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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 1-100 of 279 results in ClinicalTrials
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NCT05394064 Terminated Phase2, Phase1
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Adrenomyeloneuropathy (AMN)
Sponsor SwanBio Therapeutics, Inc. (Industry)
Compound SBT101
Therapeutic Modality Gene transfer
Target Gene ABCD1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-05-16 Last Modified Date: 1787720400000 Enrollment: 9 Netherlands, United States
NCT07667387 Recruiting Phase2, Phase1
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Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
Sponsor Rebecca Ahrens-Nicklas (Other)
Compound LNP.UCD.ABE
Therapeutic Modality Gene editing
Target Gene CPS1,OTC,ASS1,ASL,ARG,NAGS,SLC25A15
Delivery System Endocytosis
Mechanism of Action Mutation correction
First Submitted: 2026-06-18 Last Modified Date: 1787720400000 Enrollment: 7 United States
NCT07287397 Recruiting Phase2, Phase1
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Amyotrophic Lateral Sclerosis
Sponsor Vector Y Therapeutics (Industry)
Compound VTx-002
Therapeutic Modality Gene transfer
Target Gene Antibody to TDP-43
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Fast Track
First Submitted: 2025-11-19 Last Modified Date: 1787720400000 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 more
NCT05230459 Active not recruiting Phase2, Phase1
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Limb-Girdle Muscular Dystrophy, Type 2I/R9
Sponsor AskBio Inc (Industry)
Compound AB-1003
Therapeutic Modality Gene transfer
Target Gene FKRP
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: 2022-01-27 Last Modified Date: 1787720400000 Enrollment: 9 United States
NCT07429240 Recruiting Phase2, Phase1
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Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
Sponsor Precision BioSciences, Inc. (Industry)
Compound PBGENE-DMD
Therapeutic Modality Gene editing
Target Gene DMD del ex45-55
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Exon skipping/splice editor
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2026-02-17 Last Modified Date: 1787634000000 Enrollment: 18 United States
NCT06680232 Recruiting Phase1
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Chronic Hepatitis B
Sponsor Precision BioSciences, Inc. (Industry)
Compound PBGENE-HBV
Therapeutic Modality Gene editing
Target Gene HBV cccDNA
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Fast Track
First Submitted: 2024-10-24 Last Modified Date: 1787547600000 Enrollment: 45 France, Hong Kong, Moldova + 3 more
NCT06063850 Recruiting Phase2, Phase1
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Mesial Temporal Lobe Epilepsy
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-260
Therapeutic Modality Gene transfer
Target Gene MiGRIK2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
First Submitted: 2023-09-01 Last Modified Date: 1787547600000 Enrollment: 12 United States
NCT01306019 Recruiting Phase2, Phase1
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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
Therapeutic Modality Gene transfer
Target Gene IL2RG
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: 2011-02-26 Last Modified Date: 1787547600000 Enrollment: 40 United States
NCT07113743 Enrolling by invitation Phase2, Phase1
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Chronic Granulomatous Disease (CGD)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound VSVG-PCCLChimGp91
Therapeutic Modality Gene transfer
Target Gene CYBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2025-08-08 Last Modified Date: 1787547600000 Enrollment: 10 United States
NCT07643844 Recruiting Phase1
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Propionic Acidemia
Sponsor Mayo Clinic (Other)
Compound AAVrh10-PCCA
Therapeutic Modality Gene transfer
Target Gene PCCA
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: 2026-06-08 Last Modified Date: 1787288400000 Enrollment: 9 United States
NCT05419492 Recruiting Phase2, Phase1
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Dravet Syndrome
Sponsor Encoded Therapeutics (Industry)
Compound ETX101
Therapeutic Modality Gene transfer
Target Gene SCN1A
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-06-10 Last Modified Date: 1787288400000 Enrollment: 47 Australia, United Kingdom, United States
NCT05432310 Active not recruiting Phase2, Phase1
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Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Sponsor University of California, Los Angeles (Other)
Compound Simoladagene autotemcel
Therapeutic Modality Gene transfer
Target Gene ADA
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: 2022-06-04 Last Modified Date: 1787202000000 Enrollment: 20 United States
NCT07218887 Recruiting Phase2, Phase1
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BAG3 Mutation Associated Dilated Cardiomyopathy
Sponsor Alexion Pharmaceuticals, Inc. (Industry)
Compound ALXN2350
Therapeutic Modality Gene transfer
Target Gene BAG3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-10-17 Last Modified Date: 1787115600000 Enrollment: 6 Italy, Spain, United Kingdom + 1 more
NCT06109181 Active not recruiting Phase2, Phase1
