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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 278 results in ClinicalTrials
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NCT07491172 Recruiting Phase1
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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: 1788930000000 Enrollment: 90 Australia, New Zealand, United Kingdom + 1 more
NCT05243017 Active not recruiting Phase2, Phase1
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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: 1788930000000 Enrollment: 14 Poland, United Kingdom
NCT07447557 Recruiting Phase2, Phase1
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Charcot-Marie-Tooth Disease Type 4J
Sponsor Elpida Therapeutics SPC (Industry)
Compound ELP-02
Therapeutic Modality Gene transfer
Target Gene FIG4
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: 2026-01-20 Last Modified Date: 1788930000000 Enrollment: 8 United States
NCT06253507 Enrolling by invitation Phase2, Phase1
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Autosomal Recessive Chronic Granulomatous Disease (CGD)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound PCHIM-p47
Therapeutic Modality Gene transfer
Target Gene NCF1
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-02-09 Last Modified Date: 1788930000000 Enrollment: 10 United States
NCT05454566 Withdrawn Phase2, Phase1
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Osteoarthritis, Knee
Sponsor ICM Co. Ltd. (Industry)
Compound ICM-203
Therapeutic Modality Gene transfer
Target Gene NKX3-2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2022-07-04 Last Modified Date: 1788843600000
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: 1788843600000 Enrollment: 10 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: 1788843600000 Enrollment: 40 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: 1788411600000 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: 1788411600000 Enrollment: 3 Austria, Canada, Germany + 4 more
NCT06662188 Recruiting Phase2, Phase1
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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: 1788411600000 Enrollment: 6 United States
NCT06851767 Enrolling by invitation Phase2, Phase1
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X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound BE-HSPC-IL2RG
Therapeutic Modality Gene editing
Target Gene IL2RG (p.Q144X)
Delivery System Undisclosed
Mechanism of Action Mutation correction
First Submitted: 2025-02-26 Last Modified Date: 1788411600000 Enrollment: 18 United States
NCT04174105 Active not recruiting Phase2, Phase1
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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: 1788325200000 Enrollment: 11 United Kingdom, 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: 1788325200000 Enrollment: 85 Australia, Canada, Israel + 3 more
NCT02935517 Unknown Phase2, Phase1
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Achromatopsia
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-402
Therapeutic Modality Gene transfer
Target Gene CNGA3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2016-10-13 Last Modified Date: 1788238800000 Enrollment: 24 Israel, United States
NCT06539208 Unknown 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: 1788152400000 Enrollment: 31 China
NCT07479953 Not yet recruiting Phase1
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GM1 Gangliosidosis - Type I, GM1 Gangliosidosis - Type 2
Sponsor Tippi Mackenzie (Other)
Compound Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase
Therapeutic Modality Gene transfer
Target Gene GLB1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2026-03-14 Last Modified Date: 1788152400000 Enrollment: 5
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: 1787893200000 Enrollment: 6 Italy, Spain, United Kingdom + 1 more
NCT05986864 Recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Skyline Therapeutics (US) Inc. (Industry)
Compound SKG0106
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: 2023-08-03 Last Modified Date: 1787893200000 Enrollment: 68 China, United States
NCT06237777 Completed Phase1
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Diabetic Macular Edema
Sponsor Wang Min (Other)
Compound SKG0106
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: 2024-01-11 Last Modified Date: 1787806800000 Enrollment: 3 China
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: 1787806800000 Enrollment: 10 United States
NCT07267065 Not yet recruiting Phase1
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Parkinson's Disease (PD), Early Onset Parkinson Disease
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 Overexpression of protective allele/gene
First Submitted: 2025-11-24 Last Modified Date: 1787806800000 Enrollment: 9 United States
NCT03217617 Not yet recruiting Phase2, Phase1
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X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor Shenzhen Geno-Immune Medical Institute (Other)
Compound Ivlv-X1 lentiviral vector
Therapeutic Modality Gene transfer
Target Gene IL2RG
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-07-03 Last Modified Date: 1787806800000 Enrollment: 10 China
NCT02852213 Recruiting Phase1
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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: 1787806800000 Enrollment: 42 United States
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
NCT07195825 Recruiting Phase1
View Report
