SCGE Platform - Gene Therapy Clinical Trials

NCT05788536 Recruiting Phase2, Phase1

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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 dual AAV1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT07667387 Not yet 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

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 AAV8

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT04713475 Active not recruiting Phase2, Phase1

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GM1 Gangliosidosis, Beta-Galactosidase-1 (GLB1) Deficiency

Sponsor Gemma Biotherapeutics (Industry)

Compound PBGM01

Therapeutic Modality Gene transfer

Target Gene GLB1

Vector Type AAVhu68

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05693142 Recruiting Phase3, Phase2

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Duchenne Muscular Dystrophy (DMD)

Sponsor REGENXBIO Inc. (Industry)

Compound RGX-202

Therapeutic Modality Gene transfer

Target Gene Micro-dystrophin

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

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 VSV-G

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06285643 Recruiting Phase2

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Parkinson's Disease

Sponsor AskBio Inc (Industry)

Compound AB-1005

Therapeutic Modality Gene transfer

Target Gene GDNF

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

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

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 AAV9

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

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

NCT01054339 Completed Phase2

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Alpha-1 Antitrypsin Deficiency (AATD)

Sponsor Beacon Therapeutics (Industry)

Compound AGTC-0106

Therapeutic Modality Gene transfer

Target Gene SERPINA1

Vector Type AAV1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04124042 Completed Phase2

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Osteoarthritis, Knee

Sponsor Xalud Therapeutics, Inc. (Industry)

Compound XT-150

Therapeutic Modality Gene transfer

Target Gene Recombinant IL10

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track

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

NCT06559176 Enrolling by invitation Phase2, Phase1

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Chronic Granulomatous Disease

Sponsor Prime Medicine, Inc. (Industry)

Compound PM359

Therapeutic Modality Gene editing

Target Gene NCF1 (c.75_76delGT)

Delivery System Electroporation

Mechanism of Action Mutation correction

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT03720418 Terminated Phase2, Phase1

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Parkinson's Disease

Sponsor Sio Gene Therapies (Industry)

Compound OXB-102

Therapeutic Modality Gene transfer

Target Gene TH-GCH1.DDC (tricistronic)

Vector Type EIAV

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

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 AAV9

Delivery System Viral transduction

Mechanism of Action MiRNA knockdown of mutant/aberrant gene

NCT03566043 Active not recruiting Phase3, Phase2

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Mucopolysaccharidosis Type II (Hunter Syndrome)

Sponsor REGENXBIO Inc. (Industry)

Compound NAVSUNLI

Therapeutic Modality Gene transfer

Target Gene IDS

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

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 LK03

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03217617 Recruiting Phase2, Phase1

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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 LV

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 AAV5.2

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

FDA Designations Fast Track

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 VSV-G

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT05836259 Recruiting Phase2, Phase1

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Hypertrophic Cardiomyopathy

Sponsor Tenaya Therapeutics (Industry)

Compound TN-201

Therapeutic Modality Gene transfer

Target Gene MYBPC3

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

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 AAV2/4

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04537377 Active not recruiting Phase2, Phase1

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Wilson Disease

Sponsor Vivet Therapeutics SAS (Industry)

Compound VTX-801

Therapeutic Modality Gene transfer

Target Gene ATP7B-minigene

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT01801709 Completed Phase2, Phase1

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Metachromatic Leukodystrophy

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

Compound AAVrh.10-CAG/cuARSA

Therapeutic Modality Gene transfer

Target Gene ARSA

Vector Type AAVrh.10

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03041324 Terminated Phase2, Phase1

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Mucopolysaccharidosis Type II (Hunter Syndrome)

Sponsor Sangamo Therapeutics (Industry)

Compound SB-913

Therapeutic Modality Gene editing

Target Gene IDS

Vector Type AAV2/6

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT03300453 Completed Phase2, Phase1

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Sanfilippo Syndrome B

Sponsor UniQure Biopharma B.V. (Industry)

Compound AMT-110

Therapeutic Modality Gene transfer

Target Gene NAGLU

Vector Type AAV2/5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06141460 Recruiting Phase2, Phase1

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

Sponsor Shanghai Refreshgene Technology Co., Ltd. (Industry)

Compound RRG001

Therapeutic Modality Gene transfer

Target Gene Anti-VEGF

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT06255782 Recruiting Phase2, Phase1

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Ornithine Transcarbamylase (OTC) Deficiency

Sponsor iECURE, Inc. (Industry)

Compound ECUR-506

Therapeutic Modality Gene transfer

Target Gene OTC/PCSK9 locus

Vector Type dual AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Fast Track

NCT06480461 Active not recruiting Phase2, Phase1

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Parkinson's Disease

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

Compound VGN-R09b

Therapeutic Modality Gene transfer

Target Gene DDC

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track

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 AAV9

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track

NCT04480567 Active not recruiting Phase2, Phase1

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Phenylketonuria (PKU)

Sponsor BioMarin Pharmaceutical (Industry)

Compound BMN 307

Therapeutic Modality Gene transfer

Target Gene PAH

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT05641610 Recruiting Phase2, Phase1

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Hemophilia B

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

Compound ZS801

Therapeutic Modality Gene transfer

Target Gene F9

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03199469 Active not recruiting Phase3, Phase2

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X-Linked Myotubular Myopathy

Sponsor Astellas Gene Therapies (Industry)