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Arrhythmogenic Right Ventricular Cardiomyopathy
Sponsor Lexeo Therapeutics (Industry)
Compound LX2020
Therapeutic Modality Gene transfer
Target Gene PKP2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2023-10-25 Last Modified Date: 1787115600000 Enrollment: 10 United States
NCT04411654 Active not recruiting Phase2, Phase1
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Gaucher Disease Type 2
Sponsor Prevail Therapeutics (Industry)
Compound LY3884961
Therapeutic Modality Gene transfer
Target Gene GBA1
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: 2020-05-11 Last Modified Date: 1787115600000 Enrollment: 7 United Kingdom, United States
NCT06332807 Recruiting Phase2, Phase1
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Phenylketonuria (PKU)
Sponsor NGGT INC. (Industry)
Compound NGGT002
Therapeutic Modality Gene transfer
Target Gene PAH
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-02-07 Last Modified Date: 1787115600000 Enrollment: 12 United States
NCT06831825 Recruiting Phase1
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Heart Failure With Reduced Ejection Fraction
Sponsor Medley Therapeutics (Industry)
Compound YAP101
Therapeutic Modality Gene transfer
Target Gene SAV1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2025-01-23 Last Modified Date: 1786942800000 Enrollment: 24 United States
NCT07180355 Recruiting Phase1
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Friedreich's Ataxia
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-212
Therapeutic Modality Gene transfer
Target Gene FXN
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: 2025-08-22 Last Modified Date: 1786942800000 Enrollment: 10 United States
NCT04046224 Completed Phase2, Phase1
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Fabry Disease
Sponsor Sangamo Therapeutics (Industry)
Compound ST-920
Therapeutic Modality Gene transfer
Target Gene GLA
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: 2019-08-01 Last Modified Date: 1786683600000 Enrollment: 36 Australia, Canada, Germany + 4 more
NCT05616793 Recruiting Phase2, Phase1
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Leber Congenital Amaurosis-Type 5
Sponsor Opus Genetics, Inc (Industry)
Compound OPGx-001
Therapeutic Modality Gene transfer
Target Gene LCA5
Vector Type Adeno-associated virus
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: 2022-11-07 Last Modified Date: 1786683600000 Enrollment: 22 United States
NCT07627971 Recruiting Phase2, Phase1
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GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A
Sponsor Skylark Bio Inc. (Industry)
Compound SKY-GJB2
Therapeutic Modality Gene transfer
Target Gene GJB2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2026-05-28 Last Modified Date: 1786683600000 Enrollment: 10 United States
NCT03720418 Terminated Phase2, Phase1
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Parkinson's Disease
Sponsor Sio Gene Therapies (Industry)
Compound OXB-102
Therapeutic Modality Gene transfer
Target Gene TH-GCH1.DDC (tricistronic)
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2018-10-16 Last Modified Date: 1786683600000 Enrollment: 6 France, United Kingdom
NCT06018558 Active not recruiting Phase2, Phase1
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Geographic Atrophy
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: 2023-06-30 Last Modified Date: 1786683600000 Enrollment: 60 United States
NCT06064890 Recruiting Phase2, Phase1
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Frontotemporal Dementia, FTD, FTD-GRN
Sponsor AviadoBio Ltd (Industry)
Compound AVB-101
Therapeutic Modality Gene transfer
Target Gene GRN
Vector Type Adeno-associated virus
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: 1786597200000 Enrollment: 18 Belgium, Canada, Italy + 6 more
NCT06325709 Recruiting Phase2, Phase1
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X-Linked Chronic Granulomatous Disease
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound Base-edited autologous CD34+ cells
Therapeutic Modality Gene editing
Target Gene CYBB c.676 C>T
Delivery System Electroporation
Mechanism of Action Mutation correction
First Submitted: 2024-03-21 Last Modified Date: 1786597200000 Enrollment: 15 United States
NCT06706427 Active not recruiting Phase2, Phase1
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Bietti Crystalline Dystrophy
Sponsor NGGT (Suzhou) Biotechnology Co., Ltd. (Industry)
Compound NGGT001
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: 2024-11-14 Last Modified Date: 1786510800000 Enrollment: 12 China
NCT05444894 Active not recruiting Phase2, Phase1
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Beta-Thalassemia Major
Sponsor Editas Medicine, Inc. (Industry)
Compound EDIT-301
Therapeutic Modality Gene editing
Target Gene HBG1/HBG2
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-06-27 Last Modified Date: 1786424400000 Enrollment: 9 Canada, United States
NCT05228145 Active not recruiting Phase2, Phase1
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Neuronal Ceroid Lipofuscinosis CLN5
Sponsor Neurogene Inc. (Industry)
Compound NGN-101
Therapeutic Modality Gene transfer
Target Gene CLN5
