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

Showing 1-100 of 125 results in ClinicalTrials

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NCT06467344 Recruiting Phase2, Phase1

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Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration

Sponsor Ascidian Therapeutics, Inc (Industry)

Compound ACDN-01

Therapeutic Modality Gene editing

Target Gene ABCA4 pre-mRNA

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Exon skipping/splice editor

FDA Designations Fast Track, Rare Pediatric Disease Designation

NCT01306019 Recruiting Phase2, Phase1

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X-linked Severe Combined Immunodeficiency (XSCID)

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

Compound VSV-G pseudotyped CL20-i4-EF1α-hγc-OPT vector

Therapeutic Modality Gene transfer

Target Gene IL2RG

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06092034 Recruiting Phase2

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Danon Disease

Sponsor Rocket Pharmaceuticals Inc. (Industry)

Compound RP-A501

Therapeutic Modality Gene transfer

Target Gene LAMP2B

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06389877 Recruiting Phase2, Phase1

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Alpha-1 Antitrypsin Deficiency (AATD)

Sponsor Beam Therapeutics Inc. (Industry)

Compound BEAM-302

Therapeutic Modality Gene editing

Target Gene SERPINA1 (p.Glu366Lys)

Delivery System Endocytosis

Mechanism of Action Mutation correction

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

NCT07137338 Recruiting Phase1

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BAG3-Associated Dilated Cardiomyopathy (DCM)

Sponsor Rocket Pharmaceuticals Inc. (Industry)

Compound RP-A701

Therapeutic Modality Gene transfer

Target Gene BAG3

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track

NCT05878860 Recruiting Phase3

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X-Linked Retinoschisis

Sponsor Atsena Therapeutics Inc. (Industry)

Compound ATSN-201

Therapeutic Modality Gene transfer

Target Gene RS1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT07180355 Recruiting Phase1

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Friedreich's Ataxia

Sponsor Solid Biosciences Inc. (Industry)

Compound SGT-212

Therapeutic Modality Gene transfer

Target Gene FXN

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT05821959 Recruiting Phase3

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Bilateral Sensorineural Hearing Loss

Sponsor Eli Lilly and Company (Industry)

Compound AK-OTOF

Therapeutic Modality Gene transfer

Target Gene OTOF

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT07160634 Recruiting Phase3

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Duchenne Muscular Dystrophy (DMD)

Sponsor Solid Biosciences Inc. (Industry)

Compound SGT-003

Therapeutic Modality Gene transfer

Target Gene Micro-dystrophin

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

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

NCT07592273 Recruiting Phase3, Phase2

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Diabetic Retinopathy

Sponsor AbbVie (Industry)

Compound Surabgene lomparvovec

Therapeutic Modality Gene transfer

Target Gene Anti-VEGF

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT06270316 Recruiting Phase2, Phase1

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Fabry Disease

Sponsor UniQure Biopharma B.V. (Industry)

Compound AMT-191

Therapeutic Modality Gene transfer

Target Gene GLA

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT07148089 Recruiting Phase1

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Catecholaminergic Polymorphic Ventricular Tachycardia

Sponsor Solid Biosciences Inc. (Industry)

Compound SGT-501

Therapeutic Modality Gene transfer

Target Gene CASQ2

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

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

NCT07185256 Recruiting Phase2, Phase1

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

Sponsor Opus Genetics, Inc (Industry)

Compound OPGx-BEST1

Therapeutic Modality Gene transfer

Target Gene BEST1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT07587021 Recruiting Phase2

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Primary Hyperoxaluria Type 1, PH1

Sponsor YolTech Therapeutics Co., Ltd (Industry)

Compound YOLT-203

Therapeutic Modality Gene editing

Target Gene HAO1

Delivery System Endocytosis

Mechanism of Action Gene inactivation

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

NCT06364774 Recruiting Phase2, Phase1

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Beta-Thalassemia Major

Sponsor Children's Hospital of Philadelphia (Other)

Compound CHOP-ALS20

Therapeutic Modality Gene transfer

Target Gene HBB

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06910813 Recruiting Phase2, Phase1

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Nephropathic Cystinosis

Sponsor Novartis Pharmaceuticals (Industry)

Compound DFT383

Therapeutic Modality Gene transfer

Target Gene CTNS

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05419492 Recruiting Phase2, Phase1

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Dravet Syndrome

Sponsor Encoded Therapeutics (Industry)

Compound ETX101

Therapeutic Modality Gene transfer

Target Gene SCN1A

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT02716246 Recruiting Phase3, Phase2

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Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)

Sponsor Ultragenyx Pharmaceutical Inc (Industry)

Compound UX111

Therapeutic Modality Gene transfer

Target Gene SGSH

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06844214 Recruiting Phase2, Phase1

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Myotonic Dystrophy

Sponsor Sanofi (Industry)

Compound SAR446268

Therapeutic Modality Gene transfer

Target Gene DMPK

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Gene inactivation

FDA Designations Fast Track, Orphan Drug Designation

NCT03952637 Recruiting Phase2, Phase1

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GM1 Gangliosidosis

Sponsor National Human Genome Research Institute (NHGRI) (NIH)

Compound AXO-AAV-GM1

Therapeutic Modality Gene transfer

Target Gene GLB1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT07491172 Recruiting Phase1

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Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Uncontrolled Hypercholesterolemia, Mixed Dyslipidemia

Sponsor CRISPR Therapeutics AG (Industry)

Compound CTX310

Therapeutic Modality Gene editing

Target Gene ANGPTL3

Delivery System Endocytosis

Mechanism of Action Gene inactivation

NCT07007065 Recruiting Phase3

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Neovascular Age-related Macular Degeneration

Sponsor AbbVie (Industry)

Compound ABBV-RGX-314

Therapeutic Modality Gene transfer

Target Gene Anti-VEGF

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT07226206 Recruiting Phase2, Phase1

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Hemophilia A

Sponsor Hoffmann-La Roche (Industry)

Compound SPK-8011QQ

Therapeutic Modality Gene transfer

Target Gene F8

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT07048808 Recruiting Phase2

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Refractory Angina, Coronary Artery Disease

Sponsor XyloCor Therapeutics, Inc. (Industry)

Compound XC001

Therapeutic Modality Gene transfer

Target Gene VEGF

Vector Type Adenovirus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT07287397 Recruiting Phase2, Phase1

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Amyotrophic Lateral Sclerosis

Sponsor Vector Y Therapeutics (Industry)

Compound VTx-002

Therapeutic Modality Gene transfer

Target Gene Antibody to TDP-43

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

FDA Designations Fast Track

NCT07447557 Recruiting Phase2, Phase1

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Charcot-Marie-Tooth Disease Type 4J

Sponsor Elpida Therapeutics SPC (Industry)

Compound ELP-02

Therapeutic Modality Gene transfer

Target Gene FIG4

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT07770828 Recruiting Phase3

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Dry Age-related Macular Degeneration

Sponsor Ocugen (Industry)

Compound OCU410

Therapeutic Modality Gene transfer

Target Gene RORA

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT06761183 Recruiting Early phase1

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Ischemic Stroke

Sponsor Beijing Tiantan Hospital (Other)

Compound NXL-001

Therapeutic Modality Gene transfer

Target Gene NeuroD1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT06662188 Recruiting Phase2, Phase1

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SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome

Sponsor Jaguar Gene Therapy, LLC (Industry)

Compound JAG201

Therapeutic Modality Gene transfer

Target Gene SHANK3

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Rare Pediatric Disease Designation

NCT07223944 Recruiting Phase3

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Gaucher Disease Type 1

Sponsor Spur Therapeutics (Industry)

Compound FLT201

Therapeutic Modality Gene transfer

Target Gene GBA1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT07052929 Recruiting Phase2, Phase1

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X-Linked Myotubular Myopathy

Sponsor Astellas Gene Therapies (Industry)

Compound ASP2957

Therapeutic Modality Gene transfer

Target Gene MTM1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06164730 Recruiting Phase1

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Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease

Sponsor Verve Therapeutics, Inc. (Industry)

Compound VERVE-102

Therapeutic Modality Gene editing

Target Gene PCSK9

Delivery System Endocytosis

Mechanism of Action Gene inactivation

FDA Designations Fast Track

NCT07721025 Recruiting Phase2

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Friedreich Ataxia with Secondary Cardiomyopathy

Sponsor Lexeo Therapeutics (Industry)

Compound LX2006

Therapeutic Modality Gene transfer

Target Gene FXN

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT07064759 Recruiting Phase3

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Neovascular Age-Related Macular Degeneration (nAMD)

Sponsor 4D Molecular Therapeutics (Industry)

Compound 4D-150

Therapeutic Modality Gene transfer

Target Gene Anti-VEGF

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT07218887 Recruiting Phase2, Phase1

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BAG3 Mutation Associated Dilated Cardiomyopathy

Sponsor Alexion Pharmaceuticals, Inc. (Industry)

Compound ALXN2350

Therapeutic Modality Gene transfer

Target Gene BAG3

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05986864 Recruiting Phase2, Phase1

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

Sponsor Skyline Therapeutics (US) Inc. (Industry)

Compound SKG0106

Therapeutic Modality Gene transfer

Target Gene Anti-VEGF

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT07627971 Recruiting Phase2, Phase1

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GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A

Sponsor Skylark Bio Inc. (Industry)

Compound SKY-GJB2

Therapeutic Modality Gene transfer

Target Gene GJB2

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT02852213 Recruiting Phase1

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Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

Sponsor Krzysztof Bankiewicz (Other)

Compound AAV2-hAADC

Therapeutic Modality Gene transfer

Target Gene DDC

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT07667387 Recruiting Phase2, Phase1

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Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency

Sponsor Rebecca Ahrens-Nicklas (Other)

Compound LNP.UCD.ABE

Therapeutic Modality Gene editing

Target Gene CPS1,OTC,ASS1,ASL,ARG,NAGS,SLC25A15

Delivery System Endocytosis

Mechanism of Action Mutation correction

NCT06884865 Recruiting Phase2

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Osteoarthritis (OA) of the Knee

Sponsor Pacira Pharmaceuticals, Inc (Industry)

Compound PCRX-201

Therapeutic Modality Gene transfer

Target Gene IL1RA

Vector Type Adenovirus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT05248230 Recruiting Phase2

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Cystic Fibrosis

Sponsor 4D Molecular Therapeutics (Industry)

Compound 4D-710

Therapeutic Modality Gene transfer

Target Gene CFTR

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT07429240 Recruiting Phase2, Phase1

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Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD

Sponsor Precision BioSciences, Inc. (Industry)

Compound PBGENE-DMD

Therapeutic Modality Gene editing

Target Gene DMD del ex45-55

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Exon skipping/splice editor

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

NCT06680232 Recruiting Phase1

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Chronic Hepatitis B

Sponsor Precision BioSciences, Inc. (Industry)

Compound PBGENE-HBV

Therapeutic Modality Gene editing

Target Gene HBV cccDNA

Delivery System Endocytosis

Mechanism of Action Gene inactivation

FDA Designations Fast Track

NCT06063850 Recruiting Phase2, Phase1

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Mesial Temporal Lobe Epilepsy

