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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 402 results in ClinicalTrials
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
NCT07721025 Recruiting Phase2
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Friedreich Ataxia with Secondary Cardiomyopathy
Sponsor Lexeo Therapeutics (Industry)
Compound LX2006
Therapeutic Modality Gene transfer
Target Gene FXN
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2026-06-24 Last Modified Date: 1788325200000 Enrollment: 26 United States
NCT03612869 Unknown Phase3, Phase2
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Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor LYSOGENE (Industry)
Compound LYS-SAF301
Therapeutic Modality Gene transfer
Target Gene SGSH
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-07-04 Last Modified Date: 1788238800000 Enrollment: 20 France, Germany, Netherlands + 2 more
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: 1788238800000 Enrollment: 10 United States
NCT03727555 Not yet recruiting Na
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X-linked Adrenoleukodystrophy
Sponsor Shenzhen Geno-Immune Medical Institute (Other)
Compound (LV) TYF-ABCD1
Therapeutic Modality Gene transfer
Target Gene ABCD1
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-09-25 Last Modified Date: 1788238800000 Enrollment: 30 China
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
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: 1788238800000 Enrollment: 40 United States
NCT07064759 Recruiting Phase3
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Neovascular Age-Related Macular Degeneration (nAMD)
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-150
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-07-07 Last Modified Date: 1788238800000 Enrollment: 480 Argentina, Australia, Bulgaria + 11 more
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: 1788238800000 Enrollment: 18 United States
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
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
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
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
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
NCT07592273 Recruiting Phase3, Phase2
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Diabetic Retinopathy
Sponsor AbbVie (Industry)
Compound Surabgene lomparvovec
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2026-04-24 Last Modified Date: 1787806800000 Enrollment: 576 Puerto Rico, 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
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
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
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
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
NCT05353647 Active not recruiting Phase2
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Sickle Cell Disease
Sponsor David Williams (Other)
Compound Autologous CD34+ HSC cells transduced with the lentiviral vector containing BCL11a-targeted shRNA
Therapeutic Modality Gene transfer
Target Gene BCL11A
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2022-04-21 Last Modified Date: 1787720400000 Enrollment: 25 United States
NCT03645460 Not yet recruiting Na
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Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Sponsor Shenzhen Geno-Immune Medical Institute (Other)
Compound ADA lentiviral vector
Therapeutic Modality Gene transfer
Target Gene ADA
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-07-17 Last Modified Date: 1787720400000 Enrollment: 10 China
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
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
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
NCT03566043 Active not recruiting Phase3, Phase2
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Mucopolysaccharidosis Type II (Hunter Syndrome)
Sponsor REGENXBIO Inc. (Industry)
Compound NAVSUNLI
Therapeutic Modality Gene transfer
Target Gene IDS
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-05-01 Last Modified Date: 1787634000000 Enrollment: 48 Brazil, United States
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
NCT05248230 Recruiting Phase2
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Cystic Fibrosis
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-710
Therapeutic Modality Gene transfer
Target Gene CFTR
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: 2022-02-10 Last Modified Date: 1787634000000 Enrollment: 30 United States
NCT05139316 Completed Phase3
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Glycogen Storage Disease Type Ia
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound GENGLYCOS
Therapeutic Modality Gene transfer
Target Gene G6PC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-07-14 Last Modified Date: 1787634000000 Enrollment: 49 Brazil, Canada, Denmark + 6 more
NCT06884865 Recruiting Phase2