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-12-17 Last Modified Date: 1786424400000 Enrollment: 6 United Kingdom, United States
NCT06765980 Recruiting Phase2, Phase1
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Geographic Atrophy Secondary to Age-related Macular Degeneration
Sponsor Kriya Therapeutics, Inc. (Industry)
Compound KRIYA-825
Therapeutic Modality Gene transfer
Target Gene CR2-CR1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2024-12-19 Last Modified Date: 1786424400000 Enrollment: 62 Canada, Israel, New Zealand + 1 more
NCT05603312 Completed Phase2, Phase1
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Parkinson's Disease
Sponsor MeiraGTx, LLC (Industry)
Compound AAV-GAD
Therapeutic Modality Gene transfer
Target Gene GAD1/2
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: 2022-10-12 Last Modified Date: 1786424400000 Enrollment: 14 United States
NCT07148089 Recruiting Phase1
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Catecholaminergic Polymorphic Ventricular Tachycardia
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-501
Therapeutic Modality Gene transfer
Target Gene CASQ2
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: 2025-08-22 Last Modified Date: 1786424400000 Enrollment: 18 Canada, United States
NCT06517888 Recruiting Phase2, Phase1
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Vestibular Schwannoma
Sponsor Akouos, Inc. (Industry)
Compound AAVAnc80-antiVEGF
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-07-19 Last Modified Date: 1786424400000 Enrollment: 27 United States
NCT05821959 Recruiting Phase2, Phase1
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Bilateral Sensorineural Hearing Loss
Sponsor Akouos, Inc. (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: 1786424400000 Enrollment: 22 Canada, Taiwan, United Kingdom + 1 more
NCT06539208 Recruiting Phase2, Phase1
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Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy
Sponsor YolTech Therapeutics Co., Ltd (Industry)
Compound YOLT-201
Therapeutic Modality Gene editing
Target Gene TTR
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2024-08-01 Last Modified Date: 1786424400000 Enrollment: 31 China
NCT04853576 Active not recruiting Phase2, Phase1
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Sickle Cell Disease
Sponsor Editas Medicine, Inc. (Industry)
Compound EDIT-301
Therapeutic Modality Gene editing
Target Gene HBG1/HBG2
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-04-16 Last Modified Date: 1786424400000 Enrollment: 45 Canada, United States
NCT03368742 Active not recruiting Phase2, Phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-001
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-12-05 Last Modified Date: 1786424400000 Enrollment: 12 United States
NCT06217861 Recruiting Phase1
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Glutaric Acidemia Type I
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGM-R02b
Therapeutic Modality Gene transfer
Target Gene GCDH
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: 2023-12-12 Last Modified Date: 1786424400000 Enrollment: 12 China
NCT05441553 Unknown Phase2, Phase1
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Hemophilia B
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGB-R04
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
First Submitted: 2022-06-15 Last Modified Date: 1786424400000 Enrollment: 26 China
NCT03634007 Completed Phase2, Phase1
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Alzheimer's Disease, Early Onset Alzheimer's Disease
Sponsor Lexeo Therapeutics (Industry)
Compound LX1001
Therapeutic Modality Gene transfer
Target Gene APOE2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2018-08-03 Last Modified Date: 1786424400000 Enrollment: 15 United States
NCT05820152 Terminated Phase2, Phase1
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Leber Hereditary Optic Neuropathy (LHON)
Sponsor Neurophth Therapeutics Inc (Other)
Compound NFS-02
Therapeutic Modality Gene transfer
Target Gene ND1 m.3460G>A
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-04-06 Last Modified Date: 1786424400000 Enrollment: 11 China, United States
NCT06611436 Terminated Phase2, Phase1
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Hemophilia B
Sponsor Be Biopharma (Industry)
Compound BE-101
Therapeutic Modality Gene editing
Target Gene F9
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2024-09-18 Last Modified Date: 1786424400000 Enrollment: 5 United States
NCT07052929 Recruiting Phase2, Phase1
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X-Linked Myotubular Myopathy
Sponsor Astellas Gene Therapies (Industry)
Compound ASP2957
Therapeutic Modality Gene transfer
Target Gene MTM1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-06-30 Last Modified Date: 1786078800000 Enrollment: 9 Canada, United States
NCT04040049 Terminated Phase2, Phase1
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Fabry Disease
Sponsor Spur Therapeutics (Industry)
Compound FLT190
Therapeutic Modality Gene transfer
Target Gene GLA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2019-07-26 Last Modified Date: 1785992400000 Enrollment: 3 Austria, Canada, Germany + 4 more
NCT05788536 Recruiting Phase2, Phase1
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Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Sponsor Regeneron Pharmaceuticals (Industry)
