SCGE Platform - Gene Therapy Clinical Trials

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, patent applications, and more to give a comprehensive overview of the gene therapy clinical trial landscape.

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.

Updates in the last 14 days

NCT07282847

2026-05-28

Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD

Recruiting

FDA accepted IND in January 2026

NCT06461702

2026-05-28

Familial Hypercholesterolemia

Unknown

FDA cleared IND application in June 2025

NCT06856759

2026-05-28

Rett Syndrome

Active not recruiting

NCT07052929

2026-05-28

X-Linked Myotubular Myopathy

Recruiting

Astellas has restarted their XLMTM program by creating a new drug product ASP2957, with substantial modifications to the capsid to detarget the liver,...

NCT07592273

2026-05-28

Diabetic Retinopathy

Recruiting

This product is also under evaluation for wet AMD

Showing 1-100 of 266 results in ClinicalTrials

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NCT07282847 Recruiting Phase2, Phase1

View Report

Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD

Sponsor AskBio Inc (Industry)

Compound AB-1009

Therapy Type Gene transfer

Target Gene GAA

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT07052929 Recruiting Phase2, Phase1

View Report

X-Linked Myotubular Myopathy

Sponsor Astellas Gene Therapies (Industry)

Compound ASP2957

Therapy Type Gene transfer

Target Gene MTM1

Vector Type AAV3.8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05419492 Recruiting Phase2, Phase1

View Report

Dravet Syndrome

Sponsor Encoded Therapeutics (Industry)

Compound ETX101

Therapy Type Gene transfer

Target Gene SCN1A

Vector Type AAV9

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

NCT02725580 Completed Phase2, Phase1

View Report

Variant Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease)

Sponsor Emily de los Reyes (Other)

Compound AT-GTX-501

Therapy Type Gene transfer

Target Gene CLN6

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT02599922 Active not recruiting Phase2, Phase1

View Report

Achromatopsia

Sponsor Beacon Therapeutics (Industry)

Compound AGTC-401

Therapy Type Gene transfer

Target Gene CNGB3

Vector Type AAV2tYF

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT05361031 Completed Phase2, Phase1

View Report

Charcot-Marie-Tooth Disease 1A

Sponsor Helixmith Co., Ltd. (Industry)

Compound ENGENSIS

Therapy Type Gene transfer

Target Gene HGF

Mechanism of Action Overexpression of protective allele/gene

NCT06539208 Recruiting Phase2, Phase1

View Report

Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy

Sponsor YolTech Therapeutics Co., Ltd (Industry)

Compound YOLT-201

Therapy Type Gene editing

Target Gene TTR

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Gene inactivation

NCT06389877 Recruiting Phase2, Phase1

View Report

Alpha-1 Antitrypsin Deficiency (AATD)

Sponsor Beam Therapeutics Inc. (Industry)

Compound BEAM-302

Therapy Type Gene editing

Target Gene SERPINA1 (p.Glu366Lys)

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Mutation correction

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation

NCT06164730 Recruiting Phase1

View Report

Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease

Sponsor Verve Therapeutics, Inc. (Industry)

Compound VERVE-102

Therapy Type Gene editing

Target Gene PCSK9

Vector Type LDLR + GalNAc

Delivery System Endocytosis

Mechanism of Action Gene inactivation

FDA Designations Fast Track

NCT06622668 Withdrawn Phase2, Phase1

View Report

Alpha-1 Antitrypsin Deficiency (AATD)

Sponsor Intellia Therapeutics (Industry)

Compound NTLA-3001

Therapy Type Gene editing

Target Gene SERPINA1

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Functional gene replacement

NCT06451770 Recruiting Phase1

View Report

Hypercholesterolemia

Sponsor Verve Therapeutics, Inc. (Industry)

Compound VERVE-201

Therapy Type Gene editing

Target Gene ANGPTL3

Vector Type LDLR + GalNAc

Delivery System Endocytosis

Mechanism of Action Gene inactivation

NCT06735755 Recruiting Phase2, Phase1

View Report

Glycogen Storage Disease Type Ia

Sponsor Beam Therapeutics Inc. (Industry)

Compound BEAM-301

Therapy Type Gene editing

Target Gene G6PC1 (p.R83C)

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Mutation correction

NCT06680232 Recruiting Phase1

View Report

Chronic Hepatitis B

Sponsor Precision BioSciences, Inc. (Industry)