Sponsor UniQure Biopharma B.V. (Industry)

Compound AMT-260

Therapeutic Modality Gene transfer

Target Gene MiGRIK2

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action MiRNA knockdown of mutant/aberrant gene

NCT07643844 Recruiting Phase1

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Propionic Acidemia

Sponsor Mayo Clinic (Other)

Compound AAVrh10-PCCA

Therapeutic Modality Gene transfer

Target Gene PCCA

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06332807 Recruiting Phase2, Phase1

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Phenylketonuria (PKU)

Sponsor NGGT INC. (Industry)

Compound NGGT002

Therapeutic Modality Gene transfer

Target Gene PAH

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT06831825 Recruiting Phase1

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Heart Failure With Reduced Ejection Fraction

Sponsor Medley Therapeutics (Industry)

Compound YAP101

Therapeutic Modality Gene transfer

Target Gene SAV1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Gene inactivation

NCT06128629 Recruiting Phase3

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Transthyretin Amyloidosis (ATTR) with Cardiomyopathy

Sponsor Intellia Therapeutics (Industry)

Compound NTLA-2001

Therapeutic Modality Gene editing

Target Gene TTR

Delivery System Endocytosis

Mechanism of Action Gene inactivation

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

NCT05616793 Recruiting Phase2, Phase1

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Leber Congenital Amaurosis-Type 5

Sponsor Opus Genetics, Inc (Industry)

Compound OPGx-001

Therapeutic Modality Gene transfer

Target Gene LCA5

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06325709 Recruiting Phase2, Phase1

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X-Linked Chronic Granulomatous Disease

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

Compound Base-edited autologous CD34+ cells

Therapeutic Modality Gene editing

Target Gene CYBB c.676 C>T

Delivery System Electroporation

Mechanism of Action Mutation correction

NCT06064890 Recruiting Phase2, Phase1

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Frontotemporal Dementia, FTD, FTD-GRN

Sponsor AviadoBio Ltd (Industry)

Compound AVB-101

Therapeutic Modality Gene transfer

Target Gene GRN

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT06292650 Recruiting Early phase1

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Retinitis Pigmentosa

Sponsor Zhongmou Therapeutics (Industry)

Compound ZM-02

Therapeutic Modality Gene transfer

Target Gene Ch2.0

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Orphan Drug Designation

NCT06217861 Recruiting Phase1

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Glutaric Acidemia Type I

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

Compound VGM-R02b

Therapeutic Modality Gene transfer

Target Gene GCDH

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06517888 Recruiting Phase2, Phase1

View Report

Vestibular Schwannoma

Sponsor Akouos, Inc. (Industry)

Compound AAVAnc80-antiVEGF

Therapeutic Modality Gene transfer

Target Gene Anti-VEGF

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT06272149 Recruiting Early phase1

View Report

Type II Gaucher Disease

Sponsor Xinhua Hospital, Shanghai Jiao Tong University School of Medicine (Other)

Compound VGN-R08b

Therapeutic Modality Gene transfer

Target Gene GBA1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06765980 Recruiting Phase2, Phase1

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Geographic Atrophy Secondary to Age-related Macular Degeneration

Sponsor Kriya Therapeutics, Inc. (Industry)

Compound KRIYA-825

Therapeutic Modality Gene transfer

Target Gene CR2-CR1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT05788536 Recruiting Phase2, Phase1

View Report

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

Sponsor Regeneron Pharmaceuticals (Industry)

Compound OTARMENI

Therapeutic Modality Gene transfer

Target Gene OTOF

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT07195825 Recruiting Phase1

View Report

Parkinson's Disease

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

Compound BBM-P002

Therapeutic Modality Gene transfer

Target Gene TH90del-AADC

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT06185673 Recruiting Phase2, Phase1

View Report

Oculopharyngeal Muscular Dystrophy

Sponsor Benitec Biopharma, Inc. (Industry)

Compound BB-301

Therapeutic Modality Gene transfer

Target Gene PABPN1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement, Gene inactivation

FDA Designations Fast Track, Orphan Drug Designation

NCT05791864 Recruiting Phase2, Phase1

View Report

Neuronal Ceroid Lipofuscinosis Type 2

Sponsor Tern Therapeutics, LLC (Industry)

Compound TTX-381

Therapeutic Modality Gene transfer

Target Gene TPP1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT04676048 Recruiting Phase2, Phase1

View Report

Hemophilia A

Sponsor ASC Therapeutics (Industry)

Compound ASC618

Therapeutic Modality Gene transfer

Target Gene F8

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT07058662 Recruiting Phase2, Phase1

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Belief BioMed (Beijing) Co., Ltd (Industry)

Compound BBM-D101

Therapeutic Modality Gene transfer

Target Gene Undisclosed

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Undisclosed

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06291935 Recruiting Phase1

View Report

Retinitis Pigmentosa

Sponsor VeonGen Therapeutics GmbH (Industry)

Compound VG901

Therapeutic Modality Gene transfer

Target Gene CNGA1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT07543991 Recruiting Phase1

View Report

Giant Axonal Neuropathy (GAN)

Sponsor University of Texas Southwestern Medical Center (Other)

Compound ScAAV9/JeT-GAN

Therapeutic Modality Gene transfer

Target Gene GAN

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06280378 Recruiting Phase2, Phase1

View Report

Beta-Thalassemia Major

Sponsor Kanglin Biotechnology (Hangzhou) Co., Ltd. (Industry)

Compound KL003

Therapeutic Modality Gene transfer

Target Gene HBB

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06526923 Recruiting Phase2, Phase1

View Report

Cystic Fibrosis

Sponsor Spirovant Sciences, Inc. (Industry)

Compound SP-101

Therapeutic Modality Gene transfer

Target Gene CFTRΔR

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06492876 Recruiting Phase2, Phase1

View Report

Diabetic Macular Edema

Sponsor Frontera Therapeutics (Industry)

Compound FT-003

Therapeutic Modality Gene transfer

Target Gene Aflibercept

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT06833983 Recruiting Phase3

View Report

Hemophilia A

Sponsor Gritgen Therapeutics Co., Ltd. (Industry)

Compound GS1191-0445

Therapeutic Modality Gene transfer

Target Gene F8

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05241444 Recruiting Phase1

View Report

Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome (IPEX)

Sponsor Bacchetta, Rosa, MD (Other)

Compound CD4^LVFOXP3

Therapeutic Modality Gene transfer

Target Gene FOXP3

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06942572 Recruiting Phase2, Phase1

View Report

Stargardt Disease

Sponsor Splice Bio (Industry)

Compound SB-007

Therapeutic Modality Gene transfer

Target Gene ABCA4

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT05762510 Recruiting Early phase1

View Report

Beta-Thalassemia Major

Sponsor First Affiliated Hospital of Guangxi Medical University (Other)

Compound GMCN-508B

Therapeutic Modality Gene transfer

Target Gene HBB

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05860569 Recruiting Phase1

View Report

Hypertriglyceridemia

Sponsor GeneCradle Inc (Industry)

Compound GC304

Therapeutic Modality Gene transfer

Target Gene GPIHBP1 or LPL

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement, Overexpression of protective allele/gene

NCT05672121 Recruiting Phase2, Phase1

View Report

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

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

Compound KH631

Therapeutic Modality Gene transfer

Target Gene VEGFR1/R2 fusion protein

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT03466463 Recruiting Na

View Report

Crigler-Najjar Syndrome

Sponsor Genethon (Other)

Compound GNT0003

Therapeutic Modality Gene transfer

Target Gene UGT1A1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03311503 Recruiting Phase2, Phase1

View Report

X-linked Severe Combined Immunodeficiency (XSCID)

Sponsor David Williams (Other)

Compound G2SCID lentiviral vector transduced CD34+ cells

Therapeutic Modality Gene transfer

Target Gene IL2RG

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03538899 Recruiting Phase2, Phase1

View Report

Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency

Sponsor University of California, San Francisco (Other)

Compound AProArt

Therapeutic Modality Gene transfer

Target Gene DCLRE1C

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05824169 Recruiting Phase2, Phase1

View Report

Spinal Muscular Atrophy

Sponsor GeneCradle Inc (Industry)

Compound GC101

Therapeutic Modality Gene transfer

Target Gene SMN1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT06345898 Recruiting Early phase1

View Report

X-Linked Retinoschisis

Sponsor West China Hospital (Other)

Compound JWK002

Therapeutic Modality Gene transfer

Target Gene RS1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05757245 Recruiting Phase1

View Report

Transfusion-dependent Alpha-Thalassemia

Sponsor First Affiliated Hospital of Guangxi Medical University (Other)

Compound GMCN-508A

Therapeutic Modality Gene transfer

Target Gene HBA

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06519552 Recruiting Phase1

View Report

Mucopolysaccharidosis Type I (Hurler Syndrome)

Sponsor West China Hospital (Other)

Compound JWK-008

Therapeutic Modality Gene transfer

Target Gene IDUA

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06238908 Recruiting Early phase1

View Report

Hemophilia A

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

Compound NGGT003

Therapeutic Modality Gene transfer

Target Gene F8

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06650319 Recruiting Early phase1

View Report

Wilson Disease

Sponsor Chaohui Yu (Other)

Compound LY-M003

Therapeutic Modality Gene transfer

Target Gene ATP7B

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05858983 Recruiting Phase2, Phase1

View Report

Biallelic RPE65 Mutation-associated Retinal Dystrophy

Sponsor Frontera Therapeutics (Industry)

Compound FT-001

Therapeutic Modality Gene transfer

Target Gene RPE65

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05092685 Recruiting Phase2, Phase1

View Report

Ornithine Transcarbamylase (OTC) Deficiency

Sponsor University College, London (Other)

Compound BGT-OTCD

Therapeutic Modality Gene transfer

Target Gene OTC

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT06641154 Recruiting Phase2, Phase1

View Report

Crigler-Najjar Syndrome Type I

Sponsor Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare (Other gov)

Compound GT-UGT1A1-AAV8-02

Therapeutic Modality Gene transfer

Target Gene UGT1A1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04819841 Recruiting Phase2, Phase1

View Report

Sickle Cell Disease

Sponsor Kamau Therapeutics (Industry)

Compound KMAU-001

Therapeutic Modality Gene editing

Target Gene HBB

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Mutation correction

NCT06818838 Recruiting Phase2, Phase1

View Report

Gaucher Disease Type 1

Sponsor Lingyi Biotech Co., Ltd. (Industry)

Compound LY-M001

Therapeutic Modality Gene transfer

Target Gene GBA1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04998396 Recruiting Phase2, Phase1

View Report

Canavan Disease

Sponsor Aspa Therapeutics (Industry)

Compound BBP-812

Therapeutic Modality Gene transfer

Target Gene ASPA

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06391736 Recruiting Phase2, Phase1

View Report

Pompe Disease (Late-onset)

Sponsor GeneCradle Inc (Industry)

