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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 243 results in ClinicalTrials
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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: 1785906000000 Enrollment: 576 Puerto Rico, United States
NCT05788536 Recruiting Phase2, Phase1
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Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Sponsor Regeneron Pharmaceuticals (Industry)
Compound OTARMENI
Therapeutic Modality Gene transfer
Target Gene OTOF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-03-15 Last Modified Date: 1785906000000 Enrollment: 36 Germany, Japan, Spain + 2 more
NCT07226206 Recruiting Phase2, Phase1
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Hemophilia A
Sponsor Hoffmann-La Roche (Industry)
Compound SPK-8011QQ
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-10-28 Last Modified Date: 1785819600000 Enrollment: 5 United States
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: 1785819600000 Enrollment: 15 United States
NCT02599922 Unknown Phase2, Phase1
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Achromatopsia
Sponsor Beacon Therapeutics (Industry)
Compound AGTC-401
Therapeutic Modality Gene transfer
Target Gene CNGB3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2015-11-05 Last Modified Date: 1785733200000 Enrollment: 32 United States
NCT03920007 Active not recruiting Phase2, Phase1
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Leber Congenital Amaurosis, LCA, LCA1
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-101
Therapeutic Modality Gene transfer
Target Gene GUCY2D
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-04-10 Last Modified Date: 1785733200000 Enrollment: 15 United States
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
First Submitted: 2023-06-30 Last Modified Date: 1785474000000 Enrollment: 60 United States
NCT05164471 Terminated Phase2, Phase1
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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
NCT05956626 Active not recruiting Phase3, Phase2
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Stargardt Disease
Sponsor Ocugen (Industry)
Compound OCU410ST
Therapeutic Modality Gene transfer
Target Gene RORA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2023-06-30 Last Modified Date: 1785474000000 Enrollment: 51 United States
NCT06910813 Recruiting Phase2, Phase1
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Nephropathic Cystinosis
Sponsor Novartis Pharmaceuticals (Industry)
Compound DFT383
Therapeutic Modality Gene transfer
Target Gene CTNS
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-03-17 Last Modified Date: 1785474000000 Enrollment: 30 United States
NCT07667387 Not yet 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: 1785474000000 Enrollment: 7 United States
NCT06492850 Unknown Phase2, Phase1
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X-Linked Retinitis Pigmentosa (XLRP)
Sponsor Frontera Therapeutics (Industry)
Compound FT-002
Therapeutic Modality Gene transfer
Target Gene RPGRORF15
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2024-07-01 Last Modified Date: 1785474000000 Enrollment: 32 China
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: 1785301200000 Enrollment: 47 Australia, United Kingdom, United States
NCT06063850 Recruiting Phase2, Phase1
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Mesial Temporal Lobe Epilepsy
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-260
Therapeutic Modality Gene transfer
Target Gene MiGRIK2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
First Submitted: 2023-09-01 Last Modified Date: 1785301200000 Enrollment: 12 United States
NCT01306019 Recruiting Phase2, Phase1
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X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound VSV-G pseudotyped CL20-i4-EF1α-hγc-OPT vector
Therapeutic Modality Gene transfer
Target Gene IL2RG
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2011-02-26 Last Modified Date: 1785214800000 Enrollment: 40 United States
NCT06185673 Recruiting Phase2, Phase1
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Oculopharyngeal Muscular Dystrophy
Sponsor Benitec Biopharma, Inc. (Industry)
Compound BB-301
Therapeutic Modality Gene transfer
Target Gene PABPN1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement, Gene inactivation
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2023-12-15 Last Modified Date: 1784869200000 Enrollment: 30 United States
NCT01422772 Completed Phase2, Phase1
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Ischemic Heart Disease
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2011-08-21 Last Modified Date: 1784869200000 Enrollment: 9 South Korea
NCT05361031 Completed Phase2, Phase1
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Charcot-Marie-Tooth Disease 1A
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2022-03-31 Last Modified Date: 1784869200000 Enrollment: 12 South Korea
NCT03363165 Completed Phase2
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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
NCT07293546 Enrolling by invitation Phase2, Phase1