Compound PBGENE-HBV

Therapy Type Gene editing

Target Gene HBV DNA

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Gene inactivation

FDA Designations Fast Track

NCT06517888 Recruiting Phase2, Phase1

View Report

Vestibular Schwannoma

Sponsor Akouos, Inc. (Industry)

Compound AAVAnc80-antiVEGF

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAVAnc80

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT06839235 Recruiting Phase2, Phase1

View Report

Primary Hyperoxaluria Type 1 (PH1)

Sponsor Arbor Biotechnologies (Industry)

Compound ABO-101

Therapy Type Gene editing

Target Gene HAO1

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Gene inactivation

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT01422772 Completed Phase2, Phase1

View Report

Ischemic Heart Disease

Sponsor Helixmith Co., Ltd. (Industry)

Compound ENGENSIS

Therapy Type Gene transfer

Target Gene HGF

Vector Type none

Mechanism of Action Overexpression of protective allele/gene

NCT05398029 Completed Phase1

View Report

Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease

Sponsor Verve Therapeutics, Inc. (Industry)

Compound VERVE-101

Therapy Type Gene editing

Target Gene PCSK9

Vector Type LDLR

Delivery System Endocytosis

Mechanism of Action Gene inactivation

NCT07226206 Recruiting Phase2, Phase1

View Report

Hemophilia A

Sponsor Hoffmann-La Roche (Industry)

Compound SPK-8011QQ

Therapy Type Gene transfer

Target Gene F8

Vector Type LK03

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01306019 Recruiting Phase2, Phase1

View Report

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

Therapy Type Gene transfer

Target Gene IL2RG

Vector Type VSV-G

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06736080 Not yet recruiting Phase2, Phase1

View Report

Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3)

Sponsor Assistance Publique - Hôpitaux de Paris (Other)

Compound Autologous Cells Transduced Ex Vivo with LV-UNC13D

Therapy Type Gene transfer

Target Gene UNC13D

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT07113743 Enrolling by invitation Phase2, Phase1

View Report

Chronic Granulomatous Disease (CGD)

Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)

Compound VSVG-PCCLChimGp91

Therapy Type Gene transfer

Target Gene CYBB

Vector Type VSV-G

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT03580083 Suspended Phase2, Phase1

View Report

Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome

Sponsor REGENXBIO Inc. (Industry)

Compound RGX-111

Therapy Type Gene transfer

Target Gene IDUA

Vector Type AAV2/9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT03588299 Active not recruiting Phase2, Phase1

View Report

Hemophilia A

Sponsor Bayer (Industry)

Compound DTX201

Therapy Type Gene transfer

Target Gene F8

Vector Type AAVhu37

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06817382 Recruiting Phase1

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Insmed Gene Therapy LLC (Industry)

Compound INS1201

Therapy Type Gene transfer

Target Gene Micro-dystrophin

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04747431 Active not recruiting Phase2, Phase1

View Report

Frontotemporal Dementia, FTD, FTD-GRN, C9orf72

Sponsor Passage Bio, Inc. (Industry)

Compound PBFT02

Therapy Type Gene transfer

Target Gene GRN

Vector Type AAV1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT05748873 Active not recruiting Phase2, Phase1

View Report

Retinitis Pigmentosa

Sponsor SparingVision (Industry)

Compound SPVN06

Therapy Type Gene transfer

Target Gene NXNL1

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT06217861 Recruiting Phase1

View Report

Glutaric Acidemia Type I

Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)

Compound VGM-R02b

Therapy Type Gene transfer

Target Gene GCDH

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT07287397 Recruiting Phase2, Phase1

View Report

Amyotrophic Lateral Sclerosis

Sponsor Vector Y Therapeutics (Industry)

Compound VTx-002

Therapy Type Gene transfer

Target Gene Antibody to TDP-43

Vector Type AAV5.2

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

FDA Designations Fast Track

NCT05541627 Active not recruiting Phase2, Phase1

View Report

Huntington's Disease

Sponsor AskBio France, SAS, a subsidiary of AskBio Inc. (Industry)

Compound AB-1001

Therapy Type Gene transfer

Target Gene CYP46A1

Vector Type AAVrh10

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT05504837 Recruiting Phase1

View Report

Cystic Fibrosis

Sponsor Krystal Biotech, Inc. (Industry)

Compound KB407

Therapy Type Gene transfer

Target Gene CFTR

Vector Type HSV-1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT03368742 Active not recruiting Phase2, Phase1

