: 1787634000000 Enrollment: 48 Brazil, United States
NCT04370054 Active not recruiting Phase3
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Hemophilia A
Sponsor Pfizer (Industry)
Compound Giroctogogene fitelparvovec
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2020-04-21 Last Modified Date: 1787115600000 Enrollment: 77 Australia, Brazil, Canada + 13 more
NCT05606614 Active not recruiting Phase3
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Rett Syndrome
Sponsor Taysha Gene Therapies, Inc. (Industry)
Compound TSHA-102
Therapeutic Modality Gene transfer
Target Gene MiniMECP2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-10-28 Last Modified Date: 1787115600000 Enrollment: 17 Canada, United States
NCT06128629 Recruiting Phase3
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Transthyretin Amyloidosis (ATTR) with Cardiomyopathy
Sponsor Intellia Therapeutics (Industry)
Compound NTLA-2001
Therapeutic Modality Gene editing
Target Gene TTR
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2023-11-08 Last Modified Date: 1786683600000 Enrollment: 1200 Argentina, Australia, Austria + 23 more
NCT04283227 Active not recruiting Phase3
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Metachromatic Leukodystrophy
Sponsor Orchard Therapeutics (Industry)
Compound LENMELDY
Therapeutic Modality Gene transfer
Target Gene ARSA
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-02-13 Last Modified Date: 1786683600000 Enrollment: 6 Italy
NCT05898620 Active not recruiting Phase3
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Rett Syndrome
Sponsor Neurogene Inc. (Industry)
Compound NGN-401
Therapeutic Modality Gene transfer
Target Gene MECP2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-06-01 Last Modified Date: 1786424400000 Enrollment: 33 Australia, United Kingdom, United States
NCT06699108 Active not recruiting Phase3
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Bietti Crystalline Dystrophy
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGR-R01
Therapeutic Modality Gene transfer
Target Gene CYP4V2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-11-19 Last Modified Date: 1786424400000 Enrollment: 45 China
NCT05693142 Active not recruiting Phase3, Phase2
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Duchenne Muscular Dystrophy (DMD)
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-202
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-01-04 Last Modified Date: 1786424400000 Enrollment: 65 Canada, United States
NCT06388200 Active not recruiting Phase3
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Retinitis Pigmentosa
Sponsor Ocugen (Industry)
Compound OCU400
Therapeutic Modality Gene transfer
Target Gene NR2E3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-04-09 Last Modified Date: 1785474000000 Enrollment: 140 Canada, United States
NCT05956626 Active not recruiting Phase3, Phase2
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Stargardt Disease
Sponsor Ocugen (Industry)
Compound OCU410ST
Therapeutic Modality Gene transfer
Target Gene RORA
Vector Type Adeno-associated virus
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: 1785474000000 Enrollment: 51 United States
NCT06634420 Active not recruiting Phase3
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Hereditary Angioedema
Sponsor Intellia Therapeutics (Industry)
Compound NTLA-2002
Therapeutic Modality Gene editing
Target Gene KLKB1
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-10-07 Last Modified Date: 1785301200000 Enrollment: 80 Australia, Canada, France + 6 more
NCT05968118 Unknown Phase3
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Peripheral Artery Disease
Sponsor Beijing Northland Biotech. Co., Ltd. (Industry)
Compound NL003
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-01-04 Last Modified Date: 1784869200000 Enrollment: 36 China
NCT02563522 Terminated Phase3
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Diabetic Foot Ulcer
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2015-09-28 Last Modified Date: 1784869200000 Enrollment: 44 United States
NCT04275323 Completed Phase3
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Critical Limb Ischemia
Sponsor Beijing Northland Biotech. Co., Ltd. (Industry)
Compound NL003
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2020-01-18 Last Modified Date: 1784869200000 Enrollment: 302 China
NCT04469270 Completed Phase3
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Diabetic Neuropathy
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2020-06-25 Last Modified Date: 1784869200000 Enrollment: 162 United States
NCT06199531 Active not recruiting Phase3
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NGLY1 Deficiency
Sponsor Grace Science, LLC (Industry)
Compound GS-100
Therapeutic Modality Gene transfer
Target Gene NGLY1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-11-21 Last Modified Date: 1784782800000 Enrollment: 10 United States
