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 277 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

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

NCT05614531 Unknown Phase2, Phase1

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Spinal Muscular Atrophy Type I

Sponsor Guangzhou Jiayin Biotech Ltd (Industry)

Compound EXG001-307

Therapeutic Modality Gene transfer

Target Gene SMN1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05906953 Unknown Phase2, Phase1

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Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations

Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)

Compound HG004

Therapeutic Modality Gene transfer

Target Gene RPE65

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations 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

NCT04884815 Active not recruiting Phase2, Phase1

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Wilson Disease

Sponsor Ultragenyx Pharmaceutical Inc (Industry)

Compound UX701

Therapeutic Modality Gene transfer

Target Gene ATP7B

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

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

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

NCT06851767 Enrolling by invitation Phase2, Phase1

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X-linked Severe Combined Immunodeficiency (XSCID)

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

Compound BE-HSPC-IL2RG

Therapeutic Modality Gene editing

Target Gene IL2RG (p.Q144X)

Delivery System Undisclosed

Mechanism of Action Mutation correction

NCT03588299 Completed Phase2, Phase1

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Hemophilia A

Sponsor Bayer (Industry)

Compound DTX201

Therapeutic Modality Gene transfer

Target Gene F8

Vector Type Adeno-associated virus

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

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

NCT04127578 Active not recruiting Phase2, Phase1

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Parkinson's Disease

Sponsor Prevail Therapeutics (Industry)

Compound PR001

Therapeutic Modality Gene transfer

Target Gene GBA1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track

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

NCT06817382 Active not recruiting Phase1

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Duchenne Muscular Dystrophy (DMD)

Sponsor Insmed Gene Therapy LLC (Industry)

Compound INS1201

Therapeutic Modality Gene transfer

Target Gene Micro-dystrophin

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06253507 Enrolling by invitation Phase2, Phase1

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Autosomal Recessive Chronic Granulomatous Disease (CGD)

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

Compound PCHIM-p47

Therapeutic Modality Gene transfer

Target Gene NCF1

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01496040 Completed Phase2, Phase1

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Leber Congenital Amaurosis

Sponsor Nantes University Hospital (Other)

Compound RAAV2/4.hRPE65

Therapeutic Modality Gene transfer

Target Gene RPE65

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

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

NCT05984927 Active not recruiting Phase2, Phase1

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

Sponsor Elisigen, Inc. (Industry)

Compound NG101

Therapeutic Modality Gene transfer

Target Gene Codon optimized aflibercept

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

FDA Designations Fast Track

NCT06114056 Active not recruiting Phase1

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Duchenne Muscular Dystrophy (DMD)

Sponsor West China Hospital (Other)

Compound JWK007

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

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

NCT05243017 Active not recruiting Phase2, Phase1

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Huntington's Disease

Sponsor UniQure Biopharma B.V. (Industry)

Compound AMT-130

Therapeutic Modality Gene transfer

Target Gene MiHTT

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action MiRNA knockdown of mutant/aberrant gene

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

NCT05454566 Withdrawn Phase2, Phase1

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Osteoarthritis, Knee

Sponsor ICM Co. Ltd. (Industry)

Compound ICM-203

Therapeutic Modality Gene transfer

Target Gene NKX3-2

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT07113743 Enrolling by invitation Phase2, Phase1

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Chronic Granulomatous Disease (CGD)

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

Compound VSVG-PCCLChimGp91

Therapeutic Modality Gene transfer

Target Gene CYBB

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

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

NCT04040049 Terminated Phase2, Phase1

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Fabry Disease

Sponsor Spur Therapeutics (Industry)

Compound FLT190

Therapeutic Modality Gene transfer

Target Gene GLA

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT04174105 Active not recruiting Phase2, Phase1

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Pompe Disease (Late-onset)

Sponsor Astellas Gene Therapies (Industry)

Compound AT845

Therapeutic Modality Gene transfer

Target Gene GAA

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

NCT02935517 Unknown Phase2, Phase1

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Achromatopsia

Sponsor Beacon Therapeutics (Industry)

Compound AGTC-402

Therapeutic Modality Gene transfer

Target Gene CNGA3

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT07479953 Not yet recruiting Phase1