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Solid Biosciences Inc. (Industry)

Compound SGT-001

Therapy Type Gene transfer

Target Gene Micro-dystrophin

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06255782 Recruiting Phase2, Phase1

View Report

Ornithine Transcarbamylase (OTC) Deficiency

Sponsor iECURE, Inc. (Industry)

Compound ECUR-506

Therapy Type Gene transfer

Target Gene OTC/PCSK9 locus

Vector Type dual AAV8

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

NCT05682144 Recruiting Phase1

View Report

Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS

Sponsor Immusoft of CA, Inc. (Industry)

Compound ISP-001

Therapy Type Gene transfer

Target Gene IDUA

Vector Type Sleeping Beauty

Delivery System Electroporation

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05456880 Active not recruiting Phase2, Phase1

View Report

Sickle Cell Disease

Sponsor Beam Therapeutics Inc. (Industry)

Compound BEAM-101

Therapy Type Gene editing

Target Gene HBG1/HBG2

Vector Type none

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

NCT06064890 Recruiting Phase2, Phase1

View Report

Frontotemporal Dementia, FTD, FTD-GRN

Sponsor AviadoBio Ltd (Industry)

Compound AVB-101

Therapy Type Gene transfer

Target Gene GRN

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT05444894 Active not recruiting Phase2, Phase1

View Report

Beta-Thalassemia Major

Sponsor Editas Medicine, Inc. (Industry)

Compound EDIT-301

Therapy Type Gene editing

Target Gene HBG1/HBG2

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT04581785 Terminated Phase2, Phase1

View Report

Methylmalonic Acidemia

Sponsor LogicBio Therapeutics, Inc (Industry)

Compound LB-001

Therapy Type Gene transfer

Target Gene MMUT

Vector Type AAV-LK03

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT04680065 Active not recruiting Phase1

View Report

Multiple System Atrophy

Sponsor Brain Neurotherapy Bio, Inc. (Industry)

Compound AB-1005

Therapy Type Gene transfer

Target Gene GDNF

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT04853576 Active not recruiting Phase2, Phase1

View Report

Sickle Cell Disease

Sponsor Editas Medicine, Inc. (Industry)

Compound EDIT-301

Therapy Type Gene editing

Target Gene HBG1/HBG2

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06291961 Completed Phase1

View Report

Beta-Thalassemia Major

Sponsor CorrectSequence Therapeutics Co., Ltd (Industry)

Compound CS-101

Therapy Type Gene editing

Target Gene BCL11A

Vector Type none

Delivery System Electroporation

Mechanism of Action Overexpression of protective allele/gene

NCT06018558 Active not recruiting Phase2, Phase1

View Report

Geographic Atrophy

Sponsor Ocugen (Industry)

Compound OCU410

Therapy Type Gene transfer

Target Gene RORA

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT05788536 Recruiting Phase2, Phase1

View Report

Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

Sponsor Regeneron Pharmaceuticals (Industry)

Compound OTARMENI

Therapy Type Gene transfer

Target Gene OTOF

Vector Type dual AAV1

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

NCT06506461 Recruiting Phase1

View Report

Sickle Cell Disease

Sponsor St. Jude Children's Research Hospital (Other)

Compound Gene-modified CD34+ cells

Therapy Type Gene editing

Target Gene BCL11A

Delivery System Electroporation

Mechanism of Action Overexpression of protective allele/gene

NCT06826612 Recruiting Phase2, Phase1

View Report

Huntington's Disease

Sponsor Hoffmann-La Roche (Industry)

Compound RG6662

Therapy Type Gene transfer

Target Gene MiHTT

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Gene inactivation

NCT06765980 Recruiting Phase2, Phase1

View Report

Geographic Atrophy Secondary to Age-related Macular Degeneration

Sponsor Kriya Therapeutics, Inc. (Industry)

Compound KRIYA-825

Therapy Type Gene transfer

Target Gene CR2-CR1

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT02362438 Completed Phase1

View Report

Giant Axonal Neuropathy

Sponsor National Institute of Neurological Disorders and Stroke (NINDS) (NIH)

Compound ScAAV9/JeT-GAN

Therapy Type Gene transfer

Target Gene GAN

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06237777 Unknown Phase1

View Report

Diabetic Macular Edema

Sponsor Wang Min (Other)

Compound SKG0106

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT04408625 Active not recruiting Phase2, Phase1

