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 1-86 of 86 results in ClinicalTrials

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NCT05073133 Completed Phase4

View Report

Spinal Muscular Atrophy

Sponsor Novartis Pharmaceuticals (Industry)

Compound ZOLGENSMA

Therapy Type Gene transfer

Target Gene SMN1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT05386680 Completed Phase3

View Report

Spinal Muscular Atrophy

Sponsor Novartis Pharmaceuticals (Industry)

Compound ITVISMA

Therapy Type Gene transfer

Target Gene SMN1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Orphan Drug Designation

NCT05139316 Completed Phase3

View Report

Glycogen Storage Disease Type Ia

Sponsor Ultragenyx Pharmaceutical Inc (Industry)

Compound DTX401

Therapy Type Gene transfer

Target Gene G6PC

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT04671433 Completed Phase3

View Report

X-Linked Retinitis Pigmentosa (XLRP)

Sponsor Janssen Research & Development, LLC (Industry)

Compound Botaretigene sparoparvovec

Therapy Type Gene transfer

Target Gene RPGR

Vector Type AAV2/5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug 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

NCT03496012 Completed Phase3

View Report

Choroideremia

Sponsor Biogen (Industry)

Compound BIIB111

Therapy Type Gene transfer

Target Gene CHM

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy

NCT02652767 Completed Phase3

View Report

Leber Hereditary Optic Neuropathy (LHON)

Sponsor GenSight Biologics (Industry)

Compound LUMEVOQ

Therapy Type Gene transfer

Target Gene ND4G11778A

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06066008 Completed Early phase1

View Report

X-Linked Retinoschisis

Sponsor Zhongmou Therapeutics (Industry)

Compound ZM-01

Therapy Type Gene transfer

Target Gene RS1

Vector Type AAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03569891 Completed Phase3

View Report

Hemophilia B

Sponsor CSL Behring (Industry)

Compound HEMGENIX

Therapy Type Gene transfer

Target Gene F9-R338L

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Priority Review, Orphan Drug 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

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

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

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

NCT00028236 Completed Phase1

View Report

X-linked Severe Combined Immunodeficiency (XSCID)

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

Compound GALV MFGS-gc retrovirus

Therapy Type Gene transfer

Target Gene IL2RG

Vector Type MFGS/GALV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01344798 Completed Phase1

View Report

Limb-Girdle Muscular Dystrophy, Type 2C/R5

Sponsor Genethon (Other)

Compound AAV1.DES.hSGCG

Therapy Type Gene transfer

Target Gene SGCG

Vector Type AAV1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04275323 Completed Phase3

View Report

Critical Limb Ischemia

Sponsor Beijing Northland Biotech. Co., Ltd. (Industry)

Compound NL003

Therapy Type Gene transfer

Target Gene HGF

Delivery System None (naked plasmid)

Mechanism of Action Overexpression of protective allele/gene

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

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

NCT03207009 Completed Phase3

View Report

Beta-Thalassemia Major

Sponsor Genetix Biotherapeutics Inc. (Industry)

Compound ZYNTEGLO

Therapy Type Gene transfer

Target Gene HBB

Vector Type BB305 LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT02161380 Completed Phase1

View Report

Leber Hereditary Optic Neuropathy (LHON)

Sponsor Byron Lam (Other)

Compound ScAAV2-P1ND4v2

Therapy Type Gene transfer

Target Gene ND4

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

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

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 Lipid encapsulation

Mechanism of Action Gene inactivation

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

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

NCT06594094 Completed Early phase1

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)

Compound HG302

Therapy Type Gene editing

Target Gene DMD (exon 51 splice site)

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Exon skipping/splice editor

FDA Designations Orphan Drug Designation, Rare Pediatric Disease 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

NCT02082860 Completed Phase1

View Report

Acute Intermittent Porphyria

Sponsor Digna Biotech S.L. (Industry)

Compound RAAV2/5-PBGD

Therapy Type Gene transfer

Target Gene PBGD

Vector Type AAV2/5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

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

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

NCT00891306 Completed Phase3, Phase2

View Report

Familial Lipoprotein Lipase Deficiency

Sponsor Amsterdam Molecular Therapeutics (Industry)

Compound GLYBERA

Therapy Type Gene transfer

Target Gene LPL

Vector Type AAV1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT03964792 Completed Phase2, Phase1

View Report

Sickle Cell Disease

Sponsor Assistance Publique - Hôpitaux de Paris (Other)

Compound DREPAGLOBE

Therapy Type Gene transfer

Target Gene HBB

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT02132130 Completed Phase2, Phase1

View Report

Unilateral OR Bilateral Severe to Profound Hearing Loss

Sponsor Novartis Pharmaceuticals (Industry)

Compound CGF166

Therapy Type Gene transfer

Target Gene ATOH1

Vector Type Ad5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT00598481 Completed Phase2

View Report

Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Sponsor Fondazione Telethon (Other)

Compound STRIMVELIS

Therapy Type Gene transfer

Target Gene ADA

Vector Type MoMLV (RV)

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01054339 Completed Phase2

View Report

Alpha-1 Antitrypsin Deficiency (AATD)

Sponsor Beacon Therapeutics (Industry)

Compound AGTC-0106

Therapy Type Gene transfer

Target Gene SERPINA1

Vector Type AAV1

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

NCT00272857 Completed Phase1

View Report

Fanconi Anemia Complementation Group A

Sponsor Boston Children's Hospital (Other)

Compound MSCV-FANCA

Therapy Type Gene transfer

Target Gene FANCA

Vector Type MSCV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03734588 Completed Phase2, Phase1

View Report

Hemophilia A

Sponsor Spark Therapeutics, Inc. (Industry)

Compound SPK-8016

Therapy Type Gene transfer

Target Gene F8

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01482195 Completed Phase1

View Report

Retinal Disease, Retinitis Pigmentosa

Sponsor King Khaled Eye Specialist Hospital (Other gov)

Compound AAV2-VMD2-hMERTK

Therapy Type Gene transfer

Target Gene MERTK

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04469270 Completed Phase3

View Report

Diabetic Neuropathy

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 Regenerative Medicine Advanced Therapy

NCT03173521 Completed Phase2, Phase1

View Report

Mucopolysaccharidosis Type VI

Sponsor Fondazione Telethon (Other)

Compound AAV2/8.TBG.hARSB

Therapy Type Gene transfer

Target Gene ARSB

Vector Type AAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01301443 Completed Phase1

View Report

Neovascular (Wet) Age-Related Macular Degeneration

Sponsor Oxford BioMedica (Industry)

Compound RetinoStat

Therapy Type Gene transfer

Target Gene COL18A1+PLG

Vector Type EIAV

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT00876863 Completed Phase2

View Report

Alzheimer's Disease

Sponsor Sangamo Therapeutics (Industry)

Compound CERE-110

Therapy Type Gene transfer

Target Gene NGF

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT00494195 Completed Phase1

View Report

Limb-Girdle Muscular Dystrophy, Type 2D/R3

Sponsor Nationwide Children's Hospital (Other)

Compound RAAV1.tMCK.human-alpha-sarcoglycan

Therapy Type Gene transfer

Target Gene SGCA

Vector Type AAV1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01161576 Completed Phase1

View Report

CLN2 Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis

Sponsor Weill Medical College of Cornell University (Other)

Compound LX1004

Therapy Type Gene transfer

Target Gene TPP1

Vector Type AAVrh10

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT02234934 Completed Phase2, Phase1

View Report

X-Linked Chronic Granulomatous Disease

Sponsor University of California, Los Angeles (Other)

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

NCT05265767 Completed Phase1

View Report

Hemophilia A

Sponsor Christian Medical College, Vellore, India (Other)

Compound CD68-ET3-LV

Therapy Type Gene transfer

Target Gene F8

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03634007 Completed Phase2, Phase1

View Report

Alzheimer's Disease, Early Onset Alzheimer's Disease

Sponsor Lexeo Therapeutics (Industry)

Compound LX1001

Therapy Type Gene transfer

Target Gene APOE2

Vector Type AAVrh10

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track

NCT00428935 Completed Phase1

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Nationwide Children's Hospital (Other)

Compound RAAV2.5-CMV-mini-Dystrophin

Therapy Type Gene transfer

Target Gene Micro-dystrophin

Vector Type AAV2.5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01801709 Completed Phase2, Phase1