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GM1 Gangliosidosis - Type I, GM1 Gangliosidosis - Type 2

Sponsor Tippi Mackenzie (Other)

Compound Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase

Therapeutic Modality Gene transfer

Target Gene GLB1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06539208 Unknown Phase2, Phase1

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Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy

Sponsor YolTech Therapeutics Co., Ltd (Industry)

Compound YOLT-201

Therapeutic Modality Gene editing

Target Gene TTR

Delivery System Endocytosis

Mechanism of Action Gene inactivation

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

NCT06237777 Completed Phase1

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Diabetic Macular Edema

Sponsor Wang Min (Other)

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

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

NCT07267065 Not yet recruiting Phase1

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Parkinson's Disease (PD), Early Onset Parkinson Disease

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 Overexpression of protective allele/gene

NCT03217617 Not yet recruiting Phase2, Phase1

View Report

X-linked Severe Combined Immunodeficiency (XSCID)

Sponsor Shenzhen Geno-Immune Medical Institute (Other)

Compound Ivlv-X1 lentiviral vector

Therapeutic Modality Gene transfer

Target Gene IL2RG

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05230459 Active not recruiting Phase2, Phase1

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Limb-Girdle Muscular Dystrophy, Type 2I/R9

Sponsor AskBio Inc (Industry)

Compound AB-1003

Therapeutic Modality Gene transfer

Target Gene FKRP

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

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

NCT05394064 Terminated Phase2, Phase1

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Adrenomyeloneuropathy (AMN)

Sponsor SwanBio Therapeutics, Inc. (Industry)

Compound SBT101

Therapeutic Modality Gene transfer

Target Gene ABCD1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

View Report

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

NCT05432310 Active not recruiting Phase2, Phase1

View Report

Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Sponsor University of California, Los Angeles (Other)

Compound Simoladagene autotemcel

Therapeutic Modality Gene transfer

Target Gene ADA

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, 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

NCT06109181 Active not recruiting Phase2, Phase1

View Report

Arrhythmogenic Right Ventricular Cardiomyopathy

Sponsor Lexeo Therapeutics (Industry)

Compound LX2020

Therapeutic Modality Gene transfer

Target Gene PKP2

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT04411654 Active not recruiting Phase2, Phase1

View Report

Gaucher Disease Type 2

Sponsor Prevail Therapeutics (Industry)

Compound LY3884961

Therapeutic Modality Gene transfer

Target Gene GBA1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06831825 Recruiting Phase1

View Report

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

NCT03720418 Terminated Phase2, Phase1

View Report

Parkinson's Disease

Sponsor Sio Gene Therapies (Industry)

Compound OXB-102

Therapeutic Modality Gene transfer

Target Gene TH-GCH1.DDC (tricistronic)

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT05616793 Recruiting Phase2, Phase1

View Report

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

NCT04046224 Completed Phase2, Phase1

View Report

Fabry Disease

Sponsor Sangamo Therapeutics (Industry)

Compound ST-920

Therapeutic Modality Gene transfer

Target Gene GLA

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06325709 Recruiting Phase2, Phase1

View Report

X-Linked Chronic Granulomatous Disease

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

Compound Base-edited autologous CD34+ cells

Therapeutic Modality Gene editing

Target Gene CYBB c.676 C>T

Delivery System Electroporation

Mechanism of Action Mutation correction

NCT06064890 Recruiting Phase2, Phase1

View Report

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

NCT06706427 Active not recruiting Phase2, Phase1

View Report

Bietti Crystalline Dystrophy

Sponsor NGGT (Suzhou) Biotechnology Co., Ltd. (Industry)

Compound NGGT001

Therapeutic Modality Gene transfer

Target Gene CYP4V2

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT06217861 Recruiting Phase1

View Report

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

NCT05441553 Unknown Phase2, Phase1

View Report

Hemophilia B

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

Compound VGB-R04

Therapeutic Modality Gene transfer

Target Gene F9

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT06611436 Terminated Phase2, Phase1

View Report

Hemophilia B

Sponsor Be Biopharma (Industry)