Compound GC301

Therapeutic Modality Gene transfer

Target Gene GAA

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT06692712 Recruiting Phase3

View Report

Hereditary Spastic Paraplegia Type 50

Sponsor Elpida Therapeutics SPC (Industry)

Compound MELPIDA

Therapeutic Modality Gene transfer

Target Gene AP4M1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT07476183 Recruiting Phase1

View Report

RPS19 Deficient Diamond-Blackfan Anemia

Sponsor Apriligen, Inc. (Industry)

Compound APR-2020

Therapeutic Modality Gene transfer

Target Gene RPS19

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06492863 Recruiting Phase2, Phase1

View Report

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

Sponsor Frontera Therapeutics (Industry)

Compound FT-003

Therapeutic Modality Gene transfer

Target Gene Aflibercept

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT05761899 Recruiting Phase2, Phase1

View Report

Hereditary Pulmonary Alveolar Proteinosis

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

Compound Gene-Corrected Macrophages

Therapeutic Modality Gene transfer

Target Gene CSF2RA

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05885412 Recruiting Phase1

View Report

PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Sponsor Rocket Pharmaceuticals Inc. (Industry)

Compound RP-A601

Therapeutic Modality Gene transfer

Target Gene PKP2

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT04278131 Recruiting Phase2, Phase1

View Report

Retinitis Pigmentosa

Sponsor Bionic Sight LLC (Industry)

Compound BS01

Therapeutic Modality Gene transfer

Target Gene ChronosFP

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT05682144 Recruiting Phase1

View Report

Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS

Sponsor Immusoft of CA, Inc. (Industry)

Compound ISP-001

Therapeutic Modality Gene transfer

Target Gene IDUA

Delivery System Electroporation

Mechanism of Action Functional gene replacement

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

NCT05071222 Recruiting Phase2, Phase1

View Report

Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency

Sponsor Assistance Publique - Hôpitaux de Paris (Other)

Compound ARTEGENE

Therapeutic Modality Gene transfer

Target Gene DCLRE1C

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06228924 Recruiting Phase1

View Report

Arrhythmogenic Right Ventricular Cardiomyopathy

Sponsor Tenaya Therapeutics (Industry)

Compound TN-401

Therapeutic Modality Gene transfer

Target Gene PKP2

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06399107 Recruiting Phase2, Phase1

View Report

Sickle Cell Disease, Sickle-Cell Disease With Crisis

Sponsor Essen Biotech (Other)

Compound BAH243

Therapeutic Modality Gene transfer

Target Gene HBB

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT06465550 Recruiting Phase1

View Report

Beta-Thalassemia Major

Sponsor Shanghai BDgene Co., Ltd. (Industry)

Compound BD211

Therapeutic Modality Gene transfer

Target Gene HBB

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

Visual summary of all 125 matching trials (across every page). Click a slice or bar to filter the results by it.

Therapeutic Modality

Current Phase

Recruitment Status

Funder Type

Vector Type

Conditions Studied

Age Group

Top Locations

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Updates in the last 14 days

NCT06467344

1791349200000

Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration

Recruiting

Rare Pediatric Disease designation granted 4/25/24

NCT01306019

1791262800000

X-linked Severe Combined Immunodeficiency (XSCID)

Recruiting

Developed in partnership with St. Jude, and licensed to Mustang Bio, Mustang Bio discontinued this program

NCT06092034

1791262800000

Danon Disease

Recruiting

Clinical hold lifted August 2025

NCT06389877

1791176400000

Alpha-1 Antitrypsin Deficiency (AATD)

Recruiting

Data from Parts A and B of Phase 1/2 trial released in March 2026, Beam intends to pursue an accelerated approval for BEAM-302

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.