Compound AT132

Therapeutic Modality Gene transfer

Target Gene MTM1

Vector Type AAV8

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

NCT06611436 Terminated Phase2, Phase1

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Hemophilia B

Sponsor Be Biopharma (Industry)

Compound BE-101

Therapeutic Modality Gene editing

Target Gene F9

Vector Type AAV6

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT00598481 Completed Phase2

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Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Sponsor Fondazione Telethon (Other)

Compound STRIMVELIS

Therapeutic Modality Gene transfer

Target Gene ADA

Vector Type MoMLV (RV)

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05353647 Active not recruiting Phase2

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Sickle Cell Disease

Sponsor David Williams (Other)

Compound Autologous CD34+ HSC cells transduced with the lentiviral vector containing BCL11a-targeted shRNA

Therapeutic Modality Gene transfer

Target Gene BCL11A

Vector Type VSV-G

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT03580083 Suspended Phase2, Phase1

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

Sponsor REGENXBIO Inc. (Industry)

Compound RGX-111

Therapeutic Modality Gene transfer

Target Gene IDUA

Vector Type AAV2/9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT04581785 Terminated Phase2, Phase1

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Methylmalonic Acidemia

Sponsor LogicBio Therapeutics, Inc (Industry)

Compound LB-001

Therapeutic Modality Gene transfer

Target Gene MMUT

Vector Type AAV-LK03

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06422351 Suspended Phase2

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Pyruvate Kinase Deficiency

Sponsor Rocket Pharmaceuticals Inc. (Industry)

Compound RP-L301

Therapeutic Modality Gene transfer

Target Gene PKLR

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug 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 AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT05739643 Active not recruiting Phase2, Phase1

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Krabbe Disease

Sponsor Forge Biologics, Inc (Industry)

Compound FBX-101

Therapeutic Modality Gene transfer

Target Gene GALC

Vector Type AAVrh10

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06492863 Recruiting Phase2, Phase1

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

Sponsor Frontera Therapeutics (Industry)

Compound FT-003

Therapeutic Modality Gene transfer

Target Gene Aflibercept

Vector Type AAV2.7m8

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT05541627 Active not recruiting Phase2, Phase1

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Huntington's Disease

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

Compound AB-1001

Therapeutic Modality Gene transfer

Target Gene CYP46A1

Vector Type AAVrh10

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT03612869 Unknown Phase3, Phase2

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Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)

Sponsor LYSOGENE (Industry)

Compound LYS-SAF301

Therapeutic Modality Gene transfer

Target Gene SGSH

Vector Type AAV2rh.10

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT03315182 Terminated Phase2, Phase1

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Mucopolysaccharidosis Type 3 B

Sponsor Abeona Therapeutics, Inc (Industry)

Compound ABO-101

Therapeutic Modality Gene transfer

Target Gene NAGLU

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06983158 Terminated Phase2, Phase1

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Developmental and Epileptic Encephalopathy

Sponsor Capsida Biotherapeutics, Inc. (Industry)

Compound CAP-002

Therapeutic Modality Gene transfer

Target Gene STXBP1

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT02317887 Completed Phase2, Phase1

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X-Linked Retinoschisis

Sponsor VegaVect, Inc. (Industry)

Compound RS1 AAV Vector

Therapeutic Modality Gene transfer

Target Gene RS1

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06942572 Recruiting Phase2, Phase1

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Stargardt Disease

Sponsor Splice Bio (Industry)

Compound SB-007

Therapeutic Modality Gene transfer

Target Gene ABCA4

Vector Type dual AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT01505062 Terminated Phase2, Phase1

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Usher Syndrome, Retinitis Pigmentosa

Sponsor Sanofi (Industry)

Compound SAR421869

Therapeutic Modality Gene transfer

Target Gene MYO7A

Vector Type EIAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06517888 Recruiting Phase2, Phase1

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Vestibular Schwannoma

Sponsor Akouos, Inc. (Industry)

Compound AAVAnc80-antiVEGF

Therapeutic Modality Gene transfer

Target Gene Anti-VEGF

Vector Type AAVAnc80

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT03538899 Recruiting Phase2, Phase1

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Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency

Sponsor University of California, San Francisco (Other)

Compound AProArt

Therapeutic Modality Gene transfer

Target Gene DCLRE1C

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05616793 Recruiting Phase2, Phase1

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Leber Congenital Amaurosis-Type 5

Sponsor Opus Genetics, Inc (Industry)

Compound OPGx-LCA5

Therapeutic Modality Gene transfer

Target Gene LCA5

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT04248439 Active not recruiting Phase2

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Fanconi Anemia Complementation Group A

Sponsor Rocket Pharmaceuticals Inc. (Industry)

Compound RP-L102

Therapeutic Modality Gene transfer

Target Gene FANCA

Vector Type LV

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

NCT05071222 Recruiting Phase2, Phase1

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Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency

Sponsor Assistance Publique - Hôpitaux de Paris (Other)

Compound ARTEGENE

Therapeutic Modality Gene transfer

Target Gene DCLRE1C

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03328130 Terminated Phase2, Phase1

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Retinitis Pigmentosa

Sponsor eyeDNA Therapeutics (Industry)

Compound CTX-PDE6B

Therapeutic Modality Gene transfer

Target Gene PDE6B

Vector Type AAV2/5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Rare Pediatric Disease Designation

NCT04774536 Recruiting Phase2, Phase1

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Sickle Cell Disease

Sponsor Mark Walters, MD (Other)