View Report

Metachromatic Leukodystrophy

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

Compound AAVrh.10-CAG/cuARSA

Therapy Type Gene transfer

Target Gene ARSA

Vector Type AAVrh.10

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01024998 Completed Phase1

View Report

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

Sponsor Genzyme, a Sanofi Company (Industry)

Compound RAAV2-sFLT01

Therapy Type Gene transfer

Target Gene SFLT01

Vector Type AAV2

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

NCT03852498 Completed Phase3

View Report

Cerebral Adrenoleukodystrophy (CALD)

Sponsor Genetix Biotherapeutics Inc. (Industry)

Compound SKYSONA

Therapy Type Gene transfer

Target Gene ABCD1

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT00821340 Completed Phase1

View Report

Leber Congenital Amaurosis-Type 2

Sponsor Hadassah Medical Organization (Other)

Compound RAAV2-hRPE65

Therapy Type Gene transfer

Target Gene RPE65

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01410019 Completed Phase2, Phase1

View Report

X-linked Severe Combined Immunodeficiency (XSCID)

Sponsor Assistance Publique - Hôpitaux de Paris (Other)

Compound Autologous CD34+ cells transduced with the self-inactivating (SIN) GAMMARETROVIRAL vector pSRS11.EFS.IL2RG.pre

Therapy Type Gene transfer

Target Gene IL2RG

Vector Type RV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04917874 Completed Phase3

View Report

Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Sponsor Krystal Biotech, Inc. (Industry)

Compound VYJUVEK

Therapy Type Gene transfer

Target Gene COL7A1

Vector Type HSV1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT05144386 Completed Phase1

View Report

HIV-1-infection

Sponsor Excision BioTherapeutics (Industry)

Compound EBT-101

Therapy Type Gene editing

Target Gene HIV genes

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Gene excision

FDA Designations Fast Track, Regenerative Medicine Advanced Therapy

NCT02556736 Completed Phase2, Phase1

View Report

Advanced Retinitis Pigmentosa

Sponsor AbbVie (Industry)

Compound RST-001

Therapy Type Gene transfer

Target Gene ChR2

Vector Type AAV2.7m8

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Orphan Drug Designation

NCT00749957 Completed Phase2, Phase1

View Report

Leber Congenital Amaurosis-Type 2

Sponsor Beacon Therapeutics (Industry)

Compound RAAV2-CB-hRPE65

Therapy Type Gene transfer

Target Gene RPE65

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03363165 Completed Phase2

View Report

Peripheral Artery Disease

Sponsor Northwestern University (Other)

Compound ENGENSIS

Therapy Type Gene transfer

Target Gene HGF

Delivery System None (naked plasmid)

Mechanism of Action Overexpression of protective allele/gene

NCT00976352 Completed Phase2, Phase1

View Report

Glycogen Storage Disease Type 2 (Pompe Disease)

Sponsor University of Florida (Other)

Compound AAV1-CMV-hGAA

Therapy Type Gene transfer

Target Gene GAA

Vector Type AAV2/1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03300453 Completed Phase2, Phase1

View Report

Sanfilippo Syndrome B

Sponsor UniQure Biopharma B.V. (Industry)

Compound AMT-110

Therapy Type Gene transfer

Target Gene NAGLU

Vector Type AAV2/5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT02240407 Completed Phase1

View Report

Glycogen Storage Disease Type 2 (Pompe Disease)

Sponsor University of Florida (Other)

Compound RAAV9-DES-hGAA

Therapy Type Gene transfer

Target Gene GAA

Vector Type AAV2/9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03562494 Completed Phase1

View Report

Parkinson's Disease

Sponsor Neurocrine Biosciences (Industry)

Compound VY-AADC01

Therapy Type Gene transfer

Target Gene DDC

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy

NCT02168686 Completed Phase2, Phase1

View Report

Alpha-1 Antitrypsin Deficiency (AATD)

Sponsor Adverum Biotechnologies, Inc. (Industry)

Compound ADVM-043

Therapy Type Gene transfer

Target Gene SERPINA1

Vector Type AAVrh10

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01496040 Completed Phase2, Phase1

View Report

Leber Congenital Amaurosis

Sponsor Nantes University Hospital (Other)

Compound RAAV2/4.hRPE65

Therapy Type Gene transfer

Target Gene RPE65

Vector Type AAV2/4

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT01064440 Completed Phase2

View Report

Critical Limb Ischemia

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

NCT03333590 Completed Phase2, Phase1

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Kevin Flanigan (Other)

Compound RAAVrh74.MCK.GALGT2

Therapy Type Gene transfer

Target Gene GALGT2

Vector Type AAVrh74

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT00372320 Completed Phase1

View Report

Parotid Salivary Dysfunction

Sponsor National Institute of Dental and Craniofacial Research (NIDCR) (NIH)

Compound AdhAQP1

Therapy Type Gene transfer

Target Gene AQP1

Vector Type Ad5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT01494805 Completed Phase2, Phase1

View Report

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

Sponsor Lions Eye Institute, Perth, Western Australia (Other)

Compound RAAV.sFlt-1

Therapy Type Gene transfer

Target Gene Soluble VEGFR1

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT03812263 Completed Phase2, Phase1

View Report

Leukocyte Adhesion Defect - Type I

Sponsor Rocket Pharmaceuticals Inc. (Industry)

Compound KRESLADI

Therapy Type Gene transfer

Target Gene ITGB2

Vector Type Chim.hCD18-LV

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

NCT00787059 Completed Phase2, Phase1

View Report

Congestive Heart Failure

Sponsor Hammond, H. Kirk, M.D. (Indiv)

Compound RT-100

Therapy Type Gene transfer

Target Gene ADCY6

Vector Type Ad5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track

NCT01643330 Completed Phase2

View Report

Ischemic Cardiomyopathy, Non-ischemic Cardiomyopathy, Heart Failure, Cardiomyopathies

Sponsor Celladon Corporation (Industry)

Compound AAV1-CMV-Serca2a

Therapy Type Gene transfer

Target Gene SERCA2A

Vector Type AAV1

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT04323098 Completed Phase3

View Report

Hemophilia A

Sponsor BioMarin Pharmaceutical (Industry)

Compound ROCTAVIAN

Therapy Type Gene transfer

Target Gene F8

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT03001310 Completed Phase2, Phase1

View Report

Achromatopsia

Sponsor MeiraGTx UK II Ltd (Industry)

Compound AAV-CNGB3

Therapy Type Gene transfer

Target Gene CNGB3

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

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

Filters ..