View Report

Frontotemporal Dementia

Sponsor Prevail Therapeutics (Industry)

Compound LY3884963

Therapy Type Gene transfer

Target Gene GRN

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT06844214 Recruiting Phase2, Phase1

View Report

Myotonic Dystrophy

Sponsor Sanofi (Industry)

Compound SAR446268

Therapy Type Gene transfer

Target Gene DMPK

Vector Type AAV.SAN011

Delivery System Viral transduction

Mechanism of Action Gene inactivation

FDA Designations Fast Track, Orphan Drug Designation

NCT07180355 Recruiting Phase1

View Report

Friedreich's Ataxia

Sponsor Solid Biosciences Inc. (Industry)

Compound SGT-212

Therapy Type Gene transfer

Target Gene FXN

Vector Type AAVhu68

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06910813 Recruiting Phase2, Phase1

View Report

Nephropathic Cystinosis

Sponsor Novartis Pharmaceuticals (Industry)

Compound DFT383

Therapy Type Gene transfer

Target Gene CTNS

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT04998396 Recruiting Phase2, Phase1

View Report

Canavan Disease

Sponsor Aspa Therapeutics (Industry)

Compound BBP-812

Therapy Type Gene transfer

Target Gene ASPA

Vector Type AAV9

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

NCT04774536 Recruiting Phase2, Phase1

View Report

Sickle Cell Disease

Sponsor Mark Walters, MD (Other)

Compound CRISPR_SCD001

Therapy Type Gene editing

Target Gene HBB

Vector Type none

Delivery System Electroporation

Mechanism of Action Mutation correction

NCT06515002 Terminated Phase2, Phase1

View Report

Cystic Fibrosis

Sponsor Boehringer Ingelheim (Industry)

Compound BI 3720931

Therapy Type Gene transfer

Target Gene CFTR

Vector Type rSIV.F/HN

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06364774 Recruiting Phase2, Phase1

View Report

Beta-Thalassemia Major

Sponsor Children's Hospital of Philadelphia (Other)

Compound CHOP-ALS20

Therapy Type Gene transfer

Target Gene HBB

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05885412 Recruiting Phase1

View Report

PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Sponsor Rocket Pharmaceuticals Inc. (Industry)

Compound RP-A601

Therapy Type Gene transfer

Target Gene PKP2

Vector Type AAVrh.74

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation

NCT03533673 Completed Phase1

View Report

Pompe Disease

Sponsor AskBio Inc (Industry)

Compound ACTUS-101

Therapy Type Gene transfer

Target Gene GAA

Vector Type AAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track

NCT04046224 Completed Phase2, Phase1

View Report

Fabry Disease

Sponsor Sangamo Therapeutics (Industry)

Compound ST-920

Therapy Type Gene transfer

Target Gene GLA

Vector Type AAV2/6

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT05903794 Completed Phase1

View Report

Neovascular (Wet) Age-Related Macular Degeneration (nAMD)

Sponsor Exegenesis Bio (Industry)

Compound EXG102-031

Therapy Type Gene transfer

Target Gene ABD-VEGFR

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT05835895 Active not recruiting Phase1

View Report

Osteoarthritis, Knee

Sponsor Genascence Corporation (Industry)

Compound GNSC-001

Therapy Type Gene transfer

Target Gene IL1RN

Vector Type AAV2.5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track

NCT05432310 Recruiting Phase2, Phase1

View Report

Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Sponsor University of California, Los Angeles (Other)

Compound Simoladagene autotemcel

Therapy Type Gene transfer

Target Gene ADA

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06559176 Enrolling by invitation Phase2, Phase1

View Report

Chronic Granulomatous Disease

Sponsor Prime Medicine, Inc. (Industry)

Compound PM359

Therapy Type Gene editing

Target Gene NCF1 (c.75_76delGT)

Delivery System Electroporation

Mechanism of Action Mutation correction

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

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

Therapy Type Gene editing

Target Gene CYBB c.676 C>T

Vector Type none

Delivery System Electroporation

Mechanism of Action Mutation correction

NCT02651675 Terminated Phase2, Phase1

View Report

Homozygous Familial Hypercholesterolemia (HoFH)

Sponsor REGENXBIO Inc. (Industry)

Compound RGX-501

Therapy Type Gene transfer

Target Gene LDLR

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT04145037 Terminated Phase2, Phase1

View Report

Gaucher Disease Type 1

Sponsor AVROBIO (Industry)