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Osteoarthritis (OA) of the Knee
Sponsor Pacira Pharmaceuticals, Inc (Industry)
Compound PCRX-201
Therapeutic Modality Gene transfer
Target Gene IL1RA
Vector Type Adenovirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-03-13 Last Modified Date: 1787634000000 Enrollment: 135 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
NCT02716246 Recruiting Phase3, Phase2
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Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound UX111
Therapeutic Modality Gene transfer
Target Gene SGSH
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-03-17 Last Modified Date: 1787288400000 Enrollment: 36 Australia, Spain, 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
NCT05878860 Recruiting Phase3
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X-Linked Retinoschisis
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-201
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-12 Last Modified Date: 1787288400000 Enrollment: 97 United Kingdom, United States
NCT05930561 Active not recruiting Phase2
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Diabetic Macular Edema, Diabetic Retinopathy
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-150
Therapeutic Modality Gene transfer
Target Gene VEGF Trap + VEGF-C RNAi
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2023-06-26 Last Modified Date: 1787288400000 Enrollment: 72 Puerto Rico, 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
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
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
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
NCT04370054 Active not recruiting Phase3
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Hemophilia A
Sponsor Pfizer (Industry)
Compound Giroctogogene fitelparvovec
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2020-04-21 Last Modified Date: 1787115600000 Enrollment: 77 Australia, Brazil, Canada + 13 more
NCT05606614 Active not recruiting Phase3
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Rett Syndrome
Sponsor Taysha Gene Therapies, Inc. (Industry)
Compound TSHA-102
Therapeutic Modality Gene transfer
Target Gene MiniMECP2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-10-28 Last Modified Date: 1787115600000 Enrollment: 17 Canada, United States
NCT04671433 Completed Phase3
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X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Janssen Research & Development, LLC (Industry)
Compound Botaretigene sparoparvovec
Therapeutic Modality Gene transfer
Target Gene RPGR
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-11-05 Last Modified Date: 1787115600000 Enrollment: 105 Belgium, Canada, Denmark + 8 more
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
NCT07007065 Recruiting Phase3
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Neovascular Age-related Macular Degeneration
Sponsor AbbVie (Industry)
Compound ABBV-RGX-314
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2025-05-28 Last Modified Date: 1786683600000 Enrollment: 561 Austria, Belgium, Bulgaria + 14 more
NCT06594094 Active not recruiting Early phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)
Compound HG302
Therapeutic Modality Gene editing
Target Gene DMD (exon 51 splice site)
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Exon skipping/splice editor
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-09-10 Last Modified Date: 1786683600000 Enrollment: 4 China
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
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
NCT04283227 Active not recruiting Phase3
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Metachromatic Leukodystrophy
Sponsor Orchard Therapeutics (Industry)
Compound LENMELDY
Therapeutic Modality Gene transfer
Target Gene ARSA
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-02-13 Last Modified Date: 1786683600000 Enrollment: 6 Italy
NCT06128629 Recruiting Phase3
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Transthyretin Amyloidosis (ATTR) with Cardiomyopathy
Sponsor Intellia Therapeutics (Industry)
Compound NTLA-2001
Therapeutic Modality Gene editing
Target Gene TTR
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2023-11-08 Last Modified Date: 1786683600000 Enrollment: 1200 Argentina, Australia, Austria + 23 more
NCT07223944 Recruiting Phase3
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Gaucher Disease Type 1
Sponsor Spur Therapeutics (Industry)
Compound FLT201
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-10-30 Last Modified Date: 1786683600000 Enrollment: 45 Argentina, Australia, Brazil + 7 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
NCT07160634 Recruiting Phase3
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-003
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-08-29 Last Modified Date: 1786597200000 Enrollment: 80 Australia, Canada, United States
NCT06663878 Active not recruiting Na