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FOXG1 Syndrome
Sponsor FOXG1 Research Foundation (Other)
Compound FRF-001
Therapeutic Modality Gene transfer
Target Gene FOXG1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-12-16 Last Modified Date: 1784869200000 Enrollment: 12 United States
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: 1784869200000 Enrollment: 10 United States
NCT05176093 Completed Phase2
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Amyotrophic Lateral Sclerosis (ALS)
Sponsor Helixmith Co., Ltd. (Industry)
Compound ENGENSIS
Therapeutic Modality Gene transfer
Target Gene HGF
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2021-11-05 Last Modified Date: 1784869200000 Enrollment: 8 South Korea, United States
NCT05821959 Recruiting Phase2, Phase1
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Bilateral Sensorineural Hearing Loss
Sponsor Akouos, Inc. (Industry)
Compound AK-OTOF
Therapeutic Modality Gene transfer
Target Gene OTOF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-02-08 Last Modified Date: 1784869200000 Enrollment: 22 Canada, Taiwan, United Kingdom + 1 more
NCT05603312 Completed Phase2, Phase1
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Parkinson's Disease
Sponsor MeiraGTx, LLC (Industry)
Compound AAV-GAD
Therapeutic Modality Gene transfer
Target Gene GAD1/2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2022-10-12 Last Modified Date: 1784869200000 Enrollment: 14 United States
NCT01064440 Completed Phase2
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Critical Limb Ischemia
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: 2010-02-04 Last Modified Date: 1784869200000 Enrollment: 52 South Korea, United States
NCT05791864 Recruiting Phase2, Phase1
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Neuronal Ceroid Lipofuscinosis Type 2
Sponsor Tern Therapeutics, LLC (Industry)
Compound TTX-381
Therapeutic Modality Gene transfer
Target Gene TPP1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-03-17 Last Modified Date: 1784869200000 Enrollment: 16 Germany, United Kingdom
NCT05926765 Active not recruiting Phase2
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Grade 2 and 3 Radiation-Induced Late Xerostomia
Sponsor MeiraGTx, LLC (Industry)
Compound AAV-hAQP1
Therapeutic Modality Gene transfer
Target Gene AQP1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2023-06-22 Last Modified Date: 1784869200000 Enrollment: 290 Canada, United Kingdom, United States
NCT04124042 Completed Phase2
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Osteoarthritis, Knee
Sponsor Xalud Therapeutics, Inc. (Industry)
Compound XT-150
Therapeutic Modality Gene transfer
Target Gene Recombinant IL10
Delivery System Not Applicable
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2019-10-09 Last Modified Date: 1784869200000 Enrollment: 289 Australia, United States
NCT05445323 Active not recruiting Phase2, Phase1
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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: 2022-06-23 Last Modified Date: 1784869200000 Enrollment: 8 United States
NCT07185256 Recruiting Phase2, Phase1
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ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
Sponsor Opus Genetics, Inc (Industry)
Compound OPGx-BEST1
Therapeutic Modality Gene transfer
Target Gene BEST1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-07-17 Last Modified Date: 1784782800000 Enrollment: 10 United States
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: 1784782800000 Enrollment: 10 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: 1784782800000 Enrollment: 54 United States
NCT02186418 Terminated Phase2, Phase1
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Sickle Cell Disease
Sponsor Children's Hospital Medical Center, Cincinnati (Other)
Compound ARU-1801
Therapeutic Modality Gene transfer
Target Gene HBG(G16D)
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2014-06-30 Last Modified Date: 1784782800000 Enrollment: 7 United States
NCT06611436 Terminated Phase2, Phase1
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Hemophilia B
Sponsor Be Biopharma (Industry)
Compound BE-101
Therapeutic Modality Gene editing
Target Gene F9
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2024-09-18 Last Modified Date: 1784782800000 Enrollment: 5 United States
NCT05739643 Active not recruiting Phase2, Phase1
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Krabbe Disease
Sponsor Forge Biologics, Inc (Industry)
Compound FBX-101
Therapeutic Modality Gene transfer
Target Gene GALC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-02-13 Last Modified Date: 1784782800000 Enrollment: 9 United States
NCT05293626 Active not recruiting Phase2, Phase1
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Leber Hereditary Optic Neuropathy (LHON)
Sponsor Neurophth Therapeutics Inc (Other)
Compound OPVIKA
Therapeutic Modality Gene transfer
Target Gene ND4 m.11778G>A
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2021-12-09 Last Modified Date: 1784782800000 Enrollment: 12 United States
NCT07058662 Recruiting Phase2, Phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor Belief BioMed (Beijing) Co., Ltd (Industry)
Compound BBM-D101