Compound BE-101

Therapeutic Modality Gene editing

Target Gene F9

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

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

NCT03634007 Completed Phase2, Phase1

View Report

Alzheimer's Disease, Early Onset Alzheimer's Disease

Sponsor Lexeo Therapeutics (Industry)

Compound LX1001

Therapeutic Modality Gene transfer

Target Gene APOE2

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track

NCT05603312 Completed Phase2, Phase1

View Report

Parkinson's Disease

Sponsor MeiraGTx, LLC (Industry)

Compound AAV-GAD

Therapeutic Modality Gene transfer

Target Gene GAD1/2

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT05820152 Terminated Phase2, Phase1

View Report

Leber Hereditary Optic Neuropathy (LHON)

Sponsor Neurophth Therapeutics Inc (Other)

Compound NFS-02

Therapeutic Modality Gene transfer

Target Gene ND1 m.3460G>A

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06765980 Recruiting Phase2, Phase1

View Report

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

NCT05228145 Active not recruiting Phase2, Phase1

View Report

Neuronal Ceroid Lipofuscinosis CLN5

Sponsor Neurogene Inc. (Industry)

Compound NGN-101

Therapeutic Modality Gene transfer

Target Gene CLN5

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04853576 Active not recruiting Phase2, Phase1

View Report

Sickle Cell Disease

Sponsor Editas Medicine, Inc. (Industry)

Compound EDIT-301

Therapeutic Modality Gene editing

Target Gene HBG1/HBG2

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

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

NCT05444894 Active not recruiting Phase2, Phase1

View Report

Beta-Thalassemia Major

Sponsor Editas Medicine, Inc. (Industry)

Compound EDIT-301

Therapeutic Modality Gene editing

Target Gene HBG1/HBG2

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT03368742 Active not recruiting Phase2, Phase1

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Solid Biosciences Inc. (Industry)

Compound SGT-001

Therapeutic Modality Gene transfer

Target Gene Micro-dystrophin

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT02599922 Unknown Phase2, Phase1

View Report

Achromatopsia

Sponsor Beacon Therapeutics (Industry)

Compound AGTC-401

Therapeutic Modality Gene transfer

Target Gene CNGB3

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT03920007 Active not recruiting Phase2, Phase1

View Report

Leber Congenital Amaurosis, LCA, LCA1

Sponsor Atsena Therapeutics Inc. (Industry)

Compound ATSN-101

Therapeutic Modality Gene transfer

Target Gene GUCY2D

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06492850 Unknown Phase2, Phase1

View Report

X-Linked Retinitis Pigmentosa (XLRP)

Sponsor Frontera Therapeutics (Industry)

Compound FT-002

Therapeutic Modality Gene transfer

Target Gene RPGRORF15

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT05164471 Terminated Phase2, Phase1

View Report

Hemophilia B

Sponsor Spur Therapeutics (Industry)

Compound FLT180a

Therapeutic Modality Gene transfer

Target Gene F9

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT01422772 Completed Phase2, Phase1

View Report

Ischemic Heart Disease

Sponsor Helixmith Co., Ltd. (Industry)

Compound ENGENSIS

Therapeutic Modality Gene transfer

Target Gene HGF

Delivery System Not Applicable

Mechanism of Action Overexpression of protective allele/gene

NCT05361031 Completed Phase2, Phase1

View Report

Charcot-Marie-Tooth Disease 1A

Sponsor Helixmith Co., Ltd. (Industry)

Compound ENGENSIS

Therapeutic Modality Gene transfer

Target Gene HGF

Delivery System Not Applicable

Mechanism of Action Overexpression of protective allele/gene

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

NCT07293546 Enrolling by invitation Phase2, Phase1

View Report

FOXG1 Syndrome

Sponsor FOXG1 Research Foundation (Other)

Compound FRF-001

Therapeutic Modality Gene transfer

Target Gene FOXG1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT02341807 Completed Phase2, Phase1

View Report

Choroideremia

Sponsor Spark Therapeutics, Inc. (Industry)

Compound SPK-7001

Therapeutic Modality Gene transfer

Target Gene CHM

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

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

NCT05144386 Completed Phase1

View Report

HIV-1-infection

Sponsor Excision BioTherapeutics (Industry)