NCT05881408 Active not recruiting Phase3
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Duchenne Muscular Dystrophy (DMD)
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound ELEVIDYS
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
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: 1784782800000 Enrollment: 148 Australia, Belgium, Canada + 11 more
NCT06856577 Active not recruiting Phase3
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Adverum Biotechnologies, Inc. (Industry)
Compound ADVM-022
Therapeutic Modality Gene transfer
Target Gene Codon optimized aflibercept
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2025-02-26 Last Modified Date: 1784782800000 Enrollment: 311 United States
NCT05203679 Active not recruiting Phase3, Phase2
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Hemophilia B
Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound BBM-H901
Therapeutic Modality Gene transfer
Target Gene F9
Vector Type Adeno-associated virus
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: 1784782800000 Enrollment: 32 China
NCT00999609 Active not recruiting Phase3
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Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Sponsor Spark Therapeutics, Inc. (Industry)
Compound LUXTURNA
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type Adeno-associated virus
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: 1784696400000 Enrollment: 31 United States
NCT04323098 Completed Phase3
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Hemophilia A
Sponsor BioMarin Pharmaceutical (Industry)
Compound ROCTAVIAN
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2020-03-24 Last Modified Date: 1784696400000 Enrollment: 22 Australia, Brazil, Taiwan + 1 more
NCT06833983 Recruiting Phase3
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Hemophilia A
Sponsor Gritgen Therapeutics Co., Ltd. (Industry)
Compound GS1191-0445
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-01-28 Last Modified Date: 1784696400000 Enrollment: 50 China
NCT06246513 Active not recruiting Phase3
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Limb-Girdle Muscular Dystrophy, Type 2E/R4
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-9003
Therapeutic Modality Gene transfer
Target Gene SGCB
Vector Type Adeno-associated virus
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: 1784696400000 Enrollment: 17 Belgium, Germany, Italy + 3 more
NCT03569891 Completed Phase3
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Hemophilia B
Sponsor CSL Behring (Industry)
Compound HEMGENIX
Therapeutic Modality Gene transfer
Target Gene F9-R338L
Vector Type Adeno-associated virus
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: 1784696400000 Enrollment: 67 Belgium, Denmark, Germany + 5 more
NCT06743646 Active not recruiting Phase3
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Bietti Crystalline Dystrophy
Sponsor Chigenovo Co., Ltd (Network)
Compound ZVS101e
Therapeutic Modality Gene transfer
Target Gene CYP4V2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-12-17 Last Modified Date: 1784696400000 Enrollment: 62 China
NCT04281485 Active not recruiting Phase3
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Duchenne Muscular Dystrophy (DMD)
Sponsor Pfizer (Industry)
Compound PF-06939926
Therapeutic Modality Gene transfer
Target Gene Mini-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-02-11 Last Modified Date: 1784696400000 Enrollment: 114 Australia, Belgium, Canada + 12 more
NCT06149403 Active not recruiting Phase3
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Mucopolysaccharidosis Type I (Hurler Syndrome)
Sponsor Orchard Therapeutics (Industry)
Compound OTL-203
Therapeutic Modality Gene transfer
Target Gene IDUA
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-11-17 Last Modified Date: 1784696400000 Enrollment: 41 Italy, Netherlands, United Kingdom + 1 more
NCT04850118 Active not recruiting Phase3, Phase2
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X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-501
Therapeutic Modality Gene transfer
Target Gene RPGR
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2021-04-05 Last Modified Date: 1784696400000 Enrollment: 85 Australia, Canada, United Kingdom + 1 more
NCT06432140 Active not recruiting Phase3, Phase2
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Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGN-R09b
Therapeutic Modality Gene transfer
Target Gene DDC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-05-17 Last Modified Date: 1784696400000 Enrollment: 13 China
NCT03207009 Completed Phase3
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Beta-Thalassemia Major
Sponsor Genetix Biotherapeutics Inc. (Industry)
Compound ZYNTEGLO
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