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

SCGE Platform GTCT
Trial ID	Indication	Compound Name	Sponsor	Funder Type	Target Gene/Variant	Therapy Type	Therapy Route	Mechanism of Action	Route of Administration ^>	Drug Product Type	Target Tissue/Cell	Delivery System	Vector Type	Editor Type	Dosage/s	Current Stage	Recruitment Status	Development Status	Submit Date ^>	Completion Date	Last_Update	Eligibility Age	Enrollment Count	No. of Trial Sites	Locations	Has US IND	FDA Designation	Recent Updates	Resources/Links
Trial ID	Indication	Compound Name	Sponsor	Funder Type	Target Gene/Variant	Therapy Type	Therapy Route	Mechanism of Action	Route of Administration ^>	Drug Product Type	Target Tissue/Cell	Delivery System	Vector Type	Editor Type	Dosage/s	Current Stage	Recruitment Status	Development Status	Submit Date ^>	Completion Date	Last_Update	Eligibility Age	Enrollment Count	No. of Trial Sites	Locations	Has US IND	FDA Designation	Recent Updates	Resources/Links
NCT05073133	Spinal Muscular Atrophy	ZOLGENSMA	Novartis Pharmaceuticals	Industry	SMN1	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV9		1.1E14 vg/kg	Phase4	Completed	Approved	2021-09-20	2023-08-08	2024-10-09	<= 24 Months	16	5	Locations:Argentina + 1 more Argentina, Brazil		Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	FDA approved 5/24/19, price/treatment $2.1M	Preclinical Publications : Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery A large animal model of spinal muscular atrophy and correction of phenotype Clinical Publications : AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue (Abstract) Long-term Safety of Onasemnogene Abeparvovec for Patients with Spinal Muscular Atrophy: Real-world Findings from the RESTORE Registry - MDA 2025 Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1 Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1 Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes Health outcomes in spinal muscular atrophy type 1 following AVXS-101 gene replacement therapy (Abstract) Outcomes and need for supplemental therapy after gene therapy in patients with spinal muscular atrophy type 1 identified by newborn screen - MDA 2025 Fatal outcomes following onasemnogene abeparvovec in advanced-stage spinal muscular atrophy Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG) Respiratory outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy: national real-world cohort study Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial Protocol : FDA Approval Documents Related NCTID : Long Term Follow-Up: NCT04042025 Long Term Follow-Up: NCT03421977 Phase 4: NCT04488133 (study subjects were given nusinersen after receiving Zolgensma) Phase 3: NCT04851873 Phase 4: NCT05861999 (study subjects were given risdiplam after receiving Zolgensma) Phase 4: NCT05861986 (study subjects were given risdiplam after receiving Zolgensma) Long Term Follow-Up: NCT05335876 Phase 1: NCT02122952 Phase 3: NCT03837184 Phase 3: NCT03306277 Phase 3: NCT03461289 Phase 3: NCT03505099 Patient Registry: NCT04174157
NCT05903794	Neovascular (Wet) Age-Related Macular Degeneration (nAMD)	EXG102-031	Exegenesis Bio	Industry	ABD-VEGFR	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Subretinal	Viral vector		Viral transduction	AAV8		Dose 1: Undisclosed dose 1 \| Dose 2: Undisclosed dose 2	Phase1	Completed	Active	2023-06-06	2026-02-16	2026-04-08	>= 50 Years	12	2	United States			FDA cleared IND 1/18/23	Preclinical Publications : (Abstract) Efficacy Evaluation of EXG102-031, A Novel AAV-based Gene Therapy for Neovascular AMD in a Mouse Model of VEGF-A-induced Exudative Retinal Detachment vascular leakage Mouse Model - ARVO 2023 News and Press Releases : Exegenesis Bio Announces FDA Clearance of Investigational New Drug (IND) Application for EXG102-031: A Novel Gene Therapy for the Treatment of neovascular Age-Related Macular Degeneration (nAMD) Related NCTID : Long Term Follow-Up: NCT06817343 Long Term Follow-Up: NCT06859515
NCT05386680	Spinal Muscular Atrophy	ITVISMA	Novartis Pharmaceuticals	Industry	SMN1	Gene transfer	In-vivo	Functional gene replacement	Intrathecal	Viral vector		Viral transduction	AAV9		Dose 1: Phase 1/2: 6.0E13 vg, 1.2E14 vg, 2.4E14 vg \| Dose 2: Phase 3: 1.2E14 vg \| Dose 3: Approved Dose: 1.2E14 vg	Phase3	Completed	Approved	2022-05-18	2024-11-29	2026-01-13	2 Years - 17 Years	27	13	Locations:United States + 8 more Australia, Belgium, Canada, France, Italy, Japan, Netherlands, Spain, United States		Breakthrough Therapy, Orphan Drug Designation	Same drug product as Zolgensma (dosing and route of administration differs), FDA approved 11/24/25 for list price $2.59M	Preclinical Publications : Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys Clinical Publications : Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG) Protocol : FDA Approval Documents Statistical Analysis Plan Clinical Trial Protocol Related NCTID : Phase 4: NCT07448610 Patient Registry: NCT04174157 Phase 3: NCT05089656 Long Term Follow Up: NCT05335876 Phase 1: NCT03381729
NCT05139316	Glycogen Storage Disease Type Ia	DTX401	Ultragenyx Pharmaceutical Inc	Industry	G6PC	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV8		Dose 1: Phase 1/2: 2E12 GC/kg \| Dose 2: Phase 1/2: 6E12 GC/kg \| Dose 3: Phase 3: 1.0E13 GC/kg	Phase3	Completed	Active	2021-07-14	2026-02-20	2026-03-25	>= 8 Years	49	20	Locations:United States + 8 more Brazil, Canada, Denmark, Germany, Italy, Japan, Netherlands, Spain, United States		Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy	BLA Submission completed 12/30/25, PDUFA Action Date set for 8/23/26	Preclinical Publications : An evolutionary approach to optimizing glucose-6-phosphatase-α enzymatic activity for gene therapy of glycogen storage disease type Ia Gene therapy using a novel G6PC-S298C variant enhances the long-term efficacy for treating glycogen storage disease type Ia News and Press Releases : Ultragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update Ultragenyx Reports Third Quarter 2025 Financial Results and Corporate Update Ultragenyx Announces U.S. FDA Acceptance and Priority Review of the Biologics License Application (BLA) for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa) Ultragenyx Initiates Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for the Treatment of Glycogen Storage Disease Type Ia (GSDIa) Ultragenyx Completes Rolling Submission of Biologics License Application (BLA) to U.S. FDA for DTX401 AAV Gene Therapy for Glycogen Storage Disease Type Ia (GSDIa) Clinical Publications : Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa) (Corporate Presentation) Top-Line Phase 3 Results DTX401 for Glycogen Storage Disease Type Ia (GSDIa) - May 2024 The multiple faces of urinary glucose tetrasaccharide as biomarker for patients with hepatic glycogen storage diseases Trial Interviews to Explore Glycogen Storage Disease Type Ia Patient Experiences Following Gene Therapy Protocol : Statistical Analysis Plan Clinical Trial Protocol Related NCTID : Long Term Follow-Up: NCT03970278 Long Term Follow-Up: NCT06636383 Phase 1/2: NCT03517085
NCT04671433	X-Linked Retinitis Pigmentosa (XLRP)	Botaretigene sparoparvovec	Janssen Research & Development, LLC	Industry	RPGR	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2/5		Dose 1: 2.0E10 vg/mL \| Dose 2: 2.0E11 vg/mL \| Dose 3: 2.0E12 vg/mL	Phase3	Completed	Active	2020-11-05	2024-09-30	2026-03-16	>= 3 Years	105	28	Locations:United States + 10 more Belgium, Canada, Denmark, France, Israel, Italy, Netherlands, Spain, Switzerland, United Kingdom, United States		Fast Track, Orphan Drug Designation	Phase 3 dosing complete, phase 3 study did not meet its primary efficacy endpoint	Preclinical Publications : (Poster) AAV-RPGRGene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa (XLRP): Human retinal organoid vector efficacy data - ARVO 2022 News and Press Releases : Corporate Presentation J&J gene therapy fails to improve visual navigation in late-stage rare eye disease trial Clinical Publications : Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa (Presentation) Ph1/2 AAV5-RPGR (Botaretigene Sparoparvovec) Gene Therapy Trial in RPGR-associated X-linked Retinitis Pigmentosa (XLRP) - AAO 2022 Related NCTID : Phase 3: NCT04794101 Phase 2: NCT06646289 Phase 1/2: NCT03252847 Long Term Follow-Up: NCT04671433