Compound EBT-101

Therapeutic Modality Gene editing

Target Gene HIV genes

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Gene excision

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

NCT06460844 Active not recruiting Phase1

View Report

Retinitis Pigmentosa, Choroideremia

Sponsor Ray Therapeutics, Inc. (Industry)

Compound RTx-015

Therapeutic Modality Gene transfer

Target Gene Codon optimized ChR-3M

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT05456880 Active not recruiting Phase2, Phase1

View Report

Sickle Cell Disease

Sponsor Beam Therapeutics Inc. (Industry)

Compound BEAM-101

Therapeutic Modality Gene editing

Target Gene HBG1/HBG2

Delivery System Electroporation

Mechanism of Action Mutation correction

FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation

NCT04408625 Active not recruiting Phase2, Phase1

View Report

Frontotemporal Dementia

Sponsor Prevail Therapeutics (Industry)

Compound LY3884963

Therapeutic Modality Gene transfer

Target Gene GRN

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT03223194 Terminated Phase1

View Report

Crigler-Najjar Syndrome

Sponsor Audentes Therapeutics (Industry)

Compound AT342

Therapeutic Modality Gene transfer

Target Gene UGT1A1

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

NCT05739643 Active not recruiting Phase2, Phase1

View Report

Krabbe Disease

Sponsor Forge Biologics, Inc (Industry)

Compound FBX-101

Therapeutic Modality Gene transfer

Target Gene GALC

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT04783181 Active not recruiting Phase2, Phase1

View Report

Congenital Adrenal Hyperplasia

Sponsor Adrenas Therapeutics Inc (Industry)

Compound BBP-631

Therapeutic Modality Gene transfer

Target Gene CYP21A2

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT01301443 Completed Phase1

View Report

Neovascular (Wet) Age-Related Macular Degeneration

Sponsor Oxford BioMedica (Industry)

Compound RetinoStat

Therapeutic Modality Gene transfer

Target Gene COL18A1+PLG

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT05293626 Active not recruiting Phase2, Phase1

View Report

Leber Hereditary Optic Neuropathy (LHON)

Sponsor Neurophth Therapeutics Inc (Other)

Compound OPVIKA

Therapeutic Modality Gene transfer

Target Gene ND4 m.11778G>A

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT02168686 Completed Phase2, Phase1

View Report

Alpha-1 Antitrypsin Deficiency (AATD)

Sponsor Adverum Biotechnologies, Inc. (Industry)

Compound ADVM-043

Therapeutic Modality Gene transfer

Target Gene SERPINA1

Vector Type Adeno-associated virus

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT02186418 Terminated Phase2, Phase1

View Report

Sickle Cell Disease

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

Compound ARU-1801

Therapeutic Modality Gene transfer

Target Gene HBG(G16D)