Compound CRISPR_SCD001

Therapeutic Modality Gene editing

Target Gene HBB

Delivery System Electroporation

Mechanism of Action Mutation correction

NCT03964792 Completed Phase2, Phase1

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Sickle Cell Disease

Sponsor Assistance Publique - Hôpitaux de Paris (Other)

Compound DREPAGLOBE

Therapeutic Modality Gene transfer

Target Gene HBB

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT02559830 Unknown Phase2, Phase1

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Metachromatic Leukodystrophy, Adrenoleukodystrophy

Sponsor Shenzhen Second People's Hospital (Other)

Compound CD34+ hematopoietic stem cells transduced with ABCD1 or ARSA

Therapeutic Modality Gene transfer

Target Gene ABCD1 or ARSA

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT00749957 Completed Phase2, Phase1

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Leber Congenital Amaurosis-Type 2

Sponsor Beacon Therapeutics (Industry)

Compound RAAV2-CB-hRPE65

Therapeutic Modality Gene transfer

Target Gene RPE65

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05121376 Active not recruiting Phase2, Phase1

View Report

Hereditary Angioedema

Sponsor BioMarin Pharmaceutical (Industry)

Compound BMN 331

Therapeutic Modality Gene transfer

Target Gene SERPING1

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT01422772 Completed Phase2, Phase1

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Ischemic Heart Disease

Sponsor Helixmith Co., Ltd. (Industry)

Compound ENGENSIS

Therapeutic Modality Gene transfer

Target Gene HGF

Vector Type none

Mechanism of Action Overexpression of protective allele/gene

NCT05164471 Terminated Phase2, Phase1

View Report

Hemophilia B

Sponsor Spur Therapeutics (Industry)

Compound FLT180a

Therapeutic Modality Gene transfer

Target Gene F9

Vector Type AAVS3

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03920007 Active not recruiting Phase2, Phase1

View Report

Leber Congenital Amaurosis, LCA, LCA1

Sponsor Atsena Therapeutics Inc. (Industry)

Compound ATSN-101

Therapeutic Modality Gene transfer

Target Gene GUCY2D

Vector Type AAV2/5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT00643747 Completed Phase2, Phase1

View Report

Leber Congenital Amaurosis-Type 2

Sponsor University College, London (Other)

Compound TgAAG76

Therapeutic Modality Gene transfer

Target Gene RPE65

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT02781480 Completed Phase2, Phase1

View Report

Leber Congenital Amaurosis

Sponsor MeiraGTx UK II Ltd (Industry)

Compound Cevaretigene ritoparvovec

Therapeutic Modality Gene transfer

Target Gene RPE65

Vector Type AAV2/5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05445323 Active not recruiting Phase2, Phase1

View Report

Friedreich Ataxia, Cardiomyopathy, Secondary

Sponsor Lexeo Therapeutics (Industry)

Compound LX2006

Therapeutic Modality Gene transfer

Target Gene FXN

Vector Type AAVrh10

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

NCT02716246 Recruiting Phase3, Phase2

View Report

Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)

Sponsor Ultragenyx Pharmaceutical Inc (Industry)

Compound UX111

Therapeutic Modality Gene transfer

Target Gene SGSH

Vector Type AAV9

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

NCT06196840 Active not recruiting Phase2

View Report

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

Sponsor Innostellar Biotherapeutics Co.,Ltd (Industry)

Compound LX102

Therapeutic Modality Gene transfer

Target Gene VEGF-trap

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

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 AAV9-PL

Delivery System Viral transduction

Mechanism of Action Functional gene replacement, Gene inactivation

FDA Designations Fast Track, Orphan Drug Designation

NCT03326336 Active not recruiting Phase2, Phase1

View Report

Non-syndromic Retinitis Pigmentosa

Sponsor GenSight Biologics (Industry)

Compound GS030

Therapeutic Modality Gene transfer

Target Gene ChR-tdT

Vector Type AAV2.7m8

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track, Orphan Drug Designation

NCT04945772 Completed Phase2

View Report

Retinitis Pigmentosa, Retinal Dystrophies, Retinal Degeneration

Sponsor Nanoscope Therapeutics Inc. (Industry)

Compound MCO-010

Therapeutic Modality Gene transfer

Target Gene MCO

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track, Orphan Drug Designation

NCT04850118 Active not recruiting Phase3, Phase2

View Report

X-Linked Retinitis Pigmentosa (XLRP)

Sponsor Beacon Therapeutics (Industry)

Compound AGTC-501

Therapeutic Modality Gene transfer

Target Gene RPGR

Vector Type AAV2tYF

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT05417126 Completed Phase2

View Report

Stargardt Disease

Sponsor Nanoscope Therapeutics Inc. (Industry)

Compound MCO-010

Therapeutic Modality Gene transfer

Target Gene MCO

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track, Orphan Drug Designation

NCT05956626 Active not recruiting Phase3, Phase2

View Report

Stargardt Disease

Sponsor Ocugen (Industry)

Compound OCU410ST

Therapeutic Modality Gene transfer

Target Gene RORA

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Orphan Drug Designation

NCT02725580 Completed Phase2, Phase1

View Report

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

Sponsor Emily de los Reyes (Other)

Compound AT-GTX-501

Therapeutic Modality Gene transfer

Target Gene CLN6

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06458595 Recruiting Phase2, Phase1

View Report

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

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

Compound KH658

Therapeutic Modality Gene transfer

Target Gene Anti-VEGF

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT04201405 Active not recruiting Phase2, Phase1