Compound OTARMENI
Therapeutic Modality Gene transfer
Target Gene OTOF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-03-15 Last Modified Date: 1785906000000 Enrollment: 36 Germany, Japan, Spain + 2 more
NCT07226206 Recruiting Phase2, Phase1
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Hemophilia A
Sponsor Hoffmann-La Roche (Industry)
Compound SPK-8011QQ
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-10-28 Last Modified Date: 1785819600000 Enrollment: 5 United States
NCT07195825 Recruiting Phase1
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Parkinson's Disease
Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound BBM-P002
Therapeutic Modality Gene transfer
Target Gene TH90del-AADC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2025-09-10 Last Modified Date: 1785819600000 Enrollment: 18 China
NCT03920007 Active not recruiting Phase2, Phase1
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Leber Congenital Amaurosis, LCA, LCA1
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-101
Therapeutic Modality Gene transfer
Target Gene GUCY2D
Vector Type Adeno-associated virus
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: 2019-04-10 Last Modified Date: 1785733200000 Enrollment: 15 United States
NCT02599922 Unknown Phase2, Phase1
View Report
Achromatopsia
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-401
Therapeutic Modality Gene transfer
Target Gene CNGB3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2015-11-05 Last Modified Date: 1785733200000 Enrollment: 32 United States
NCT06164730 Recruiting Phase1
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Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-102
Therapeutic Modality Gene editing
Target Gene PCSK9
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Fast Track
First Submitted: 2023-12-01 Last Modified Date: 1785733200000 Enrollment: 85 Australia, Canada, Israel + 3 more
NCT06910813 Recruiting Phase2, Phase1
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Nephropathic Cystinosis
Sponsor Novartis Pharmaceuticals (Industry)
Compound DFT383
Therapeutic Modality Gene transfer
Target Gene CTNS
Vector Type Lentivirus
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: 1785474000000 Enrollment: 30 United States
NCT06492850 Unknown Phase2, Phase1
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X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Frontera Therapeutics (Industry)
Compound FT-002
Therapeutic Modality Gene transfer
Target Gene RPGRORF15
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2024-07-01 Last Modified Date: 1785474000000 Enrollment: 32 China
NCT05164471 Terminated Phase2, Phase1
View Report
Hemophilia B
Sponsor Spur Therapeutics (Industry)
Compound FLT180a
Therapeutic Modality Gene transfer
Target Gene F9
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-11-22 Last Modified Date: 1785474000000 Enrollment: 6 United Kingdom, United States
NCT06817382 Recruiting Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Insmed Gene Therapy LLC (Industry)
Compound INS1201
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-02-04 Last Modified Date: 1785214800000 Enrollment: 12 United States
NCT06185673 Recruiting Phase2, Phase1
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Oculopharyngeal Muscular Dystrophy
Sponsor Benitec Biopharma, Inc. (Industry)
Compound BB-301
Therapeutic Modality Gene transfer
Target Gene PABPN1
Vector Type Adeno-associated virus
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: 1784869200000 Enrollment: 30 United States
NCT05361031 Completed Phase2, Phase1
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Charcot-Marie-Tooth Disease 1A
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: 2022-03-31 Last Modified Date: 1784869200000 Enrollment: 12 South Korea
NCT07491172 Recruiting Phase1
View Report
Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Uncontrolled Hypercholesterolemia, Mixed Dyslipidemia
Sponsor CRISPR Therapeutics AG (Industry)
Compound CTX310
Therapeutic Modality Gene editing
Target Gene ANGPTL3
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2026-03-18 Last Modified Date: 1784869200000 Enrollment: 90 Australia, New Zealand, United Kingdom + 1 more
NCT07293546 Enrolling by invitation Phase2, Phase1
View Report
FOXG1 Syndrome
Sponsor FOXG1 Research Foundation (Other)
Compound FRF-001
Therapeutic Modality Gene transfer
Target Gene FOXG1
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: 2025-12-16 Last Modified Date: 1784869200000 Enrollment: 12 United States
NCT01422772 Completed Phase2, Phase1
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Ischemic Heart Disease
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: 2011-08-21 Last Modified Date: 1784869200000 Enrollment: 9 South Korea
NCT05791864 Recruiting Phase2, Phase1
View Report
Neuronal Ceroid Lipofuscinosis Type 2
Sponsor Tern Therapeutics, LLC (Industry)
Compound TTX-381
Therapeutic Modality Gene transfer
Target Gene TPP1
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: 2023-03-17 Last Modified Date: 1784869200000 Enrollment: 16 Germany, United Kingdom
NCT07185256 Recruiting Phase2, Phase1
View Report
ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
Sponsor Opus Genetics, Inc (Industry)
Compound OPGx-BEST1