Vector Type Lentivirus

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

Visual summary of all 277 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

Page 1 of 3

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
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
NCT05614531	Spinal Muscular Atrophy Type I	EXG001-307	Guangzhou Jiayin Biotech Ltd	Industry	SMN1	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 1.1E14 vg/kg \| Dose 2: 1.5E14 vg/kg	Phase2, Phase1	Unknown	Active	2022-11-02	2025-08	2024-09-19	1 Day - 180 Days	12	2	China			null	Preclinical Publications : Efficacy and preliminary safety assessment of EXG001-307 AAV gene therapy for spinal muscular atrophy News and Press Releases : Exegenesis Bio to Present 9-Patient Data from a Phase 1/2 Clinical Trial of EXG001-307, a Novel rAAV Gene Therapy for Spinal Muscular Atrophy (SMA) Type 1: Improved Head Control and Sitting Without External Assistance Clinical Publications : (Abstract #627) Clinical Safety and Efficacy of EXG001-307 in SMA Type 1 Patients: A Next-Generation AAV-Based Gene Therapy - ASGCT 2024 Related NCTID : Early Phase 1: NCT06888661 (using intrathecal administration)
NCT05906953	Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations	HG004	HuidaGene Therapeutics Co., Ltd.	Industry	RPE65	Gene transfer	in vivo	Functional gene replacement	Subretinal	Viral vector	null	Viral transduction	Adeno-associated virus		Undisclosed dose escalation, 3 levels	Phase2, Phase1	Unknown	Active	2023-05-09	2025-12	2024-09-19	6 Years - 50 Years	20	3	Locations:United States + 1 more China, United States		Orphan Drug Designation, Rare Pediatric Disease Designation	First patient dosed November 2023	Preclinical Publications : (Abstract # 5091-A0348) In-vivo Validation of HG004 Gene Replacement Therapy for Inherited Blindness - ARVO 2024 News and Press Releases : HuidaGene Therapeutics Announces First Patient Dosed in Multnational Phase 1/2 Trial of HG004 for Inherited Blindness Clinical Publications : (Abstract #3286) HG004 Gene Therapy for Patients with Leber Congenital Amaurosis - ARVO 2024 Related NCTID : Early Phase 1: NCT06088992
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
NCT04884815	Wilson Disease	UX701	Ultragenyx Pharmaceutical Inc	Industry	ATP7B	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 5.0E12 GC/kg \| Dose 2: 1.0E13 GC/kg \| Dose 3: 2.0E13 GC/kg \| Dose 4: Undisclosed dose 4	Phase2, Phase1	Active not recruiting	Active	2021-05-07	2034-03	2026-09-25	>= 18 Years	82	16	Locations:United States + 3 more Portugal, Spain, United Kingdom, United States		Fast Track, Orphan Drug Designation	Enrollment for 4th cohort is complete, data expected 2026	News and Press Releases : Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update Ultragenyx Reports Third Quarter 2025 Financial Results and Corporate Update UX701 (rivunatpagene miziparvovec): Gene therapy for the potential treatment of Wilson disease Ultragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease Ultragenyx Reports Fourth Quarter and Full Year 2025 Financial Results and Corporate Update Ultragenyx Announces Completion of Dosing Across Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease
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
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
NCT06851767	X-linked Severe Combined Immunodeficiency (XSCID)	BE-HSPC-IL2RG	National Institute of Allergy and Infectious Diseases (NIAID)	NIH	IL2RG (p.Q144X)	Gene editing	ex vivo	Mutation correction	Intravenous	Autologous cells	CD34+ cells	Undisclosed	null	undisclosed base editor	>5E6 cells/kg	Phase2, Phase1	Enrolling by invitation	Active	2025-02-26	2034-12-31	2026-09-23	3 Years - 99 Years	18	1	United States			null
NCT03588299	Hemophilia A	DTX201	Bayer	Industry	F8	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 0.5E13 GC/kg \| Dose 2: 1E13 GC/kg \| Dose 3: 2E13 GC/kg \| Dose 4: 4E13 GC/kg	Phase2, Phase1	Completed	Active	2018-07-06	2026-09-15	2026-09-23	>= 18 Years	11	13	Locations:United States + 5 more Bulgaria, France, Germany, Netherlands, United Kingdom, United States			License reverted to Ultragenyx in 2022, uncertain development status	Preclinical Publications : Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice Optimized Adeno-Associated Viral-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques News and Press Releases : Ultragenyx Pharmaceutical Inc. SEC Filing for FY2023 Clinical Publications : Characteristics of BAY 2599023 in the Current Treatment Landscape of Hemophilia A Gene Therapy (Abstract) First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A - BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII - ASH 2020 (Abstract # 1539) First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A—BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII - ASH 2020