View Report

Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)

Sponsor University of Manchester (Other)

Compound OTL-201

Therapeutic Modality Gene transfer

Target Gene SGSH

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05243017 Active not recruiting Phase2, Phase1

View Report

Huntington's Disease

Sponsor UniQure Biopharma B.V. (Industry)

Compound AMT-130

Therapeutic Modality Gene transfer

Target Gene MiHTT

Vector Type AAV5

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

NCT02599922 Active not recruiting Phase2, Phase1

View Report

Achromatopsia

Sponsor Beacon Therapeutics (Industry)

Compound AGTC-401

Therapeutic Modality Gene transfer

Target Gene CNGB3

Vector Type AAV2tYF

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

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 LV

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

NCT06539208 Recruiting Phase2, Phase1

View Report

Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy

Sponsor YolTech Therapeutics Co., Ltd (Industry)

Compound YOLT-201

Therapeutic Modality Gene editing

Target Gene TTR

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Gene inactivation

NCT06300476 Active not recruiting Phase2, Phase1

View Report

Stargardt Disease

Sponsor West China Hospital (Other)

Compound JWK006

Therapeutic Modality Gene transfer

Target Gene ABCA4

Vector Type dual AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06948019 Not yet recruiting Phase2, Phase1

View Report

Hereditary Spastic Paraplegia Type 47

Sponsor BlackfinBio Ltd (Industry)

Compound BFB-101

Therapeutic Modality Gene transfer

Target Gene AP4B1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

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 ND1G3460A

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05230459 Recruiting Phase2, Phase1

View Report

Limb-Girdle Muscular Dystrophy, Type 2I/R9

Sponsor AskBio Inc (Industry)

Compound AB-1003

Therapeutic Modality Gene transfer

Target Gene FKRP

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, 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 AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04611503 Active not recruiting Phase2, Phase1

View Report

Retinitis Pigmentosa

Sponsor STZ eyetrial (Other)

Compound RAAV.hPDE6A

Therapeutic Modality Gene transfer

Target Gene PDE6A

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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 ND4G11778A

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

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 AAV-LK03

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations 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 AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT04517149 Active not recruiting Phase2, Phase1

View Report

X-Linked Retinitis Pigmentosa (XLRP)

Sponsor 4D Molecular Therapeutics (Industry)

Compound 4D-125

Therapeutic Modality Gene transfer

Target Gene RPGR

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT06492850 Recruiting 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 AAV5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT03173521 Completed Phase2, Phase1

View Report

Mucopolysaccharidosis Type VI

Sponsor Fondazione Telethon (Other)

Compound AAV2/8.TBG.hARSB

Therapeutic Modality Gene transfer

Target Gene ARSB

Vector Type AAV2/8

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 HIV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

NCT06550011 Active not recruiting Phase2, Phase1

View Report

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

Sponsor Avirmax Biopharma Inc (Industry)

Compound ABI-110

Therapeutic Modality Gene transfer

Target Gene VEGFtrap

Vector Type AAV.N54

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT02416622 Completed Phase2, Phase1

View Report

X-Linked Retinoschisis

Sponsor Beacon Therapeutics (Industry)

Compound RAAV2tYF-CB-hRS1

Therapeutic Modality Gene transfer

Target Gene RS1

Vector Type AAV2tYF

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT06467344 Recruiting Phase2, Phase1

View Report

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 AAV

Delivery System Viral transduction

Mechanism of Action Exon skipping/splice editor

FDA Designations Fast Track, Rare Pediatric Disease Designation

NCT05432310 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 LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

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

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 AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT02651675 Terminated Phase2, Phase1

View Report

Homozygous Familial Hypercholesterolemia (HoFH)

Sponsor REGENXBIO Inc. (Industry)