View Report
Wilson Disease
Sponsor Xinhua Hospital, Shanghai Jiao Tong University School of Medicine (Other)
Compound MWAV201
Therapeutic Modality Gene transfer
Target Gene Undisclosed
Vector Type Undisclosed
Delivery System Undisclosed
Mechanism of Action Undisclosed
First Submitted: 2024-10-10 Last Modified Date: 1786510800000 Enrollment: 9 China
NCT06255782 Recruiting Phase3
View Report
Ornithine Transcarbamylase (OTC) Deficiency
Sponsor iECURE, Inc. (Industry)
Compound ECUR-506
Therapeutic Modality Gene transfer
Target Gene OTC/PCSK9 locus
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2023-12-19 Last Modified Date: 1786510800000 Enrollment: 20 Australia, Spain, United Kingdom + 1 more
NCT06860672 Terminated Early phase1
View Report
Snijders Blok-Campeau syndrome
Sponsor Yongguo Yu (Other)
Compound Dual Vector AAV-CHD3-R1025W Base Editor
Therapeutic Modality Gene editing
Target Gene CDH3/R1025W
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Mutation correction
First Submitted: 2025-02-24 Last Modified Date: 1786510800000 Enrollment: 1 China
NCT06706427 Active not recruiting Phase2, Phase1
View Report
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
NCT07587021 Recruiting Phase2
View Report
Primary Hyperoxaluria Type 1, PH1
Sponsor YolTech Therapeutics Co., Ltd (Industry)
Compound YOLT-203
Therapeutic Modality Gene editing
Target Gene HAO1
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2026-05-01 Last Modified Date: 1786510800000 Enrollment: 36 United States
NCT05444894 Active not recruiting Phase2, Phase1
View Report
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
NCT06517888 Recruiting Phase2, Phase1
View Report
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
NCT06611436 Terminated Phase2, Phase1
View Report
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
NCT06292650 Recruiting Early phase1
View Report
Retinitis Pigmentosa
Sponsor Zhongmou Therapeutics (Industry)
Compound ZM-02
Therapeutic Modality Gene transfer
Target Gene Ch2.0
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2024-02-27 Last Modified Date: 1786424400000 Enrollment: 12 China
NCT03368742 Active not recruiting Phase2, Phase1
View Report
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
View Report
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
NCT06699108 Active not recruiting Phase3
View Report
Bietti Crystalline Dystrophy
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGR-R01
Therapeutic Modality Gene transfer
Target Gene CYP4V2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-11-19 Last Modified Date: 1786424400000 Enrollment: 45 China
NCT05441553 Unknown Phase2, Phase1
View Report
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
NCT05693142 Active not recruiting Phase3, Phase2
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-202
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-01-04 Last Modified Date: 1786424400000 Enrollment: 65 Canada, United States
NCT05820152 Terminated Phase2, Phase1
View Report
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
NCT07148089 Recruiting Phase1
View Report
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
NCT06272149 Recruiting Early phase1
View Report
Type II Gaucher Disease
Sponsor Xinhua Hospital, Shanghai Jiao Tong University School of Medicine (Other)
Compound VGN-R08b
Therapeutic Modality Gene transfer
Target Gene GBA1
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: 2023-07-17 Last Modified Date: 1786424400000 Enrollment: 6 China
NCT06392724 Active not recruiting Early phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Peking Union Medical College Hospital (Other)
Compound GEN6050X
Therapeutic Modality Gene editing
Target Gene DMD
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Exon skipping/splice editor
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-04-26 Last Modified Date: 1786424400000 Enrollment: 3 China
NCT04853576 Active not recruiting Phase2, Phase1
View Report
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
NCT06765980 Recruiting Phase2, Phase1
View Report
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
NCT05898620 Active not recruiting Phase3
View Report
Rett Syndrome
Sponsor Neurogene Inc. (Industry)
Compound NGN-401
Therapeutic Modality Gene transfer
Target Gene MECP2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-06-01 Last Modified Date: 1786424400000 Enrollment: 33 Australia, United Kingdom, United States
NCT05821959 Recruiting Phase2, Phase1
View Report
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
NCT03634007 Completed Phase2, Phase1
View Report
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
NCT06217861 Recruiting Phase1
View Report
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
NCT05603312 Completed Phase2, Phase1
View Report
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
NCT07052929 Recruiting Phase2, Phase1
View Report
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
View Report
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