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)
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)
NCT04127578	Parkinson's Disease	PR001	Prevail Therapeutics	Industry	GBA1	Gene transfer	in vivo	Overexpression of protective allele/gene	Intracisternal	Viral vector	null	Viral transduction	Adeno-associated virus		Undisclosed dose escalation, 2 levels	Phase2, Phase1	Active not recruiting	Active	2019-10-14	2031-08-31	2026-09-18	35 Years - 80 Years	32	15	Locations:United States + 1 more Israel, United States		Fast Track	null	Preclinical Publications : (Abstract) PR001 gene therapy improved phenotypes in models of Parkinson's disease with GBA1 mutation News and Press Releases : Gene Therapy for Parkinson's Disease Associated with GBA1 Mutations Protocol : (Abstract) Design of Phase 1/2a Study of AAV9-Based Gene Therapy for Parkinson's Disease with Pathogenic GBA1 Mutations (PROPEL Trial) - AAN 2020
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
NCT06817382	Duchenne Muscular Dystrophy (DMD)	INS1201	Insmed Gene Therapy LLC	Industry	Micro-dystrophin	Gene transfer	in vivo	Functional gene replacement	Intrathecal	Viral vector	null	Viral transduction	Adeno-associated virus		Undisclosed dose escalation, 2 levels	Phase1	Active not recruiting	Active	2025-02-04	2028-03-31	2026-09-16	2 Years - 4 Years	12	10	United States			IND cleared in 4Q2024, Clinical trial initiation planned in 1H2025	News and Press Releases : Insmed's gene therapy poised to challenge DMD landscape after IND clearance
NCT06253507	Autosomal Recessive Chronic Granulomatous Disease (CGD)	PCHIM-p47	National Institute of Allergy and Infectious Diseases (NIAID)	NIH	NCF1	Gene transfer	ex vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	Lentivirus		Transduced CD34+ cells, minimum dose: 3E6 CD34+ cells/kg	Phase2, Phase1	Enrolling by invitation	Active	2024-02-09	2027-03-31	2026-09-15	3 Years - 65 Years	10	1	United States			null	Preclinical Publications : Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease Related NCTID : Phase 1/2: NCT05207657 (UK trial)
NCT01496040	Leber Congenital Amaurosis	RAAV2/4.hRPE65	Nantes University Hospital	Other	RPE65	Gene transfer	in vivo	Functional gene replacement	Subretinal	Viral vector	null	Viral transduction	Adeno-associated virus		Dose range: 1.22E10 -4.8E10 vg (n=9)	Phase2, Phase1	Completed	Inactive	2011-11-24	2014-08	2026-09-14	6 Years - 50 Years	9	1	France			Clinical study demonstrated safety, inconsistent findings on long-term efficacy	Preclinical Publications : (Abstract 207) Evaluation of the Immune Responses in the Treatment of RPE65 Deficient Dogs by Subretinal Injection of a Recombinant AAV Serotype 4 - ASGCT 2011 Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium Gene therapeutic prospects in early onset of severe retinal dystrophy: restoration of vision in RPE65 Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium Clinical Publications : Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis
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
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
NCT05984927	Neovascular (Wet) Age-Related Macular Degeneration (nAMD)	NG101	Elisigen, Inc.	Industry	Codon optimized aflibercept	Gene transfer	in vivo	Genetic delivery of therapeutic protein	Subretinal	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 1E9 vg/eye \| Dose 2: 3E9 vg/eye \| Dose 3: 8E9 vg/eye	Phase2, Phase1	Active not recruiting	Active	2023-07-24	2030-01	2026-09-10	50 Years - 89 Years	20	4	Locations:United States + 1 more Canada, United States		Fast Track	Plans to expand trial into the USA	Preclinical Publications : (Poster) Preclinical evaluation of NG101 for treatment of wet age-related macular degeneration - ASGCT 2024 Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration (Abstract) Low-Dose AAV Gene Therapy NG101 for Wet AMD: Preclinical Results and Ongoing Phase 1/2a Trial Design - ARVO 2025 News and Press Releases : Neuracle Genetics Received IND Approval for Phase 1/2a Clinical Trials of AAV Gene Therapy in the USA A New Milestone for Accelerating Drug Development with FDA Fast Track Designation Phase 1/2a Clinical Trials Progressing Smoothly in the U.S. and Canada