Compound RGX-501

Therapeutic Modality Gene transfer

Target Gene LDLR

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

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

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

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

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

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

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

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

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

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

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

SCGE Platform GTCT
Trial ID	Indication	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
NCT05788536	Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)	OTARMENI	Regeneron Pharmaceuticals	Industry	OTOF	Gene transfer	in vivo	Functional gene replacement	Intracochlear	Viral vector	Hair cell	Viral transduction	dual AAV1		7.2E12 vg/ear (one or both ears); concentration: 3E13 vg/ml	Phase2, Phase1	Recruiting	Approved	2023-03-15	2032-02-25	2026-07-01		36	16	Locations:United States + 4 more Germany, Japan, Spain, United Kingdom, United States		Regenerative Medicine Advanced Therapy (RMAT), Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	Otarmeni was approved 4/23/26 under the National Priority Voucher Program, Marketing Authorization Application was accepted for review by the EMA	Preclinical Publications : Functional, sustained recovery of hearing in Otoferlin-deficient mice using DB-OTO, a hair-cell-specific AAV-based gene therapy Recovery kinetics of dual AAV-mediated human otoferlin expression News and Press Releases : FDA Approval Documents Latest DB-OTO Results Demonstrate Clinically Meaningful Hearing Improvements in Nearly All Children with Profound Genetic Hearing Loss in CHORD Trial FDA Approves First-Ever Gene Therapy for Treatment of Genetic Hearing Loss Under National Priority Voucher Program Otarmeni™ (lunsotogene parvec) Receives EMA Filing Acceptance for Genetic Hearing Loss DB-OTO Results in the New England Journal of Medicine Showcase Dramatic and Sustained Improvements in Hearing and Speech Perception in Children with Profound Genetic Hearing Loss Latest DB-OTO Results Show Dramatically Improved Hearing to Normal Levels in a Child with Profound Genetic Deafness within 24 Weeks and Initial Hearing Improvements in a Second Child at 6 Weeks Clinical Publications : DB-OTO Gene Therapy for Inherited Deafness (Abstract #847) Advances in Gene Therapy for Profound Hearing Loss: An Update on the CHORD Phase 1/2 Trial - ASGCT 2025 Protocol : Clinical Trial Protocol
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		Endocytosis		ABE mRNA	Undisclosed dose based on body weight with potential for repeat dosing	Phase2, Phase1	Not yet recruiting	Active	2026-06-18	2028-07	2026-06-29	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 How to create personalized gene editing platforms: Next steps toward interventional genetics - this paper outlines the sponsor's thinking on creating umbrella clinical trials 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
NCT07592273	Diabetic Retinopathy	Surabgene lomparvovec	AbbVie	Industry	Anti-VEGF	Gene transfer	in vivo	Genetic delivery of therapeutic protein	Suprachoroidal	Viral vector		Viral transduction	AAV8		Dose 1: Phase 2 dose: 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	2026-07-01	>= 18 Years	576	11	Locations:United States + 1 more Puerto Rico, United States			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 Clinical Publications : (Presentation) Suprachoroidal Surabgene Lomparvovec (Sura-vec, ABBV-RGX-314) First Time 2-Year Results in NPDR -AAO 2025 Related NCTID :* Phase 2: NCT06942520 (uses subretinal injection) Phase 2: NCT04567550 Long Term Follow-Up: NCT05296447
NCT04713475	GM1 Gangliosidosis, Beta-Galactosidase-1 (GLB1) Deficiency	PBGM01	Gemma Biotherapeutics	Industry	GLB1	Gene transfer	in vivo	Functional gene replacement	Intracisternal	Viral vector		Viral transduction	AAVhu68		Dose 1: 3.3E10 GC/g brain weight \| Dose 2: 1.1E11 GC/g brain weight \| Dose 3: 2.2E11 GC/g brain weight \| Dose 4: Undisclosed dose 4	Phase2, Phase1	Active not recruiting	Active	2021-01-04	2029-02	2026-06-30	1 Month - 24 Months	26	7	Locations:United States + 3 more Brazil, Turkey (Türkiye), United Kingdom, United States			Out-licensed development to Gemma Biotherapeutics, uncertain development status	News and Press Releases : (Corporate Presentation) PBGM01 Study in Infantile GM1 Gangliosidosis Interim Clinical Results from Cohorts 1-4 and Program Update of Imagine-1 Clinical Study - August 2023 Passage Bio Out-licenses Three Pediatric Gene Therapy Programs to GEMMA Biotherapeutics and Enters New Research Collaboration