SCGE Platform GTCT
Trial ID	Indication	Drug Product Name	Sponsor	Funder Type	Target Gene/Variant	Therapeutic Modality	Therapy Route	Mechanism of Action	Route of Administration ^>	Drug Product Type	Target Tissue/Cell	Gene Delivery System Type	Vector Type	Editor Type	Dosage/s	Current Phase	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	Drug Product Name	Sponsor	Funder Type	Target Gene/Variant	Therapeutic Modality	Therapy Route	Mechanism of Action	Route of Administration ^>	Drug Product Type	Target Tissue/Cell	Gene Delivery System Type	Vector Type	Editor Type	Dosage/s	Current Phase	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
NCT06467344	Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration	ACDN-01	Ascidian Therapeutics, Inc	Industry	ABCA4 pre-mRNA	Gene editing	in vivo	Exon skipping/splice editor	Subretinal	Viral vector	null	Viral transduction	Adeno-associated virus		Undisclosed dose escalation, 3 levels	Phase2, Phase1	Recruiting	Active	2024-05-31	2030-12-01	2026-10-07	>= 12 Years	15	11	United States		Fast Track, Rare Pediatric Disease Designation	Rare Pediatric Disease designation granted 4/25/24	Preclinical Publications : RNA-rewriting candidate moves into the clinic (Abstract #706) Exon Editing of ABCA4 RNA in Human Retinal Explants and Non-Human Primate Retina for the Treatment of Stargardt Disease - ASGCT 2024 News and Press Releases : Ascidian Therapeutics Announces First-Ever IND for an RNA Exon Editor as FDA Approves Trial Plan and Fast Tracks ACDN-01 in Stargardt Disease and Other ABCA4 Retinopathies Clinical Publications : (Abstract #351) Rewriting ABCA4 RNA for the Treatment of Stargardt Disease - ASGCT 2023
NCT01306019	X-linked Severe Combined Immunodeficiency (XSCID)	VSV-G pseudotyped CL20-i4-EF1α-hγc-OPT vector	National Institute of Allergy and Infectious Diseases (NIAID)	NIH	IL2RG	Gene transfer	ex vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	Lentivirus		Dose 1: Transduced CD34+ cells (median dose: 6.73E6 cells/kg; range: 4.46-15.10E6 cells/kg, n=8) \| Dose 2: Transduced CD34+ cells (median dose: 20.4E6 cells/kg; range: 16-25E6 cells/kg, n=5)	Phase2, Phase1	Recruiting	Active	2011-02-26	2032-12-31	2026-10-06	2 Years - 50 Years	40	1	United States		Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation	Developed in partnership with St. Jude, and licensed to Mustang Bio, Mustang Bio discontinued this program	Grant : P01 HL053749 NIAID (Z01-AI-00988 & U54-AI082973) National Cancer Institute (CA21765) CLIN2-09504 Preclinical Publications : A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection (Abstract 308) Correction of SCID-X1 in a Canine Transplant Model and in Primary Human Cells from a SCID-X1 Patient Using SIN Lentiviral Vectors Containing Cellular Transcription Control Elements - ASGCT 2009 (Abstract 309) Efficient Transduction of Human CD34+ Cells with an Insulated SIN Lentiviral Vector for SCID-X1 Produced from a Stable Producer Cell Line - ASGCT 2009 News and Press Releases : Mustang Bio Announces Strategic Manufacturing Partnership and Portfolio Updates Mustang Bio Reports Full-Year 2023 Financial Results and Recent Corporate Highlights Mustang Bio, Inc. SEC Filing for Q32024 Mustang Bio Provides an Update on its IND Application for MB-207, a Lentiviral Gene Therapy for Treatment of X-linked Severe Combined Immunodeficiency (“XSCID”) in Previously Transplanted Patients Clinical Publications : Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1 Innate-like memory T cells rapidly emerge in humans after gene therapy for SCID-X1 Protocol : Clinical Trial Protocol Related NCTID : Phase 1/2: NCT03315078 Phase 1/2: NCT01512888
NCT06092034	Danon Disease	RP-A501	Rocket Pharmaceuticals Inc.	Industry	LAMP2B	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 3.8E13 GC/kg \| Dose 2: 6.7E13 GC/kg \| Dose 3: 1.1E14 GC/kg (dose discontinued)	Phase2	Recruiting	Active	2023-10-11	2032-06	2026-10-06	>= 8 Years	18	6	Locations:United States + 3 more Germany, Italy, United Kingdom, United States		Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	Clinical hold lifted August 2025	Grant : CLIN2-15218 Preclinical Publications : Systemic AAV9.LAMP2B injection reverses metabolic and physiologic multiorgan dysfunction in a murine model of Danon disease News and Press Releases : Corporate Presentation - November 2024 Rocket Pharmaceuticals Announces Completion of Enrollment in Phase 2 Pivotal Trial of RP-A501 for the Treatment of Danon Disease Clinical Publications : Rocket Pharmaceuticals Announces FDA Has Lifted the Clinical Hold on the Pivotal Phase 2 Trial of RP-A501 for the Treatment of Danon Disease Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease Protocol : Clinical Trial Protocol (Corporate Presentation) RP-A501 Program Update Related NCTID : Phase 1: NCT03882437
NCT06389877	Alpha-1 Antitrypsin Deficiency (AATD)	BEAM-302	Beam Therapeutics Inc.	Industry	SERPINA1 (p.Glu366Lys)	Gene editing	in vivo	Mutation correction	Intravenous	Encapsulated mRNA	Liver	Endocytosis	null	ABE mRNA	Dose 1: Part A: 15mg (n=3); 30mg (n=3); 60mg (n=6); 75mg (n=9); 2X 60mg (n=3) \| Dose 2: Part B: 30mg (n=3); 60mg (n=2) \| Dose 3: Pivotal dose selected: 60mg	Phase2, Phase1	Recruiting	Active	2024-04-22	2030-05	2026-10-05	18 Years - 75 Years	192	12	Locations:United States + 5 more Australia, Ireland, Netherlands, New Zealand, United Kingdom, United States		Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation	Data from Parts A and B of Phase 1/2 trial released in March 2026, Beam intends to pursue an accelerated approval for BEAM-302	Preclinical Publications : (Poster) BEAM-302 decreases hepatic aggregates of mutant AAT and increases circulating functional AAT in rodent models of Alpha-1 Antitrypsin Deficiency - ESGCT 2023 (Presentation) BEAM-302 Base editing as a potential therapeutic approach for alpha-1 antitrypsin deficiency (Alpha-1) - Alpha-1 National Conference 2024 News and Press Releases : Beam Therapeutics Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) Beam Therapeutics Announces Compelling Updated Clinical Data from the Ongoing Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) to Support Advancement to Pivotal Development Beam Therapeutics Announces Progress in Hematology and Genetic Disease Franchises and Outlines Key 2025 Anticipated Catalysts Beam Therapeutics Reports Second Quarter 2025 Financial Results and Provides Update on BEAM-302 Development Progress in Alpha-1 Antitrypsin Deficiency (AATD) Corporate Presentation - January 2026 Beam Therapeutics Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency (AATD), Demonstrating First Ever Clinical Genetic Correction of a Disease-causing Mutation Beam Therapeutics Reports Fourth Quarter and Year-End 2025 Financial Results and Announces New Liver-Targeted Genetic Disease Program in Phenylketonuria (PKU) Beam Therapeutics Announces Clearance of Investigational New Drug Application for BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) by the United States (U.S.) Food and Drug Administration Clinical Publications : (Presentation) The potential for base editing to treat diseases impacting the lungs: Ongoing data from the BEAM-302 clinical trial in alpha-1 antitrypsin deficiency (AATD) - ATS 2026
NCT07137338	BAG3-Associated Dilated Cardiomyopathy (DCM)	RP-A701	Rocket Pharmaceuticals Inc.	Industry	BAG3	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Undisclosed	Phase1	Recruiting	Active	2025-07-25	2029-06	2026-10-01	18 Years - 65 Years	8	3	United States		Fast Track	First patient dosing in Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy anticipated in mid-2026	News and Press Releases : Rocket Pharmaceuticals Reports Second Quarter 2025 Financial Results and Highlights Recent Progress Rocket Pharmaceuticals Reports First Quarter 2026 Financial Results and Highlights Recent Progress Rocket Pharmaceuticals Announces FDA IND Clearance of RP-A701 for the Treatment of BAG3-associated Dilated Cardiomyopathy
NCT05878860	X-Linked Retinoschisis	ATSN-201	Atsena Therapeutics Inc.	Industry	RS1	Gene transfer	in vivo	Functional gene replacement	Subretinal	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: Phase 1/2 Part A: 1.1E10 vg/eye, 3.8E10 vg/eye, 2.3E10 vg/eye; n=3/cohort \| Dose 2: Phase 1/2 Part B: 1.1E10 vg/eye, 1.7 vg/eye; n=3/cohort \| Dose 3: Phase 3: 1.1E10 vg/eye; n=39	Phase3	Recruiting	Active	2023-05-12	2033-04	2026-09-29	>= 5 Years	97	21	Locations:United States + 2 more Canada, United Kingdom, United States		Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	First patient in pivotal cohort has been dosed. Enrollment in pivotal cohort is expected to complete by end of Q1 2027, topline results expected 1H2028, BLA filing targeted for 2H2028	Preclinical Publications : (Presentation) IND-enabling Studies to Support the Clinical Development of ATSN-201, a Subretinally Delivered, Laterally Spreading Gene Replacement Therapy For X-linked Retinoschisis (XLRS) - ESGCT 2023 News and Press Releases : Atsena Therapeutics Doses First Patient and Reports Rapid Early Enrollment in Pivotal Trial of ATSN-201 for X-Linked Retinoschisis Atsena Therapeutics Initiates Part B of Phase I/II Clinical Trial Evaluating Gene Therapy ATSN-201 to Treat X-linked Retinoschisis Atsena Therapeutics Granted U.S. FDA Regenerative Medicine Advanced Therapy Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis Atsena Therapeutics Granted U.S. FDA Fast Track Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis Atsena Presents Positive Clinical Data from Its XLRS and LCA1 Gene Therapy Programs at ARVO 2026 Atsena Therapeutics Receives Data Monitoring Committee Recommendation to Proceed with Pivotal Part C Cohort of Phase 1/2/3 LIGHTHOUSE Trial Evaluating ATSN-201 in Patients with X-linked Retinoschisis Atsena Presents Positive Interim Six-Month Results from Part B of Phase 1/2/3 LIGHTHOUSE Trial Evaluating ATSN-201 in Patients with X-linked Retinoschisis Atsena Therapeutics Announces Dosing Complete for Adults in Part B of the Phase I/II/III LIGHTHOUSE Trial Evaluating ATSN-201 to Treat X-linked Retinoschisis Clinical Publications : (Presentation) Safety and Efficacy of ATSN-201 Dose Escalation in Patients with X-Linked Retinoschisis (XLRS) - ASGCT 2025 (Presentation) Safety and efficacy of ATSN-201 in patients with X-linked retinoschisis: 12-month dose finding results of the LIGHTHOUSE study - ASGCT 2026 (Presentation) Safety and efficacy of ATSN-201 in patients with X-linked retinoschisis: 12-month dose finding results of the LIGHTHOUSE study - ARVO 2026 (Presentation) Interim Safety and Efficacy of ATSN-201 Dose Escalation Study in Patients with X-linked Retinoschisis - AAO 2024
NCT07180355	Friedreich's Ataxia	SGT-212	Solid Biosciences Inc.	Industry	FXN	Gene transfer	in vivo	Functional gene replacement	Intraparenchymal, Intravenous	Viral vector	Dentate nucleus	Viral transduction	Adeno-associated virus		Undisclosed	Phase1	Recruiting	Active	2025-08-22	2032-02-29	2026-09-29	18 Years - 40 Years	10	3	United States		Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	First patient dosed January 2026, initial data expected H2 2026	Preclinical Publications : (Poster) Efficacy and Safety of a Novel AAV FXN Gene Therapy (SGT-212) for the Treatment of Friedreich’s Ataxia - Society for Neuroscience 2025 News and Press Releases : Solid Biosciences Receives FDA Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia (Corporate Presentation) January 2026