Therapeutic Modality Gene transfer
Target Gene Undisclosed
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Undisclosed
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-07-01 Last Modified Date: 1784782800000 Enrollment: 9 China
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: 1784782800000 Enrollment: 7 United Kingdom, United States
NCT02168686 Completed Phase2, Phase1
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Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Adverum Biotechnologies, Inc. (Industry)
Compound ADVM-043
Therapeutic Modality Gene transfer
Target Gene SERPINA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2014-06-10 Last Modified Date: 1784782800000 Enrollment: 6 United States
NCT06389877 Recruiting Phase2, Phase1
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Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-302
Therapeutic Modality Gene editing
Target Gene SERPINA1 (p.Glu366Lys)
Delivery System Endocytosis
Mechanism of Action Mutation correction
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-04-22 Last Modified Date: 1784782800000 Enrollment: 106 Australia, Ireland, Netherlands + 3 more
NCT05203679 Active not recruiting Phase3, Phase2
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Hemophilia B
Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound BBM-H901
Therapeutic Modality Gene transfer
Target Gene F9
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-12-29 Last Modified Date: 1784782800000 Enrollment: 32 China
NCT04408625 Active not recruiting Phase2, Phase1
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Frontotemporal Dementia
Sponsor Prevail Therapeutics (Industry)
Compound LY3884963
Therapeutic Modality Gene transfer
Target Gene GRN
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-05-21 Last Modified Date: 1784782800000 Enrollment: 35 Australia, Belgium, France + 3 more
NCT05456880 Active not recruiting Phase2, Phase1
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Sickle Cell Disease
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-101
Therapeutic Modality Gene editing
Target Gene HBG1/HBG2
Delivery System Electroporation
Mechanism of Action Mutation correction
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2022-06-23 Last Modified Date: 1784782800000 Enrollment: 15 United States
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: 1784782800000 Enrollment: 22 United States
NCT04783181 Active not recruiting Phase2, Phase1
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Congenital Adrenal Hyperplasia
Sponsor Adrenas Therapeutics Inc (Industry)
Compound BBP-631
Therapeutic Modality Gene transfer
Target Gene CYP21A2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-03-01 Last Modified Date: 1784782800000 Enrollment: 8 United States
NCT02341807 Completed Phase2, Phase1
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Choroideremia
Sponsor Spark Therapeutics, Inc. (Industry)
Compound SPK-7001
Therapeutic Modality Gene transfer
Target Gene CHM
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2015-01-12 Last Modified Date: 1784782800000 Enrollment: 15 United States
NCT04676048 Recruiting Phase2, Phase1
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Hemophilia A
Sponsor ASC Therapeutics (Industry)
Compound ASC618
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-12-15 Last Modified Date: 1784782800000 Enrollment: 12 United States
NCT02453477 Unknown Phase2, Phase1
View Report
Beta-Thalassemia Major
Sponsor IRCCS San Raffaele (Other)
Compound OTL-300
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2015-05-15 Last Modified Date: 1784782800000 Enrollment: 10 Italy
NCT04611503 Active not recruiting Phase2, Phase1
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Retinitis Pigmentosa
Sponsor STZ eyetrial (Other)
Compound RAAV.hPDE6A
Therapeutic Modality Gene transfer
Target Gene PDE6A
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2020-05-20 Last Modified Date: 1784696400000 Enrollment: 9 Germany
NCT03368742 Active not recruiting Phase2, Phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-001
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-12-05 Last Modified Date: 1784696400000 Enrollment: 12 United States
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: 1784696400000 Enrollment: 9 France
NCT03300453 Completed Phase2, Phase1
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Sanfilippo Syndrome B
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-110
Therapeutic Modality Gene transfer
Target Gene NAGLU
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-06-15 Last Modified Date: 1784696400000 Enrollment: 4 France
NCT06480461 Active not recruiting Phase2, Phase1
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Parkinson's Disease
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGN-R09b
Therapeutic Modality Gene transfer
Target Gene DDC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track
First Submitted: 2024-06-14 Last Modified Date: 1784696400000 Enrollment: 39 China
NCT04278131 Recruiting Phase2, Phase1
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Retinitis Pigmentosa
Sponsor Bionic Sight LLC (Industry)
Compound BS01
Therapeutic Modality Gene transfer
Target Gene ChronosFP
