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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
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: 1790139600000 Enrollment: 18 United States
NCT06364774 Recruiting Phase2, Phase1
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Beta-Thalassemia Major
Sponsor Children's Hospital of Philadelphia (Other)
Compound CHOP-ALS20
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-03-26 Last Modified Date: 1790139600000 Enrollment: 12 United States
NCT03588299 Completed Phase2, Phase1
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Hemophilia A
Sponsor Bayer (Industry)
Compound DTX201
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-07-06 Last Modified Date: 1790139600000 Enrollment: 11 Bulgaria, France, Germany + 3 more
NCT07587021 Recruiting Phase2
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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: 1790139600000 Enrollment: 36 United States
NCT06910813 Recruiting Phase2, Phase1
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Nephropathic Cystinosis
Sponsor Novartis Pharmaceuticals (Industry)
Compound DFT383
Therapeutic Modality Gene transfer
Target Gene CTNS
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-03-17 Last Modified Date: 1790053200000 Enrollment: 30 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: 1790053200000 Enrollment: 47 Australia, United Kingdom, 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: 1790053200000 Enrollment: 36 Australia, Spain, 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: 1790053200000 Enrollment: 40 United States
NCT06844214 Recruiting Phase2, Phase1
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Myotonic Dystrophy
Sponsor Sanofi (Industry)
Compound SAR446268
Therapeutic Modality Gene transfer
Target Gene DMPK
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Gene inactivation
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2025-02-19 Last Modified Date: 1789966800000 Enrollment: 32 Argentina, Australia, Canada + 3 more
NCT05878860 Recruiting Phase3
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X-Linked Retinoschisis
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-201
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-12 Last Modified Date: 1789966800000 Enrollment: 97 Canada, United Kingdom, United States
NCT03952637 Recruiting Phase2, Phase1
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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: 1789966800000 Enrollment: 54 United States
NCT04127578 Active not recruiting Phase2, Phase1
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Parkinson's Disease
Sponsor Prevail Therapeutics (Industry)
Compound PR001
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2019-10-14 Last Modified Date: 1789707600000 Enrollment: 32 Israel, United States
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: 1789707600000 Enrollment: 90 Australia, New Zealand, United Kingdom + 1 more
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: 1789621200000 Enrollment: 576 Puerto Rico, United States
NCT06817382 Active not recruiting Phase1
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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: 1789534800000 Enrollment: 12 United States
NCT06739434 Enrolling by invitation Na
View Report
Rett Syndrome
Sponsor Genecombio Ltd. (Other)
Compound GCB-002
Therapeutic Modality Gene transfer
Target Gene MECP2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-12-12 Last Modified Date: 1789448400000 Enrollment: 6 China
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: 1789448400000 Enrollment: 10 United States
NCT07007065 Recruiting Phase3
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Neovascular Age-related Macular Degeneration
Sponsor AbbVie (Industry)
Compound ABBV-RGX-314
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2025-05-28 Last Modified Date: 1789448400000 Enrollment: 561 Austria, Belgium, Bulgaria + 14 more
NCT01496040 Completed Phase2, Phase1
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Leber Congenital Amaurosis
Sponsor Nantes University Hospital (Other)
Compound RAAV2/4.hRPE65
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2011-11-24 Last Modified Date: 1789362000000 Enrollment: 9 France
NCT07048808 Recruiting Phase2
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Refractory Angina, Coronary Artery Disease
Sponsor XyloCor Therapeutics, Inc. (Industry)
Compound XC001
Therapeutic Modality Gene transfer
Target Gene VEGF
Vector Type Adenovirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2025-06-24 Last Modified Date: 1789102800000 Enrollment: 106 United States
NCT07226206 Recruiting Phase2, Phase1
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Hemophilia A
Sponsor Hoffmann-La Roche (Industry)
Compound SPK-8011QQ
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-10-28 Last Modified Date: 1789102800000 Enrollment: 5 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: 1789016400000 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 more
NCT06114056 Active not recruiting Phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor West China Hospital (Other)
Compound JWK007
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-10-29 Last Modified Date: 1789016400000 Enrollment: 3 China
NCT05984927 Active not recruiting Phase2, Phase1
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Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Elisigen, Inc. (Industry)
Compound NG101
Therapeutic Modality Gene transfer
Target Gene Codon optimized aflibercept
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Fast Track
First Submitted: 2023-07-24 Last Modified Date: 1789016400000 Enrollment: 20 Canada, United States
NCT02144610 Terminated Phase3
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Critical Limb Ischemia
Sponsor AnGes USA, Inc. (Industry)
Compound COLLATEGENE
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2014-05-20 Last Modified Date: 1789016400000 Enrollment: 46 Belgium, Canada, Denmark + 8 more
NCT05345171 Active not recruiting Phase3
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Ornithine Transcarbamylase (OTC) Deficiency
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound DTX301
Therapeutic Modality Gene transfer
Target Gene OTC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2022-04-18 Last Modified Date: 1788930000000 Enrollment: 37 Argentina, Brazil, Canada + 7 more
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
NCT07770828 Recruiting Phase3
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Dry Age-related Macular Degeneration
Sponsor Ocugen (Industry)
Compound OCU410
Therapeutic Modality Gene transfer
Target Gene RORA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2026-08-13 Last Modified Date: 1788930000000 Enrollment: 237 United States
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
NCT06761183 Recruiting Early phase1
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Ischemic Stroke
Sponsor Beijing Tiantan Hospital (Other)
Compound NXL-001
Therapeutic Modality Gene transfer
Target Gene NeuroD1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-12-27 Last Modified Date: 1788930000000 Enrollment: 9 China