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
NCT07226206 Recruiting Phase2, Phase1
View Report
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
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
NCT03920007 Active not recruiting Phase2, Phase1
View Report
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
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
NCT06910813 Recruiting Phase2, Phase1
View Report
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
View Report
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
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
NCT05361031 Completed Phase2, Phase1
View Report
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
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
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
View Report
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
NCT06185673 Recruiting Phase2, Phase1
View Report
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
NCT02556736 Completed Phase2, Phase1
View Report
Advanced Retinitis Pigmentosa
Sponsor AbbVie (Industry)
Compound RST-001
Therapeutic Modality Gene transfer
Target Gene ChR2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2015-09-21 Last Modified Date: 1784696400000 Enrollment: 14 United States
NCT03533673 Completed Phase1
View Report
Pompe Disease
Sponsor AskBio Inc (Industry)
Compound ACTUS-101
Therapeutic Modality Gene transfer
Target Gene GAA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track
First Submitted: 2018-04-13 Last Modified Date: 1784696400000 Enrollment: 7 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
NCT00979238 Completed Phase1
View Report
Hemophilia B
Sponsor St. Jude Children's Research Hospital (Other)
Compound ScAAV2/8-LP1-hFIXco
Therapeutic Modality Gene transfer
Target Gene F9
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2009-09-16 Last Modified Date: 1784696400000 Enrollment: 14 United Kingdom, United States
NCT00076557 Terminated Phase2, Phase1
View Report
Hemophilia B
Sponsor Avigen (Industry)
Compound AAV2-hFIX16
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: 2004-01-26 Last Modified Date: 1784696400000 Enrollment: 15 United States
NCT03001830 Active not recruiting Phase2, Phase1
View Report
Hemophilia A
Sponsor University College, London (Other)
Compound AAV2/8-HLP-FVIII-V3
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-12-09 Last Modified Date: 1784696400000 Enrollment: 14 United Kingdom, United States
NCT04145037 Terminated Phase2, Phase1
View Report
Gaucher Disease Type 1
Sponsor AVROBIO (Industry)
Compound AVR-RD-02
Therapeutic Modality Gene transfer
Target Gene GBA1
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: 2019-08-27 Last Modified Date: 1784696400000 Enrollment: 8 Canada, United States
NCT00643747 Completed Phase2, Phase1
View Report
Leber Congenital Amaurosis-Type 2
Sponsor University College, London (Other)
Compound TgAAG76
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2008-03-20 Last Modified Date: 1784696400000 Enrollment: 12 United Kingdom
NCT03562494 Completed Phase1
View Report
Parkinson's Disease
Sponsor Neurocrine Biosciences (Industry)
Compound VY-AADC01
Therapeutic Modality Gene transfer
Target Gene DDC
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: 2018-05-16 Last Modified Date: 1784696400000 Enrollment: 14 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

Clinical Trials Daily Digest

Updates in the last 14 days

NCT07491172
Last update post date Previous: 2026-07-23 Current: 2026-09-09 api
Number of locations Previous: 19 Current: 17 api
Recent updates Previous: IND was cleared in 1Q26 Current: CRISPR Therapeutics continues to anticipate sharin... curator
Dose 2 Previous: Current: Phase 1b: 0.8mg/kg curator
Dose 1 Previous: Phase 1: 0.1 mg/kg (n=3); 0.3 mg/kg (n=3), 0.6mg/k... Current: Phase 1a: 0.1 mg/kg (n=3); 0.3 mg/kg (n=3), 0.6mg/... curator
Clinical publications Previous: null Current: LinkName:Durability of CRISPR-Cas9 Gene Editing Ta... curator
News and press releases Previous: null Current: LinkName:CRISPR Therapeutics Presents Phase 1a Dat... curator
Protocol Previous: null Current: LinkName:Clinical Trial Protocol;LinkUrl:https://w... curator
Clinical publications Previous: null Current: LinkName:Phase 1 Trial of CRISPR-Cas9 Gene Editing... curator
News and press releases Previous: null Current: LinkName:CRISPR Therapeutics Announces Positive Ph... curator
News and press releases Previous: null Current: LinkName:CRISPR Therapeutics Provides Business Upd... curator
Clinical publications Previous: null Current: LinkName:(Presentation) Phase 1 Trial of CRISPR-Ca... curator
Indication doid Previous: Current: DOID:13810; DOID:13809; DOID:3146 curator
Development status Previous: Current: Active curator
Record status Previous: Draft Current: Active curator
Indication Previous: Cardiovascular, Metabolic Disease, Dyslipidemias, ... Current: Hypertriglyceridemia, Heterozygous Familial Hyperc... curator
Compound name Previous: Current: CTX310 curator
Editor type Previous: Current: Cas9 mRNA curator
Delivery system Previous: Current: Endocytosis curator
Target tissue Previous: Current: Hepatocytes curator
Drug product type Previous: Current: Encapsulated mRNA curator
Route of administration Previous: Current: Intravenous curator
Mechanism of action Previous: Current: Gene inactivation curator
Therapy route Previous: Current: in vivo curator
Therapy type Previous: Current: Gene editing curator
Target gene Previous: Current: ANGPTL3 curator
1788930000000
Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozyg...