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: 2017-06-29 Last Modified Date: 1784696400000 Enrollment: 19 France, Germany, Greece + 3 more
NCT06692712 Recruiting Phase3
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Hereditary Spastic Paraplegia Type 50
Sponsor Elpida Therapeutics SPC (Industry)
Compound MELPIDA
Therapeutic Modality Gene transfer
Target Gene AP4M1
Vector Type Adeno-associated virus
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: 1784696400000 Enrollment: 24 Spain, United States
NCT05386680 Completed Phase3
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Spinal Muscular Atrophy
Sponsor Novartis Pharmaceuticals (Industry)
Compound ITVISMA
Therapeutic Modality Gene transfer
Target Gene SMN1
Vector Type Adeno-associated virus
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: 1784696400000 Enrollment: 27 Australia, Belgium, Canada + 6 more
NCT03837483 Active not recruiting Phase3
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Wiskott-Aldrich Syndrome
Sponsor Fondazione Telethon (Other)
Compound WASKYRA
Therapeutic Modality Gene transfer
Target Gene WAS
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-02-08 Last Modified Date: 1784696400000 Enrollment: 10 Italy, United States
NCT04293185 Active not recruiting Phase3
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Sickle Cell Disease
Sponsor Genetix Biotherapeutics Inc. (Industry)
Compound LYFGENIA
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-02-12 Last Modified Date: 1784696400000 Enrollment: 35 United States
NCT02652767 Completed Phase3
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Leber Hereditary Optic Neuropathy (LHON)
Sponsor GenSight Biologics (Industry)
Compound LUMEVOQ
Therapeutic Modality Gene transfer
Target Gene ND4 m.11778G>A
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-01-07 Last Modified Date: 1784696400000 Enrollment: 39 France, Germany, Italy + 2 more
NCT03861273 Active not recruiting Phase3
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Hemophilia B
Sponsor Pfizer (Industry)
Compound BEQVEZ
Therapeutic Modality Gene transfer
Target Gene F9-R338L
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2019-03-01 Last Modified Date: 1784696400000 Enrollment: 51 Australia, Brazil, Canada + 13 more
NCT03852498 Completed Phase3
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Cerebral Adrenoleukodystrophy (CALD)
Sponsor Genetix Biotherapeutics Inc. (Industry)
Compound SKYSONA
Therapeutic Modality Gene transfer
Target Gene ABCD1
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-02-13 Last Modified Date: 1784696400000 Enrollment: 35 France, Germany, Italy + 3 more
NCT03496012 Completed Phase3
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Choroideremia
Sponsor Biogen (Industry)
Compound BIIB111
Therapeutic Modality Gene transfer
Target Gene CHM
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2018-03-07 Last Modified Date: 1784696400000 Enrollment: 169 Canada, Denmark, Finland + 5 more
NCT06111638 Recruiting Phase3, Phase2
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Hemophilia A
Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound BBM-H803
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2023-10-27 Last Modified Date: 1784610000000 Enrollment: 55 China
NCT04917874 Completed Phase3
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Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor Krystal Biotech, Inc. (Industry)
Compound VYJUVEK
Therapeutic Modality Gene transfer
Target Gene COL7A1
Vector Type Herpesvirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-06-02 Last Modified Date: 1784610000000 Enrollment: 47 United States
NCT05725018 Active not recruiting Phase3
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Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor Abeona Therapeutics, Inc (Industry)
Compound ZEVASKYN
Therapeutic Modality Gene transfer
Target Gene COL7A1
Vector Type Retrovirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-01-23 Last Modified Date: 1784610000000 Enrollment: 12 United States
NCT00891306 Completed Phase3, Phase2
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Familial Lipoprotein Lipase Deficiency
Sponsor Amsterdam Molecular Therapeutics (Industry)
Compound GLYBERA
Therapeutic Modality Gene transfer
Target Gene LPL
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2009-04-30 Last Modified Date: 1784610000000 Enrollment: 5 Canada
NCT05477563 Recruiting Phase3
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Beta-Thalassemia, Sickle Cell Disease
Sponsor Vertex Pharmaceuticals Incorporated (Industry)
Compound CASGEVY
Therapeutic Modality Gene editing
Target Gene BCL11A
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-07-26 Last Modified Date: 1783400400000 Enrollment: 26 Germany, Italy, Saudi Arabia + 1 more
Visual summary of all 59 matching trials (across every page). Click a slice or bar to filter the results by it.