Compound AVR-RD-02

Therapy Type Gene transfer

Target Gene GBA1

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05739643 Active not recruiting Phase2, Phase1

View Report

Krabbe Disease

Sponsor Forge Biologics, Inc (Industry)

Compound FBX-101

Therapy Type Gene transfer

Target Gene GALC

Vector Type AAVrh10

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06063850 Recruiting Phase2, Phase1

View Report

Mesial Temporal Lobe Epilepsy

Sponsor UniQure Biopharma B.V. (Industry)

Compound AMT-260

Therapy Type Gene transfer

Target Gene MiGRIK2

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action MiRNA knockdown of mutant/aberrant gene

NCT00979238 Completed Phase1

View Report

Hemophilia B

Sponsor St. Jude Children's Research Hospital (Other)

Compound ScAAV2/8-LP1-hFIXco

Therapy Type Gene transfer

Target Gene F9

Vector Type AAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06458595 Recruiting Phase2, Phase1

View Report

Neovascular (Wet) Age-Related Macular Degeneration (nAMD)

Sponsor Chengdu Origen Biotechnology Co., Ltd. (Industry)

Compound KH658

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT06662188 Recruiting Phase2, Phase1

View Report

SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome

Sponsor Jaguar Gene Therapy, LLC (Industry)

Compound JAG201

Therapy Type Gene transfer

Target Gene SHANK3

Vector Type AAV2/9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Rare Pediatric Disease Designation

NCT04411654 Active not recruiting Phase2, Phase1

View Report

Gaucher Disease Type 2

Sponsor Prevail Therapeutics (Industry)

Compound LY3884961

Therapy Type Gene transfer

Target Gene GBA1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT04611503 Active not recruiting Phase2, Phase1

View Report

Retinitis Pigmentosa

Sponsor STZ eyetrial (Other)

Compound RAAV.hPDE6A

Therapy Type Gene transfer

Target Gene PDE6A

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06185673 Recruiting Phase2, Phase1

View Report

Oculopharyngeal Muscular Dystrophy

Sponsor Benitec Biopharma, Inc. (Industry)

Compound BB-301

Therapy Type Gene transfer

Target Gene PABPN1

Vector Type AAV9-PL

Delivery System Viral transduction

Mechanism of Action Functional gene replacement, Gene inactivation

FDA Designations Fast Track, Orphan Drug Designation

NCT06480461 Active not recruiting Phase2, Phase1

View Report

Parkinson's Disease

Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)

Compound VGN-R09b

Therapy Type Gene transfer

Target Gene DDC

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track

NCT05665166 Active not recruiting Phase2, Phase1

View Report

Mucopolysaccharidosis II

Sponsor University of Manchester (Other)

Compound AVR-RD-05

Therapy Type Gene transfer

Target Gene IDS

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05454566 Not yet recruiting Phase2, Phase1

View Report

Osteoarthritis, Knee

Sponsor ICM Co. Ltd. (Industry)

Compound ICM-203

Therapy Type Gene transfer

Target Gene NKX3-2

Vector Type AAV5.2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT05071222 Recruiting Phase2, Phase1

View Report

Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency

Sponsor Assistance Publique - Hôpitaux de Paris (Other)

Compound ARTEGENE

Therapy Type Gene transfer

Target Gene DCLRE1C

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT07185256 Recruiting Phase2, Phase1

View Report

ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy

Sponsor Opus Genetics, Inc (Industry)

Compound OPGx-BEST1

Therapy Type Gene transfer

Target Gene BEST1

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05836259 Recruiting Phase2, Phase1

View Report

Hypertrophic Cardiomyopathy

Sponsor Tenaya Therapeutics (Industry)

Compound TN-201

Therapy Type Gene transfer

Target Gene MYBPC3

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06747273 Terminated Phase1

View Report

Limb-Girdle Muscular Dystrophy, Type 2D/R3

Sponsor Sarepta Therapeutics, Inc. (Industry)

Compound SRP-9004

Therapy Type Gene transfer

Target Gene SGCA

Vector Type AAVrh74

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT05973630 Active not recruiting Phase1

View Report

Limb-Girdle Muscular Dystrophy, Type 2C/R5

Sponsor Atamyo Therapeutics (Industry)

Compound ATA-200

Therapy Type Gene transfer

Target Gene SGCG

Vector Type AAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT04884815 Active not recruiting Phase2, Phase1