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: 2020-02-13 Last Modified Date: 1784696400000 Enrollment: 20 United States
NCT04046224 Completed Phase2, Phase1
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Fabry Disease
Sponsor Sangamo Therapeutics (Industry)
Compound ST-920
Therapeutic Modality Gene transfer
Target Gene GLA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2019-08-01 Last Modified Date: 1784696400000 Enrollment: 36 Australia, Canada, Germany + 4 more
NCT03612869 Unknown Phase3, Phase2
View Report
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: 1784696400000 Enrollment: 20 France, Germany, Netherlands + 2 more
NCT04240314 Completed Phase2, Phase1
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Duchenne Muscular Dystrophy (DMD)
Sponsor Megan Waldrop (Other)
Compound AT702
Therapeutic Modality Gene transfer
Target Gene DMD (exon 2)
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Exon skipping/splice editor
First Submitted: 2020-01-22 Last Modified Date: 1784696400000 Enrollment: 3 United States
NCT02935517 Active not recruiting 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: 1784696400000 Enrollment: 24 Israel, United States
NCT05071222 Recruiting Phase2, Phase1
View Report
Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency
Sponsor Assistance Publique - Hôpitaux de Paris (Other)
Compound ARTEGENE
Therapeutic Modality Gene transfer
Target Gene DCLRE1C
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-08-27 Last Modified Date: 1784696400000 Enrollment: 7 France
NCT00076557 Terminated Phase2, Phase1
View Report
Hemophilia B
Sponsor Avigen (Industry)
Compound AAV2-hFIX16
Therapeutic Modality Gene transfer
Target Gene F9
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2004-01-26 Last Modified Date: 1784696400000 Enrollment: 15 United States
NCT04145037 Terminated Phase2, Phase1
View Report
Gaucher Disease Type 1
Sponsor AVROBIO (Industry)
Compound AVR-RD-02
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-08-27 Last Modified Date: 1784696400000 Enrollment: 8 Canada, United States
NCT06285643 Recruiting Phase2
View Report
Parkinson's Disease
Sponsor AskBio Inc (Industry)
Compound AB-1005
Therapeutic Modality Gene transfer
Target Gene GDNF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2024-02-14 Last Modified Date: 1784696400000 Enrollment: 127 Germany, Poland, United Kingdom + 1 more
NCT04945772 Completed Phase2
View Report
Retinitis Pigmentosa, Retinal Dystrophies, Retinal Degeneration
Sponsor Nanoscope Therapeutics Inc. (Industry)
Compound MCO-010
Therapeutic Modality Gene transfer
Target Gene MCO
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2021-06-15 Last Modified Date: 1784696400000 Enrollment: 27 Puerto Rico, United States
NCT05092685 Recruiting Phase2, Phase1
View Report
Ornithine Transcarbamylase (OTC) Deficiency
Sponsor University College, London (Other)
Compound BGT-OTCD
Therapeutic Modality Gene transfer
Target Gene OTC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2021-09-28 Last Modified Date: 1784696400000 Enrollment: 12 United Kingdom
NCT04394286 Terminated Phase2, Phase1
View Report
Hemophilia B
Sponsor Baxalta now part of Shire (Industry)
Compound SHP648
Therapeutic Modality Gene transfer
Target Gene F9
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2020-05-15 Last Modified Date: 1784696400000 Enrollment: 2 Spain, Turkey (Türkiye)
NCT03580083 Suspended Phase2, Phase1
View Report
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
Sponsor REGENXBIO Inc. (Industry)
Compound RGX-111
Therapeutic Modality Gene transfer
Target Gene IDUA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-06-18 Last Modified Date: 1784696400000 Enrollment: 21 Brazil, United States
NCT05243017 Active not recruiting Phase2, Phase1
View Report
Huntington's Disease
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-130
Therapeutic Modality Gene transfer
Target Gene MiHTT
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2021-11-01 Last Modified Date: 1784696400000 Enrollment: 14 Poland, United Kingdom
NCT05454566 Not yet recruiting Phase2, Phase1
View Report
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: 1784696400000 Enrollment: 18
NCT04819841 Recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Kamau Therapeutics (Industry)
Compound KMAU-001
Therapeutic Modality Gene editing
Target Gene HBB
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Mutation correction
First Submitted: 2021-03-24 Last Modified Date: 1784696400000 Enrollment: 15 United States
NCT04273269 Terminated Phase2, Phase1
View Report
GM1 Gangliosidosis
Sponsor LYSOGENE (Industry)
Compound LYS-GM101
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: 2020-01-21 Last Modified Date: 1784696400000 Enrollment: 5 France, United Kingdom, United States
NCT03374657 Completed Phase2, Phase1
View Report
Retinitis Pigmentosa
Sponsor Novartis Pharmaceuticals (Industry)
Compound CPK850
Therapeutic Modality Gene transfer
Target Gene RLBP1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-12-11 Last Modified Date: 1784696400000 Enrollment: 12 Sweden
NCT06818838 Recruiting Phase2, Phase1
View Report
Gaucher Disease Type 1
Sponsor Lingyi Biotech Co., Ltd. (Industry)
Compound LY-M001