Therapeutic Modality Gene transfer
Target Gene BEST1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-07-17 Last Modified Date: 1784782800000 Enrollment: 10 United States
NCT04783181 Active not recruiting Phase2, Phase1
View Report
Congenital Adrenal Hyperplasia
Sponsor Adrenas Therapeutics Inc (Industry)
Compound BBP-631
Therapeutic Modality Gene transfer
Target Gene CYP21A2
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: 2021-03-01 Last Modified Date: 1784782800000 Enrollment: 8 United States
NCT02186418 Terminated Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Children's Hospital Medical Center, Cincinnati (Other)
Compound ARU-1801
Therapeutic Modality Gene transfer
Target Gene HBG(G16D)
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2014-06-30 Last Modified Date: 1784782800000 Enrollment: 7 United States
NCT05739643 Active not recruiting Phase2, Phase1
View Report
Krabbe Disease
Sponsor Forge Biologics, Inc (Industry)
Compound FBX-101
Therapeutic Modality Gene transfer
Target Gene GALC
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: 2023-02-13 Last Modified Date: 1784782800000 Enrollment: 9 United States
NCT02453477 Unknown Phase2, Phase1
View Report
Beta-Thalassemia Major
Sponsor IRCCS San Raffaele (Other)
Compound OTL-300
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2015-05-15 Last Modified Date: 1784782800000 Enrollment: 10 Italy
NCT01301443 Completed Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration
Sponsor Oxford BioMedica (Industry)
Compound RetinoStat
Therapeutic Modality Gene transfer
Target Gene COL18A1+PLG
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2011-02-21 Last Modified Date: 1784782800000 Enrollment: 21 United States
NCT02341807 Completed Phase2, Phase1
View Report
Choroideremia
Sponsor Spark Therapeutics, Inc. (Industry)
Compound SPK-7001
Therapeutic Modality Gene transfer
Target Gene CHM
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2015-01-12 Last Modified Date: 1784782800000 Enrollment: 15 United States
NCT06460844 Active not recruiting Phase1
View Report
Retinitis Pigmentosa, Choroideremia
Sponsor Ray Therapeutics, Inc. (Industry)
Compound RTx-015
Therapeutic Modality Gene transfer
Target Gene Codon optimized ChR-3M
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-06-10 Last Modified Date: 1784782800000 Enrollment: 10 United States
NCT05144386 Completed Phase1
View Report
HIV-1-infection
Sponsor Excision BioTherapeutics (Industry)
Compound EBT-101
Therapeutic Modality Gene editing
Target Gene HIV genes
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Gene excision
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2021-11-22 Last Modified Date: 1784782800000 Enrollment: 6 United States
NCT03952637 Recruiting Phase2, Phase1
View Report
GM1 Gangliosidosis
Sponsor National Human Genome Research Institute (NHGRI) (NIH)
Compound AXO-AAV-GM1
Therapeutic Modality Gene transfer
Target Gene GLB1
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: 2019-05-15 Last Modified Date: 1784782800000 Enrollment: 54 United States
NCT05456880 Active not recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-101
Therapeutic Modality Gene editing
Target Gene HBG1/HBG2
Delivery System Electroporation
Mechanism of Action Mutation correction
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2022-06-23 Last Modified Date: 1784782800000 Enrollment: 15 United States
NCT02168686 Completed Phase2, Phase1
View Report
Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Adverum Biotechnologies, Inc. (Industry)
Compound ADVM-043
Therapeutic Modality Gene transfer
Target Gene SERPINA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2014-06-10 Last Modified Date: 1784782800000 Enrollment: 6 United States
NCT04408625 Active not recruiting Phase2, Phase1
View Report
Frontotemporal Dementia
Sponsor Prevail Therapeutics (Industry)
Compound LY3884963
Therapeutic Modality Gene transfer
Target Gene GRN
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-05-21 Last Modified Date: 1784782800000 Enrollment: 35 Australia, Belgium, France + 3 more
NCT03223194 Terminated Phase1
View Report
Crigler-Najjar Syndrome
Sponsor Audentes Therapeutics (Industry)
Compound AT342
Therapeutic Modality Gene transfer
Target Gene UGT1A1
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: 2017-07-17 Last Modified Date: 1784782800000 Enrollment: 1 Israel, United Kingdom, United States
NCT06389877 Recruiting Phase2, Phase1
View Report
Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-302
Therapeutic Modality Gene editing
Target Gene SERPINA1 (p.Glu366Lys)
Delivery System Endocytosis
Mechanism of Action Mutation correction
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-04-22 Last Modified Date: 1784782800000 Enrollment: 106 Australia, Ireland, Netherlands + 3 more
NCT04676048 Recruiting Phase2, Phase1
View Report
Hemophilia A
Sponsor ASC Therapeutics (Industry)
Compound ASC618
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-12-15 Last Modified Date: 1784782800000 Enrollment: 12 United States
NCT05293626 Active not recruiting Phase2, Phase1
View Report
Leber Hereditary Optic Neuropathy (LHON)
Sponsor Neurophth Therapeutics Inc (Other)
Compound OPVIKA
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
FDA Designations Orphan Drug Designation