NCT05598333 Active not recruiting Phase2
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Congestive Heart Failure
Sponsor AskBio Inc (Industry)
Compound AB-1002
Therapeutic Modality Gene transfer
Target Gene PPP1R1A
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2022-10-25 Last Modified Date: 1788843600000 Enrollment: 150 Austria, Belgium, Bulgaria + 9 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: 1788843600000 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
NCT05139316 Completed Phase3
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Glycogen Storage Disease Type Ia
Sponsor Ultragenyx Pharmaceutical Inc (Industry)
Compound GENGLYCOS
Therapeutic Modality Gene transfer
Target Gene G6PC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-07-14 Last Modified Date: 1788843600000 Enrollment: 49 Brazil, Canada, Denmark + 6 more
NCT03199469 Active not recruiting Phase3, Phase2
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X-Linked Myotubular Myopathy
Sponsor Astellas Gene Therapies (Industry)
Compound AT132
Therapeutic Modality Gene transfer
Target Gene MTM1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-06-21 Last Modified Date: 1788411600000 Enrollment: 27 Canada, France, Germany + 1 more
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
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
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
NCT07223944 Recruiting Phase3
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Gaucher Disease Type 1
Sponsor Spur Therapeutics (Industry)
Compound FLT201
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-10-30 Last Modified Date: 1788411600000 Enrollment: 45 Argentina, Australia, Brazil + 7 more
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
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
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
NCT07064759 Recruiting Phase3
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Neovascular Age-Related Macular Degeneration (nAMD)
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-150
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-07-07 Last Modified Date: 1788238800000 Enrollment: 480 Argentina, Australia, Bulgaria + 11 more
NCT03612869 Unknown Phase3, Phase2
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Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor LYSOGENE (Industry)
Compound LYS-SAF301
Therapeutic Modality Gene transfer
Target Gene SGSH
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-07-04 Last Modified Date: 1788238800000 Enrollment: 20 France, Germany, Netherlands + 2 more
NCT02935517 Unknown Phase2, Phase1
View Report
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
View Report
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
View Report
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
View Report
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
NCT03217617 Not yet recruiting Phase2, Phase1
View Report
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
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
NCT02852213 Recruiting Phase1
View Report
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor Krzysztof Bankiewicz (Other)
Compound AAV2-hAADC
Therapeutic Modality Gene transfer
Target Gene DDC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-07-20 Last Modified Date: 1787806800000 Enrollment: 42 United States
NCT07267065 Not yet recruiting Phase1
View Report
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
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
NCT03645460 Not yet recruiting Na
View Report
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
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
NCT07667387 Recruiting Phase2, Phase1
View Report
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
View Report
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
NCT07429240 Recruiting Phase2, Phase1
View Report
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
View Report
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
NCT06884865 Recruiting Phase2
View Report
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
NCT03566043 Active not recruiting Phase3, Phase2
View Report
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
NCT06680232 Recruiting Phase1
View Report
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
View Report
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
NCT05930561 Active not recruiting Phase2
View Report
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
NCT07643844 Recruiting Phase1
View Report
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
View Report
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
View Report
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
NCT05606614 Active not recruiting Phase3
View Report
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
View Report
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
NCT06332807 Recruiting Phase2, Phase1
View Report
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
NCT04370054 Active not recruiting Phase3
View Report
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
NCT04411654 Active not recruiting Phase2, Phase1
View Report
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
NCT07180355 Recruiting Phase1
View Report
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
View Report
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
View Report
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
NCT06128629 Recruiting Phase3
View Report
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
NCT03720418 Terminated Phase2, Phase1
View Report
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
NCT04283227 Active not recruiting Phase3
View Report
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
NCT04046224 Completed Phase2, Phase1
View Report
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
NCT06594094 Active not recruiting Early phase1
View Report
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
NCT06064890 Recruiting Phase2, Phase1
View Report
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
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
NCT06325709 Recruiting Phase2, Phase1
View Report
X-Linked Chronic Granulomatous Disease
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound Base-edited autologous CD34+ cells
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
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
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
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
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
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
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
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
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
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
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
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
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
Visual summary of all 402 matching trials (across every page). Click a slice or bar to filter the results by it.