NCT06114056	Duchenne Muscular Dystrophy (DMD)	JWK007	West China Hospital	Other	Micro-dystrophin	Gene transfer	in vivo	Overexpression of protective allele/gene	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 1.0E14 vg/kg \| Dose 2: 2.0E14 vg/kg	Phase1	Active not recruiting	Active	2023-10-29	2029-11-13	2026-09-10	5 Years - 10 Years	3	1	China			Investigator-initiated trial conducted at West China Hospital
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
NCT05243017	Huntington's Disease	AMT-130	UniQure Biopharma B.V.	Industry	MiHTT	Gene transfer	in vivo	MiRNA knockdown of mutant/aberrant gene	Intraparenchymal	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: Low dose cohort: 6E12 gc/subject \| Dose 2: High dose cohort: 6E13 gc/subject	Phase2, Phase1	Active not recruiting	Active	2021-11-01	2029-10-07	2025-03-10	25 Years - 65 Years	14	4	Locations:Poland + 1 more Poland, United Kingdom		Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation	Sponsor announced in June 2026 that FDA has reversed previous decision, and 3-year analysis from Phase I/II study is acceptable as the primary basis of a BLA for accelerated approval, BLA was submitted in September 2026 and sponsor has requested priority review	Preclinical Publications : Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington's disease models Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease News and Press Releases : uniQure Announces FDA Breakthrough Therapy Designation Granted to AMT-130 for the Treatment of Huntingtonâs Disease uniQure Announces Submission of Biologics License Application for Ifezuntirgene Inilparvovec (AMT-130) in Huntington’s Disease uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease uniQure Announces Alignment with FDA on Key Elements of Accelerated Approval Pathway for AMT-130 in Huntington's Disease uniQure Announces Plan for BLA Submission for AMT-130 in Huntington’s Disease uniQure Announces 2025 Financial Results and Provides Recent Company Updates uniQure Announces Positive Topline Results from Pivotal Phase I/II Study of AMT-130 in Patients with Huntington’s Disease uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease Clinical Publications : Huntington's Disease Clinical Trials Corner: March 2024 (Corporate Presentation) Phase I/II AMT-130 Huntington's Disease Program Update - July 2024 Related NCTID : Phase 1/2: NCT04120493
NCT05454566	Osteoarthritis, Knee	ICM-203	ICM Co. Ltd.	Industry	NKX3-2	Gene transfer	in vivo	Overexpression of protective allele/gene	Intra-articular	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 6E12 vg \| Dose 2: 2E13 vg \| Dose 3: 6E13 vg	Phase2, Phase1	Withdrawn	Active	2022-07-04	2028-11-15	2026-09-08	50 Years - 80 Years	null	null				Update on Phase 1/2a study given at ASGCT 2024	Clinical Publications : (Abstract #1847) Interim Analysis of a Phase 1/2a Study of ICM-203 AAV Gene Therapy for Osteoarthritis - ASGCT 2025 INTERIM REVIEW OF EFFICACY FROM A FIRST-IN-HUMAN PHASE 1/2A CLINICAL STUDY OF ICM-203, AN INTRA-ARTICULAR, AAV GENE THERAPY FOR OSTEOARTHRITIS Related NCTID : Phase 1/2: NCT04875754 Long Term Follow Up: NCT05752032
NCT07113743	Chronic Granulomatous Disease (CGD)	VSVG-PCCLChimGp91	National Institute of Allergy and Infectious Diseases (NIAID)	NIH	CYBB	Gene transfer	ex vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	Lentivirus		Transduced CD34+ cells (6.5-32.6E6 cells/kg)	Phase2, Phase1	Enrolling by invitation	Inactive	2025-08-08	2029-09-01	2026-09-08	3 Years - 60 Years	10	1	United States		Orphan Drug Designation	Commercial rights are owned by Orchard, which has deprioritized the program	Preclinical Publications : Gene correction of induced pluripotent stem cells derived from a murine model of X-linked chronic granulomatous disorder Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells News and Press Releases : Orchard Therapeutics Extends Runway into 2024, Focusing HSC Gene Therapy Platform Exclusively on Severe Neurometabolic Diseases and Research Platform Orchard Therapeutics Announces FDA Granted Orphan Drug Designation for OTL-102 for the Treatment of X-linked Chronic Granulomatous Disease (X-CGD) Clinical Publications : Lentiviral gene therapy for X-linked chronic granulomatous disease Related NCTID : Phase 1/2: NCT02757911 Phase 1/2: NCT02234934 Phase 1/2: NCT01855685