First Submitted: 2021-12-09 Last Modified Date: 1784782800000 Enrollment: 12 United States
NCT07058662 Recruiting Phase2, Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Belief BioMed (Beijing) Co., Ltd (Industry)
Compound BBM-D101
Therapeutic Modality Gene transfer
Target Gene Undisclosed
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Undisclosed
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-07-01 Last Modified Date: 1784782800000 Enrollment: 9 China
NCT03328130 Terminated Phase2, Phase1
View Report
Retinitis Pigmentosa
Sponsor eyeDNA Therapeutics (Industry)
Compound CTX-PDE6B
Therapeutic Modality Gene transfer
Target Gene PDE6B
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Rare Pediatric Disease Designation
First Submitted: 2017-10-05 Last Modified Date: 1784696400000 Enrollment: 19 France
NCT02702115 Terminated Phase2, Phase1
View Report
Mucopolysaccharidosis Type I (Hurler Syndrome)
Sponsor Sangamo Therapeutics (Industry)
Compound SB-318
Therapeutic Modality Gene editing
Target Gene IDUA
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: 2016-02-29 Last Modified Date: 1784696400000 Enrollment: 3 United States
NCT03041324 Terminated Phase2, Phase1
View Report
Mucopolysaccharidosis Type II (Hunter Syndrome)
Sponsor Sangamo Therapeutics (Industry)
Compound SB-913
Therapeutic Modality Gene editing
Target Gene IDS
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: 2017-01-13 Last Modified Date: 1784696400000 Enrollment: 9 United States
NCT05241444 Recruiting Phase1
View Report
Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome (IPEX)
Sponsor Bacchetta, Rosa, MD (Other)
Compound CD4^LVFOXP3
Therapeutic Modality Gene transfer
Target Gene FOXP3
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-01-12 Last Modified Date: 1784696400000 Enrollment: 30 United States
NCT05224505 Active not recruiting Phase1
View Report
Limb-Girdle Muscular Dystrophy, Type 2I/R9
Sponsor Atamyo Therapeutics (Industry)
Compound GNT0006
Therapeutic Modality Gene transfer
Target Gene FKRP
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Rare Pediatric Disease Designation
First Submitted: 2021-12-15 Last Modified Date: 1784696400000 Enrollment: 6 Denmark, France, United Kingdom
NCT06662188 Recruiting Phase2, Phase1
View Report
SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Sponsor Jaguar Gene Therapy, LLC (Industry)
Compound JAG201
Therapeutic Modality Gene transfer
Target Gene SHANK3
Vector Type Adeno-associated virus
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: 1784696400000 Enrollment: 6 United States
NCT02852213 Recruiting Phase1
View Report
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor Krzysztof Bankiewicz (Other)
Compound AAV2-hAADC
Therapeutic Modality Gene transfer
Target Gene DDC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-07-20 Last Modified Date: 1784696400000 Enrollment: 42 United States
NCT01024998 Completed Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Genzyme, a Sanofi Company (Industry)
Compound RAAV2-sFLT01
Therapeutic Modality Gene transfer
Target Gene SFLT01
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2009-12-02 Last Modified Date: 1784696400000 Enrollment: 19 United States
NCT06291935 Recruiting Phase1
View Report
Retinitis Pigmentosa
Sponsor VeonGen Therapeutics GmbH (Industry)
Compound VG901
Therapeutic Modality Gene transfer
Target Gene CNGA1
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: 2024-02-01 Last Modified Date: 1784696400000 Enrollment: 6 Germany
NCT05222178 Terminated Phase1
View Report
Phenylketonuria (PKU)
Sponsor Homology Medicines, Inc (Industry)
Compound HMI-103
Therapeutic Modality Gene transfer
Target Gene PAH
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track
First Submitted: 2021-12-21 Last Modified Date: 1784696400000 Enrollment: 3 United States
NCT05243017 Active not recruiting Phase2, Phase1
View Report
Huntington's Disease
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-130
Therapeutic Modality Gene transfer
Target Gene MiHTT
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2021-11-01 Last Modified Date: 1784696400000 Enrollment: 14 Poland, United Kingdom
NCT06519552 Recruiting Phase1
View Report
Mucopolysaccharidosis Type I (Hurler Syndrome)
Sponsor West China Hospital (Other)
Compound JWK-008
Therapeutic Modality Gene transfer
Target Gene IDUA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-06-23 Last Modified Date: 1784696400000 Enrollment: 6 China
NCT06465550 Recruiting Phase1
View Report
Beta-Thalassemia Major
Sponsor Shanghai BDgene Co., Ltd. (Industry)
Compound BD211
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-06-12 Last Modified Date: 1784696400000 Enrollment: 9 China
NCT04174105 Active not recruiting Phase2, Phase1
View Report
Pompe Disease (Late-onset)
Sponsor Astellas Gene Therapies (Industry)
Compound AT845
Therapeutic Modality Gene transfer
Target Gene GAA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2019-11-13 Last Modified Date: 1784696400000 Enrollment: 11 United Kingdom, United States
NCT03116113 Completed Phase2, Phase1
View Report
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Biogen (Industry)
Compound BIIB112
Therapeutic Modality Gene transfer
Target Gene RPGR
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2017-03-29 Last Modified Date: 1784696400000 Enrollment: 50 United Kingdom, United States