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Recruitment Status
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Clinical Trials Daily Digest

Updates in the last 14 days

NCT06851767
Last update post date Previous: 2026-09-03 Current: 2026-09-23 api
Eligibility criteria Previous: * inclusion criteria: in order to be eligible to ... Current: * inclusion criteria: in order to be eligible to ... api
Description Previous: Background: x-linked severe combined immunodefici... Current: Background: x-linked severe combined immunodefici... api
Editor type Previous: base editor Current: undisclosed base editor curator
1790139600000
X-linked Severe Combined Immunodeficiency (XSCID)
National Institute of Allergy and Infectious Diseases (NIAID) Enrolling by invitation
NCT06364774
Last update post date Previous: 2026-04-15 Current: 2026-09-23 api
Vector type Previous: LV Current: Lentivirus curator
Editor type Previous: none Current: curator
Eligibility max age Previous: 35 years Current: 40 years api
Eligibility criteria Previous: Inclusion criteria: 1. age 18 to \< 36 years at t... Current: Inclusion criteria: 1. age 18 to \< 40 years at t... api
1790139600000
Beta-Thalassemia Major
Children's Hospital of Philadelphia Recruiting
NCT03588299
Last update post date Previous: 2026-07-06 Current: 2026-09-23 api
Overall status Previous: Active not recruiting Current: Completed api
Vector type Previous: AAVhu37 Current: Adeno-associated virus curator
Editor type Previous: none Current: curator
Estimated completion date Previous: 2026-11-03 Current: 2026-09-15 api
Eligibility criteria Previous: null Current: Inclusion criteria: * males age 18 years or older... api
1790139600000
Hemophilia A
Bayer Completed
License reverted to Ultragenyx in 2022, uncertain development status
NCT07587021
Last update post date Previous: 2026-09-08 Current: 2026-09-23 api
Number of locations Previous: 1 Current: 4 api
Overall status Previous: Not yet recruiting Current: Recruiting api
Locations Previous: Current: United states api
Description Previous: This study will be conducted to evaluate the effic... Current: This study will be conducted to evaluate the effic... api
Vector type Previous: LNP Current: curator
Indication doid Previous: Current: DOID:0111670 curator
Development status Previous: Current: Active curator
Record status Previous: Draft Current: Active curator
Compound name Previous: Current: YOLT-203 curator
Recent updates Previous: Current: US IND application cleared November 2025 curator
Dose 1 Previous: Current: Early Phase 1 doses: 0.3 mg/kg, 0.6 mg/kg, 1.0 mg/... curator
Editor type Previous: Current: YolCas12 curator
Delivery system Previous: Current: Endocytosis curator
Drug product type Previous: Current: Encapsulated RNP 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: HAO1 curator
Fda designation Previous: Current: Orphan Drug Designation, Rare Pediatric Disease De... curator
Related nctid Previous: null Current: LinkName:Early Phase 1: NCT06511349;LinkUrl:https:... curator
News and press releases Previous: null Current: LinkName:YolTech Receives U.S. FDA ODD for YOLT-20... curator
News and press releases Previous: null Current: LinkName:YolTech Therapeutics Receives FDA IND Cle... curator
News and press releases Previous: null Current: LinkName:Success for YolTech's hyperoxaluria in vi... curator
News and press releases Previous: null Current: LinkName:YolTech Therapeutics Administers First Pa... curator
News and press releases Previous: null Current: LinkName:YolTech Receives U.S. FDA RPDD for YOLT-2... curator
News and press releases Previous: null Current: LinkName:YolTech Therapeutics Receives FDA IND Cle... curator
Preclinical publications Previous: null Current: LinkName:Efficient and safe in vivo treatment of p... curator
Preclinical publications Previous: null Current: LinkName:Library-Assisted Evolution in Eukaryotic ... curator
1790139600000
Primary Hyperoxaluria Type 1, PH1
YolTech Therapeutics Co., Ltd Recruiting
US IND application cleared November 2025
NCT06910813
Last update post date Previous: 2026-07-31 Current: 2026-09-22 api
Number of locations Previous: 5 Current: 6 api
Estimated completion date Previous: 2044-03-14 Current: 2044-05-28 api
Vector type Previous: LV Current: Lentivirus curator
Compound description Previous: Current: pCCL-EFS-CTNS-WPRE curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: Key inclusion criteria: participants eligible for... api
Recent updates Previous: AVROBIO sold product to Novartis in May 2023 Current: clinical results published 2/18/26 curator
Clinical publications Previous: null Current: LinkName:Hematopoietic Stem-Cell Gene Therapy for ... curator
1790053200000
Nephropathic Cystinosis
Novartis Pharmaceuticals Recruiting
Clinical results published 2/18/26
14 more update(s) recently — View All
Disclaimer: The information on this dashboard has been collected for the convenience of patients and researchers. The SCGE team are not medical doctors and cannot provide medical advice. Please discuss with your provider the risks/benefits of participating in a clinical trial, and do not send us your personal medical information. The information contained within this table does not make use of any confidential or privileged information-all data is collected from publicly available sources. The SCGE makes no comment as to the efficacy and safety of the items listed, as these are not known at the time of publication. For the most up to date information, or to inquire about enrollment, please refer to clinicaltrials.gov or the Sponsor's website for contact information.