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
NCT04040049	Fabry Disease	FLT190	Spur Therapeutics	Industry	GLA	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		7.5E11 vg/kg	Phase2, Phase1	Terminated	Inactive	2019-07-26	2023-05-02	2026-09-03	>= 18 Years	3	13	Locations:United States + 6 more Austria, Canada, Germany, Italy, Norway, United Kingdom, United States			Development cancelled to prioritize other programs	Preclinical Publications : Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease News and Press Releases : Freeline pauses development of Fabry disease gene therapy FLT190 Freeline Reports New Data from Fabry Disease Program, Pipeline and Company Updates, Including Appointment of Pamela Foulds, MD as Chief Medical Officer, and Third Quarter 2021 Financial Results
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
NCT04174105	Pompe Disease (Late-onset)	AT845	Astellas Gene Therapies	Industry	GAA	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	null	Viral transduction	Adeno-associated virus		Dose 1: 3E13 vg/kg \| Dose 2: 6E13 vg/kg \| Dose 3: 1E14 vg/kg	Phase2, Phase1	Active not recruiting	Active	2019-11-13	2035-02-28	2026-09-02	18 Years - 80 Years	11	4	Locations:United States + 1 more United Kingdom, United States			Anticipated PoC judgement timing: 2H/FY2025	Preclinical Publications : Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicity News and Press Releases : Q3 YTD/FY2024 Financial Results - February 2025 Astellas Announces Update on Preliminary Safety and Efficacy Data from FORTIS Study of Investigational AT845 in Adults with Late-Onset Pompe Disease Clinical Publications : (Abstract) Two-year safety and exploratory efficacy of AT845 gene replacement therapy for late onset Pompe disease: FORTIS, a phase 1/2 open-label clinical study - MDA 2024
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
NCT02935517	Achromatopsia	AGTC-402	Beacon Therapeutics	Industry	CNGA3	Gene transfer	in vivo	Functional gene replacement	Subretinal	Viral vector	Cone cells	Viral transduction	Adeno-associated virus		Dose 1: 4.0E10 vg/mL \| Dose 2: 1.2E11 vg/mL \| Dose 3: 3.6E11 vg/mL \| Dose 4: 1.1E12 vg/mL \| Dose 5: 3.2E12 vg/mL	Phase2, Phase1	Unknown	Inactive	2016-10-13	2026-08	2022-07-22	>= 4 Years	24	6	Locations:United States + 1 more Israel, United States		Orphan Drug Designation	Development discontinued by Sponsor	Preclinical Publications : Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep News and Press Releases : Orphan Drug Designation Beacon Completed Clinical Trials SEC Form 10-K: AGTC Annual Report for FY 2022 Clinical Publications : (Abstract) Interim Safety Results in Two Phase 1/2 Open-label, Dose-escalation Clinical Trials of Subretinal Gene Therapy with AGTC-401 (rAAV2tYF-PR1.7-hCNGB3) and AGTC-402 (rAAV2tYF-PR1.7-hCNGA3) in Subjects with Achromatopsia (ACHM) - ARVO 2022
NCT07479953	GM1 Gangliosidosis - Type I, GM1 Gangliosidosis - Type 2	Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase	Tippi Mackenzie	Other	GLB1	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	Umbilical vein	Viral transduction	Adeno-associated virus		Undisclosed	Phase1	Not yet recruiting	Active	2026-03-14	2055-06-01	2026-08-31		5	null				This trial is enrolling a maternal subject and dosing a fetus through the umbilical vein to treat severe GM1 Gangliosidosis symptoms prior to the birth of the fetus	Preclinical Publications : Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus
NCT06539208	Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy	YOLT-201	YolTech Therapeutics Co., Ltd	Industry	TTR	Gene editing	in vivo	Gene inactivation	Intravenous	Encapsulated mRNA	Liver	Endocytosis	null	Cas9 mRNA	Undisclosed dose escalation	Phase2, Phase1	Unknown	Active	2024-08-01	2026-06-30	2024-08-06	18 Years - 80 Years	31	3	China			Dose escalation completed December 2024	Preclinical Publications : Library-Assisted Evolution in Eukaryotic Cells Yield Adenine Base Editors with Enhanced Editing Specificity News and Press Releases : YolTech Completes Dose Escalation in YOLT-201 Phase I Trial YolTech Therapeutics Announces Positive Clinical Results for YOLT-201, an Investigational In Vivo Gene Editing Therapy for Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

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