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-01-24 Last Modified Date: 1784696400000 Enrollment: 12 China
NCT00985517 Completed Phase2, Phase1
View Report
Idiopathic Parkinson's Disease
Sponsor Sangamo Therapeutics (Industry)
Compound CERE-120
Therapeutic Modality Gene transfer
Target Gene NRTN
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2009-09-25 Last Modified Date: 1784696400000 Enrollment: 57 United States
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: 1784696400000 Enrollment: 12 United States
NCT04998396 Recruiting Phase2, Phase1
View Report
Canavan Disease
Sponsor Aspa Therapeutics (Industry)
Compound BBP-812
Therapeutic Modality Gene transfer
Target Gene ASPA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-08-05 Last Modified Date: 1784696400000 Enrollment: 26 United States
NCT00976352 Completed Phase2, Phase1
View Report
Glycogen Storage Disease Type 2 (Pompe Disease)
Sponsor University of Florida (Other)
Compound AAV1-CMV-hGAA
Therapeutic Modality Gene transfer
Target Gene GAA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2009-07-13 Last Modified Date: 1784696400000 Enrollment: 9 United States
NCT06819514 Not yet recruiting Phase2, Phase1
View Report
Fabry Disease
Sponsor Guangzhou Jiayin Biotech Ltd (Industry)
Compound EXG110
Therapeutic Modality Gene transfer
Target Gene GLA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2025-01-22 Last Modified Date: 1784696400000 Enrollment: 16 China
NCT04127578 Active not recruiting Phase2, Phase1
View Report
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: 1784696400000 Enrollment: 32 Israel, United States
NCT03311503 Recruiting Phase2, Phase1
View Report
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor David Williams (Other)
Compound G2SCID lentiviral vector transduced CD34+ cells
Therapeutic Modality Gene transfer
Target Gene IL2RG
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-10-12 Last Modified Date: 1784696400000 Enrollment: 12 United States
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: 1784696400000 Enrollment: 45 Canada, United States
NCT02416622 Completed Phase2, Phase1
View Report
X-Linked Retinoschisis
Sponsor Beacon Therapeutics (Industry)
Compound RAAV2tYF-CB-hRS1
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2015-04-02 Last Modified Date: 1784696400000 Enrollment: 27 United States
NCT04797260 Suspended Phase2, Phase1
View Report
Severe Combined Immunodeficiency Due to RAG1 Deficiency
Sponsor Videja B.V. (Industry)
Compound Autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector
Therapeutic Modality Gene transfer
Target Gene RAG1
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-03-11 Last Modified Date: 1784696400000 Enrollment: 10 Netherlands, Poland, Spain + 1 more
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: 1784696400000 Enrollment: 26 China
NCT03454893 Terminated Phase2, Phase1
View Report
Fabry Disease
Sponsor AVROBIO (Industry)
Compound AVR-RD-01
Therapeutic Modality Gene transfer
Target Gene GLA
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2017-12-20 Last Modified Date: 1784696400000 Enrollment: 15 Australia, Brazil, United States
NCT05748873 Active not recruiting Phase2, Phase1
View Report
Retinitis Pigmentosa
Sponsor SparingVision (Industry)
Compound SPVN06
Therapeutic Modality Gene transfer
Target Gene NXNL1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-02-07 Last Modified Date: 1784696400000 Enrollment: 33 France, United States
NCT04566445 Terminated Phase2
View Report
Dry Age-related Macular Degeneration
Sponsor Gyroscope Therapeutics Limited (Industry)
Compound GT005
Therapeutic Modality Gene transfer
Target Gene CFI
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2020-09-09 Last Modified Date: 1784696400000 Enrollment: 255 Australia, France, Germany + 4 more
NCT04418427 Completed Phase2
View Report
Diabetic Macular Edema, Diabetic Retinopathy
Sponsor Adverum Biotechnologies, Inc. (Industry)
Compound Ixoberogene soroparvovec
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
First Submitted: 2020-05-28 Last Modified Date: 1784696400000 Enrollment: 36 Puerto Rico, United States
NCT05824169 Recruiting Phase2, Phase1
View Report
Spinal Muscular Atrophy
Sponsor GeneCradle Inc (Industry)
Compound GC101
Therapeutic Modality Gene transfer
Target Gene SMN1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2023-04-10 Last Modified Date: 1784696400000 Enrollment: 18 China
NCT06515002 Terminated Phase2, Phase1
View Report
Cystic Fibrosis
Sponsor Boehringer Ingelheim (Industry)
Compound BI 3720931
Therapeutic Modality Gene transfer
Target Gene CFTR
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-07-18 Last Modified Date: 1784696400000 Enrollment: 5 France, Italy, Netherlands + 2 more
NCT02247843 Completed Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Donald B. Kohn, M.D. (Other)
Compound LentiG-βAS3-FB (contains anti-sickling mutations: T87Q, G16D, E22A)
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2014-09-20 Last Modified Date: 1784696400000 Enrollment: 4 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: 1784696400000 Enrollment: 11 China, United States
NCT03326336 Active not recruiting Phase2, Phase1
View Report
Non-syndromic Retinitis Pigmentosa
Sponsor GenSight Biologics (Industry)
Compound GS030