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Clinical Trials Daily Digest

4 Updates

Updates in the last 14 days

NCT06467344
Last update post date Previous: 2025-12-02 Current: 2026-10-07 api
Number of locations Previous: 10 Current: 11 api
Vector type Previous: AAV Current: Adeno-associated virus curator
Dose 3 Previous: Undisclosed high dose Current: curator
Dose 2 Previous: Undisclosed medium dose Current: curator
Dose 1 Previous: Undisclosed low dose Current: Undisclosed dose escalation, 3 levels curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: Key inclusion criteria: * presence of mutations i... api
Indication doid Previous: DOID:0050817 Current: DOID:0050817,DOID:0061241 curator
1791349200000
Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration
Ascidian Therapeutics, Inc Recruiting
Rare Pediatric Disease designation granted 4/25/24
NCT01306019
Last update post date Previous: 2026-10-06 Current: 2026-10-13 api
Description Previous: = 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... Current: = 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
Vector type Previous: VSV-G Current: Lentivirus curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: * inclusion criteria: * a proven mutation in the c... api
1791262800000
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
NCT06092034
Recent updates Previous: Clinical hold lifted August 2025 Current: Clinical hold lifted August 2025. Sponsor expects ... curator
News and press releases Previous: null Current: LinkName:Rocket Pharmaceuticals Announces FDA Alig... curator
Last update post date Previous: 2026-05-04 Current: 2026-10-06 api
Estimated completion date Previous: 2032-04 Current: 2032-06 api
Eligibility criteria Previous: Key inclusion criteria: 1. documentation of a pat... Current: Key inclusion criteria: 1. documentation of a pat... api
Enrollment count Previous: 14 Current: 18 api
Intervention description Previous: Rp-a501 is a gene therapy product consisting of a ... Current: Rp-a501 is a gene therapy product consisting of an... api
Description Previous: This is a single arm phase 2 trial to evaluate the... Current: This is a single arm phase 2 trial to evaluate the... api
Vector type Previous: AAV9 Current: Adeno-associated virus curator
Editor type Previous: none Current: curator
1791262800000
Danon Disease
Rocket Pharmaceuticals Inc. Recruiting
Clinical hold lifted August 2025
NCT06389877
Dose 2 Previous: Part B: 30mg (n=3); 60mg (n=2) Current: Part B: 30mg (n=3); 60mg (n=5) curator
Clinical publications Previous: null Current: LinkName:(Presentation) First-in-human in vivo gen... curator
Last update post date Previous: 2026-03-20 Current: 2026-10-05 api
Eligibility max age Previous: 70 years Current: 75 years api
Eligibility criteria Previous: Part a: inclusion criteria: * males or females 1... Current: Part a: key inclusion criteria: * age 18-70 year... api
Number of locations Previous: 11 Current: 12 api
Enrollment count Previous: 106 Current: 192 api
Description Previous: This is a phase 1/2, Multicenter, Open-label, Dose... Current: This is a phase 1/2, Multicenter, Open-label study... api
Preclinical publications Previous: LinkName:(Presentation) BEAM-302 Base editing as a... Current: LinkName:(Presentation) BEAM-302 Base editing as a... curator
Preclinical publications Previous: LinkName:(Corporate Presentation) BEAM-302: Target... Current: null curator
Preclinical publications Previous: LinkName:(Poster) BEAM-302 decreases hepatic aggre... Current: LinkName:(Poster) BEAM-302 decreases hepatic aggre... curator
Vector type Previous: LNP Current: curator
Editor type Previous: ABE Current: ABE mRNA curator
Clinical publications Previous: null Current: LinkName:(Presentation) The potential for base edi... curator
Drug product type Previous: Encapsulated RNP Current: Encapsulated mRNA curator
Delivery system Previous: Lipid encapsulation Current: Endocytosis curator
Fda designation Previous: Orphan Drug Designation, Regenerative Medicine Adv... Current: Orphan Drug Designation, Regenerative Medicine Adv... curator
Recent updates Previous: Data from Parts A and B of Phase 1/2 trial expecte... Current: Data from Parts A and B of Phase 1/2 trial release... curator
Dose 4 Previous: 75mg Current: curator
Dose 3 Previous: 60mg Current: Pivotal dose selected: 60mg curator
Dose 1 Previous: 15mg Current: Part A: 15mg (n=3); 30mg (n=3); 60mg (n=6); 75mg (... curator
News and press releases Previous: null Current: LinkName:Corporate Presentation - January 2026;Lin... curator
News and press releases Previous: null Current: LinkName:Beam Therapeutics Announces Compelling Up... curator
Estimated completion date Previous: 2027-08 Current: 2030-05 curator
Locations Previous: New zealand, Netherlands, United kingdom, Australi... Current: New zealand, Netherlands, United states, Ireland, ... curator
News and press releases Previous: null Current: LinkName:Beam Therapeutics Reports Fourth Quarter ... curator
1791176400000
Alpha-1 Antitrypsin Deficiency (AATD)
Beam Therapeutics Inc. Recruiting
Data from Parts A and B of Phase 1/2 trial released in March 2026, Beam intends to pursue an accelerated approval for BEAM-302
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.