CRISPR Therapeutics AG Recruiting
CRISPR Therapeutics continues to anticipate sharing an update from the CTX310 Phase 1b clinical trial in the second half of 2026, focused on severe hy...
NCT05243017
Recent updates Previous: Sponsor announced in June 2026 that FDA has revers... Current: Sponsor announced in June 2026 that FDA has revers... curator
Dose 2 Previous: 6E13 gc/subject Current: High dose cohort: 6E13 gc/subject curator
Dose 1 Previous: 6E12 gc/subject Current: Low dose cohort: 6E12 gc/subject curator
Alias type Previous: null Current: proper name curator
Alias value Previous: null Current: Ifezuntirgene Inilparvovec curator
News and press releases Previous: null Current: LinkName:uniQure Announces Submission of Biologics... curator
Vector type Previous: AAV5 Current: Adeno-associated virus curator
News and press releases Previous: null Current: LinkName:uniQure Announces Plan for BLA Submission... curator
Therapy route Previous: Current: in vivo curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: 2 × upper limit of normal (uln) 2. aspartate aminotransferase (ast) \>2 × uln 3. total bilirubin \>2 × uln 4. alkaline phosphatase (alp) \>2 × uln 5. creatinine \>1.5 × uln 6. platelet count \<100, 000/mm3 7. prothrombin time (pt) \>1.2 × uln 8. partial thromboplastin time (ptt) \>1.2 × uln 15. known immunocompromised status including participants who have undergone organ transplantation or who test positive at screening for the human immunodeficiency virus (hiv); or who are at risk of pathogen reactivation if immunosuppressed, Including participants who test positive at screening for hepatitis c virus antibody (anti-hcv), Hepatitis c virus ribonucleic acid (hcv rna), Hepatitis b surface antigen (hbsag) or hepatitis b core antibody (anti-hbc); or who have history of active tuberculosis or a positive tuberculosis blood test during screening. for participants with an indeterminate tuberculosis blood test result or positive tuberculosis test result, Repeat testing is recommended. 16. known allergy, Sensitivity, Or other contraindication to immunosuppression regimens in this protocol. 17. any participant with an active infection (e.g., Coronavirus disease 2019 \[covid-19\]) at screening or at the time of treatment that requires medical intervention. participants may rescreen, Or if screened eligible and an open surgical slot is available, May receive treatment after recovery.">Inclusion criteria: 1. able and willing to provid... api
News and press releases Previous: null Current: LinkName:uniQure Announces 2025 Financial Results ... curator
1788930000000
Huntington's Disease
UniQure Biopharma B.V. Active not recruiting
Sponsor announced in June 2026 that FDA has reversed previous decision, and 3-year analysis from Phase I/II study is acceptable as the primary basis o...