NCT04046224	Fabry Disease	ST-920	Sangamo Therapeutics	Industry	GLA	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV2/6		Dose 1: 0.26E13 vg/kg (n=2) \| Dose 2: 0.53E13 vg/kg (n=2) \| Dose 3: 1.58E13 vg/kg (n=2) \| Dose 4: 2.63E13 vg/kg (n=26)	Phase2, Phase1	Completed	Active	2019-08-01	2025-04-10	2025-05-15	>= 18 Years	33	18	Locations:United States + 6 more Australia, Canada, Germany, Italy, Taiwan, United Kingdom, United States		Orphan Drug Designation	Rolling BLA submission initiated December 2025, the company expects submission to be completed 2Q2026	Preclinical Publications : AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction News and Press Releases : Sangamo Therapeutics Announces Updated Phase 1/2 STAAR Study Data in Fabry Disease Showing Sustained Benefit, Improvements in Kidney Function and Favorable Safety Profile Sangamo Therapeutics Reports Recent Business Highlights and Third Quarter 2025 Financial Results Sangamo Therapeutics Initiates Rolling Submission of BLA to U.S. FDA for ST-920 in Fabry Disease Sangamo Therapeutics Announces Alignment With FDA on Accelerated Approval Pathway for ST-920 in Fabry Disease With BLA Submission Expected in 2025 Sangamo Therapeutics Announces U.S. FDA Alignment on Abbreviated Pathway to Potential Approval and EMA Prime Eligibility for ST-920 in Fabry Disease Clinical Publications : (Presentation) Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing Phase 1/2 study (STAAR) - WORLDSymposium 2024 Isaralgagene Civaparvovec (ST-920) Shows Positive Mean Annualized eGFR Slope in Adults with Fabry Disease: Updated Results from the Registrational Phase 1/2 STAAR Gene Therapy Study (Poster) Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing Phase 1/2 study (STAAR) - WORLDSymposium 2025 Related NCTID : Long Term Follow-Up: NCT05039866
NCT03496012	Choroideremia	BIIB111	Biogen	Industry	CHM	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2		Dose 1: 1.0E10 gp (n=34) \| Dose 2: 1.0E11 gp (n=69)	Phase3	Completed	Inactive	2018-03-07	2020-12-01	2023-12-07	>= 18 Years	169	18	Locations:United States + 7 more Canada, Denmark, Finland, France, Germany, Netherlands, United Kingdom, United States		Regenerative Medicine Advanced Therapy	Phase III study did not meet its primary endpoint or demonstrate efficacy on key secondary endpoints	Preclinical Publications : Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo News and Press Releases : Biogen Announces Agreement to Acquire Nightstar Therapeutics to Establish Clinical Pipeline of Gene Therapy Candidates in Ophthalmology Biogen Announces Topline Results from Phase 3 Gene Therapy Study in Choroideremia Biogen 2021 Annual Report Clinical Publications : Choroideremia Gene Therapy Phase 2 Clinical Trial: 24-Month Results Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia CHANGES IN RETINAL SENSITIVITY AFTER GENE THERAPY IN CHOROIDEREMIA (Review) Gene therapy for choroideremia using an adeno-associated viral vector encoding Rab escort protein 1: the REGENERATE open-label trial Two-Year Results After AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience Efficacy and Safety of Retinal Gene Therapy Using Adeno-Associated Virus Vector for Patients With Choroideremia: A Randomized Clinical Trial Protocol : Clinical Trial Protocol Clinical Trial Protocol and Statistical Analysis Plan (NCT02553135) Statistical Analysis Plan Related NCTID : Phase 2: NCT02407678 Phase 1/2: NCT02077361 Phase 1/2: NCT01461213 Long Term Follow-Up: NCT03584165 Phase 2: NCT02553135 Phase 2: NCT02671539 Phase 2: NCT03507686
NCT02652767	Leber Hereditary Optic Neuropathy (LHON)	LUMEVOQ	GenSight Biologics	Industry	ND4G11778A	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV2		9E10 vg in 90uL	Phase3	Completed	Active	2016-01-07	2019-07-04	2026-03-02	>= 15 Years	39	7	Locations:United States + 4 more France, Germany, Italy, United Kingdom, United States			Granted compassionate use authorization for named patient in France, early access use authorized in Israel; MAA in UK goal in 2026	Preclinical Publications : Biodistribution of intravitreal lenadogene nolparvovec gene therapy in nonhuman primates Nuclear expression of mitochondrial ND4 leads to the protein assembling in complex I and prevents optic atrophy and visual loss News and Press Releases : GenSight Biologics Announces the Granting of Compassionate Use Authorization (CUA/AAC) for GS010/LUMEVOQ® in France GenSight Biologics Submits EU Marketing Authorisation Application for LUMEVOQ® Gene Therapy to Treat Vision Loss due to Leber Hereditary Optic Neuropathy (LHON) GenSight Biologics Announces Publication of 5-Year Outcomes for Patients Treated Unilaterally with LUMEVOQ® Gene Therapy GenSight Biologics Announces Regulatory Authorization for Early Access Treatment with GS010/LUMEVOQ® in Israel Clinical Publications : Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy Long-Term Follow-Up After Unilateral Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy: The RESTORE Study Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy Meta-analysis of treatment outcomes for patients with m.11778G>A MT-ND4 Leber hereditary optic neuropathy Absence of lenadogene nolparvovec DNA in a brain tumor biopsy from a patient in the REVERSE clinical study, a case report Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy Safety of Intravitreal Gene Therapy for Treatment of Subjects with Leber Hereditary Optic Neuropathy due to Mutations in the Mitochondrial ND4 Gene: The REVEAL Study Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy Related NCTID : Phase 3: NCT02652780 Phase 3: NCT03293524 Long Term Follow-Up: NCT03406104 Phase 1/2: NCT02064569
NCT00979238	Hemophilia B	ScAAV2/8-LP1-hFIXco	St. Jude Children's Research Hospital	Other	F9	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV2/8		Dose 1: 2E11 vg/kg (n=2) \| Dose 2: 6E11 vg/kg (n=2) \| Dose 3: 2E12 vg/kg (n=6) \| Dose 4: Extension phase with new process: 2E12 vg/kg (n=2) \| Dose 5: Extension phase with new process: 5E12 vg/kg (n=2)	Phase1	Completed	Inactive	2009-09-16	2026-03-26	2026-04-02	>= 18 Years	14	4	Locations:United States + 1 more United Kingdom, United States			Was the first Hemophilia B trial to show sustained efficacy	Clinical Publications : (Abstract) Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy - ASH 2023 Adenovirus-associated virus vector-mediated gene transfer in hemophilia B Long-term safety and efficacy of factor IX gene therapy in hemophilia B (Abstract) Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer - ASH 2018 Protocol : Clinical Trial Protocol
NCT06066008	X-Linked Retinoschisis	ZM-01	Zhongmou Therapeutics	Industry	RS1	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV2/8		Dose 1: 2.07E11 vg/eye \| Dose 2: Undisclosed high dose	Early phase1	Completed	Active	2023-09-25	2026-01-31	2026-03-31	3 Years - 18 Years	2	1	China				Clinical Publications : (Abstract) Novel Gene Therapy for XLRS: Initial Findings from a Clinical Trial Showing Visual Improvement - ARVO 2024 (Abstract 2003) AAV-Mediated Gene Therapy Restores Retinal Structure and Function in X-linked Retinoschisis: From Bench to Bedside - ASGCT 2025
NCT03569891	Hemophilia B	HEMGENIX	CSL Behring	Industry	F9-R338L	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector	Liver	Viral transduction	AAV5		Dose 1: Phase 1/2: 5E12 vg/kg (n=5) or 2E13 vg/kg (n=5) \| Dose 2: Phase 2b: 2E13 vg/kg (n=3) \| Dose 3: Open label Phase 3: 2E13 vg/kg (n=54) \| Dose 4: Approved dose: 2E13 vg/kg	Phase3	Completed	Approved	2018-06-14	2025-03-19	2026-03-27	>= 18 Years	67	33	Locations:United States + 7 more Belgium, Denmark, Germany, Ireland, Netherlands, Sweden, United Kingdom, United States		Breakthrough Therapy, Priority Review, Orphan Drug Designation	FDA approved 11/22/22; Price/treatment: $3.5M	Preclinical Publications : Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs Clinical Publications : Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy Indirect treatment comparisons of the gene therapy etranacogene dezaparvovec versus extended half-life factor IX therapies for severe or moderately severe haemophilia B Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B Final Analysis of a Study of Etranacogene Dezaparvovec for Hemophilia B Protocol : Clinical Trial Protocol Statistical Analysis Plan FDA Approval Documents Related NCTID : Phase 1/2: NCT02396342 Phase 2: NCT03489291 Phase 3: NCT06003387