NCT00985517 Completed Phase2, Phase1
View Report
Idiopathic Parkinson's Disease
Sponsor Sangamo Therapeutics (Industry)
Compound CERE-120
Therapeutic Modality Gene transfer
Target Gene NRTN
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2009-09-25 Last Modified Date: 1784696400000 Enrollment: 57 United States
NCT06114056 Active not recruiting Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor West China Hospital (Other)
Compound JWK007
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: 2023-10-29 Last Modified Date: 1784696400000 Enrollment: 3 China
NCT05906251 Terminated Phase1
View Report
Limb Girdle Muscular Dystrophy, Type 2B/R2
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-6004
Therapeutic Modality Gene transfer
Target Gene DYSF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-06-07 Last Modified Date: 1784696400000 Enrollment: 2 United States
NCT06747273 Terminated Phase1
View Report
Limb-Girdle Muscular Dystrophy, Type 2D/R3
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-9004
Therapeutic Modality Gene transfer
Target Gene SGCA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-12-18 Last Modified Date: 1784696400000 Enrollment: 4 United States
NCT00976352 Completed Phase2, Phase1
View Report
Glycogen Storage Disease Type 2 (Pompe Disease)
Sponsor University of Florida (Other)
Compound AAV1-CMV-hGAA
Therapeutic Modality Gene transfer
Target Gene GAA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2009-07-13 Last Modified Date: 1784696400000 Enrollment: 9 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
Therapeutic Modality Gene transfer
Target Gene CLN6
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: 2016-03-16 Last Modified Date: 1784696400000 Enrollment: 13 United States
NCT04525352 Terminated Phase1
View Report
Infantile Malignant Osteopetrosis
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-L401
Therapeutic Modality Gene transfer
Target Gene TCIRG1
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track
First Submitted: 2020-08-11 Last Modified Date: 1784696400000 Enrollment: 1 United States
NCT05665166 Active not recruiting Phase2, Phase1
View Report
Mucopolysaccharidosis II
Sponsor University of Manchester (Other)
Compound AVR-RD-05
Therapeutic Modality Gene transfer
Target Gene IDS
Vector Type Lentivirus
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: 1784696400000 Enrollment: 5 United Kingdom

Clinical Trials Daily Digest

Updates in the last 14 days

NCT05394064
With has results Previous: False Current: True api
Last update post date Previous: 2025-10-31 Current: 2026-08-26 api
Enrollment count Previous: 8 Current: 9 api
Intervention description Previous: Sbt101 treatmentprocedure that mimics sbt101 infus... Current: Sbt101 treatment api
Intervention name Previous: Sbt101, Imitation procedure Current: Sbt101 treatment api
Vector type Previous: AAV9 Current: Adeno-associated virus curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: Inclusion criteria: 1. diagnosed with x-linked ad... api
1787720400000
Adrenomyeloneuropathy (AMN)
SwanBio Therapeutics, Inc. Terminated
Study was terminated for business/strategic reasons, not due to safety concerns
NCT07667387
Last update post date Previous: 2026-08-11 Current: 2026-08-26 api
Estimated completion date Previous: 2028-07 Current: 2028-09 api
Overall status Previous: Not yet recruiting Current: Recruiting api
Brief title Previous: A phase i/ii open-label safety and efficacy study ... Current: Study of lnp.ucd.abe in patients with urea cycle d... api
Eligibility criteria Previous: Inclusion criteria: 1. diagnosis of a severe urea... Current: Inclusion criteria: 1. diagnosis of a severe urea... api
Record status Previous: Draft Current: Active curator
Compound description Previous: Current: personalized variant-specific LNP.UCD.ABE curator
Indication doid Previous: Current: DOID:9267 curator
Development status Previous: Current: Active curator
Compound name Previous: Current: LNP.UCD.ABE curator
Recent updates Previous: Current: This clinical trial protocol is a master protocol ... curator
Dose 1 Previous: Current: undisclosed dose based on body weight with potenti... curator
Editor type Previous: Current: ABE mRNA curator
Delivery system Previous: Current: Endocytosis curator
Drug product type Previous: Current: Encapsulated mRNA curator
Route of administration Previous: Current: Intravenous curator
Mechanism of action Previous: Current: Mutation correction curator
Therapy route Previous: Current: in vivo curator
Therapy type Previous: Current: Gene editing curator
Target gene Previous: Current: CPS1,OTC,ASS1,ASL,ARG,NAGS,SLC25A15 curator
Preclinical publications Previous: null Current: LinkName:Pre-IND Official Responses/Meeting Summar... curator
Preclinical publications Previous: null Current: LinkName:Pre-IND Briefing Book;LinkUrl:https://scg... curator
Preclinical publications Previous: null Current: LinkName:Patient-Specific In Vivo Gene Editing to ... curator
Preclinical publications Previous: null Current: LinkName:How to create personalized gene editing p... curator
1787720400000
Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
Rebecca Ahrens-Nicklas Recruiting
This clinical trial protocol is a master protocol for participants with a mutation in one of the 7 genes that cause a urea cycle disorder. Participant...