NCT07770828NEW
Recent updates Previous: Topline Phase 2 data announced in March 2026, Ocug... Current: Topline Phase 2 data announced in March 2026, Ocug... curator
Dose 3 Previous: 1.5E11 vg/mL (200ul) Current: curator
Dose 2 Previous: 5E10 vg/mL (200ul) Current: Phase 3 study: 1E10 vg/eye (200ul injection volume... curator
Dose 1 Previous: 2.5E10 vg/mL Current: Phase 1/2 study: 5E9 vg/eye, 1E10 vg/eye, 3E10 vg/... curator
News and press releases Previous: null Current: LinkName:Ocugen Announces First Patient Dosed in P... curator
Indication doid Previous: null Current: DOID:10871 curator
Development status Previous: null Current: Active curator
Record status Previous: Provisional Current: Active curator
Compound name Previous: null Current: OCU410 curator
Vector type Previous: null Current: Adeno-associated virus curator
Delivery system Previous: null Current: Viral transduction curator
Drug product type Previous: null Current: Viral vector curator
Route of administration Previous: null Current: Subretinal curator
Mechanism of action Previous: null Current: Overexpression of protective allele/gene curator
Therapy route Previous: null Current: in vivo curator
Therapy type Previous: null Current: Gene transfer curator
Target gene Previous: null Current: RORA curator
Fda designation Previous: Current: Regenerative Medicine Advanced Therapy (RMAT) curator
Preclinical publications Previous: null Current: LinkName:(Abstract) OCU410, a Potential Therapeuti... curator
Preclinical publications Previous: null Current: LinkName:Retinoic acid related orphan receptor &al... curator
News and press releases Previous: null Current: LinkName:Ocugen Announces Topline 12-month Data fr... curator
News and press releases Previous: null Current: LinkName:Ocugen, Inc. Announces Dosing Completion ... curator
News and press releases Previous: null Current: LinkName:Ocugen Announces Positive Preliminary Pha... curator
News and press releases Previous: null Current: LinkName:Ocugen Provides Business Update with Four... curator
News and press releases Previous: null Current: LinkName:Ocugen Provides Business Update with Thir... curator
News and press releases Previous: null Current: LinkName:Ocugen Provides Business Update with Thir... curator
News and press releases Previous: null Current: LinkName:Ocugen Announces that the U.S. Food and D... curator
Clinical publications Previous: null Current: LinkName:(Abstract) Preliminary Safety and Efficac... curator
1788930000000
Dry Age-related Macular Degeneration
Ocugen Recruiting
Topline Phase 2 data announced in March 2026, Ocugen announce in September 2026 that the first patient in the registrational trial had been dosed
NCT06761183
Last update post date Previous: 2025-01-07 Current: 2026-09-09 api
Estimated completion date Previous: 2026-12-31 Current: 2027-12-31 api
Overall status Previous: Not yet recruiting Current: Recruiting api
Eligibility criteria Previous: Inclusion criteria: * age 40-75 years, Inclusive,... Current: Inclusion criteria: * age 40-75 years, Inclusive,... api
Vector type Previous: AAV9 Current: Adeno-associated virus curator
Therapy route Previous: Current: in vivo curator
Dose 1 Previous: Dose escalation with 3 levels, unknown concentrati... Current: Undisclosed dose escalation, 3 levels curator
Editor type Previous: none Current: curator
1788930000000
Ischemic Stroke
Beijing Tiantan Hospital Recruiting
This product is also being evaluated in Alzheimer's Disease indication in IIT trial in China
NCT05345171
Last update post date Previous: 2026-06-02 Current: 2026-09-09 api
Dose 4 Previous: Phase 3: 1.7E13 GC/kg Current: curator
Dose 3 Previous: Phase 1/2: 1.7E13 GC/kg Current: curator
Dose 2 Previous: Phase 1/2: 1.0E13 GC/kg Current: Phase 3: 1.7E13 GC/kg curator
Dose 1 Previous: Phase 1/2: 3.4E12 GC/kg Current: Phase 1/2 doses: 3.4E12 GC/kg, 1.0E13 GC/kg, 1.7E1... curator
Vector type Previous: AAV8 Current: Adeno-associated virus curator
Editor type Previous: none Current: curator
Enrollment type Previous: Estimated Current: Actual api
Enrollment count Previous: 32 Current: 37 api
Related nctid Previous: null Current: LinkName:Long Term Follow-Up: NCT03636438;LinkUrl:... curator
Recent updates Previous: Enrollment of Phase 3 study complete in February 2... Current: Interim results announced March 2026, data are exp... curator
News and press releases Previous: null Current: LinkName:Ultragenyx Announces Positive 36-Week Dat... curator
Eligibility criteria Previous: null Current: Key inclusion criteria: * confirmed clinical diag... api
1788930000000
Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Pharmaceutical Inc Active not recruiting
Interim results announced March 2026, data are expected first half of 2027
26 more update(s) recently — View All
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