View Report
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
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
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
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
NCT05956626 Active not recruiting Phase3, Phase2
View Report
Stargardt Disease
Sponsor Ocugen (Industry)
Compound OCU410ST
Therapeutic Modality Gene transfer
Target Gene RORA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2023-06-30 Last Modified Date: 1785474000000 Enrollment: 51 United States
NCT06388200 Active not recruiting Phase3
View Report
Retinitis Pigmentosa
Sponsor Ocugen (Industry)
Compound OCU400
Therapeutic Modality Gene transfer
Target Gene NR2E3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-04-09 Last Modified Date: 1785474000000 Enrollment: 140 Canada, United States
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
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
NCT06634420 Active not recruiting Phase3
View Report
Hereditary Angioedema
Sponsor Intellia Therapeutics (Industry)
Compound NTLA-2002
Therapeutic Modality Gene editing
Target Gene KLKB1
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-10-07 Last Modified Date: 1785301200000 Enrollment: 80 Australia, Canada, France + 6 more
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
NCT03363165 Completed Phase2
View Report
Peripheral Artery Disease
Sponsor Northwestern University (Other)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2017-11-30 Last Modified Date: 1784869200000 Enrollment: 39 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

Clinical Trials Daily Digest

Updates in the last 14 days

NCT04174105
Last update post date Previous: 2026-06-10 Current: 2026-09-02 api
Vector type Previous: AAV8 Current: Adeno-associated virus curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: Inclusion criteria: * participant is aged ≥ 18 ye... api
1788325200000
Pompe Disease (Late-onset)
Astellas Gene Therapies Active not recruiting
Anticipated PoC judgement timing: 2H/FY2025
NCT06164730
Last update post date Previous: 2026-08-03 Current: 2026-09-02 api
Estimated completion date Previous: 2027-08 Current: 2027-10 api
Number of locations Previous: 24 Current: 27 api
Vector type Previous: LDLR + GalNAc Current: curator
Drug product type Previous: Encapsulated RNP Current: Encapsulated mRNA curator
Recent updates Previous: Verve was acquired by Lilly in June 2025 Current: Phase 1 study results published May 2026, Lilly pl... curator
Dose 4 Previous: Undisclosed dose 4 Current: curator
Dose 3 Previous: 0.6mg/kg Current: curator
Dose 2 Previous: 0.45mg/kg Current: curator
Dose 1 Previous: 0.3mg/kg Current: Phase 1 trial: 0.3 mg/kg (n=4); 0.45 mg/kg (n=6); ... curator
Editor type Previous: base editor Current: ABE8.8 mRNA curator
News and press releases Previous: null Current: LinkName:A single dose of Lilly's PCSK9 base edito... curator
Clinical publications Previous: null Current: LinkName:In Vivo Base Editing of PCSK9 with VERVE-... curator
Delivery system Previous: Lipid encapsulation Current: Endocytosis curator
Eligibility criteria Previous: null Current: Inclusion criteria: * diagnosis of hefh or premat... api
1788325200000
Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Verve Therapeutics, Inc. Recruiting
Phase 1 study results published May 2026, Lilly plans to initiate Phase 2 study by the end of 2026
NCT07721025
Last update post date Previous: 2026-07-22 Current: 2026-09-02 api
News and press releases Previous: LinkName:(Corporate Presentation) Corporate Overvi... Current: LinkName:(Corporate Presentation) Corporate Overvi... curator
Record status Previous: Draft Current: Active curator
Compound description Previous: null Current: AAVrh.10-CAG-hFXN curator
Indication doid Previous: null Current: DOID:0111218 curator
Development status Previous: null Current: Active curator
Indication Previous: Friedreich Ataxia, Cardiomyopathy - Secondary Current: Friedreich Ataxia with Secondary Cardiomyopathy curator
Compound name Previous: null Current: LX2006 curator
Recent updates Previous: null Current: Finalized pivotal trial protocol and statistical a... curator
Dose 2 Previous: null Current: Pivotal dose: 1.2E12 vg/kg curator
Dose 1 Previous: null Current: Dose escalation study: 1.8E11 vg/kg (n=6), 5.6E11 ... 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: Intravenous curator
Mechanism of action Previous: null Current: Functional gene replacement curator
Therapy route Previous: null Current: in vivo curator
Therapy type Previous: null Current: Gene transfer curator
Target gene Previous: null Current: FXN curator
Fda designation Previous: Current: Breakthrough Therapy, Fast Track, Orphan Drug Desi... curator
News and press releases Previous: null Current: LinkName:Lexeo Therapeutics Announces Positive Int... curator
News and press releases Previous: null Current: LinkName:Lexeo Therapeutics Announces Positive Int... curator