NCT05176093	Amyotrophic Lateral Sclerosis (ALS)	ENGENSIS	Helixmith Co., Ltd.	Industry	HGF	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intramuscular	Plasmid		None (naked plasmid)			64mg/cycle, 3 cycles/patient, 2 days/cycle, 128 injections/cycle	Phase2	Completed	Active	2021-11-05	2024-08-23	2025-10-06	18 Years - 80 Years	8	2	Locations:United States + 1 more South Korea, United States		Fast Track, Orphan Drug Designation	Same product as NL003	News and Press Releases : Helixmith Announces Topline Results from Phase 2A Study of Engensis for Treatment of ALS (Amyotrophic Lateral Sclerosis) VM BioPharma Announces FDA Fast Track Designation Granted for Investigational Gene Therapy VM202 for Patients with Amyotrophic Lateral Sclerosis (ALS) Clinical Publications : Open label study to assess the safety of VM202 in subjects with amyotrophic lateral sclerosis Protocol : Clinical Trial Protocol and Statistical Analysis Plan Related NCTID : Phase 2: NCT04632225 Phase 1/2: NCT02039401 Long Term Follow-Up: NCT05176093
NCT02725580	Variant Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease)	AT-GTX-501	Emily de los Reyes	Other	CLN6	Gene transfer	In-vivo	Functional gene replacement	Intrathecal	Viral vector		Viral transduction	AAV9		1.5E13 vg	Phase2, Phase1	Completed	Inactive	2016-03-16	2024-10	2025-10-24	>= 1 Year	13	1	United States		Orphan Drug Designation, Rare Pediatric Disease Designation	Program was discontinued due to lack of sustained efficacy in LTFU study, Amicus returned development rights to Abigail Wexner Research Institute at Nationwide Children's Hospital in January 2024	Preclinical Publications : Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease News and Press Releases : Letter to Batten community from Amicus Therapeutics Letter to Batten community from Nationwide Children's Hospital Amicus Therapeutics Reports Preliminary 2021 Revenue and Provides 2022 Strategic Outlook and Revenue Guidance Clinical Publications : (Presentation) Single-dose AAV9-CLN6 gene transfer stabilizes motor and language function in variant late infantile neuronal ceroid lipofuscinosis 6 (vLINCL6) disease: interim results from the first clinical gene therapy trial - CNS 2020 Protocol : Statistical Analysis Plan Clinical Trial Protocol Related NCTID : Long Term Follow-Up: NCT04273243
NCT01422772	Ischemic Heart Disease	ENGENSIS	Helixmith Co., Ltd.	Industry	HGF	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intramyocardial	Plasmid		None (naked plasmid)	none		Dose 1: 0.5mg/1ml (4 sites) \| Dose 2: 1mg/2ml (8 sites) \| Dose 3: 2mg/4ml (8 sites)	Phase2, Phase1	Completed	Active	2011-08-21	2014-08	2025-10-03	19 Years - 75 Years	9	1	South Korea			Same product as NL003	Preclinical Publications : MR assessment of myocardial perfusion, viability, and function after intramyocardial transfer of VM202, a new plasmid human hepatocyte growth factor in ischemic swine myocardium Enhanced cardioprotective effects by coexpression of two isoforms of hepatocyte growth factor from naked plasmid DNA in a rat ischemic heart disease model Quantitative MR measurements of regional and global left ventricular function and strain after intramyocardial transfer of VM202 into infarcted swine myocardium Clinical Publications : Intramyocardial transfer of hepatocyte growth factor as an adjunct to CABG: phase I clinical study
NCT02354781	Duchenne Muscular Dystrophy (DMD)	RAAV1.CMV.huFollistatin344	Jerry R. Mendell	Other	FST (FS344)	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intramuscular (gluteus, quadriceps, tibialis anterior)	Viral vector		Viral transduction	AAV1		Total dose: 2.4E12 vg/kg (1.2E12vg/kg/limb)	Phase2, Phase1	Completed	Inactive	2015-01-26	2017-11	2023-10-11	>= 7 Years	3	1	United States			Study drug was previously given in the trial for Becker Muscular Dystrophy (NCT01519349). Was well tolerated in DMD patients but not tested further	Preclinical Publications : Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model Follistatin gene delivery enhances muscle growth and strength in nonhuman primates Protocol : Clinical Trial Protocol and Statistical Analysis Plan
NCT04945772	Retinitis Pigmentosa, Retinal Dystrophies, Retinal Degeneration	MCO-010	Nanoscope Therapeutics Inc.	Industry	MCO	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravitreal	Viral vector	Bipolar cells	Viral transduction	AAV2		Dose 1: 0.9E11 gc/eye \| Dose 2: 1.2E11 gc/eye	Phase2	Completed	Active	2021-06-15	2024-01-18	2024-03-22	>= 18 Years	27	6	Locations:United States + 1 more Puerto Rico, United States		Fast Track, Orphan Drug Designation	First modules of BLA submitted in July 2025, full submission anticipated in early 2026	Grant : R01 EY028216 R01EY025717 R44 EY025905 Preclinical Publications : Biodistribution of adeno-associated virus type 2 carrying multi-characteristic opsin in dogs following intravitreal injection Multi-Characteristic Opsin Therapy to Functionalize Retina, Attenuate Retinal Degeneration, and Restore Vision in Mouse Models of Retinitis Pigmentosa Efficacy of Intravitreal Multi-Characteristic Opsin (MCO-010) Optogenetic Gene Therapy in a Mouse Model of Leber Congenital Amaurosis Topology and Mechanism of Broadband and Fast Multi-Characteristic Opsin for Neuromodulation News and Press Releases : Nanoscope Announces Plans to Submit BLA for MCO-010 to Treat Retinitis Pigmentosa Nanoscope Therapeutics Initiates Rolling Submission of Biologics License Application to FDA for MCO-010, the First Gene-Agnostic Therapy to Treat Retinitis Pigmentosa Clinical Publications : (Abstract) Longitudinal AUC analysis of BCVA in patients treated with low- or high-dose MCO-010 mutation agnostic optogenetic therapy for retinitis pigmentosa: 100-week TOPLINE results from a Phase 2b/3 randomized, sham-controlled clinical trial (RESTORE) - EURETINA 2024 (Abstract) Longitudinal BCVA analysis of low- or high-dose MCO-010 mutation agnostic optogenetic therapy for retinitis pigmentosa: 12-month results from a Phase 2b/3 randomized, sham-controlled, patient- and assessor-masked clinical trial (RESTORE) - ARVO 2024 Related NCTID : Phase 1/2: NCT04919473 Long Term Follow Up: NCT06162585
NCT05361031	Charcot-Marie-Tooth Disease 1A	ENGENSIS	Helixmith Co., Ltd.	Industry	HGF	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intramuscular	Plasmid		None (naked plasmid)			0.25mg/0.5ml (56 injections on days 0,14,90,104)	Phase2, Phase1	Completed	Active	2022-03-31	2022-02-22	2025-10-06	19 Years - 65 Years	12	1	South Korea			Same product as NL003	News and Press Releases : Helixmith Co. Ltd announces positive topline data from world's first gene therapy phase 1 study for CMT
NCT00028236	X-linked Severe Combined Immunodeficiency (XSCID)	GALV MFGS-gc retrovirus	National Institute of Allergy and Infectious Diseases (NIAID)	NIH	IL2RG	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	MFGS/GALV		Dose: 30E6 transduced CD34+ cells/kg (n=3)	Phase1	Completed	Inactive	2001-12-17	2011-07-25	2017-07-02	18 Months - 20 Years	3	1	United States			Enrolled 3/8 planned patients	Preclinical Publications : Retroviral transduction of IL2RG into CD34(+) cells from X-linked severe combined immunodeficiency patients permits human T- and B-cell development in sheep chimeras Lymphoid development and function in X-linked severe combined immunodeficiency mice after stem cell gene therapy Comparison of five retrovirus vectors containing the human IL-2 receptor gamma chain gene for their ability to restore T and B lymphocytes in the X-linked severe combined immunodeficiency mouse model Clinical Publications : Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency
NCT01344798	Limb-Girdle Muscular Dystrophy, Type 2C/R5	AAV1.DES.hSGCG	Genethon	Other	SGCG	Gene transfer	In-vivo	Functional gene replacement	Intramuscular (carpi radialis)	Viral vector		Viral transduction	AAV1		Dose 1: 3E9 vg/100ul \| Dose 2: 1.5E10 vg/100ul \| Dose 3: 4.5E10 vg/300ul	Phase1	Completed	Inactive	2011-03-17	2010-06	2011-04-29	>= 15 Years	9	1	France			Vector was well tolerated but expression of transgene was not detectable in most patients after 30 days	Clinical Publications : A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C