Therapeutic Modality Gene transfer
Target Gene ChR-tdT
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2017-10-11 Last Modified Date: 1784696400000 Enrollment: 10 France, United Kingdom, United States
NCT04713475 Active not recruiting Phase2, Phase1
View Report
GM1 Gangliosidosis, Beta-Galactosidase-1 (GLB1) Deficiency
Sponsor Gemma Biotherapeutics (Industry)
Compound PBGM01
Therapeutic Modality Gene transfer
Target Gene GLB1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-01-04 Last Modified Date: 1784696400000 Enrollment: 26 Brazil, Turkey (Türkiye), United Kingdom + 1 more
NCT01801709 Completed Phase2, Phase1
View Report
Metachromatic Leukodystrophy
Sponsor Institut National de la Santé Et de la Recherche Médicale, France (Other gov)
Compound AAVrh.10-CAG/cuARSA
Therapeutic Modality Gene transfer
Target Gene ARSA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2013-01-28 Last Modified Date: 1784696400000 Enrollment: 5 France
NCT03315182 Terminated Phase2, Phase1
View Report
Mucopolysaccharidosis Type 3 B
Sponsor Abeona Therapeutics, Inc (Industry)
Compound ABO-101
Therapeutic Modality Gene transfer
Target Gene NAGLU
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-10-10 Last Modified Date: 1784696400000 Enrollment: 11 France, Germany, Spain + 1 more
NCT06196840 Active not recruiting Phase2
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Innostellar Biotherapeutics Co.,Ltd (Industry)
Compound LX102
Therapeutic Modality Gene transfer
Target Gene VEGF-trap
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2023-12-25 Last Modified Date: 1784696400000 Enrollment: 50 China
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: 1784696400000 Enrollment: 9 Canada, United States
NCT03199469 Active not recruiting Phase3, Phase2
View Report
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: 1784696400000 Enrollment: 27 Canada, France, Germany + 1 more
NCT06662188 Recruiting Phase2, Phase1
View Report
SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Sponsor Jaguar Gene Therapy, LLC (Industry)
Compound JAG201
Therapeutic Modality Gene transfer
Target Gene SHANK3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Rare Pediatric Disease Designation
First Submitted: 2024-10-16 Last Modified Date: 1784696400000 Enrollment: 6 United States
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: 1784696400000 Enrollment: 25 United States

Clinical Trials Daily Digest

Updates in the last 14 days

NCT07592273
Last update post date Previous: 2026-07-29 Current: 2026-08-05 api
Number of locations Previous: 18 Current: 23 api
Vector type Previous: AAV8 Current: Adeno-associated virus curator
Recent updates Previous: First patient has been dosed in the Phase IIb/III ... Current: First patient has been dosed in the Phase IIb/III ... curator
Dose 1 Previous: Phase 2 doses: 2.5E11 GC/eye, 5.0E11 GC/eye,1.0E12... Current: Phase 2 doses: 2.5E11 GC/eye, 5.0E11 GC/eye, 1.0E1... curator
Clinical publications Previous: null Current: LinkName:(Presentation) Suprachoroidal Delivery of... curator
News and press releases Previous: null Current: LinkName:REGENXBIO Presents Positive Long-Term Dat... curator
Related nctid Previous: LinkName:Phase 2: NCT06942520 (uses subretinal inj... Current: LinkName:Phase 2: NCT06942520 (using subretinal in... curator
News and press releases Previous: null Current: LinkName:REGENXBIO Announces First Patient Dosed i... curator
Locations Previous: United states Current: Puerto rico, United states api
Indication doid Previous: DOID:9191, DOID:8947 Current: DOID:8947 curator
Record status Previous: Draft Current: Active curator
Dose 3 Previous: 1.0E12 GC/eye Current: curator
Dose 2 Previous: 5.0E11 GC/eye Current: Phase 3 dose: 1.0E12 GC/eye curator
News and press releases Previous: null Current: LinkName:REGENXBIO Announces Pivotal Program for S... curator
Development status Previous: Current: Active curator
Compound name Previous: Current: Surabgene lomparvovec curator
Delivery system Previous: Current: Viral transduction curator
Drug product type Previous: Current: Viral vector curator
Route of administration Previous: Current: Suprachoroidal curator
Mechanism of action Previous: Current: Genetic delivery of therapeutic protein curator
Therapy route Previous: Current: In-vivo curator
Therapy type Previous: Current: Gene transfer curator
Target gene Previous: Current: Anti-VEGF curator
Alias type Previous: null Current: proprietary name curator
Alias value Previous: null Current: ABBV-RGX-314 curator
Related nctid Previous: null Current: LinkName:Long Term Follow-Up: NCT05296447;LinkUrl:... curator
Related nctid Previous: null Current: LinkName:Phase 2: NCT04567550;LinkUrl:https://clin... curator
News and press releases Previous: null Current: LinkName:REGENXBIO Reports Third Quarter 2025 Fina... curator
Clinical publications Previous: null Current: LinkName:(Presentation) Suprachoroidal Surabgene L... curator
Estimated completion date Previous: 2036-02 Current: 2036-01 api
Overall status Previous: Not yet recruiting Current: Recruiting api
1785906000000
Diabetic Retinopathy
AbbVie Recruiting
First patient has been dosed in the Phase IIb/III trial using suprachoroidal delivery. Phase IIb data was presented at the ASRS annual meeting in July...