News and press releases Previous: null Current: LinkName:(Corporate Presentation) LX2006 Regulator... curator
News and press releases Previous: null Current: LinkName:Lexeo Therapeutics Announces Progress in ... curator
News and press releases Previous: null Current: LinkName:Lexeo Therapeutics Announces FDA Breakthr... curator
Clinical publications Previous: null Current: LinkName:(Corporate Presentation) Lexeo Therapeuti... curator
Clinical publications Previous: null Current: LinkName:AAVrh.10hFXN Gene Therapy for the Cardiom... curator
Preclinical publications Previous: null Current: LinkName:Expression and processing of mature human... curator
Preclinical publications Previous: null Current: LinkName:Stress-Induced Mouse Model of the Cardiac... curator
Preclinical publications Previous: null Current: LinkName:Identification of Safe and Effective Intr... curator
Protocol Previous: null Current: LinkName:Clinical Trial Protocol;LinkUrl:https://c... curator
Related nctid Previous: null Current: LinkName:Phase 1: NCT05302271;LinkUrl:https://www.... curator
Related nctid Previous: null Current: LinkName:Phase 1/2: NCT05445323;LinkUrl:https://ww... curator
1788325200000
Friedreich Ataxia with Secondary Cardiomyopathy
Lexeo Therapeutics Recruiting
Finalized pivotal trial protocol and statistical analysis plan in June 2026, 1.2E12 will be pivotal dose, topline data readout expected 2H2027
NCT03612869
Preclinical publications Previous: LinkName:(Abstract 211) AAV gene therapy LYS-SAF30... Current: LinkName:(Abstract 211) AAV gene therapy LYS-SAF30... curator
Vector type Previous: AAV2rh.10 Current: Adeno-associated virus curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: Inclusion criteria: * documented mps iiia diagnos... api
1788238800000
Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
LYSOGENE Unknown
Phase 2/3 study failed primary efficacy endpoint
NCT07113743
Last update post date Previous: 2026-08-24 Current: 2026-09-01 api
Description Previous: Background: x-linked chronic granulomatous diseas... Current: Background: x-linked chronic granulomatous diseas... api
Eligibility criteria Previous: 2 x the upper limits of normal (uln) (patients with a correctable deficiency controlled on medication will not be excluded). v. cytogenetic abnormalities known to be associated with hematopoietic defect on peripheral blood or bone marrow. b. infectious i. evidence of infection with hiv-1 and -2, Hepatitis b, Hepatitis c, Adenovirus, Parvovirus b 19 or toxoplasmosis within 8 weeks prior to mobilization/apheresis or bone marrow harvest. cytomegalovirus (cmv) infection is allowable as long as the infection is under control. ii. history of infection with mycobacteria or bacille calmette-guerin (bcg) vaccination. c. pulmonary i. resting o2 saturation by pulse oximetry \< 90% on room air. d. cardiac i. abnormal electrocardiogram (ecg) indicating cardiac pathology. ii. uncorrected congenital cardiac malformation with clinical symptomatology. iii. active cardiac disease, Including clinical evidence of congestive heart failure, Cyanosis, Hypotension. iv. poor cardiac function as evidenced by lv ejection fraction \<40% on echocardiogram. e. neurological i. significant neurologic abnormality by examination. ii. uncontrolled seizure disorder. f. renal i. renal insufficiency: serum creatinine \>=2.5 mg/dl, Or \>=3+ proteinuria. * chemistry lab abnormalities: serum sodium \>= 156 mmol/l or \<= 129 mmol/l, Potassium \>= 6.1 mmol/l or \<= 2.9 mmol/l, Calcium \>= 3.2 mmol/l or \< 1.74 mmol/l, Magnesium \>= 1.24 mmol/l or \< 0.39 mmol/l, Phosphate \>= 5.1 mmol/l or \< 1.9 mmol/l. * serum transaminases \> 5x the upper limit of normal (uln). serum bilirubin \> 2x the upper limit of normal (uln). serum glucose \> 1.5x the upper limit of normal (uln). * general * expected survival \< 6 months. * major congenital anomaly. * known allergic reactions to components of busulfan or dimethyl sulfoxide (dmso) or contraindication for administration of conditioning medication. * evidence of active malignant disease. * treatment with another investigational drug or other intervention within 6 months. * unable to undergo apheresis as per the nih cc department of transfusion medicine standard of care apheresis procedures. 1. patients who are hemodynamically unstable (systolic or diastolic blood pressure fall of 20 mm hg from the stable patient's baseline measurement) or requiring mechanical respiratory assistance are excluded. history of vasculitis. * administration of gamma-interferon within 30 days before the infusion of transduced, Autologous cd34+ cells. * any other condition that, In the opinion of the investigator, May compromise the safety or compliance of the patient or would preclude the patient from successful study completion.">-inclusion criteria: in order to be eligible to p... Current: 2 x the upper limits of normal (uln) (patients with a correctable deficiency controlled on medication will not be excluded). v. cytogenetic abnormalities known to be associated with hematopoietic defect on