NCT04275323	Critical Limb Ischemia	NL003	Beijing Northland Biotech. Co., Ltd.	Industry	HGF	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intramuscular	Plasmid		None (naked plasmid)			Dose 1: Concentration: 0.5mg/1ml (32 sites x 0.5ml), Total 8mg \| Dose 2: Total dose: 12mg \| Dose 3: Total dose: 18mg \| Dose 4: Total dose: 24mg	Phase3	Completed	Active	2020-01-18	2024-06-03	2024-09-19	20 Years - 80 Years	302	13	China			Same product as VM202	Clinical Publications : Nine-year follow-up of patients receiving recombinant human hepatocyte growth factor nude plasmid DNA for critical limb ischemia: Updated safety and efficacy results A Randomized, Double-Blind, Placebo-Controlled Phase II Study of Hepatocyte Growth Factor in the Treatment of Critical Limb Ischemia Rationale and design for the study of recombinant human hepatocyte growth factor plasmid in the treatment of patients with chronic limb-threatening ischemia (HOPE CLTI): Randomized, placebo-controlled, double-blind, phase III clinical trials Related NCTID : Phase 2: NCT01548378 Phase 3: NCT04274049
NCT00985517	Idiopathic Parkinson's Disease	CERE-120	Sangamo Therapeutics	Industry	NRTN	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraparenchymal	Viral vector	Putamen, substantia nigra	Viral transduction	AAV2		Dose 1: Phase 1: 1.3E11 vg (n=6) \| Dose 2: Phase 1: 5.4E11 vg (n=6) \| Dose 3: Phase 2: 5.4E11 vg (n=38) \| Dose 4: Phase 2: 1.0E12 vg (putamen) + 2.0E11 vg (substantia nigra)	Phase2, Phase1	Completed	Inactive	2009-09-25	2017-11-16	2020-04-16	35 Years - 70 Years	57	11	United States			Therapy was originally developed by Ceregene, Inc. Company was acquired by Sangamo Therapeutics in 2013 after CERE-120 failed its Phase 2b trial for Parkinson's disease	Preclinical Publications : Striatal delivery of neurturin by CERE-120, an AAV2 vector for the treatment of dopaminergic neuron degeneration in Parkinson's disease Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys AAV2-mediated delivery of human neurturin to the rat nigrostriatal system: long-term efficacy and tolerability of CERE-120 for Parkinson's disease Issues regarding gene therapy products for Parkinson's disease: the development of CERE-120 (AAV-NTN) as one reference point Transgene expression, bioactivity, and safety of CERE-120 (AAV2-neurturin) following delivery to the monkey striatum Expression, bioactivity, and safety 1 year after adeno-associated viral vector type 2-mediated delivery of neurturin to the monkey nigrostriatal system support cere-120 for Parkinson's disease Bioactivity of AAV2-neurturin gene therapy (CERE-120): differences between Parkinson's disease and nonhuman primate brains Properly scaled and targeted AAV2-NRTN (neurturin) to the substantia nigra is safe, effective and causes no weight loss: support for nigral targeting in Parkinson's disease Enhanced neurotrophic distribution, cell signaling and neuroprotection following substantia nigral versus striatal delivery of AAV2-NRTN (CERE-120) News and Press Releases : Sangamo BioSciences to Acquire Ceregene Clinical Publications : Advancing neurotrophic factors as treatments for age-related neurodegenerative diseases: developing and demonstrating "clinical proof-of-concept" for AAV-neurturin (CERE-120) in Parkinson's disease Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial Post-Mortem Studies of Neurturin Gene Therapy for Parkinson's Disease: Two Subjects with 10 Years CERE120 Delivery Long-term post-mortem studies following neurturin gene therapy in patients with advanced Parkinson's disease Long-Term Safety of Patients with Parkinson's Disease Receiving rAAV2-Neurturin (CERE-120) Gene Transfer Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: A double-blind, randomized, controlled trial Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients Related NCTID : Phase 1: NCT00252850
NCT05417126	Stargardt Disease	MCO-010	Nanoscope Therapeutics Inc.	Industry	MCO	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravitreal	Viral vector	Bipolar cells	Viral transduction	AAV2		1.2E11 gc/eye	Phase2	Completed	Active	2022-06-09	2023-09-28	2023-11-09	>= 16 Years	6	2	United States		Fast Track, Orphan Drug Designation	Phase III Study expected to begin by the end of 2025	Grant : R01 EY028216 R01EY025717 R44 EY025905 Preclinical Publications : Biodistribution of adeno-associated virus type 2 carrying multi-characteristic opsin in dogs following intravitreal injection News and Press Releases : Nanoscope Therapeutics Announces End-of-Phase 2 Meeting with U.S. FDA and Plan to Initiate a Phase 3 Clinical Trial of MCO-010 to Treat Stargardt Macular Degeneration Nanoscope Therapeutics Announces Publication of STARLIGHT Phase 2 Study Evaluating MCO-010 Gene-Agnostic Therapy in Patients with Stargardt Disease Clinical Publications : (Abstract) Longitudinal BCVA Analysis of Patients With Stargardt Disease and Macular Degeneration Treated With MCO-010, a Mutation-Agnostic Optogenetic Therapy: 48-Week Results From a Phase 2a Clinical Trial (STARLIGHT) - ARVO 2024 Safety and efficacy of MCO-010 optogenetic therapy in patients with Stargardt disease in USA (STARLIGHT): an open-label multi-center Ph2 trial Related NCTID : Long Term Follow Up: NCT06048185
NCT03207009	Beta-Thalassemia Major	ZYNTEGLO	Genetix Biotherapeutics Inc.	Industry	HBB	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	BB305 LV		Minimum dose: 5.0E6 CD34+ cells/kg	Phase3	Completed	Approved	2017-06-29	2022-11-15	2024-03-07	0 Years - 50 Years	19	9	Locations:United States + 5 more France, Germany, Greece, Italy, United Kingdom, United States		Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	FDA approval granted 8/17/22, $2.8M estimated cost/treatment	Clinical Publications : Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia Drug product attributes predict clinical efficacy in betibeglogene autotemcel gene therapy for β-thalassemia Case study of betibeglogene autotemcel gene therapy in an adult Greek patient with transfusion-dependent β-thalassaemia of a severe genotype Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia Gene therapy in transfusion-dependent non-β0/β0 genotype β-thalassemia: first real-world experience of beti-cel Protocol : Statistical Analysis Plan FDA Approved ZYNTEGLO Clinical Trial Protocol Related NCTID : Phase 1/2: NCT01745120 Long Term Follow-Up: NCT02633943 Phase 3: NCT02906202 Phase 1/2: NCT02151526
NCT02161380	Leber Hereditary Optic Neuropathy (LHON)	ScAAV2-P1ND4v2	Byron Lam	Other	ND4	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV2		Dose 1: 5.0E8 vg/eye (n=9) \| Dose 2: 2.4E9 vg/eye (n=3) \| Dose 3: 2.5E9 vg/eye (n=9) \| Dose 4: 1.0E10 vg/eye (n=7)	Phase1	Completed	Inactive	2014-06-06	2025-03-31	2025-06-05	>= 15 Years	28	1	United States			Safe but low efficacy, researchers did not advance development	Preclinical Publications : Efficient expression of self-complementary AAV in ganglion cells of the ex vivo primate retina Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial Induction of rapid and highly efficient expression of the human ND4 complex I subunit in the mouse visual system by self-complementary adeno-associated virus Mutant NADH dehydrogenase subunit 4 gene delivery to mitochondria by targeting sequence-modified adeno-associated virus induces visual loss and optic atrophy in mice LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile Clinical Publications : Gene Therapy for Leber Hereditary Optic Neuropathy: Initial Results Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results Leber Hereditary Optic Neuropathy Gene Therapy: Adverse Events and Visual Acuity Results of All Patient Groups (Abstract) Leber Hereditary Optic Neuropathy Gene Therapy: Longitudinal Relationships Among Visual Function and Anatomical Measures -ARVO 2023 Leber Hereditary Optic Neuropathy Gene Therapy: Longitudinal Relationships Among Visual Function and Anatomical Measures Protocol : Trial end points and natural history in patients with G11778A Leber hereditary optic neuropathy : preparation for gene therapy clinical trial