NCT05693142	Duchenne Muscular Dystrophy (DMD)	RGX-202	REGENXBIO Inc.	Industry	Micro-dystrophin	Gene transfer	in vivo	Overexpression of protective allele/gene	Intravenous	Viral vector		Viral transduction	AAV8		Dose 1: 1E14 GC/kg body weight \| Dose 2: 2E14 GC/kg body weight (pivotal dose)	Phase3, Phase2	Recruiting	Active	2023-01-04	2028-08	2026-06-30	>= 1 Year	65	17	Locations:United States + 1 more Canada, United States			Pivotal topline data announced May 2026, dosing completed in confirmatory study. Sponsor plans to pursue accelerated approval and submit a BLA in Q3 2026	News and Press Releases : REGENXBIO Highlights Key 2026 Catalysts and Announces Positive Long-Term Functional Outcomes in Lead Duchenne Gene Therapy Program REGENXBIO INITIATES PIVOTAL PHASE OF AFFINITY DUCHENNE TRIAL OF RGX-202 GENE THERAPY AND REPORTS POSITIVE FUNCTIONAL DATA REGENXBIO REPORTS NEW POSITIVE FUNCTIONAL DATA FROM PHASE I/II AFFINITY DUCHENNE® TRIAL OF RGX-202 REGENXBIO Completes Dosing in Confirmatory Study of RGX-202, Marking Completion of Registrational Development Program and Supporting Planned BLA Submission in Q3 2026 REGENXBIO Announces Positive Topline Results from Pivotal Phase III AFFINITY DUCHENNE® Study of RGX-202 (Corporate Presentation) RGX-202: AFFINITY DUCHENNE Pivotal Trial and Interim Functional Data Clinical Publications : (Corporate Presentation) RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data (Presentation) RGX-202, an investigational gene therapy for the treatment of Duchenne Muscular Dystrophy: Interim clinical data - MDA 2025 (Poster) RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data - WMS 2025 (Corporate Presentation) AFFINITY DUCHENNE® Pivotal Trial of RGX-202 Topline Data May 14, 2026 (Abstract #O75) RGX-202, an investigational gene therapy for the treatment of Duchenne Muscular Dystrophy: Interim clinical data - MDA 2025 Related NCTID :* Long Term Follow-Up: NCT06491927
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	VSV-G		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	2026-06-29	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 (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 (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 Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection 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
NCT06285643	Parkinson's Disease	AB-1005	AskBio Inc	Industry	GDNF	Gene transfer	in vivo	Overexpression of protective allele/gene	Intracerebral	Viral vector	Putamen	Viral transduction	AAV2		Up to 1.8mL (3.3E12 vg/ml/gadoteridol 2mM co-infusion)	Phase2	Recruiting	Active	2024-02-14	2028-08-31	2026-06-29	45 Years - 75 Years	127	46	Locations:United States + 3 more Germany, Poland, United Kingdom, United States		Regenerative Medicine Advanced Therapy (RMAT), Fast Track	AB-1005 is also being evaluated for individuals with the parkinsonian subtype of multiple system atrophy (MSA-P)	Grant : ZIA NS003051 F32 NS064692 U54 NS045309 CLIN2-11661 NIH/NHLBI K08 HL 146991 and NIH/NHLBI K08 HL 140078 Preclinical Publications : Functional effects of AAV2-GDNF on the dopaminergic nigrostriatal pathway in parkinsonian rhesus monkeys Anterograde axonal transport of AAV2-GDNF in rat basal ganglia Interventional MRI-guided putaminal delivery of AAV2-GDNF for a planned clinical trial in Parkinson's disease Evaluation of an AAV2-based rapamycin-regulated glial cell line-derived neurotrophic factor (GDNF) expression vector system Intermittent convection-enhanced delivery of GDNF into rhesus monkey putamen: absence of local or cerebellar toxicity Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF Safety evaluation of AAV2-GDNF gene transfer into the dopaminergic nigrostriatal pathway in aged and parkinsonian rhesus monkeys Safety Assessment of AAV2-hGDNF Administered Via Intracerebral Injection in Rats for Treatment of Parkinson's Disease Clinically relevant effects of convection-enhanced delivery of AAV2-GDNF on the dopaminergic nigrostriatal pathway in aged rhesus monkeys News and Press Releases : AskBio receives FDA Fast Track and MHRA Innovation Passport designations for AB-1005 investigational GDNF gene therapy for Parkinson's disease AskBio presents 18-month Phase Ib trial results of AB-1005 gene therapy for patients with Parkinson's disease First European Participants Randomized in AskBio Phase 2 Gene Therapy Trial of AB-1005 for Parkinson’s Disease AskBio Welcomes Brain Neurotherapy Bio to Expand Clinical Pipeline for Neurodegenerative Diseases AskBio's Viralgen to Supply Commercial-Scale Drug Product for REGENERATE-PD Investigational Gene Therapy Phase II Trial for Parkinson’s Disease Clinical Publications : Long-term safety of MRI-guided administration of AAV2-GDNF and gadoteridol in the putamen of individuals with Parkinson's disease Persistent GDNF Expression 45 Months after Putaminal Infusion of AAV2-GDNF in a Patient with Parkinson's Disease Trial of magnetic resonance-guided putaminal gene therapy for advanced Parkinson's disease Intraputaminal Delivery of Adeno-Associated Virus Serotype 2-Glial Cell Line-Derived Neurotrophic Factor in Mild or Moderate Parkinson's Disease (Abstract) Phase 1b Safety and Preliminary Efficacy of Bilateral Intraputaminal Delivery of AAV2 GDNF (AB-1005) in Participants With Mild or Moderate Parkinson's Disease - AAN Meeting, April 2024 Protocol : Clinical Trial Protocol Related NCTID : Phase 1: NCT04167540 Phase 1: NCT01621581 Phase 2: NCT07566429
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	AAV9		Undisclosed dose escalation, 2 levels	Phase2, Phase1	Recruiting	Active	2022-06-10	2033-01	2026-06-29	6 Months - 17 Years	47	13	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)