NCT05821959	Bilateral Sensorineural Hearing Loss	AK-OTOF	Eli Lilly and Company	Industry	OTOF	Gene transfer	in vivo	Functional gene replacement	Intracochlear	Viral vector	Hair cell	Viral transduction	Adeno-associated virus		Dose 1: 4.1E11 vg/cochlea \| Dose 2: 8.1E11 vg/cochlea	Phase3	Recruiting	Active	2023-02-08	2029-12	2026-09-29		48	9	Locations:United States + 3 more Canada, Taiwan, United Kingdom, United States		Orphan Drug Designation, Rare Pediatric Disease Designation	null	Preclinical Publications : Ancestral library identifies conserved reprogrammable liver motif on AAV capsid Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction (Presentation) Preclinical Development of a Genetic Medicine for Otoferlin Gene-mediated Hearing Loss: AK-OTOF News and Press Releases : Positive Phase 1/2 Clinical Trial Data for an Investigational Gene Therapy for Genetic Hearing Loss to be Presented at the Association for Research in Otolaryngology 2024 MidWinter Meeting SEC Form 10-K: 2024 Annual Report Clinical Publications : (Abstract) Clinical Development of AK-OTOF Gene Therapy for OTOF-Mediated Hearing Loss - ARO MidWinter Meeting 2024 Related NCTID : Long Term Follow-Up: NCT06696456
NCT07160634	Duchenne Muscular Dystrophy (DMD)	SGT-003	Solid Biosciences Inc.	Industry	Micro-dystrophin	Gene transfer	in vivo	Overexpression of protective allele/gene	Intravenous	Viral vector	Muscle cells	Viral transduction	Adeno-associated virus		1E14 vg/kg	Phase3	Recruiting	Active	2025-08-29	2034-01	2026-09-28	7 Years - 11 Years	80	7	Locations:United States + 2 more Australia, Canada, United States		Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	Drug continues to be well tolerated in 47 participants dosed as of 5/11/26, dosing is ongoing in Phase 3 trial	Preclinical Publications : (Abstract #1389) AAV-SLB101, a Novel Muscle-Tropic Capsid, Increases Gene Delivery and Expression Versus AAV9 and AAVrh74, in Mouse Models of DMD and FSHD Muscle Disease - ASGCT 2025 (Poster) Novel Pharmacokinetic Effects of POLARIS-101™,Solid Biosciences’ Rationally Designed, Next-Generation Capsid - ASGCT 2026 (Poster) SGT-003 Gene Therapy Stabilizes the DAPC and Improves Muscle Integrity in Duchenne Muscular Dystrophy - ASGCT 2026 (Poster) Systemic Administration of a Rationally Engineered Vector POLARIS-101™ Leads to Successful Transduction of Non-Human Primate Tissues in The Presence of Neutralizing Antibodies; Implications for Gene Therapy and Immune Evasion Strategies (Poster) Systemic Delivery of SGT-003 Microdystrophin Gene Therapy Using the Novel Capsid AAV-SLB101 Ameliorates Muscle Pathology and Rescues Muscle Function in the mdx Mouse Model of Duchenne Muscular Dystrophy News and Press Releases : Solid Biosciences Reports First Quarter 2026 Financial Results and Provides Business Updates Solid Biosciences Granted FDA Orphan Drug Designation for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003 Solid Biosciences Reports Third Quarter 2025 Financial Results and Provides Update on INSPIRE DUCHENNE Clinical Trial Progress and Planned Regulatory Discussions Solid Biosciences Receives Rare Pediatric Disease Designation from the FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003 Solid Biosciences Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Gene Therapy SGT-003 Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003 Solid Biosciences Announces Positive Feedback from Type C Meeting with FDA for SGT-003 Gene Therapy for Duchenne Muscular Dystrophy Solid Biosciences Awarded Innovation Passport Designation Under the New UK Innovative Licensing and Access Pathway for SGT-003, an Investigational Gene Therapy for Duchenne Muscular Dystrophy Clinical Publications : (Corporate Presentation) August 2026 (Presentation) Update on the INSPIRE DUCHENNE Phase 1/2 Study of the Next-Generation Microdystrophin Gene Therapy Candidate SGT-003 for Duchenne Muscular Dystrophy - ASGCT 2026 (Poster) Lower POLARIS-101™ (AAV-SLB101) Cross-Reactive Antibodies in Elevidys-treated Patients - ASGCT 2026 (Presentation) Update on the INSPIRE DUCHENNE Phase 1/2 Study of the Next-Generation Microdystrophin Gene Therapy Candidate SGT-003 for Duchenne Muscular Dystrophy - MDA Conference 2026 (Poster) Positive Preliminary Safety and Liver Toxicity Profile Using SGT-003, Solid Biosciences’ Next-Generation Investigational Gene Therapy for Duchenne Muscular Dystrophy - MDA Conference 2026 Related NCTID : Phase 1/2: NCT06138639
NCT07592273	Diabetic Retinopathy	Surabgene lomparvovec	AbbVie	Industry	Anti-VEGF	Gene transfer	in vivo	Genetic delivery of therapeutic protein	Suprachoroidal	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: Phase 2 doses: 2.5E11 GC/eye, 5.0E11 GC/eye, 1.0E12 GC/eye \| Dose 2: Phase 3 dose: 1.0E12 GC/eye	Phase3, Phase2	Recruiting	Active	2026-04-24	2036-01	2026-09-24	>= 18 Years	576	34	Locations:United States + 1 more Puerto Rico, United States			First patient has been dosed in the Phase IIb/III trial using suprachoroidal delivery. Phase IIb data was presented at the ASRS annual meeting in July 2026. This product is also under evaluation for wet AMD	News and Press Releases : REGENXBIO Reports Third Quarter 2025 Financial Results and Operational Highlights REGENXBIO Announces Pivotal Program for Surabgene Lomparvovec in Diabetic Retinopathy REGENXBIO Announces First Patient Dosed in Phase IIb/III NAAVIGATE Trial of Surabgene Lomparvovec in Diabetic Retinopathy; Company to Receive $100 Million Milestone REGENXBIO Presents Positive Long-Term Data for Surabgene Lomparvovec in Wet AMD and Diabetic Retinopathy at American Society of Retina Specialists Annual Meeting Clinical Publications : (Presentation) Suprachoroidal Delivery of Investigational Surabgene Lomparvovec (Sura-vec, ABBV-RGX-314): Results in NPDR at 2.5 Years - ASRS 2026 (Presentation) Suprachoroidal Surabgene Lomparvovec (Sura-vec, ABBV-RGX-314) First Time 2-Year Results in NPDR -AAO 2025 Related NCTID : Phase 2: NCT06942520 (using subretinal injection) Phase 2: NCT04567550 Long Term Follow-Up: NCT05296447
NCT06270316	Fabry Disease	AMT-191	UniQure Biopharma B.V.	Industry	GLA	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 6.0E13 gc/kg (cohort 1) \| Dose 2: 2.0E13 gc/kg (cohort 2) \| Dose 3: 4.0E13 gc/kg (cohort 3)	Phase2, Phase1	Recruiting	Active	2023-12-19	2031-11-30	2026-09-24	18 Years - 65 Years	20	10	United States		Fast Track, Orphan Drug Designation	Data on cohorts 1-3 were presented in February 2026, additional dosing paused in the mid- and high-dose cohorts pending further evaluation of asymptomatic Grade 3 liver enzyme elevations reported in two patients from the mid-dose cohort	Preclinical Publications : (Abstract #OR19) Pre‐clinical proof of concept of an AAV5‐GLA gene therapy for Fabry disease resulting in cross‐correction in GLA‐KO mice and non‐human primates in target organs - ESGCT 2021 News and Press Releases : uniQure Announces 2025 Financial Results and Provides Recent Company Updates uniQure Announces Completion of Enrollment in the First Cohort and Favorable Recommendation from the Independent Data Monitoring Committee for its Phase I/IIa Clinical Trial of AMT-191 for the Treatment of Fabry Disease uniQure Announces Initial AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease uniQure Announces Updated Preliminary AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease Clinical Publications : (Presentation) AMT-191 investigational gene therapy in adult males with classic Fabry disease; preliminary safety and biomarker data of Phase I/IIa study -WORLDSymposium 2026
NCT07148089	Catecholaminergic Polymorphic Ventricular Tachycardia	SGT-501	Solid Biosciences Inc.	Industry	CASQ2	Gene transfer	in vivo	Overexpression of protective allele/gene	Intravenous	Viral vector	Cardiomyocytes	Viral transduction	Adeno-associated virus		Undisclosed dose escalation, 2 levels	Phase1	Recruiting	Active	2025-08-22	2031-05	2026-09-24	>= 7 Years	18	5	Locations:United States + 1 more Canada, United States		Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	Company anticipates dosing the first participant in the Phase 1b ARTEMIS clinical trial evaluating SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT) in H2 2026	Preclinical Publications : (Poster) A Novel AAV Gene Therapy Strategy to Correct Calcium Dysregulation in Catecholaminergic Polymorphic Ventricular Tachycardia - ASGCT 2026 News and Press Releases : Solid Biosciences Receives FDA Fast Track Designation for SGT-501 First-in-Class Gene Therapy for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) Solid Biosciences Reports First Quarter 2026 Financial Results and Provides Business Updates SEC Form 10-K: Solid Biosciences Inc. FY 2025
NCT07185256	ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy	OPGx-BEST1	Opus Genetics, Inc	Industry	BEST1	Gene transfer	in vivo	Functional gene replacement	Subretinal	Viral vector	Retinal pigment epithelial (RPE) cells	Viral transduction	Adeno-associated virus		Dose 1: 1.5E9 vg/eye \| Dose 2: 4.5E9 vg/eye	Phase2, Phase1	Recruiting	Active	2025-07-17	2030-08	2026-09-24	>= 18 Years	10	5	United States			First participant dosed Q4 2025, final participant scheduled for dosing in June 2026, data readout expected in September 2026	Preclinical Publications : BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure (Presentation) Gene Therapy for BEST1 Inherited Retinal Disease - APAO 2026 (Poster) Development of OPGx-BEST1 AAV gene therapy for the treatment of bestrophinopathies - ARVO 2024 (Poster) Development of cell-based expression and functional potency assays for OPGx-BEST1 gene therapy - ARVO 2026 News and Press Releases : Opus Genetics Announces Financial Results for First Quarter 2026 and Provides Corporate Update (Corporate Presentation) Delivering on the Promise of Gene Therapy for Rare Inherited Retinal Diseases Opus Genetics Announces Positive Recommendation from Independent Data Monitoring Committee for Phase 1/2 Trial in Best Disease Clinical Publications : (Presentation) Preliminary Results from an Adult Participant in a Phase 1b/2a Clinical Study of OPGxBEST1 Gene Therapy for the Treatment of BVMD and ARB Due to BEST1 Mutations - Macula Society 2026 (Poster) Preliminary Results from Adult Participants in a Phase 1b/2a Clinical Study of OPGx-BEST1 Gene Therapy for ARB and BVMD due to BEST1 Mutations - ARVO 2026
NCT07587021	Primary Hyperoxaluria Type 1, PH1	YOLT-203	YolTech Therapeutics Co., Ltd	Industry	HAO1	Gene editing	in vivo	Gene inactivation	Intravenous	Encapsulated RNP	null	Endocytosis	null	YolCas12	Early Phase 1 doses: 0.3 mg/kg, 0.6 mg/kg, 1.0 mg/kg	Phase2	Recruiting	Active	2026-05-01	2028-02	2026-09-23	>= 6 Years	36	4	United States		Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation	US IND application cleared November 2025	Preclinical Publications : Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption Library-Assisted Evolution in Eukaryotic Cells Yield Adenine Base Editors with Enhanced Editing Specificity News and Press Releases : YolTech Receives U.S. FDA RPDD for YOLT-203 in Treating PH1 YolTech Receives U.S. FDA ODD for YOLT-203 in Treating PH1 YolTech Therapeutics Receives FDA IND Clearance to Initiate Global Pivotal Trial of In Vivo Gene-Editing Therapy YOLT-203 for Primary Hyperoxaluria Type 1 (PH1) YolTech Therapeutics Receives FDA IND Clearance to Initiate Global Pivotal Trial of In Vivo Gene-Editing Therapy YOLT-203 for Primary Hyperoxaluria Type 1 (PH1) YolTech Therapeutics Administers First Patient Dose in IIT of YOLT-203, the World's First In Vivo Gene Editing Therapy for PH1 Success for YolTech's hyperoxaluria in vivo gene therapy in early-stage trial Related NCTID : Early Phase 1: NCT06511349