NCT03645460
Last update post date Previous: 2026-06-23 Current: 2026-08-26 api
Estimated completion date Previous: 2027-12-31 Current: 2028-12-31 api
Overall status Previous: Recruiting Current: Not yet recruiting api
Vector type Previous: LV Current: Lentivirus curator
Number of locations Previous: 2 Current: 1 curator
Eligibility criteria Previous: null Current: Inclusion criteria: * diagnosis of classical ada-... api
1787720400000
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Shenzhen Geno-Immune Medical Institute Not yet recruiting
NCT07287397
Last update post date Previous: 2026-06-23 Current: 2026-08-26 api
Vector type Previous: AAV5.2 Current: Adeno-associated virus curator
Dose 2 Previous: Undisclosed high dose Current: curator
Dose 1 Previous: Undisclosed low dose Current: Undisclosed dose escalation, 2 levels curator
Intervention description Previous: An investigational gene therapy targeting a specif... Current: An investigational gene therapy targeting a specif... api
Intervention name Previous: Vtx-002, Optional rescue medication - corticostero... Current: Vtx-002, Preventative (prophylactic) medication - ... api
Eligibility criteria Previous: null Current: Key inclusion criteria: 1. capable of, And willin... api
Record status Previous: Draft Current: Active curator
Preclinical publications Previous: null Current: LinkName:(Poster) AAV-mediated intrabody therapy t... curator
Preclinical publications Previous: null Current: LinkName:(Poster) VecTabs® and advanced human in-v... curator
Preclinical publications Previous: null Current: LinkName:(Poster) AAV-mediated intrabody therapy t... curator
Preclinical publications Previous: null Current: LinkName:(Poster) Non-clinical development and Pha... curator
Indication doid Previous: null Current: DOID:332 curator
Development status Previous: null Current: Active curator
Compound name Previous: null Current: VTx-002 curator
Recent updates Previous: null Current: FDA cleared IND 12/25 curator
Delivery system Previous: null Current: viral transduction curator
Drug product type Previous: null Current: viral vector curator
Route of administration Previous: null Current: intracisternal curator
Mechanism of action Previous: null Current: genetic delivery of therapeutic protein curator
Therapy route Previous: null Current: in-vivo curator
Therapy type Previous: null Current: gene transfer curator
Target gene Previous: null Current: antibody to TDP-43 curator
Fda designation Previous: Current: Fast Track curator
News and press releases Previous: null Current: LinkName:VectorY Therapeutics Receives FDA Clearan... curator
News and press releases Previous: null Current: LinkName:VectorY Therapeutics Receives FDA Fast Tr... curator
1787720400000
Amyotrophic Lateral Sclerosis
Vector Y Therapeutics Recruiting
FDA cleared IND 12/25
NCT05230459
Last update post date Previous: 2026-02-20 Current: 2026-08-26 api
Overall status Previous: Recruiting Current: Active not recruiting api
Enrollment type Previous: Estimated Current: Actual api
Enrollment count Previous: 10 Current: 9 api
Vector type Previous: AAV9 Current: Adeno-associated virus curator
Dose 2 Previous: Undisclosed dose 2 Current: curator
Dose 1 Previous: Undisclosed dose 1 Current: Undisclosed dose escalation, 2 levels curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: Inclusion criteria: 1. male and female subjects a... api
1787720400000
Limb-Girdle Muscular Dystrophy, Type 2I/R9
AskBio Inc Active not recruiting
First patient dosed (8/3/23)
27 more update(s) recently — View All
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.