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

NCT05456880

2026-04-08

Sickle Cell Disease

Active not recruiting

Clinical dose manufacturing is complete, BLA submission expected as early as year-end 2026

NCT05073133

2026-04-08

Spinal Muscular Atrophy

Completed

FDA approved 5/24/19, price/treatment $2.1M

NCT05903794

2026-04-08

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

Completed

FDA cleared IND 1/18/23

NCT03199469

2026-04-08

X-Linked Myotubular Myopathy

Active not recruiting

Astellas has restarted their XLMTM program by creating a new drug product ASP2957, which is based on this prior study, with substantial modifications ...

NCT05386680

2026-04-07

Spinal Muscular Atrophy

Completed

Same drug product as Zolgensma (dosing and route of administration differs), FDA approved 11/24/25 for list price $2.59M

Showing 1-100 of 230 results in ClinicalTrials

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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 Regenerative Medicine Advanced Therapy

NCT03199469 Active not recruiting Phase3, Phase2

View Report

X-Linked Myotubular Myopathy

Sponsor Astellas Gene Therapies (Industry)

Compound AT132

Therapy Type Gene transfer

Target Gene MTM1

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy

NCT04850118 Active not recruiting Phase3, Phase2

View Report

X-Linked Retinitis Pigmentosa (XLRP)

Sponsor Beacon Therapeutics (Industry)

Compound AGTC-501

Therapy Type Gene transfer

Target Gene RPGR

Vector Type AAV2tYF

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

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

NCT03566043 Active not recruiting Phase3, Phase2

View Report

Mucopolysaccharidosis Type II (Hunter Syndrome)

Sponsor REGENXBIO Inc. (Industry)

Compound RGX-121

Therapy Type Gene transfer

Target Gene IDS

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy

NCT07048808 Recruiting Phase2

View Report

Refractory Angina, Coronary Artery Disease

Sponsor XyloCor Therapeutics, Inc. (Industry)

Compound XC001

Therapy Type Gene transfer

Target Gene VEGF

Vector Type Ad5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

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

NCT07226206 Not yet 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

NCT06884865 Active not recruiting Phase2

View Report

Osteoarthritis (OA) of the Knee

Sponsor Pacira Pharmaceuticals, Inc (Industry)

Compound PCRX-201

Therapy Type Gene transfer

Target Gene IL1RA

Vector Type Ad5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy

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

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

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 Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy

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

NCT05956626 Active not recruiting Phase3, Phase2

View Report

Stargardt Disease

Sponsor Ocugen (Industry)

Compound OCU410ST

Therapy Type Gene transfer

Target Gene RORA

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Orphan Drug Designation

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

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

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

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

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

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

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

NCT05203679 Active not recruiting Phase3, Phase2

View Report

Hemophilia B

Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)

Compound BBM-H901

Therapy Type Gene transfer

Target Gene F9

Vector Type AAV843

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations 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

NCT02716246 Recruiting Phase3, Phase2

View Report

Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)

Sponsor Ultragenyx Pharmaceutical Inc (Industry)

Compound UX111

Therapy Type Gene transfer

Target Gene SGSH

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy

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 Fast Track, Regenerative Medicine Advanced Therapy

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

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

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

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

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 Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy

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

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

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

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 Lipid encapsulation

Mechanism of Action Mutation correction

FDA Designations Orphan Drug Designation, Regenerative Medicine Advanced Therapy

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

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

NCT05176093 Completed Phase2

View Report

Amyotrophic Lateral Sclerosis (ALS)

Sponsor Helixmith Co., Ltd. (Industry)

Compound ENGENSIS

Therapy Type Gene transfer

Target Gene HGF

Delivery System None (naked plasmid)

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track, Orphan Drug 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

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

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

NCT06391736 Recruiting Phase2, Phase1

View Report

Pompe Disease (Late-onset)

Sponsor GeneCradle Inc (Industry)

Compound GC301

Therapy Type Gene transfer

Target Gene GAA

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations 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

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 Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy

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

Delivery System None (naked plasmid)

Mechanism of Action Overexpression of protective allele/gene

NCT02354781 Completed Phase2, Phase1

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Jerry R. Mendell (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

NCT05821959 Recruiting Phase2, Phase1

View Report

Sensorineural Hearing Loss, Bilateral

Sponsor Akouos, Inc. (Industry)

Compound AK-OTOF

Therapy Type Gene transfer

Target Gene OTOF

Vector Type dual Anc80L65

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06983158 Suspended Phase2, Phase1

View Report

Developmental and Epileptic Encephalopathy

Sponsor Capsida Biotherapeutics, Inc. (Industry)

Compound CAP-002

Therapy Type Gene transfer

Target Gene STXBP1

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT02935517 Active not recruiting Phase2, Phase1