View Report

Wilson Disease

Sponsor Ultragenyx Pharmaceutical Inc (Industry)

Compound UX701

Therapy Type Gene transfer

Target Gene ATP7B

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT02702115 Terminated Phase2, Phase1

View Report

Mucopolysaccharidosis Type I (Hurler Syndrome)

Sponsor Sangamo Therapeutics (Industry)

Compound SB-318

Therapy Type Gene editing

Target Gene IDUA

Vector Type AAV2/6

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05614531 Enrolling by invitation Phase2, Phase1

View Report

Spinal Muscular Atrophy Type I

Sponsor Guangzhou Jiayin Biotech Ltd (Industry)

Compound EXG001-307

Therapy Type Gene transfer

Target Gene SMN1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT02852213 Recruiting Phase1

View Report

Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

Sponsor Krzysztof Bankiewicz (Other)

Compound AAV2-hAADC

Therapy Type Gene transfer

Target Gene DDC

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01519349 Completed Phase1

View Report

Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis

Sponsor Nationwide Children's Hospital (Other)

Compound RAAV1.CMV.huFollistatin344

Therapy Type Gene transfer

Target Gene FST (FS344)

Vector Type AAV1

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT02781480 Completed Phase2, Phase1

View Report

Leber Congenital Amaurosis

Sponsor MeiraGTx UK II Ltd (Industry)

Compound Cevaretigene ritoparvovec

Therapy Type Gene transfer

Target Gene RPE65

Vector Type AAV2/5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06224660 Recruiting Phase1

View Report

DMD-Associated Dilated Cardiomyopathy

Sponsor Sardocor Corp. (Industry)

Compound SRD-001

Therapy Type Gene transfer

Target Gene SERCA2a

Vector Type AAV1

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Orphan Drug Designation

NCT04186650 Active not recruiting Phase2, Phase1

View Report

Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Sponsor Institut National de la Santé Et de la Recherche Médicale, France (Other gov)

Compound EF1a-COL7A1-SIN retroviral vector transduced autologous skin

Therapy Type Gene transfer

Target Gene COL7A1

Vector Type RV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04483440 Active not recruiting Phase1

View Report

Choroideremia

Sponsor 4D Molecular Therapeutics (Industry)

Compound 4D-110

Therapy Type Gene transfer

Target Gene CHM

Vector Type AAV R100

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT02186418 Terminated Phase2, Phase1

View Report

Sickle Cell Disease

Sponsor Children's Hospital Medical Center, Cincinnati (Other)

Compound ARU-1801

Therapy Type Gene transfer

Target Gene HBG(G16D)

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT03758404 Completed Phase2, Phase1

View Report

Achromatopsia

Sponsor MeiraGTx UK II Ltd (Industry)

Compound AAV-CNGA3

Therapy Type Gene transfer

Target Gene CNGA3

Vector Type AAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06253507 Enrolling by invitation Phase2, Phase1

View Report

Autosomal Recessive Chronic Granulomatous Disease (CGD)

Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)

Compound PCHIM-p47

Therapy Type Gene transfer

Target Gene NCF1

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT00985517 Completed Phase2, Phase1

View Report

Idiopathic Parkinson's Disease

Sponsor Sangamo Therapeutics (Industry)

Compound CERE-120

Therapy Type Gene transfer

Target Gene NRTN

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT04174105 Active not recruiting Phase2, Phase1

View Report

Pompe Disease (Late-onset)

Sponsor Astellas Gene Therapies (Industry)

Compound AT845

Therapy Type Gene transfer

Target Gene GAA

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05986864 Recruiting Phase2, Phase1

View Report

Neovascular (Wet) Age-Related Macular Degeneration (nAMD)

Sponsor Skyline Therapeutics (US) Inc. (Industry)

Compound SKG0106

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT06483802 Withdrawn Phase1

View Report

Friedreich Ataxia, Cardiomyopathy

Sponsor Astellas Gene Therapies (Industry)

Compound ASP2016

Therapy Type Gene transfer

Target Gene FXN

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track

NCT06114056 Active not recruiting Phase1

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor West China Hospital (Other)

Compound JWK007

Therapy Type Gene transfer

Target Gene Micro-dystrophin

Vector Type AAVrh74

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT04240314 Completed Phase2, Phase1

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Megan Waldrop (Other)

Compound AT702

Therapy Type Gene transfer

Target Gene DMD (exon 2)