peripheral blood or bone marrow. b. infectious i. evidence of infection with hiv-1 and -2, Hepatitis b, Hepatitis c, Adenovirus, Parvovirus b 19 or toxoplasmosis within 8 weeks prior to mobilization/apheresis or bone marrow harvest. cytomegalovirus (cmv) infection is allowable as long as the infection is under control. ii. history of infection with mycobacteria or bacille calmette-guerin (bcg) vaccination. c. pulmonary i. resting o2 saturation by pulse oximetry \< 90% on room air. d. cardiac i. abnormal electrocardiogram (ecg) indicating cardiac pathology. ii. uncorrected congenital cardiac malformation with clinical symptomatology. iii. active cardiac disease, Including clinical evidence of congestive heart failure, Cyanosis, Hypotension. iv. poor cardiac function as evidenced by lv ejection fraction \<40% on echocardiogram. e. neurological i. significant neurologic abnormality by examination. ii. uncontrolled seizure disorder. f. renal i. renal insufficiency: serum creatinine \>=2.5 mg/dl, Or \>=3+ proteinuria. * chemistry lab abnormalities: serum sodium \>= 156 mmol/l or \<= 129 mmol/l, Potassium \>= 6.1 mmol/l or \<= 2.9 mmol/l, Calcium \>= 3.2 mmol/l or \< 1.74 mmol/l, Magnesium \>= 1.24 mmol/l or \< 0.39 mmol/l, Phosphate \>= 5.1 mmol/l or \< 1.9 mmol/l. * serum transaminases \> 5x the upper limit of normal (uln). serum bilirubin \> 2x the upper limit of normal (uln). serum glucose \> 1.5x the upper limit of normal (uln). * general * expected survival \< 6 months. * major congenital anomaly. * known allergic reactions to components of busulfan or dimethyl sulfoxide (dmso) or contraindication for administration of conditioning medication. * evidence of active malignant disease. * treatment with another investigational drug or other intervention within 6 months. * unable to undergo apheresis as per the nih cc department of transfusion medicine standard of care apheresis procedures. 1. patients who are hemodynamically unstable (systolic or diastolic blood pressure fall of 20 mm hg from the stable patient's baseline measurement) or requiring mechanical respiratory assistance are excluded. history of vasculitis. * administration of gamma-interferon within 21 days before the infusion of transduced, Autologous cd34+ cells. * any other condition that, In the opinion of the investigator, May compromise the safety or compliance of the patient or would preclude the patient from successful study completion.">* inclusion criteria: in order to be eligible to ... api
Vector type Previous: Rhabdovirus Current: Lentivirus curator
Editor type Previous: none Current: curator
Record status Previous: Draft Current: Active curator
Preclinical publications Previous: null Current: LinkName:Non-Clinical Efficacy and Safety Studies ... curator
Preclinical publications Previous: null Current: LinkName:Lentiviral gene therapy for X-linked chro... curator
Preclinical publications Previous: null Current: LinkName:Biochemical correction of X-CGD by a nove... curator
Preclinical publications Previous: null Current: LinkName:Gene correction of induced pluripotent st... curator
News and press releases Previous: null Current: LinkName:Orchard Therapeutics Announces FDA Grante... curator
News and press releases Previous: null Current: LinkName:Orchard Therapeutics Extends Runway into ... curator
Clinical publications Previous: null Current: LinkName:Lentiviral gene therapy for X-linked chro... curator
Compound description Previous: Current: pCCLChimGp91/VSVg lentiviral vector curator
Indication doid Previous: Current: DOID:0070195 curator
Development status Previous: Current: Inactive curator
Compound name Previous: Current: VSVG-PCCLChimGp91 curator
Recent updates Previous: Current: Commercial rights are owned by Orchard, which has ... curator
Dose 1 Previous: Current: Transduced CD34+ cells (6.5-32.6E6 cells/kg) curator
Delivery system Previous: Current: Viral transduction curator
Target tissue Previous: Current: CD34+ cells curator
Drug product type Previous: Current: Autologous cells curator
Route of administration Previous: Current: Intravenous curator
Mechanism of action Previous: Current: Functional gene replacement curator
Therapy route Previous: Current: Ex-vivo curator
Therapy type Previous: Current: Gene transfer curator
Target gene Previous: Current: CYBB curator
Alias value Previous: null Current: OTL-102 curator
Fda designation Previous: Current: Orphan Drug Designation curator
Related nctid Previous: null Current: LinkName:Phase 1/2: NCT02234934;LinkUrl:https://ww... curator
Related nctid Previous: null Current: LinkName:Phase 1/2: NCT02757911;LinkUrl:https://ww... curator
Related nctid Previous: null Current: LinkName:Phase 1/2: NCT01855685;LinkUrl:https://ww... curator
1788238800000
Chronic Granulomatous Disease (CGD)
National Institute of Allergy and Infectious Diseases (NIAID) Enrolling by invitation
Commercial rights are owned by Orchard, which has deprioritized the program
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