View Report

Achromatopsia

Sponsor Beacon Therapeutics (Industry)

Compound AGTC-402

Therapy Type Gene transfer

Target Gene CNGA3

Vector Type AAV2tYF

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT04945772 Completed Phase2

View Report

Retinitis Pigmentosa, Retinal Dystrophies, Retinal Degeneration

Sponsor Nanoscope Therapeutics Inc. (Industry)

Compound MCO-010

Therapy Type Gene transfer

Target Gene MCO

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track, 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

Delivery System None (naked plasmid)

Mechanism of Action Overexpression of protective allele/gene

NCT04273269 Terminated Phase2, Phase1

View Report

GM1 Gangliosidosis

Sponsor LYSOGENE (Industry)

Compound LYS-GM101

Therapy Type Gene transfer

Target Gene GLB1

Vector Type AAVrh.10

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT04201405 Active not recruiting Phase2, Phase1

View Report

Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)

Sponsor University of Manchester (Other)

Compound OTL-201

Therapy Type Gene transfer

Target Gene SGSH

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

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 Lipid encapsulation

Mechanism of Action Gene inactivation

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

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

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 Lipid encapsulation

Mechanism of Action Functional gene replacement

NCT05417126 Completed Phase2

View Report

Stargardt Disease

Sponsor Nanoscope Therapeutics Inc. (Industry)

Compound MCO-010

Therapy Type Gene transfer

Target Gene MCO

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track, Orphan Drug Designation

NCT05641610 Recruiting Phase2, Phase1

View Report

Hemophilia B

Sponsor Institute of Hematology & Blood Diseases Hospital, China (Other)

Compound ZS801

Therapy Type Gene transfer

Target Gene F9

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

Therapy Type Gene transfer

Target Gene BCL11A

Vector Type VSV-G

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT03001830 Active not recruiting Phase2, Phase1

View Report

Hemophilia A

Sponsor University College, London (Other)

Compound AAV2/8-HLP-FVIII-V3

Therapy Type Gene transfer

Target Gene F8

Vector Type AAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05228145 Active not recruiting Phase2, Phase1

View Report

Neuronal Ceroid Lipofuscinosis CLN5

Sponsor Neurogene Inc. (Industry)

Compound NGN-101

Therapy Type Gene transfer

Target Gene CLN5

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT05791864 Recruiting Phase2, Phase1

View Report

Neuronal Ceroid Lipofuscinosis Type 2

Sponsor Tern Therapeutics, LLC (Industry)

Compound TTX-381

Therapy Type Gene transfer

Target Gene TPP1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy

NCT05121376 Active not recruiting Phase2, Phase1

View Report

Hereditary Angioedema

Sponsor BioMarin Pharmaceutical (Industry)

Compound BMN 331

Therapy Type Gene transfer

Target Gene SERPING1

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT02416622 Completed Phase2, Phase1

View Report

X-Linked Retinoschisis

Sponsor Beacon Therapeutics (Industry)

Compound RAAV2tYF-CB-hRS1

Therapy Type Gene transfer

Target Gene RS1

Vector Type AAV2tYF

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT03612869 Unknown Phase3, Phase2

View Report

Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)

Sponsor LYSOGENE (Industry)

Compound LYS-SAF301

Therapy Type Gene transfer

Target Gene SGSH

Vector Type AAV2rh.10

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

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

NCT03041324 Terminated Phase2, Phase1

View Report

Mucopolysaccharidosis Type II (Hunter Syndrome)

Sponsor Sangamo Therapeutics (Industry)

Compound SB-913

Therapy Type Gene editing

Target Gene IDS

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

NCT05811351 Completed Phase2

View Report

Geographic Atrophy

Sponsor Janssen Research & Development, LLC (Industry)

Compound JNJ-1887

Therapy Type Gene transfer

Target Gene CD59

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT00643747 Completed Phase2, Phase1

View Report

Leber Congenital Amaurosis-Type 2

Sponsor University College, London (Other)

Compound TgAAG76

Therapy Type Gene transfer

Target Gene RPE65

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05541627 Active not recruiting Phase2, Phase1

View Report

Huntington's Disease

Sponsor Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio) (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

NCT04127578 Active not recruiting Phase2, Phase1

View Report

Parkinson's Disease

Sponsor Prevail Therapeutics (Industry)

Compound PR001

Therapy Type Gene transfer

Target Gene GBA1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track

NCT05394064 Terminated Phase2, Phase1

View Report

Adrenomyeloneuropathy (AMN)

Sponsor SwanBio Therapeutics, Inc. (Industry)