NCT02416622	X-Linked Retinoschisis	RAAV2tYF-CB-hRS1	Beacon Therapeutics	Industry	RS1	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV2tYF		Dose 1: 1E11 vg/eye (n=6) \| Dose 2: 3E11 vg/eye (n=8) \| Dose 3: 6E11 vg/eye (n=13)	Phase2, Phase1	Completed	Active	2015-04-02	2023-05-09	2023-06-12	>= 6 Years	27	9	United States		Orphan Drug Designation	The product was well tolerated, but no signs of clinical activity were observed at six months post treatment, development discontinued in 2018; product was transferred to TeamedOn	Preclinical Publications : The dose-response relationship of subretinal gene therapy with rAAV2tYF-CB-h RS1 in a mouse model of X-linked retinoschisis Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin Safety and Biodistribution Evaluation of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin, in RS1-Deficient Mice News and Press Releases : SEC Form 8-K: Applied Genetic Technologies Corporation Report December 7, 2018 XLRS Orphan Drug Designation Received by TeamedOn Clinical Publications : Intravitreal Delivery of rAAV2tYF-CB-hRS1 Vector for Gene Augmentation Therapy in Patients with X-Linked Retinoschisis: 1-Year Clinical Results Protocol : Clinical Trial Protocol Statistical Analysis Plan
NCT05811351	Geographic Atrophy	JNJ-1887	Janssen Research & Development, LLC	Industry	CD59	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravitreal	Viral vector		Viral transduction	AAV2		Dose 1: 3.56E10 vg/eye \| Dose 2: 1.071E11 vg/mL \| Dose 3: 3.56E11 vg/eye	Phase2	Completed	Active	2023-03-07	2026-02-23	2026-03-13	>= 60 Years	305	162	Locations:United States + 16 more Australia, Belgium, Canada, Czechia, Denmark, Germany, Hungary, Italy, Netherlands, Poland, Portugal, Spain, Sweden, Switzerland, Turkey (Türkiye), United Kingdom, United States				News and Press Releases : CLINICAL TRIAL DOWNLOAD: Data on a Gene Therapy for Dry and Wet AMD Clinical Publications : (Abstract) Pooled safety analysis of a single intravitreal injection of JNJ-1887 (gene therapy, AAVCAGsCD59) in patients with age-related macular degeneration (AMD) - ARVO 2023 Phase 1 Study of JNJ-81201887 Gene Therapy in Geographic Atrophy Secondary to Age-Related Macular Degeneration (Abstract) Phase 1 Study of JNJ-81201887 Gene Therapy in Geographic Atrophy (GA) Due to Age-related Macular Degeneration (AMD) - EURETINA 2023 Related NCTID : Phase 1: NCT03585556 (with anti-VEGF for Wet AMD) Phase 1: NCT03144999 (for Dry AMD)
NCT00643747	Leber Congenital Amaurosis-Type 2	TgAAG76	University College, London	Other	RPE65	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2		Dose 1: 1E11 vg \| Dose 2: 1E12 vg	Phase2, Phase1	Completed	Inactive	2008-03-20	2014-12	2015-12-07	5 Years - 30 Years	12	1	United Kingdom			Vector was originally produced by Targeted Genetics Corporation, clinical trial showed a lack of sustained efficacy	Preclinical Publications : Gene therapy in the second eye of RPE65-deficient dogs improves retinal function RPE65 gene therapy slows cone loss in Rpe65-deficient dogs Successful gene therapy in older Rpe65-deficient dogs following subretinal injection of an adeno-associated vector expressing RPE65 Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium Clinical Publications : Spectral sensitivity measurements reveal partial success in restoring missing rod function with gene therapy Effect of gene therapy on visual function in Leber's congenital amaurosis Long-term effect of gene therapy on Leber's congenital amaurosis
NCT05398029	Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease	VERVE-101	Verve Therapeutics, Inc.	Industry	PCSK9	Gene editing	In-vivo	Gene inactivation	Intravenous	MRNA, LNP	Hepatocyte	Lipid encapsulation	LDLR	ABE8.8m	Dose 1: 0.1 mg/kg \| Dose 2: 0.3 mg/kg \| Dose 3: 0.45 mg/kg \| Dose 4: 0.6 mg/kg	Phase1	Completed	Inactive	2022-05-19	2025-02-14	2025-05-08	18 Years - 75 Years	13	3	Locations:New Zealand + 1 more New Zealand, United Kingdom			This program is deprioritized in favor of VERVE-102	Preclinical Publications : Efficacy and Safety of an Investigational Single-Course CRISPR Base-Editing Therapy Targeting PCSK9 in Nonhuman Primate and Mouse Models (Presentation) Characterization of Guide RNA Site Consistency Across Ancestries and the Potential for Off-Target Editing with the Clinical-Stage Base Editing Medicine, VERVE-101 - ASGCT 2024 Potent, Specific, and Durable Liver Editing of PCSK9 in Preclinical Studies of the CRISPR Base Editing Medicine VERVE-101 - Japanese Atherosclerosis Conference 2024 (Presentation) Proof-of-concept for in vivo Base Editing to Inactivate the PCSK9 Gene and Lower LDL-Cholesterol in Humans - TIDES 2024 News and Press Releases : Verve Therapeutics Announces Pipeline Progress and Anticipated 2025 Milestones Clinical Publications : (Presentation) Safety and Pharmacodynamic Effects of VERVE-101 - AHA Scientific Sessions 2023 (Corporate Presentation) Transforming the Care of Cardiovascular Disease Through Single-course Gene Editing Medicines Interim Results from heart-1 Trial of VERVE-101 Related NCTID : Long Term Follow-Up: NCT06112327
NCT02247843	Sickle Cell Disease	LentiG-βAS3-FB (contains anti-sickling mutations: T87Q, G16D, E22A)	Donald B. Kohn, M.D.	Other	HBB	Gene transfer	Ex-vivo	Overexpression of protective allele/gene	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells	Phase2, Phase1	Completed	Inactive	2014-09-20	2025-09-24	2025-12-17	>= 18 Years	4	1	United States			Study was closed after FDA approvals of Casgevy and Lyfgenia	Grant : DR3-06945 P30 AI028697 Preclinical Publications : β-Globin Lentiviral Vectors Have Reduced Titers due to Incomplete Vector RNA Genomes and Lowered Virion Production β-globin gene transfer to human bone marrow for sickle cell disease Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34+ cells Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study Clinical Publications : β-globin gene transfer to human bone marrow for sickle cell disease (Abstract #347) Clinical Outcomes of Lenti/G-βAS3-FB Lentiviral Vector Gene Therapy for Sickle Cell Disease - ASTCT/CIBMTR 2025
NCT03116113	X-Linked Retinitis Pigmentosa (XLRP)	BIIB112	Biogen	Industry	RPGR	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV8		Dose 1: 5E9 vg/eye (n=3) \| Dose 2: 1E10 vg/eye (n=3) \| Dose 3: 5E10 vg/eye (n=14) \| Dose 4: 1E11 vg/eye (n=3) \| Dose 5: 2.5E11 vg/eye (n=15); Dose 6: 5E11 vg/eye (n=3)	Phase2, Phase1	Completed	Inactive	2017-03-29	2020-11-18	2024-01-18	>= 10 Years	50	8	Locations:United States + 1 more United Kingdom, United States		Orphan Drug Designation	Phase 2/3 study did not meet its primary endpoint, Company suspended development in 2021	Preclinical Publications : Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa News and Press Releases : SEC Form 10-K: BIOGEN INC FY 2021 Clinical Publications : Assessment of Visual Function with Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa in the Randomized XIRIUS Phase 2/3 Study Clinical Trial Results: Clinical Trial of an Investigational Gene Therapy for X-linked Retinitis Pigmentosa Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR Protocol : Clinical Trial Protocol Statistical Analysis Plan
NCT04124042	Osteoarthritis, Knee	XT-150	Xalud Therapeutics, Inc.	Industry	Recombinant IL10	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraarticular	Plasmid		None (naked plasmid)			Dose 1: 150ug \| Dose 2: 450ug (extension study)	Phase2	Completed	Active	2019-10-09	2022-04-26	2025-03-27	45 Years - 85 Years	289	6	Locations:United States + 1 more Australia, United States		Fast Track		News and Press Releases : Xalud Therapeutics Receives FDA Fast Track Designation for XT-150 for the Treatment of Pain Associated with Osteoarthritis of the Knee Xalud Therapeutics Presents Clinical Data on XT-150 in Knee Osteoarthritis at the 2023 Osteoarthritis Research Society International World Congress Clinical Publications : (Abstract LBA-17) XT-150- A NOVEL IMMUNOMODULATORY GENE THERAPY FOR OSTEOARTHRITIS PAIN IN PHASE 2B DEVELOPMENT (Abstract) XT-150- A novel immunomodulatory gene therapy for osteoarthritis pain in phase 2b development Related NCTID : Phase 1: NCT03282149 Phase 1: NCT03477487 Phase 1: NCT04466410 (Terminated trial for neuropathic pain)