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Exon skipping/splice editor

NCT05761899 Recruiting Phase2, Phase1

View Report

Hereditary Pulmonary Alveolar Proteinosis

Sponsor Children's Hospital Medical Center, Cincinnati (Other)

Compound Gene-Corrected Macrophages

Therapy Type Gene transfer

Target Gene CSF2RA

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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AAV	Adeno-associated virus, e.g. AAV2, AAV5, AAV2/5 indicates virus containing the genome of serotype 2 packaged in the capsid from serotype 5
ABE	Adenine base editor
Ad	Adenovirus
Cas9	CRISPR associated protein 9
CBE	Cytosine base editor
CRISPR	Clustered regularly interspaced short palindromic repeats
Gc	Genome copies
HIV	Human immunodeficiency virus
HSV	Herpes simplex virus
LNP	Lipid nanoparticle
LV	Lentivirus
MRNA	Messenger ribonucleic acid
ODD	Orphan Drug Designation
PFU	Plaque forming units
RMAT	Regenerative Medicine Advanced Therapy
RNP	Ribonucleoprotein
RPDD	Rare Pediatric Disease Designation
RV	Retrovirus
START	Support for Clinical Trials Advancing Rare Disease Therapeutics
Vg	Vector genomes
VSV-G	Vesicular stomatitis virus G

Electroporation	Cell membrane permeablized by electrical field to allow gene therapy to enter the cell
Lipid encapsulation	Any lipid nanoparticle used to deliver editor, corrected gene
Microinjection	Drug is injected into individual cells
Plasmid	Any plasmid used to deliver editor, corrected gene
Viral transduction	Any virus used to deliver editor, corrected gene, etc.

Accelerated Approval	These regulations allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint
Breakthrough Therapy	A process designed to expedite the development and review of drugs which may demonstrate substantial improvement over available therapy.
Fast Track	Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need
Orphan Drug	This designation is for drugs intended to treat rare diseases or conditions that affect a small number of people, offering incentives like tax credits and market exclusivity
Priority Review	A Priority Review designation means FDA’s goal is to take action on an application within 6 months.
Rare Pediatric Disease	The rare pediatric disease PRV program aims to incentivize drug development for rare pediatric diseases.
Regenerative Medicine Advanced Therapy	Facilitates the development and expedites the review of regenerative medicine therapies, including cell therapies, therapeutic tissue engineering products, and human cell and tissue products, for serious or life-threatening conditions.
Support for Clinical Trials Advancing Rare Disease Therapeutics	(START) Pilot Program is a program designed to accelerate the development of novel drug and biological products for rare diseases by providing sponsors with enhanced communication and guidance from FDA staff.

Industry	All for-profit entities
NIH	U.S. National Institutes of Health
Other Non-Profit	Includes individuals, universities, community-based organizations

CED	Convection enhanced delivery to the brain
Inhalational	Delivered to the lungs in the form of a fine spray
Intraarterial	Into the lumen of an artery
Intraarticular	Into a joint space
Intracerebroventricular	Into the ventricles of the brain (ICV)
Intracisterna magna	Into the cisterna magna of the brain
Intracochlear	Into the cochlea of the ear
Intradermal	Into the dermal layer of the skin
Intragastric	Into the stomach
Intramuscular	Into a skeletal muscle
Intraocular	Administered into the eye
Intraparenchymal	Into the brain
Intraperitoneal	Into the peritoneal cavity
Intrastromal	Into the stroma of the cornea
Intrathecal	Into the spinal canal
Intravenous	Into a vein
Intravesicular	Into the bladder
Intravitreal	Into the eye (IVT)
Subcutaneous	Under the skin
Subretinal	Under the sensory retina
Suprachoroidal	Into the suprachoroidal space between the sclera and the choroid of the eye
Topical	Applied to the outer layer of the skin

Clinical Trials - Gene Therapy Trial Browser

Clinical Trials Daily Digest

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Early Phase I	Describes exploratory trials conducted before traditional Phase I trials to investigate how or whether a drug affects the body, have no therapeutic or diagnostic goals
Phase I	Describes clinical trials that focus on the safety of a drug
Phase II	Describes clinical trials that gather preliminary data on effectiveness, continue to monitor safety
Phase I/II	Combination Phase I/Phase II clinical trial
Phase III	Pivotal experiments to gather data on safety and effectiveness
Phase II/III	Combination Phase II/Phase III clinical trial