NCT01054339	Alpha-1 Antitrypsin Deficiency (AATD)	AGTC-0106	Beacon Therapeutics	Industry	SERPINA1	Gene transfer	in vivo	Functional gene replacement	Intramuscular	Viral vector		Viral transduction	AAV1		Dose 1: Early Phase 1(used AAV2): 2.1E12 vg, 6.9E12 vg, 2.1E13, 6.9E13; n=3/group \| Dose 2: Phase 1: 6.9E12 vg, 2.2E13 vg, 6.013 vg; n=3/group \| Dose 3: Phase 2: 6E11 vg/kg, 1.9E12 vg/kg, 6E12 vg/kg; n=3/group	Phase2	Completed	Inactive	2010-01-21	2015-10	2019-03-28	18 Years - 75 Years	9	4	Locations:United States + 1 more Ireland, United States			IM route resulted in poor efficacy, product development abandoned	Preclinical Publications : (Abstract #101) Toxicology and Biodistribution Studies of a Recombinant Adeno-Associated Virus 2 (rAAV2) Alpha-1 Antitrypsin (AAT) Vector - ASGCT 2004 Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector News and Press Releases : SEC Form 10-Q: Q1 2015 Clinical Publications : Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results Gene therapy for alpha-1 antitrypsin deficiency: an update Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy 5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression Related NCTID : Early Phase 1: NCT00377416 Phase 1: NCT00430768
NCT04124042	Osteoarthritis, Knee	XT-150	Xalud Therapeutics, Inc.	Industry	Recombinant IL10	Gene transfer	in vivo	Overexpression of protective allele/gene	Intra-articular	Naked plasmid					Dose 1: 150ug \| Dose 2: 450ug (extension study)	Phase2	Completed	Active	2019-10-09	2022-04-26	2025-03-27	45 Years - 85 Years	289	6	Locations:United States + 1 more Australia, United States		Fast Track		News and Press Releases : Xalud Therapeutics Receives FDA Fast Track Designation for XT-150 for the Treatment of Pain Associated with Osteoarthritis of the Knee Xalud Therapeutics Presents Clinical Data on XT-150 in Knee Osteoarthritis at the 2023 Osteoarthritis Research Society International World Congress Clinical Publications : (Abstract) XT-150- A novel immunomodulatory gene therapy for osteoarthritis pain in phase 2b development (Abstract LBA-17) XT-150- A NOVEL IMMUNOMODULATORY GENE THERAPY FOR OSTEOARTHRITIS PAIN IN PHASE 2B DEVELOPMENT Protocol : Clinical Trial Protocol Statistical Analysis Plan Related NCTID : Phase 1: NCT04466410 (Terminated trial for neuropathic pain) Phase 1: NCT03282149 Phase 1: NCT03477487
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		undisclosed base editor	>5E6 cells/kg	Phase2, Phase1	Enrolling by invitation	Active	2025-02-26	2034-12-31	2026-06-01	3 Years - 99 Years	18	1	United States
NCT06559176	Chronic Granulomatous Disease	PM359	Prime Medicine, Inc.	Industry	NCF1 (c.75_76delGT)	Gene editing	ex vivo	Mutation correction	Intravenous	Autologous cells	CD34+ cells	Electroporation		prime editor mRNA (encodes a SpCas9 nickase domain and a reverse-transcriptase domain)	Dose 1: Patient 1: 9.1E6 transduced CD34+ cells/kg \| Dose 2: Patient 2: 4.3E6 transduced CD34+ cells/kg	Phase2, Phase1	Enrolling by invitation	Active	2024-07-11	2030-02	2026-04-06	>= 6 Years	12	5	Locations:United States + 2 more Canada, United Kingdom, United States		Orphan Drug Designation, Rare Pediatric Disease Designation	Prime intends to submit BLA on the basis of the 2 patients treated, working on alignment with FDA	Grant : R00 HL163805 R01EB031172 R35 GM118062 U01AI142756 R01 GM065400 F32 GM 112366 RM1 HG009490 Preclinical Publications : Corporate Presentation JPM 2024 (Abstract) Prime Editing Efficiently and Precisely Corrects Causative Mutation in Chronic Granulomatous Disease, Restoring Myeloid Function: Toward Development of a Prime Edited Autologous Hematopoietic Stem Cell Therapy - ASH 2022 Efficient prime editing in mouse brain, liver and heart with dual AAVs Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage (Poster) Development of PM359, a Prime Edited CD34+ cell drug product for the treatment of p47phox Chronic Granulomatous Disease - ESGCT 2025 News and Press Releases : Prime Medicine Reports Full Year 2025 Financial Results and Provides Business Updates Prime Medicine Announces Breakthrough Clinical Data Showing Rapid Restoration of DHR Positivity After Single Infusion of PM359, an Investigational Prime Editor for Chronic Granulomatous Disease Prime Medicine Presents Preclinical Data Demonstrating Ability of PM359 to Efficiently, Reproducibly and Durably Correct Causative Mutation of Chronic Granulomatous Disease (CGD) Prime Medicine Reports Third Quarter 2024 Financial Results and Provides Business Updates Clinical Publications : Prime Editing for p47phox-Deficient Chronic Granulomatous Disease Protocol : Study Protocol (NEJM)
NCT03720418	Parkinson's Disease	OXB-102	Sio Gene Therapies	Industry	TH-GCH1.DDC (tricistronic)	Gene transfer	in vivo	Overexpression of protective allele/gene	Intraparenchymal	Viral vector	Putamen	Viral transduction	EIAV		Dose 1: 1.9E7 TU \| Dose 2: 4.0E7 TU \| Dose 3: 1E8 TU	Phase2, Phase1	Terminated	Inactive	2018-10-16	2022-04-12	2022-05-03	30 Years - 70 Years	6	3	Locations:France + 1 more France, United Kingdom			Axovant rebranded as Sio Gene Therapies, the company dissolved in 2023, returned rights to Oxford Biomedica which has shelved the program	Preclinical Publications : Gene Therapy for Parkinson's Disease: Preclinical Evaluation of Optimally Configured TH:CH1 Fusion for Maximal Dopamine Synthesis Optimizing Transgene Configuration and Protein Fusions to Maximize Dopamine Production for the Gene Therapy of Parkinson's Disease Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease News and Press Releases : Sio Gene Therapies to dissolve after years of setbacks Oxford Biomedica announces update to agreement with Sio Gene Therapies Clinical Publications : Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial Long-Term Follow-Up of a Phase I/II Study of ProSavin, a Lentiviral Vector Gene Therapy for Parkinson's Disease Related NCTID : Long Term Follow-Up: NCT01856439 Phase 1/2: NCT00627588