NCT06364774	Beta-Thalassemia Major	CHOP-ALS20	Children's Hospital of Philadelphia	Other	HBB	Gene transfer	ex vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	Lentivirus		Transduced CD34+ cells	Phase2, Phase1	Recruiting	Active	2024-03-26	2027-12-31	2026-09-23	18 Years - 40 Years	12	1	United States			null	Preclinical Publications : Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies
NCT06910813	Nephropathic Cystinosis	DFT383	Novartis Pharmaceuticals	Industry	CTNS	Gene transfer	ex vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	Lentivirus		Min dose: 3E6 CD34+ cells/kg	Phase2, Phase1	Recruiting	Active	2025-03-17	2044-05-28	2026-09-22	2 Years - 5 Years	30	6	United States		Orphan Drug Designation, Rare Pediatric Disease Designation	Clinical results published 2/18/26	Preclinical Publications : Brief reports: Lysosomal cross-correction by hematopoietic stem cell-derived macrophages via tunneling nanotubes Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis (Review) Targeted gene therapy for rare genetic kidney diseases News and Press Releases : Novartis to buy Avrobio gene therapy for $88M, leaving rest of the biotech on the shelf From Wish to Reality — The Gene Therapy Initiative at UC San Diego Cystinosis Community Statement October 2023 from Novartis Clinical Publications : Hematopoietic Stem Cell Gene Therapy for Cystinosis: From Bench-to-Bedside (Presentation) Hematopoietic Stem Cell Gene Therapy for Cystinosis: updated results from a phase 1/2 clinical trial - DoH 2022 Hematopoietic Stem-Cell Gene Therapy for Cystinosis (Abstract) Phase 1/2 Clinical Trial of Autologous Hematopoietic Stem and Progenitor Cell Gene Therapy for Cystinosis - DoH 2024 Related NCTID : Long Term Follow-Up: NCT05146830
NCT05419492	Dravet Syndrome	ETX101	Encoded Therapeutics	Industry	SCN1A	Gene transfer	in vivo	Overexpression of protective allele/gene	Intraventricular	Viral vector	GABAergic inhibitory interneurons	Viral transduction	Adeno-associated virus		Undisclosed dose escalation, 2 levels	Phase2, Phase1	Recruiting	Active	2022-06-10	2033-01	2026-09-22	6 Months - 17 Years	47	14	Locations:United States + 2 more Australia, United Kingdom, United States		Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	Announced in May 2026 that the first patient in the pivotal trial has been dosed, initial data from Part 1B is expected in 4Q26	Preclinical Publications : Cell-Selective Adeno-Associated Virus-Mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model and Is Well Tolerated in Nonhuman Primates (Abstract) ETX101, a GABAergic Interneuron Selective AAV-mediated Gene Therapy for the Treatment of SCN1A+ Dravet Syndrome: Biodistribution and Safety in Non-human Primates -AES 2020 GABA Selective AAV-Mediated Gene Therapy Provides (Abstract ) Durable Seizure Protection in Multiple Refractory Epilepsy Models - ASGCT 2024 News and Press Releases : Encoded Therapeutics Announces Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by U.S. FDA for ETX101 in SCN1A+ Dravet Syndrome Encoded Therapeutics Reports Clinical Progress of ETX101 Gene Therapy for Dravet Syndrome, Recaps 2024 Corporate Achievements and Provides 2025 Outlook Encoded Therapeutics Doses First Patient in Pivotal Study of ETX101 for Dravet Syndrome and Reports Broader Portfolio Progress Related NCTID : Phase 1/2: NCT06283212 (UK only) Phase 1/2: NCT06112275 (Australia Only)
NCT02716246	Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)	UX111	Ultragenyx Pharmaceutical Inc	Industry	SGSH	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 0.5E13 vg/kg \| Dose 2: 1.0E13 vg/kg \| Dose 3: 3.0E13 vg/kg (n=22)	Phase3, Phase2	Recruiting	Active	2016-03-17	2029-03	2026-09-22		36	5	Locations:United States + 2 more Australia, Spain, United States		Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	FDA has accepted the resubmitted BLA, and set a PDUFA date of 9/19/26	Grant : U01 NS064096 R21 NS081173 R01 NS093941 Preclinical Publications : Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease Systemic scAAV9.U1a.hSGSH Delivery Corrects Brain Biochemistry in Mucopolysaccharidosis Type IIIA at Early and Later Stages of Disease Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery News and Press Releases : Ultragenyx Announces U.S. FDA Acceptance of BLA Resubmission for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) 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 Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA FDA issues CRL for Ultragenyx’s UX111 gene therapy; company remains optimistic Clinical Publications : (Abstract) Treatment with UX111 gene therapy rapidly reduced heparan sulfate (HS) exposure in cerebrospinal fluid (CSF) and improved long-term cognitive function in children with mucopolysaccharidosis type IIIA (MPS IIIA) - WORLDSymposium 2025 Related NCTID : Phase 1/2: NCT04088734 Long Term Follow-Up: NCT04360265
NCT06844214	Myotonic Dystrophy	SAR446268	Sanofi	Industry	DMPK	Gene transfer	in vivo	Gene inactivation	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		3 cohort dose escalation, undisclosed concentrations	Phase2, Phase1	Recruiting	Active	2025-02-19	2032-04-27	2026-09-21	10 Years - 55 Years	32	10	Locations:United States + 5 more Argentina, Australia, Canada, Israel, United Kingdom, United States		Fast Track, Orphan Drug Designation	null	Preclinical Publications : Development of an AAV-delivered microRNA gene therapy for myotonic dystrophy type 1 News and Press Releases : Press Release: Sanofi’s SAR446268 earns US fast track designation for the treatment of non-congenital myotonic dystrophy type 1 Protocol : (Abstract) The BrAAVe study: a phase 1/2 study to investigate the safety, tolerability, and efficacy of SAR446268, an AAV-mediated gene therapy in non-congenital DM1 patients - WMS 2025
NCT03952637	GM1 Gangliosidosis	AXO-AAV-GM1	National Human Genome Research Institute (NHGRI)	NIH	GLB1	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 1.5E13 vg/kg (n=7) \| Dose 2: 4.5E13 vg/kg (n=3)	Phase2, Phase1	Recruiting	Active	2019-05-15	2030-01-01	2026-09-21	6 Months - 12 Years	54	1	United States		Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	New clinical data published February 2026	Grant : R01 NS076991 R01 HD060576 Preclinical Publications : Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan AAV9-coGLB1 Improves Lysosomal Storage and Rescues Central Nervous System Inflammation in a Mutant Mouse Model of GM1 Gangliosidosis Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis News and Press Releases : Sio Gene Therapies Announces Granting of FDA Fast Track Designation for Investigational AXO-AAV-GM1 (AAV9-GLB1) Gene Therapy in Patients with GM1 Gangliosidosis Clinical Publications : (Abstract #162) AXO-AAV-GM1 for the Treatment of GM1 Gangliosidosis: Preliminary Results from a Phase I-II Trial - ASGCT 2021 (Abstract #eP259) A phase 1/2 trial of AXO-AAV-GM1 gene therapy for the treatment of infantile- and juvenile-onset GM1 gangliosidosis - ACMG 2022 AAV9 Gene Therapy in Type II GM1 Gangliosidosis — A Phase 1–2 Trial Protocol : Clinical Trial Protocol (NEJM)
NCT07491172	Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Uncontrolled Hypercholesterolemia, Mixed Dyslipidemia	CTX310	CRISPR Therapeutics AG	Industry	ANGPTL3	Gene editing	in vivo	Gene inactivation	Intravenous	Encapsulated mRNA	Hepatocytes	Endocytosis	null	Cas9 mRNA	Dose 1: Phase 1a: 0.1 mg/kg (n=3); 0.3 mg/kg (n=3), 0.6mg/kg (n=3), 0.7 mg/kg (n=2), 0.8 mg/kg (n=4) \| Dose 2: Phase 1b: 0.8mg/kg	Phase1	Recruiting	Active	2026-03-18	2028-06	2026-09-18	18 Years - 75 Years	90	17	Locations:United States + 3 more Australia, New Zealand, United Kingdom, United States			CRISPR Therapeutics continues to anticipate sharing an update from the CTX310 Phase 1b clinical trial in the second half of 2026, focused on severe hypertriglyceridemia patients	News and Press Releases : CRISPR Therapeutics Provides Business Update and Reports First Quarter 2026 Financial Results CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310® Demonstrating Deep and Durable ANGPTL3 Editing, Triglyceride and Lipid Lowering CRISPR Therapeutics Presents Phase 1a Data for CTX310® Demonstrating Deep and Durable ANGPTL3 Editing, Triglyceride and LDL Lowering at ESC Congress 2026 Clinical Publications : (Presentation) Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 - AHA 2025 Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310 Protocol : Clinical Trial Protocol
NCT07007065	Neovascular Age-related Macular Degeneration	ABBV-RGX-314	AbbVie	Industry	Anti-VEGF	Gene transfer	in vivo	Genetic delivery of therapeutic protein	Subretinal	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: Phase 1/2 doses: 3E9 GC/eye (n=5); 1E10 GC/eye (n=5); 6E10 GC/eye (n=6); 1.6E11 GC/eye (n=11); 2.5E11 GC/eye (n=10) \| Dose 2: Phase 2 doses (suprachoroidal delivery): 2.5E11 GC/eye, 5.0E11 GC/eye, 1.0E12 GC/eye, 1.5E12 GC/eye	Phase3	Recruiting	Active	2025-05-28	2033-03	2026-09-15	>= 50 Years	561	133	Locations:United States + 16 more Austria, Belgium, Bulgaria, Canada, Croatia, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Portugal, Puerto Rico, Spain, United Kingdom, United States			Another Phase 3 study (NCT05407636) is active, enrollment is completed; REGENXBIO expects to announce topline data with AbbVie from the ATMOSPHERE and ASCENT pivotal trials of sura-vec in wet AMD using subretinal delivery in Q4 2026	Preclinical Publications : AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression AAV8-antiVEGFfab Ocular Gene Transfer for Neovascular Age-Related Macular Degeneration News and Press Releases : AbbVie and REGENXBIO Announce Updates on the ABBV-RGX-314 Clinical Program REGENXBIO Reports Third Quarter 2025 Financial Results and Operational Highlights REGENXBIO Presents Positive Long-Term Data for Surabgene Lomparvovec in Wet AMD and Diabetic Retinopathy at American Society of Retina Specialists Annual Meeting Clinical Publications : Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study (Presentation) Subretinal Delivery of Investigational Sura-vec Gene Therapy for Neovascular Age-Related Macular Degeneration: Long-Term Follow-Up of Phase 1/2a Participants -ASRS 2026 (Presentation) Suprachoroidal Delivery of Investigational ABBV-RGX-314 for Neovascular AMD: Results from the Phase II Study - Retina, January 2024 (Presentation) Subretinal Delivery of Investigational ABBV-RGX-314* as a Gene Therapy for nAMD One-year Results of a Fellow Eye Bilateral Dosing Study Related NCTID : Phase 2: NCT04832724 Phase 2: NCT04514653 Long Term Follow Up: NCT05210803 Phase 1/2: NCT03066258 Phase 2/3: NCT04704921 Phase 2: NCT03999801
NCT07226206	Hemophilia A	SPK-8011QQ	Hoffmann-La Roche	Industry	F8	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 5E11 vg/kg \| Dose 2: 1E12 vg/kg \| Dose 3: 1.5E12 vg/kg \| Dose 4: 2E12 vg/kg	Phase2, Phase1	Recruiting	Active	2025-10-28	2032-04-30	2026-09-11	>= 18 Years	5	3	United States			Study was withdrawn by Sponsor (no participants were enrolled), Sponsor is extensively modifying the product before clinical trials resume	Preclinical Publications : (Abstract) Preclinical evaluation of SPK-8011QQ, an adeno-associated virus gene therapy for people with hemophilia A leveraging the dirloctocogene samoparvovec platform encoding an activated Protein C-resistant B-domain deleted factor VIII - ASH 2025 Activated protein C has a regulatory role in factor VIII function Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A News and Press Releases : Roche mothballs another hemophilia A gene therapy under Spark amid plans to debut new hematologic asset Integration of Spark into Roche Group Set, Hemophilia Gene Therapy Program Continues Clinical Publications : Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A Related NCTID : Phase 3: NCT06297486 (Withdrawn) Long Term Follow-Up: NCT03432520 Phase 1/2: NCT03003533