Compound SBT101

Therapy Type Gene transfer

Target Gene ABCD1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06109181 Active not recruiting Phase2, Phase1

View Report

Arrhythmogenic Right Ventricular Cardiomyopathy

Sponsor Lexeo Therapeutics (Industry)

Compound LX2020

Therapy Type Gene transfer

Target Gene PKP2

Vector Type AAVrh10

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT05598333 Active not recruiting Phase2

View Report

Congestive Heart Failure

Sponsor AskBio Inc (Industry)

Compound AB-1002

Therapy Type Gene transfer

Target Gene PPP1R1A

Vector Type AAV2i8

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track

NCT05693142 Recruiting Phase3, Phase2

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor REGENXBIO Inc. (Industry)

Compound RGX-202

Therapy Type Gene transfer

Target Gene Micro-dystrophin

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

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)

Therapy Type Gene transfer

Target Gene HBB

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT03116113 Completed Phase2, Phase1

View Report

X-Linked Retinitis Pigmentosa (XLRP)

Sponsor Biogen (Industry)

Compound BIIB112

Therapy Type Gene transfer

Target Gene RPGR

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT04124042 Completed Phase2

View Report

Osteoarthritis, Knee

Sponsor Xalud Therapeutics, Inc. (Industry)

Compound XT-150

Therapy Type Gene transfer

Target Gene Recombinant IL10

Delivery System None (naked plasmid)

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track

NCT05603312 Completed Phase2, Phase1

View Report

Parkinson's Disease

Sponsor MeiraGTx, LLC (Industry)

Compound AAV-GAD

Therapy Type Gene transfer

Target Gene GAD1/2

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy

NCT04703842 Recruiting Phase2, Phase1

View Report

Congestive Heart Failure, Heart Failure With Reduced Ejection Fraction (HFrEF)

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

NCT04394286 Terminated Phase2, Phase1

View Report

Hemophilia B

Sponsor Baxalta now part of Shire (Industry)

Compound SHP648

Therapy Type Gene transfer

Target Gene F9

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03328130 Terminated Phase2, Phase1

View Report

Retinitis Pigmentosa

Sponsor eyeDNA Therapeutics (Industry)

Compound CTX-PDE6B

Therapy Type Gene transfer

Target Gene PDE6B

Vector Type AAV2/5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Rare Pediatric Disease Designation

NCT02341807 Completed Phase2, Phase1

View Report

Choroideremia

Sponsor Spark Therapeutics, Inc. (Industry)

Compound SPK-7001

Therapy Type Gene transfer

Target Gene CHM

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT03432364 Completed Phase2, Phase1

View Report

Beta-Thalassemia Major

Sponsor Sangamo Therapeutics (Industry)

Compound ST-400

Therapy Type Gene editing

Target Gene BCL11A

Delivery System Electroporation

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

NCT05152823 Enrolling by invitation Phase2, Phase1

View Report

Spinal Muscular Atrophy with Respiratory Distress

Sponsor Megan Waldrop (Other)

Compound ACTX-401

Therapy Type Gene transfer

Target Gene IGHMBP2

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06819514 Not yet recruiting Phase2, Phase1

View Report

Fabry Disease

Sponsor Guangzhou Jiayin Biotech Ltd (Industry)

Compound EXG110

Therapy Type Gene transfer

Target Gene GLA

Vector Type proprietary AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT00927134 Completed Phase2, Phase1

View Report

X-Linked Chronic Granulomatous Disease

Sponsor University of Zurich (Other)

Compound Retroviral SF71-gp91phox transduced CD34+ cells

Therapy Type Gene transfer

Target Gene CYBB

Vector Type SFFV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT04676048 Recruiting Phase2, Phase1

View Report

Hemophilia A

Sponsor ASC Therapeutics (Industry)

Compound ASC618

Therapy Type Gene transfer

Target Gene F8

Vector Type AAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

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

NCT04783181 Active not recruiting Phase2, Phase1

View Report

Congenital Adrenal Hyperplasia

Sponsor Adrenas Therapeutics Inc (Industry)

Compound BBP-631

Therapy Type Gene transfer

Target Gene CYP21A2

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy

NCT01621867 Completed Phase2

View Report

Cystic Fibrosis

Sponsor Imperial College London (Other)

Compound PGM169/GL67A

Therapy Type Gene transfer

Target Gene CFTR

Vector Type GL67A

Delivery System Liposome

Mechanism of Action Functional gene replacement

NCT03920007 Active not recruiting Phase2, Phase1

View Report

Leber Congenital Amaurosis, LCA, LCA1

Sponsor Atsena Therapeutics Inc. (Industry)

Compound ATSN-101

Therapy Type Gene transfer

Target Gene GUCY2D

Vector Type AAV2/5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

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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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