NCT05788536
Last update post date Previous: 2026-07-01 Current: 2026-08-05 api
Number of locations Previous: 16 Current: 17 api
Vector type Previous: dual AAV1 Current: Adeno-associated virus curator
Browse condition terms Previous: Db-oto, Gene therapy, Congenital hearing loss, Sen... Current: Db-oto, Gene therapy, Congenital hearing loss, Sen... api
Estimated completion date Previous: 2032-02-28 Current: 2032-02-25 api
Brief title Previous: A study of db-oto, An adeno-associated virus (aav)... Current: A study of db-oto, An adeno-associated virus (aav)... api
Eligibility std age Previous: Child Current: Child, Adult, Older adult api
Eligibility max age Previous: 17 years Current: null api
Eligibility criteria Previous: Key inclusion criteria: 1. willingness of at leas... Current: Key inclusion criteria: 1. willingness to provide... api
Enrollment count Previous: 30 Current: 36 api
Intervention description Previous: Db-oto will be administered as a single intracochl... Current: Administered per the protocol api
Intervention name Previous: Db-oto, Db-oto Current: Db-oto api
Description Previous: Regeneron is conducting a study of an investigatio... Current: Regeneron is conducting a study of an investigatio... api
Editor type Previous: none Current: curator
Recent updates Previous: Otarmeni was approved 4/23/26 under the National P... Current: Otarmeni was approved 4/23/26 under the National P... curator
News and press releases Previous: null Current: LinkName:Otarmeni™ (lunsotogene parvec) Receives E... curator
Locations Previous: United states, United kingdom, Germany, Spain Current: United states, Japan, United kingdom, Germany, Spa... api
Development status Previous: Active Current: Approved curator
Compound name Previous: DB-OTO Current: OTARMENI curator
Alias type Previous: null Current: proprietary name, proper name curator
Alias value Previous: null Current: DB-OTO, lunsotogene parvec curator
Fda designation Previous: Fast Track, Orphan Drug Designation, Rare Pediatri... Current: Accelerated Approval, Fast Track, Orphan Drug Desi... curator
News and press releases Previous: null Current: LinkName:FDA Approval Documents;LinkUrl:https://ww... curator
News and press releases Previous: null Current: LinkName:FDA Approves First-Ever Gene Therapy for ... curator
1785906000000
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (...
Regeneron Pharmaceuticals Recruiting
Otarmeni was approved 4/23/26 under the National Priority Voucher Program, Marketing Authorization Application was accepted for review by the EMA
NCT07195825
Last update post date Previous: 2026-01-28 Current: 2026-08-04 api
Vector type Previous: AAVT42 Current: Adeno-associated virus curator
Compound description Previous: Current: AAVT42-hSyn-TH90del-T2A-AADC-WPRE curator
Recent updates Previous: Current: New results published June 2026, safety and tolera... curator
Dose 1 Previous: Undisclosed Current: Dose escalation phase: 4.0E11 vg (Cohort 1, n=1), ... curator
Target tissue Previous: Current: Putamen curator
Mechanism of action Previous: Undisclosed Current: Overexpression of protective allele/gene curator
Therapy type Previous: Undisclosed Current: Gene transfer curator
Target gene Previous: Undisclosed Current: TH90del-AADC curator
Clinical publications Previous: null Current: LinkName:Dual-target gene therapy in Parkinson’s d... curator
Eligibility criteria Previous: null Current: Inclusion criteria: 1. participants with a diagno... api
1785819600000
Parkinson's Disease
Shanghai Xinzhi BioMed Co., Ltd. Recruiting
New results published June 2026, safety and tolerability, no treatment-related SAEs or DLTs
NCT05878860
Last update post date Previous: 2026-07-21 Current: 2026-08-04 api
Number of locations Previous: 18 Current: 19 api
Locations Previous: United states Current: United states, United kingdom api
Vector type Previous: AAV.SPR Current: Adeno-associated virus curator
News and press releases Previous: LinkName: Atsena Therapeutics Doses First Patient... Current: LinkName:Atsena Therapeutics Doses First Patient a... curator
Recent updates Previous: Enrollment in pivotal cohort is expected to comple... Current: First patient in pivotal cohort has been dosed. En... curator
Editor type Previous: none Current: curator
Dose 5 Previous: 2.3E10 vg/eye (225ul) Current: curator
Dose 4 Previous: 1.5E10 vg/eye (150ul) Current: curator
Dose 3 Previous: 5.0E10 vg/eye Current: Phase 3: 1.1E10 vg/eye; n=39 curator