NCT07048808	Refractory Angina, Coronary Artery Disease	XC001	XyloCor Therapeutics, Inc.	Industry	VEGF	Gene transfer	in vivo	Overexpression of protective allele/gene	Intracardiac	Viral vector	Myocardium	Viral transduction	Adenovirus		Dose 1: Dose escalation: 1E9 vp, 1E10 vp, 4E10 vp, 1E11 vp \| Dose 2: Dose expansion: 1E11 vp	Phase2	Recruiting	Active	2025-06-24	2027-12-30	2026-09-11	18 Years - 85 Years	106	14	United States			First patient dosed in Phase 2b trial in July 2025	Preclinical Publications : Safety and biodistribution of XC001 (encoberminogene rezmadenovec) gene therapy in rats: a potential therapy for cardiovascular diseases Alteration of splicing signals in a genomic/cDNA hybrid VEGF gene to modify the ratio of expressed VEGF isoforms enhances safety of angiogenic gene therapy Adenovirus-mediated transfer of a minigene expressing multiple isoforms of VEGF is more effective at inducing angiogenesis than comparable vectors expressing individual VEGF cDNAs Safety of direct cardiac administration of AdVEGF-All6A+, a replication-deficient adenovirus vector cDNA/genomic hybrid expressing all three major isoforms of human vascular endothelial growth factor, to the ischemic myocardium of rats News and Press Releases : https://www.xylocor.com/news.html XyloCor doses first subject in Phase IIb trial of gene therapy candidate XC001 Clinical Publications : EXACT Trial: Results of the Phase 1 Dose-Escalation Study Angiogenic Gene Therapy for Refractory Angina: Results of the EXACT Phase 2 Trial Protocol : Epicardial delivery of XC001 gene therapy for refractory angina coronary treatment (The EXACT Trial): Rationale, design, and clinical considerations Related NCTID : Phase 1/2: NCT04125732 Phase 2: NCT07118449
NCT07287397	Amyotrophic Lateral Sclerosis	VTx-002	Vector Y Therapeutics	Industry	Antibody to TDP-43	Gene transfer	in vivo	Genetic delivery of therapeutic protein	Intracisternal	Viral vector	null	Viral transduction	Adeno-associated virus		Undisclosed dose escalation, 2 levels	Phase2, Phase1	Recruiting	Active	2025-11-19	2027-10-15	2026-09-10	>= 18 Years	12	11	Locations:United States + 3 more Belgium, Netherlands, United Kingdom, United States		Fast Track	FDA cleared IND 12/25	Preclinical Publications : (Poster) AAV-mediated intrabody therapy targeting TDP-43 proteinopathy in ALS - ENCALS 2025 (Poster) VecTabs® and advanced human in-vitro models: Targeting TDP-43 aggregates and oxidized phosphocholines in Amyotrophic lateral sclerosis - ALS/MND 2022 (Poster) Non-clinical development and Phase 1/ 2 clinical trial design of VTx-002, an AAV5.2 vectorized intrabody targeting TDP-43 for Amyotrophic Lateral Sclerosis - ALS/MND 2025 (Poster) AAV-mediated intrabody therapy targeting TDP-43 proteinopathy in ALS - ASGCT 2025 News and Press Releases : VectorY Therapeutics Receives FDA Clearance of IND to Proceed with the PIONEER-ALS Phase 1/2 Trial of VTx-002, a First-in-Class Vectorized Antibody Targeting TDP-43 Pathology in ALS VectorY Therapeutics Receives FDA Fast Track Designation for VTx-002, a First-in-Class Vectorized Antibody Targeting Underlying Disease Biology in ALS
NCT07447557	Charcot-Marie-Tooth Disease Type 4J	ELP-02	Elpida Therapeutics SPC	Industry	FIG4	Gene transfer	in vivo	Functional gene replacement	Intrathecal	Viral vector	null	Viral transduction	Adeno-associated virus		Undisclosed	Phase2, Phase1	Recruiting	Active	2026-01-20	2032-12	2026-09-09	3 Years - 20 Years	8	2	United States		Orphan Drug Designation, Rare Pediatric Disease Designation	IND cleared in December 2024	Preclinical Publications : AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model News and Press Releases : Elpida Therapeutics and CureCMT4J granted Rare Pediatric and Orphan Drug Designations from the FDA for CMT4J BGTC Advancing in Pre-clinical and Clinical Milestones (CMT4J Progress graphic) Elpida Therapeutics and CureCMT4J Announce FDA Clearance of IND Application for ELP-02, an AAV9 Gene Therapy for CMT4J
NCT07770828	Dry Age-related Macular Degeneration	OCU410	Ocugen	Industry	RORA	Gene transfer	in vivo	Overexpression of protective allele/gene	Subretinal	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: Phase 1/2 study: 5E9 vg/eye, 1E10 vg/eye, 3E10 vg/eye (200ul injection volume for each) \| Dose 2: Phase 3 study: 1E10 vg/eye (200ul injection volume)	Phase3	Recruiting	Active	2026-08-13	2031-07-30	2026-08-19	>= 55 Years	237	3	United States		Regenerative Medicine Advanced Therapy (RMAT)	Topline Phase 2 data announced in March 2026, Ocugen announce in September 2026 that the first patient in the registrational trial had been dosed	Preclinical Publications : Retinoic acid related orphan receptor α is a genetic modifier that rescues retinal degeneration in a mouse model of Stargardt disease and Dry AMD (Abstract) OCU410, a Potential Therapeutic for Dry-AMD, Suppresses Inflammatory Cytokine Gene Expression in Retinal Epithelial Cells - ARVO 2022 News and Press Releases : Ocugen Provides Business Update with Third Quarter 2024 Financial Results Ocugen Provides Business Update with Third Quarter 2025 Financial Results Ocugen Announces First Patient Dosed in Phase 3 Registrational Trial of OCU410 Modifier Gene Therapy for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration Ocugen Announces Topline 12-month Data from Phase 2 ArMaDa Clinical Trial Evaluating OCU410 Modifier Gene Therapy for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration Ocugen Announces that the U.S. Food and Drug Administration Has Granted OCU410 Regenerative Medicine Advanced Therapy (RMAT) Designation for Treatment of Geographic Atrophy, Secondary to Dry Age-Related Macular Degeneration Ocugen Provides Business Update with Fourth Quarter and Full Year 2025 Financial Results Ocugen Announces Positive Preliminary Phase 2 Data from OCU410 Modifier Gene Therapy for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration Ocugen, Inc. Announces Dosing Completion in the Phase 2 ArMaDa Clinical Trial for OCU410—A Multifunctional Modifier Gene Therapy for the Treatment of Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration Clinical Publications : (Abstract) Preliminary Safety and Efficacy of OCU410 for Treatment of Geographic Atrophy: Phase 1/2 OCU410: The Age-related Macular Degeneration (ArMaDa) Study update - ARVO 2025
NCT06761183	Ischemic Stroke	NXL-001	Beijing Tiantan Hospital	Other	NeuroD1	Gene transfer	in vivo	Overexpression of protective allele/gene	Intracerebral	Viral vector	Astrocyte	Viral transduction	Adeno-associated virus		Undisclosed dose escalation, 3 levels	Early phase1	Recruiting	Active	2024-12-27	2027-12-31	2026-09-09	40 Years - 75 Years	9	1	China			This product is also being evaluated in Alzheimer's Disease indication in IIT trial in China	Preclinical Publications : Transcriptomic analyses of NeuroD1-mediated astrocyte-to-neuron conversion New AAV tools fail to detect Neurod1-mediated neuronal conversion of Muller glia and astrocytes in vivo A NeuroD1 AAV-Based Gene Therapy for Functional Brain Repair after Ischemic Injury through In Vivo Astrocyte-to-Neuron Conversion Regeneration of Functional Neurons After Spinal Cord Injury via in situ NeuroD1-Mediated Astrocyte-to-Neuron Conversion Differential neuronal reprogramming induced by NeuroD1 from astrocytes in grey matter versus white matter
NCT06662188	SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome	JAG201	Jaguar Gene Therapy, LLC	Industry	SHANK3	Gene transfer	in vivo	Functional gene replacement	Intraventricular	Viral vector	null	Viral transduction	Adeno-associated virus		Undisclosed dose escalation	Phase2, Phase1	Recruiting	Active	2024-10-16	2031-06	2026-09-03	2 Years - 9 Years	6	3	United States		Fast Track, Rare Pediatric Disease Designation	Cohort 1 (3 patients) dosing is complete, first two patients in dose-escalation cohort 2 have been dosed, completion expected in Q2 2026	Preclinical Publications : (Poster) Preclinical Assessment of JAG201, a Clinical Stage Gene Therapy for Severe Neurodevelopmental Disorders Caused by Mutations or Deletions in SHANK3 Including Phelan-McDermid Syndrome (PMS) and Autism Spectrum Disorder (ASD) - ASGCT 2024 (Poster) Biodistribution Assessment in Non-Human Primates of JAG201, a SHANK3 AAV9 Vector Delivered via ICV Injection for ASD, Phelan McDermid Syndrome, and Other SHANK3 Mutation or Deletion Related Conditions - ASGCT 2023 News and Press Releases : Completion of Dosing of First Patient Cohort in Clinical Trial Evaluating JAG201 for the Treatment of a Leading Monogenic Cause of Autism Spectrum Disorder Known as Phelan-McDermid Syndrome JAG201 Clinical Study Enrollment is Now Open Jaguar Gene Therapy Community Webinar - July 11, 2024 Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome Andelyn Biosciences Announces Partnership with Jaguar Gene Therapy to Provide Late-Stage PPQ Manufacturing Jaguar Gene Therapy to Initiate Inaugural Pediatric Clinical Trial Targeting a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome
NCT07223944	Gaucher Disease Type 1	FLT201	Spur Therapeutics	Industry	GBA1	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		4.5E11 vg/kg	Phase3	Recruiting	Active	2025-10-30	2032-10-01	2026-09-03	>= 18 Years	45	33	Locations:United States + 9 more Argentina, Australia, Brazil, Canada, Israel, Saudi Arabia, Spain, Turkey (Türkiye), United Kingdom, United States		Regenerative Medicine Advanced Therapy (RMAT)	Trial initiation expected 2H26	Preclinical Publications : FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1 Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease (note: used same AAV) News and Press Releases : Spur Therapeutics Announces Successful End-of-Phase 2 Meeting with FDA for FLT201, Its Gene Therapy Candidate for Gaucher Disease Spur Therapeutics Publishes Preclinical Proof-of-Concept Data for FLT201, its Clinical-Stage Gene Therapy Candidate in Gaucher Disease Clinical Publications : (Poster) Long-term durability of FLT201: an investigational gene therapy for Gaucher disease Type 1 encoding an engineered variant of the GCase enzyme - ESGCT 2025 (Presentation) Results from GALILEO-1, a first-in-human clinical trial of FLT201 AAV- gene therapy in adult patients with type 1 Gaucher Disease - WORLDSymposium 2025 Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B (note: used same novel AAV) (Presentation) Durability of FLT201: An Investigational Gene Therapy for Gaucher Disease Type 1 Encoding an Engineered Variant of the GCaseEnzyme - ASGCT 2025 Protocol : GALILEO-1: A Phase I/II Safety and Efficacy Study of FLT201 Gene Therapy for Gaucher Disease Type 1 Related NCTID : Phase 1: NCT05324943
NCT07052929	X-Linked Myotubular Myopathy	ASP2957	Astellas Gene Therapies	Industry	MTM1	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	Muscle cells	Viral transduction	Adeno-associated virus		Dose 1: 1E12 vg/kg \| Dose 2: 2E12 vg/kg	Phase2, Phase1	Recruiting	Active	2025-06-30	2027-10-31	2026-09-03	<= 36 Months	9	5	Locations:United States + 1 more Canada, United States			Astellas has restarted their XLMTM program by creating a new drug product ASP2957, with substantial modifications to the capsid to detarget the liver, and includes a muscle-specific promotor MHCK7, IND was cleared July 2025	Preclinical Publications : (Abstract) Efficacy, Biodistribution, and Safety of an AAV Gene Therapy in XLMTM Disease Model Mice and Juvenile Non-Human Primates - MDA Conference 2026 News and Press Releases : Astellas Sees Path Forward for Gene Therapy Despite Four Patient Deaths VALOR Clinical Trial Patient Information Astellas Takes Aim at XLMTM Again, Now with MyoAAV Capsid-Based Gene Therapy Clinical Publications : Hepatobiliary disease in XLMTM: a common comorbidity with potential impact on treatment strategies Intrahepatic Cholestasis Is a Clinically Significant Feature Associated with Natural History of X-Linked Myotubular Myopathy (XLMTM): A Case Series and Biopsy Report Protocol : (Abstract) VALOR study design: a phase 1/2 study assessing the safety and preliminary efficacy of ASP2957 gene therapy for X-linked myotubular myopathy (XLMTM) - MDA Conference 2026 (Abstract 74P) Design of a phase 1/2 study assessing the safety and preliminary efficacy of ASP2957 gene therapy for X-linked myotubular myopathy (XLMTM) - World Muscle Society 2025 Related NCTID : Phase 2/3: NCT03199469 (prior drug product AT132) Epidemiological study: NCT06581146