NCT05603312	Parkinson's Disease	AAV-GAD	MeiraGTx, LLC	Industry	GAD1/2	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraparenchymal (subthalamic nucleus)	Viral vector		Viral transduction	AAV2		Dose 1: 7.0E10 vg \| Dose 2: 21E10 vg	Phase2, Phase1	Completed	Active	2022-10-12	2024-09-06	2025-09-05	25 Years - 85 Years	14	6	United States		Regenerative Medicine Advanced Therapy	Initiation of Phase III design discussions in H2 2024	Preclinical Publications : Subthalamic GAD gene therapy in a Parkinson's disease rat model News and Press Releases : MeiraGTx Announces Positive Data from Randomized, Sham-controlled Clinical Bridging Study of AAV-GAD for the Treatment of Parkinson's Disease MeiraGTx Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for AAV-GAD for the Treatment of Parkinson’s Disease Corporate Presentation November 2025 Clinical Publications : Network modulation following sham surgery in Parkinson's disease Gene therapy reduces Parkinson's disease symptoms by reorganizing functional brain connectivity Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial Long-term follow-up of a randomized AAV2- GAD gene therapy trial for Parkinson's disease Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial Related NCTID : Phase 1: NCT00195143 Long Term Follow-Up: NCT05894343 Phase 2: NCT00643890
NCT02341807	Choroideremia	SPK-7001	Spark Therapeutics, Inc.	Industry	CHM	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2		Dose 1: 5E10 vg/eye (n=5) \| Dose 2: 1E11 vg/eye (n=10)	Phase2, Phase1	Completed	Inactive	2015-01-12	2022-10-12	2024-01-25	>= 18 Years	15	3	United States		Orphan Drug Designation	Roche acquired Spark in December 2019, announced they were halting this program in the 2021 annual report	Preclinical Publications : AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models News and Press Releases : Roche enters into definitive merger agreement to acquire Spark Therapeutics Roche 2021 Annual Report Clinical Publications : Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial Short-term Assessment of Subfoveal Injection of Adeno-Associated Virus-Mediated hCHM Gene Augmentation in Choroideremia Using Adaptive Optics Ophthalmoscopy (Abstract) Cone mosaic integrity in choroideremia following gene augmentation via subretinal injection of AAV2-hCHM - ARVO 2021 Protocol : Clinical Trial Protocol Statistical Analysis Plan
NCT03432364	Beta-Thalassemia Major	ST-400	Sangamo Therapeutics	Industry	BCL11A	Gene editing	Ex-vivo	Overexpression of protective allele/gene	Intravenous	Autologous cells	CD34+ cells	Electroporation		ZFN mRNA	Dose 1: Minimum dose: 4.5E6 cells/kg \| Dose 2: Max dose: 11.4E6 CD34+ cells/kg \| Dose 3: Average dose: 7.3E6 CD34+ cells/kg	Phase2, Phase1	Completed	Inactive	2018-02-01	2022-11-17	2023-12-14	18 Years - 40 Years	5	6	United States		Orphan Drug Designation	Development discontinued by Sponsor November 2021	Preclinical Publications : Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major News and Press Releases : Sangamo Therapeutics Reports Recent Business and Clinical Highlights and Third Quarter 2021 Financial Results Sangamo Announces Preliminary Results From the First Three Patients in a Phase 1/2 Study Evaluating ST-400 Ex Vivo Gene-edited Cell Therapy in Beta Thalassemia Clinical Publications : (Poster) Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia - ASH 2021 Protocol : Clinical Trial Protocol Statistical Analysis Plan Related NCTID : Long Term Follow-Up: NCT05145062
NCT06594094	Duchenne Muscular Dystrophy (DMD)	HG302	HuidaGene Therapeutics Co., Ltd.	Industry	DMD (exon 51 splice site)	Gene editing	In-vivo	Exon skipping/splice editor	Intravenous	Viral vector		Viral transduction	AAV	hfCas12Max	Up to 2 dose cohorts, undisclosed concentration	Early phase1	Completed	Active	2024-09-10	2025-12-02	2026-02-17	4 Years - 8 Years	4	1	China		Orphan Drug Designation, Rare Pediatric Disease Designation	First patient dosed announced in December 2024	Preclinical Publications : An engineered xCas12i with high activity, high specificity, and broad PAM range (Abstract 719LBP) Duchenne muscular dystrophy gene editing therapy with CRISPR/high-fidelity Cas12Max) - WMS 2024 News and Press Releases : HuidaGene Therapeutics Initiates M.U.S.C.L.E. Clinical Trial of HG302 for Duchenne Muscular Dystrophy and Completes First Patient Dosed HuidaGene Receives Orphan Drug Designation from FDA for HG302 for the Potential Treatment of Duchenne Muscular Dystrophy after Receiving Rare Pediatric Drug Designation HuidaGene Announces Rare Pediatric Drug Designation Granted to HG302, A Novel CRISPR DNA-editing Therapy, for the Treatment of Duchenne Muscular Dystrophy
NCT00927134	X-Linked Chronic Granulomatous Disease	Retroviral SF71-gp91phox transduced CD34+ cells	University of Zurich	Other	CYBB	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	SFFV		Transduced CD34+ cells (dose range: 3.6-5.1E6 cells/kg)	Phase2, Phase1	Completed	Inactive	2009-06-22	2011-09	2011-09-27	1 Year - 18 Years	2	1	Switzerland			Enrolled 2 patients, 2 patients enrolled under NCT00564759, same vector used in both	Preclinical Publications : Use of serum-free medium with fibronectin fragment enhanced transduction in a system of gas permeable plastic containers to achieve high levels of retrovirus transduction at clinical scale Retroviral vector integration in post-transplant hematopoiesis in mice conditioned with either submyeloablative or ablative irradiation Clinical Publications : Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1 Restoration of NET formation by gene therapy in CGD controls aspergillosis Related NCTID : Phase 1/2: NCT00564759
NCT02082860	Acute Intermittent Porphyria	RAAV2/5-PBGD	Digna Biotech S.L.	Industry	PBGD	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV2/5		Dose 1: 5.0E11 gc/kg \| Dose 2: 2E12 gc/kg \| Dose 3: 6E12 gc/kg \| Dose 4: 1.8E13 gc/kg	Phase1	Completed	Inactive	2014-02-28	2014-11	2014-12-18	18 Years - 64 Years	8	2	Spain			Therapy was safe, but failed to show efficacy, this product did not advance to Phase II, original developers working on alternatives	Preclinical Publications : Porphobilinogen deaminase over-expression in hepatocytes, but not in erythrocytes, prevents accumulation of toxic porphyrin precursors in a mouse model of acute intermittent porphyria Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria An Inducible Promoter Responsive to Different Porphyrinogenic Stimuli Improves Gene Therapy Vectors for Acute Intermittent Porphyria Bioengineered PBGD variant improves the therapeutic index of gene therapy vectors for acute intermittent porphyria Clinical Publications : Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria
NCT06291961	Beta-Thalassemia Major	CS-101	CorrectSequence Therapeutics Co., Ltd	Industry	BCL11A	Gene editing	Ex-vivo	Overexpression of protective allele/gene	Intravenous	Autologous cells	CD34+ cells	Electroporation	none	transformer BE RNP	Transduced CD34+ cells	Phase1	Completed	Active	2024-02-23	2025-11-17	2026-02-10	12 Years - 35 Years	9	3	China				Preclinical Publications : Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations Base editing of the HBG promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs News and Press Releases : Locally-Developed Gene Editing Technology Cures Boy with Serious Blood Disorder Clinical Publications : (Poster) Rapid, Efficient and Durable Fetal Hemoglobin Production Following CS-101 Treatment in Transfusion-Dependent ÃÂ²-Thalassemia Participants: An Autologous, Ex Vivo Edited CD34+ Stem Cell Product Using the Innovative Transformer Base Editor (tBE) - ASH 2024 Protocol : Protocol for examining and eliminating base editor-induced genome-wide and transcriptome-wide off-target mutations Related NCTID : Early Phase 1: NCT06065189 Early Phase 1: NCT06328764 Long Term Follow-Up: NCT06479616 Early Phase 1: NCT06024876
NCT04240314	Duchenne Muscular Dystrophy (DMD)	AT702	Megan Waldrop	Other	DMD (exon 2)	Gene transfer	In-vivo	Exon skipping/splice editor	Intravenous	Viral vector		Viral transduction	AAV9		3E13 vg/kg	Phase2, Phase1	Completed	Inactive	2020-01-22	2025-07-01	2025-09-11	6 Months - 13 Years	3	1	United States			Product was licensed to Audentes/Astellas Therapeutics in 2019, discontinuation was announced in April 2022	Preclinical Publications : (Abstract P13) Comparison of U7snRNA-induced dystrophin expression following systemic delivery with AAV9, MyoAAV 2A, and MyoAAV 3A capsids in the Dup2 mouse Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model [Webinar] Understanding Gene Therapy, Part 3 - Galgt2 and Dup2 - December 2017 Clinical Publications : (Abstract) Expression of apparent full-length dystrophin in skeletal muscle in a first-in-human gene therapy trial using the scAAV9.U7-ACCA vector

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