Active, Not Recruiting	Study has ongoing, but is not enrolling new participants
Completed	Study has concluded normally
Not Yet Recruiting	Study has not started enrollment
Recruiting	Study is actively looking for participants
Suspended	Study halted prematurely but has the potential to resume
Terminated	Study was halted prematurely and will not resume, participants are no longer recieving intervention
Unknown	Study has passed its completion date, but last known status was not listed as Completed, Terminated or Withdrawn. Status has not been verified within the past 2 years. Studies with an unknown status are considered closed studies

Actual Study Start Date (m/d/y)	The actual date on which the first participant was enrolled in a clinical study
Adult/Pediatric/Both	Variable on whether trial accepts patients who are adults, pediatric (<18 years of age) or both
Ages Eligible for Study	More specific age ranges eligible for the study
Clinical Centers in USA?	Binary variable on whether trial sites are located in the USA (Y) or not (N)
Clinical Publications	URL connections to clinical data on human subjects
Compound Name	The interventional compound given to the study subjects
Countries	List of countries that contain at least 1 clinical trial site
Current Stage	The stage of the clinical trial, determined based on the studies' objective
Date of Last Update	The most recent date on which changes to a study record were made available on ClinicalTrials.gov
Delivery System	Describes the nature of the drug substance or process that is used to deliver the editor or corrected gene
Development Status	Indicates whether or not the clinical development program is ongoing, or if the drug is approved
Dose levels (up to 5)	List of doses given in the indicated clinical trial, may be expressed in specific units, or a range of possible doses
Drug Product Type	Describes the nature of the drug product
Editor Type	For gene editing type therapies, the protein that will perform the gene correction
Estimated Primary Completion Date (m/d/y)	The anticipated date that the primary outcome measure data will be complete (last participant data collected)
FDA Designations	List of special FDA designations granted to the Sponsor for the development program (i.e. Orphan Drug Designation, Fast Track, Rare Pediatric Disease Designation, RMAT, etc.)
Funder Type	Describes the organization that provides support for the clinical study
Grants	URL connections to funding used to conduct preclinical or clinical studies, granted by NIH or other US institution
Has US IND?	Binary variable indicating whether the drug product is regulated by an approved Investigational New Drug application by the Food and Drug Administration of the United States
Indication	The disease, disorder, syndrome, illness, or injury that is being studied
Locations	List of countries that contain at least 1 clinical trial site
Mechanism of Action	Simplified description of how the drug product works
NCT Number	Unique identification code given to each clinical study upon registration at ClinicalTrials.gov
News and Press Releases	URL connections to press releases generated by drug product Sponsor
N trial sites	Number of clinical sites where the clinical trial is conducted
Patents	URL connections to patents, intellectual property related to the drug product
Phases	The stage of the clinical trial, determined based on the studies' objective
Preclinical Publications	URL connections to preclinical data (in vitro, animal data)
Protocols	URL connections to clinical trial protocols, study design papers
Recent Regulatory Updates	News, updates on recent or anticipated regulatory milestones
Recruitment Status	Indicates the current recruitment status or the expanded access status
Results Posted	Indicates if summary results are posted to the clinical trial record
Route of Administration	How the drug product is introduced to the body
Sexes Eligible for Study	A type of eligibility criteria that indicates the sex of people who may participate in a clinical study (all, female, male)
Sponsor	The organization or person who initiates the study and who has authority and control over the study
Sponsor Class	Describes the organization that provides support for the clinical study
Standard Ages	Variable on whether trial accepts patients who are adults, pediatric (<18 years of age) or both
Target Gene or Variant	The symbol of the gene that is corrected or replaced by the drug compound
Target Tissue or Cell	Lists target cells if the therapy is directed at a particular cell type (either by ex-vivo enrichment, or tissue-specific regulatory elements)
Therapy Route	Describes whether the gene therapy is introduced to cells in-vivo or ex-vivo
Therapy Type	Describes the nature of the gene therapy
Trial Enrollment	Number of study subjects planned or actually enrolled in the study
Vector Type	Gives additional specifics (if known) about delivery system (e.g. AAV serotype)

Ex-vivo	When the cells are modified outside the body
In-vivo	When the cells are modified inside the body

Gene editing	A gene therapy where disease-causing variant is corrected via a gene editor
Recent Regulatory Updates	A gene therapy where a corrected gene is administered to relevant cells/tissues via a delivery system

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