NCT06164730	Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease	VERVE-102	Verve Therapeutics, Inc.	Industry	PCSK9	Gene editing	in vivo	Gene inactivation	Intravenous	Encapsulated mRNA	null	Endocytosis	null	ABE8.8 mRNA	Phase 1 trial: 0.3 mg/kg (n=4); 0.45 mg/kg (n=6); 0.6mg/kg (n=4); 0.7 mg/kg (n=8); 0.8 mg/kg (n=6); 1.0 mg/kg (n=7)	Phase1	Recruiting	Active	2023-12-01	2027-10	2026-09-02	18 Years - 70 Years	85	27	Locations:United States + 5 more Australia, Canada, Israel, New Zealand, United Kingdom, United States		Fast Track	Phase 1 study results published May 2026, Lilly plans to initiate Phase 2 study by the end of 2026	Preclinical Publications : (Presentation) Proof-of-concept for in vivo Base Editing to Inactivate the PCSK9 Gene and Lower LDL-Cholesterol in Humans - TIDES 2024 (Abstract) VERVE-102, a clinical stage in vivo base editing medicine, leads to potent and precise inactivation of PCSK9 in preclinical studies - NLA 2025 News and Press Releases : Lilly to acquire Verve Therapeutics to advance one-time treatments for people with high cardiovascular risk Verve Therapeutics Announces Dosing of First Patient in Heart-2 Phase 1b Clinical Trial Evaluating VERVE-102 Verve Therapeutics Announces Pipeline Progress and Anticipated 2025 Milestones Verve Therapeutics Announces Clearance of Investigational New Drug Application by the U.S. FDA for VERVE-102, an Investigational Gene Editing Medicine Designed to Durably Lower Cholesterol After a Single Dose A single dose of Lilly's PCSK9 base editor, VERVE-102, reduced PCSK9 by up to 88% and LDL-C by up to 62%, with durable effects supporting its potential as a one-time treatment for hypercholesterolemia Verve Therapeutics Receives U.S. FDA Fast Track Designation for VERVE-102, an In Vivo Base Editing Medicine Targeting PCSK9 Clinical Publications : In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia Protocol : (Presentation) Design of Heart-2 A Phase 1b clinical trial of VERVE-102, an in vivo base editing medicine delivered by a GalNAc-LNP and targeting PCSK9 to durably lower LDL cholesterol - AHA Scientific Sessions 2024 Related NCTID : Long Term Follow-Up: NCT06112327
NCT07721025	Friedreich Ataxia with Secondary Cardiomyopathy	LX2006	Lexeo Therapeutics	Industry	FXN	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: Dose escalation study: 1.8E11 vg/kg (n=6), 5.6E11 vg/kg (n=7),1.2E12 vg/kg (n=4) \| Dose 2: Pivotal dose: 1.2E12 vg/kg	Phase2	Recruiting	Active	2026-06-24	2032-06	2026-09-02	>= 6 Years	26	1	United States		Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	Finalized pivotal trial protocol and statistical analysis plan in June 2026, 1.2E12 will be pivotal dose, topline data readout expected 2H2027	Preclinical Publications : Expression and processing of mature human frataxin after gene therapy in mice Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia News and Press Releases : Lexeo Therapeutics Announces Positive Interim Phase 1/2 Clinical Data of LX2006 for the Treatment of Friedreich Ataxia Cardiomyopathy Lexeo Therapeutics Announces Positive Interim Phase 1/2 Data for LX2006 in Friedreich Ataxia Cardiomyopathy Supporting Advancement to Registrational Study (Corporate Presentation) LX2006 Regulatory Update - June 15, 2026 Lexeo Therapeutics Announces FDA Breakthrough Therapy Designation for LX2006 in Friedreich Ataxia (Corporate Presentation) Corporate Overview - August 2026 Lexeo Therapeutics Announces Progress in FDA Discussions for Accelerated Approval Pathway and Positive Interim Clinical Data for LX2006 in Friedreich Ataxia Cardiomyopathy Clinical Publications : AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich Ataxia A Nonrandomized Clinical Trial (Corporate Presentation) Lexeo Therapeutics Announces Progress in FDA Discussions for Accelerated Approval Pathway and Positive Interim Clinical Data for LX2006 in FA Cardiomyopathy - October 7, 2025 Protocol : Clinical Trial Protocol Related NCTID : Phase 1: NCT05302271 Phase 1/2: NCT05445323
NCT07064759	Neovascular Age-Related Macular Degeneration (nAMD)	4D-150	4D Molecular Therapeutics	Industry	Anti-VEGF	Gene transfer	in vivo	Genetic delivery of therapeutic protein	Intravitreal	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: Phase 1/2 doses: 1E10 vg/eye, 3E10 vg/eye \| Dose 2: Phase 3 dose: 3E10 vg/eye	Phase3	Recruiting	Active	2025-07-07	2029-02	2026-04-16	>= 50 Years	480	94	Locations:United States + 13 more Argentina, Australia, Bulgaria, Germany, Hungary, Italy, Japan, Latvia, Lithuania, Portugal, Singapore, Spain, United Kingdom, United States		Regenerative Medicine Advanced Therapy (RMAT)	Sponsor announced positive 2-year data from Phase 2b trial in July 2026; 4D-150 is also being evaluated for the treatment of Diabetic macular edema in a Phase 2 trial (NCT05930561)	Preclinical Publications : Design and Characterization of a Novel Intravitreal Dual-Transgene Genetic Medicine for Neovascular Retinopathies News and Press Releases : (Corporate Presentation) August 2026 SEC Form 10-Q: 4D Molecular Therapeutics, Inc. Q3 2024 4DMT Focuses Pipeline to Prioritize 4D-150 in Wet AMD & DME 4DMT Announces Positive Long-Term Data from Phase 1/2 PRISM Clinical Trial in Wet AMD Supporting 4D-150’s Potential as a Backbone Therapy with Consistent and Durable Benefit over Multiple Years 4DMT Announces Positive 2-Year Data from PRISM Phase 2b Clinical Trial in a Broad Wet AMD Population Clinical Publications : (Presentation) Extended Follow-Up in the PRISM Clinical Trial Evaluating 4D-150 in Adults with Neovascular Age-related Macular Degeneration - Angiogenesis 2025 (Presentation) Extended Follow-Up in the PRISM Clinical Trial Evaluating 4D-150 in Adults with Neovascular Age-related Macular Degeneration - EURETINA 2024 (Corporate Presentation) Intravitreal 4D-150: Phase 2 Population Extension in Broad Disease Activity Wet AMD Patients - July 2024 (Corporate Presentation) 4D-150 Business & PRISM Wet AMD Long-term Data Update (Presentation) 2-Year Follow-Up: PRISM Phase 2b Clinical Trial Evaluating Investigational 4D-150, an Intravitreal Gene Therapy, in a Broad Neovascular AMD Population - ASRS 2026 Related NCTID : Phase 1/2: NCT05197270 Phase 3: NCT06864988
NCT07218887	BAG3 Mutation Associated Dilated Cardiomyopathy	ALXN2350	Alexion Pharmaceuticals, Inc.	Industry	BAG3	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Undisclosed dose escalation, 3 levels	Phase2, Phase1	Recruiting	Active	2025-10-17	2032-01-27	2026-08-28	18 Years - 70 Years	6	11	Locations:United States + 3 more Italy, Spain, United Kingdom, United States			null	Related NCTID : Natural History Study: NCT05981092
NCT05986864	Neovascular (Wet) Age-Related Macular Degeneration (nAMD)	SKG0106	Skyline Therapeutics (US) Inc.	Industry	Anti-VEGF	Gene transfer	in vivo	Genetic delivery of therapeutic protein	Intravitreal	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 8E9 vg/eye \| Dose 2: 2.4E10 vg/eye \| Dose 3: 7.2E10 vg/eye	Phase2, Phase1	Recruiting	Active	2023-08-03	2027-01-30	2026-08-28	>= 50 Years	68	9	Locations:United States + 1 more China, United States			Therapy is also under development to treat diabetic macular edema	Preclinical Publications : (Abstract) SKG0106, an AAV vector delivered intravitreally, for effective, safe and durable treatment of nAMD - ARVO 2024 News and Press Releases : Skyline Therapeutics Receives FDA Clearance of IND for SKG0106, a Novel Intravitreally Delivered AAV Gene Therapy Candidate for Neovascular Age-related Macular Degeneration Clinical Publications : (Abstract #1626) Preclinical and Phase I Clinical Trial Update of the Safety and Efficacy of SKG0106, a Novel AAV Gene Therapy Product for the Treatment of Neovascular Age-Related Macular Degeneration - ASGCT 2024 Related NCTID : Long Term Follow-Up: NCT06346600 Phase 1: NCT06213038
NCT07627971	GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A	SKY-GJB2	Skylark Bio Inc.	Industry	GJB2	Gene transfer	in vivo	Functional gene replacement	Intracochlear	Viral vector	null	Viral transduction	Adeno-associated virus		Undisclosed	Phase2, Phase1	Recruiting	Active	2026-05-28	2028-06	2026-08-27	9 Months - 7 Years	10	4	United States			First patient was dosed in Phase 1/2 clinical trial, preliminary data is expected by year-end 2026	News and Press Releases : Skylark Bio and Forge Biologics Announce Strategic AAV Development and cGMP Manufacturing Partnership Supporting Skylark’s Clinical Development Skylark Bio Doses First Patient in SONIX Phase 1/2 Trial of SKY-GJB2 for GJB2-Related Pediatric Deafness
NCT02852213	Aromatic L-amino Acid Decarboxylase (AADC) Deficiency	AAV2-hAADC	Krzysztof Bankiewicz	Other	DDC	Gene transfer	in vivo	Functional gene replacement	Intracerebral	Viral vector	Substantia nigra & ventral tegmental area	Viral transduction	Adeno-associated virus		Dose 1: Dose: 1.3E11 vg (<160uL); concentration of 8.3E11 vg/mL \| Dose 2: 4.2E11 vg (160ul) \| Dose 3: 1.6E12 vg (60uL) \| Dose 4: 1.3E12 vg (500uL)	Phase1	Recruiting	Active	2016-07-20	2037-08	2026-08-27	>= 24 Months	42	3	United States			Product is also under evaluation for the treatment of Parkinson's disease	Grant : R01 NS073940 R01 NS094292 1R01NS094292 5R01NS094292 Preclinical Publications : Real-Time Magnetic Resonance Imaging During Convective Gene Therapy Perfusion of the Brain SAFETY AND TOLERABILITY OF MRI-GUIDED INFUSION OF AAV2-hAADC INTO THE MID-BRAIN OF NON-HUMAN PRIMATE Clinical Publications : Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons Protocol : Clinical Trial Protocol and Statistical Analysis Plan Related NCTID : Phase 1: NCT07267065 (Parkinson's disease indication)
NCT07667387	Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency	LNP.UCD.ABE	Rebecca Ahrens-Nicklas	Other	CPS1,OTC,ASS1,ASL,ARG,NAGS,SLC25A15	Gene editing	in vivo	Mutation correction	Intravenous	Encapsulated mRNA	null	Endocytosis	null	ABE mRNA	Undisclosed dose based on body weight with potential for repeat dosing	Phase2, Phase1	Recruiting	Active	2026-06-18	2028-09	2026-08-26	24 Hours - 5 Years	7	1	United States			This clinical trial protocol is a master protocol for participants with a mutation in one of the 7 genes that cause a urea cycle disorder. Participants with a variant that is amenable to correction by adenine base editing will receive a personalized drug product	Preclinical Publications : Pre-IND Briefing Book Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease - this paper outlines a similar case where the target indication was CPS1 deficiency Pre-IND Official Responses/Meeting Summary How to create personalized gene editing platforms: Next steps toward interventional genetics - this paper outlines the sponsor's thinking on creating umbrella clinical trials

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