Dose 2 Previous: 3.0E10 vg/eye Current: Phase 1/2 Part B: 1.1E10 vg/eye, 1.7 vg/eye; n=3/c... curator
Dose 1 Previous: 1.5E10 vg/eye Current: Phase 1/2 Part A: 1.1E10 vg/eye, 3.8E10 vg/eye, 2.... curator
Clinical publications Previous: LinkName:Safety and efficacy of ATSN-101 in patien... Current: null curator
Clinical publications Previous: null Current: LinkName:(Presentation) Safety and efficacy of ATS... curator
Clinical publications Previous: null Current: LinkName:(Presentation) Safety and efficacy of ATS... curator
News and press releases Previous: null Current: LinkName:Atsena Presents Positive Clinical Data fr... curator
News and press releases Previous: LinkName: Atsena Presents Positive Interim Six-Mo... Current: LinkName:Atsena Presents Positive Interim Six-Mont... curator
News and press releases Previous: LinkName: Atsena Therapeutics Receives Data Monit... Current: LinkName:Atsena Therapeutics Receives Data Monitor... curator
Estimated completion date Previous: 2029-10 Current: 2033-04 api
Official title Previous: A phase 1/2, Open-label, Dose escalation and dose ... Current: A phase 1/2/3, Open-label, Dose escalation, Dose e... api
Eligibility criteria Previous: Inclusion criteria: 1. age ≥ 18 for cohorts 1 thr... Current: Part a and b: inclusion criteria: 1. age ≥ 18 fo... api
Eligibility sex Previous: Male Current: All api
Enrollment count Previous: 21 Current: 97 api
Phases Previous: Phase1, Phase2 Current: Phase3 api
Description Previous: This study will evaluate the safety and tolerabili... Current: This study will evaluate the safety and efficacy o... api
1785819600000
X-Linked Retinoschisis
Atsena Therapeutics Inc. Recruiting
First patient in pivotal cohort has been dosed. Enrollment in pivotal cohort is expected to complete by end of Q1 2027, topline results expected 1H202...
NCT07226206
Last update post date Previous: 2026-07-14 Current: 2026-08-04 api
Number of locations Previous: 2 Current: 3 api
Vector type Previous: LK03 Current: Adeno-associated virus curator
Editor type Previous: none Current: curator
Overall status Previous: Not yet recruiting Current: Recruiting api
Compound description Previous: AAV LK03 capsid + TTRmut-hFVIII-X07 Current: AAV LK03 capsid + TTRmut + APC-resistant FVIII var... curator
Preclinical publications Previous: null Current: LinkName:Activated protein C has a regulatory role... curator
Indication doid Previous: null Current: DOID:12134 curator
Development status Previous: null Current: Active curator
Record status Previous: Provisional Current: Active curator
Compound name Previous: null Current: SPK-8011QQ curator
Recent updates Previous: null Current: Study was withdrawn by Sponsor (no participants we... curator
Dose 4 Previous: null Current: 2E12 vg/kg curator
Dose 3 Previous: null Current: 1.5E12 vg/kg curator
Dose 2 Previous: null Current: 1E12 vg/kg curator
Dose 1 Previous: null Current: 5E11 vg/kg 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: F8 curator
Alias type Previous: null Current: proper name curator
Alias value Previous: null Current: Dirloctogene samoparvovec curator
Related nctid Previous: null Current: LinkName:Phase 3: NCT06297486 (Withdrawn);LinkUrl:... curator
Related nctid Previous: null Current: LinkName:Phase 1/2: NCT03003533;LinkUrl:https://ww... curator
Related nctid Previous: null Current: LinkName:Long Term Follow-Up: NCT03432520;LinkUrl:... curator
Preclinical publications Previous: null Current: LinkName:Preclinical assessment of an optimized AA... curator
Preclinical publications Previous: null Current: LinkName:(Abstract) Preclinical evaluation of SPK-... curator
News and press releases Previous: null Current: LinkName:Roche mothballs another hemophilia A gene... curator
News and press releases Previous: null Current: LinkName:Integration of Spark into Roche Group Set... curator
Clinical publications Previous: null Current: LinkName:Multiyear Factor VIII Expression after AA... curator
Eligibility criteria Previous: null Current: Inclusion criteria: * signed informed consent for... api
Locations Previous: Current: United states api
1785819600000
Hemophilia A
Hoffmann-La Roche Recruiting
Study was withdrawn by Sponsor (no participants were enrolled), Sponsor is extensively modifying the product before clinical trials resume
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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.