NCT06063850	Mesial Temporal Lobe Epilepsy	AMT-260	UniQure Biopharma B.V.	Industry	MiGRIK2	Gene transfer	in vivo	MiRNA knockdown of mutant/aberrant gene	Intraparenchymal	Viral vector		Viral transduction	AAV9		Dose 1: 1E12 gc/ml \| Dose 2: 3E12 gc/ml	Phase2, Phase1	Recruiting	Active	2023-09-01	2031-12	2026-06-02	18 Years - 75 Years	12	18	United States			Preliminary data on first (low dose) cohort released June 2026, enrollment is ongoing in the second (high dose) cohort and is expected to be completed mid-2026	Preclinical Publications : GluK2 Is a Target for Gene Therapy in Drug-Resistant Temporal Lobe Epilepsy (Abstract #P074) CL002, An AAV9 vector expressing engineered miRNA targeting knockdown of GluK2-containing kainate receptors as a novel gene therapy approach for treating intractable temporal lobe epilepsy - ESGCT 2021 A novel AAV9-dual microRNA-vector targeting GRIK2 in the hippocampus as a treatment for mesial temporal lobe epilepsy News and Press Releases : uniQure Announces Preliminary Data on the First Cohort in the Phase I/IIa Clinical Trial of AMT-260 in Refractory Mesial Temporal Lobe Epilepsy uniQure Presents Clinical Case Study of First Patient Dosed with AMT-260 in Refractory Mesial Temporal Lobe Epilepsy (MTLE) SEC Form 10-Q: uniQure N.V. 3Q25 uniQure Announces Dosing of First Patient in GenTLE Phase I/IIa Clinical Trial of AMT-260 for the Treatment of Refractory Mesial Temporal Lobe Epilepsy
NCT03566043	Mucopolysaccharidosis Type II (Hunter Syndrome)	NAVSUNLI	REGENXBIO Inc.	Industry	IDS	Gene transfer	in vivo	Functional gene replacement	Intracisternal	Viral vector		Viral transduction	AAV9		Dose 1: 1.3E10 GC/g brain mass (n=3) \| Dose 2: 6.5E10 GC/g brain mass (n=7) \| Dose 3: Pivotal dose: 2.9E11 GC/g brain mass (n=15)	Phase3, Phase2	Active not recruiting	Active	2018-05-01	2025-08	2025-01-28	4 Months - 5 Years	48	5	Locations:United States + 1 more Brazil, United States		Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	CRL issued 2/7/26 (issues with study eligibility criteria, comparability of natural history control arm, appropriateness of surrogate endpoint), Company appealed this decision and reached alignment with FDA, plans to resubmit the BLA in Q3 2026, which FDA has agreed to review on an expedited basis	Preclinical Publications : Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction News and Press Releases : REGENXBIO Announces Alignment with FDA on Path Forward for NAVSUNLI™ BLA Resubmission for Accelerated Approval; First Potential Gene Therapy for MPS II REGENXBIO Announces Regulatory Update on Ultra Rare MPS Programs REGENXBIO Announces Regulatory Update on RGX-121 BLA for MPS II REGENXBIO Announces Successful Pre-BLA Meeting with FDA to Support Accelerated Approval Pathway for RGX-121 for the Treatment of MPS II REGENXBIO Reports Third Quarter 2024 Financial Results and Recent Operational Updates REGENXBIO Announces Closing of Strategic Partnership with Nippon Shinyaku for MPS Diseases REGENXBIO Presents Positive Twelve-Month Pivotal Data from Phase I/II/III CAMPSIITE® Trial of RGX-121 for Treatment of MPS II Clinical Publications : (Poster) Phase I/II/III: Interim Clinical Update of RGX-121, an Investigational Gene Therapy for Treatment of Neuronopathic Mucopolysaccharidosis Type II (MPS II) - September 2024 (Presentation) CAMPSIITE™ Phase I/II/III: Interim clinical update of clemidsogene lanparvovec (RGX-121), an investigational gene therapy for treatment of neuronopathic mucopolysaccharidosis type II (MPS II) - ICIEM 2025 (Corporate Presentation) Rare Program Update - February 2024 Related NCTID : Phase 1/2: NCT04571970 Phase 3: NCT07236606
NCT07226206	Hemophilia A	SPK-8011QQ	Hoffmann-La Roche	Industry	F8	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	LK03		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	2031-07-31	2026-06-23	>= 18 Years	5	2	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 1/2: NCT03003533 Phase 3: NCT06297486 (Withdrawn) Long Term Follow-Up: NCT03432520
NCT03217617	X-linked Severe Combined Immunodeficiency (XSCID)	Ivlv-X1 lentiviral vector	Shenzhen Geno-Immune Medical Institute	Other	IL2RG	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	LV		1E9 vg/kg	Phase2, Phase1	Recruiting	Active	2017-07-03	2027-12-31	2026-06-23	1 Month - 1 Year	10	1	China
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		Viral transduction	AAV5.2		Undisclosed dose escalation, 2 levels	Phase2, Phase1	Recruiting	Active	2025-11-19	2027-10-15	2026-06-23	>= 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
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	VSV-G		Transduced CD34+ cells (6.5-32.6E6 cells/kg)	Phase2, Phase1	Enrolling by invitation	Inactive	2025-08-08	2029-09-01	2026-06-22	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 Announces FDA Granted Orphan Drug Designation for OTL-102 for the Treatment of X-linked Chronic Granulomatous Disease (X-CGD) Orchard Therapeutics Extends Runway into 2024, Focusing HSC Gene Therapy Platform Exclusively on Severe Neurometabolic Diseases and Research Platform 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
NCT05836259	Hypertrophic Cardiomyopathy	TN-201	Tenaya Therapeutics	Industry	MYBPC3	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV9		Dose 1: Dose escalation: 3E13 vg/kg, 6E13 vg/kg; n=3/group \| Dose 2: Dose expansion: 6E13 vg/kg	Phase2, Phase1	Recruiting	Active	2023-04-18	2032-08	2026-03-19	18 Years - 75 Years	30	10	United States		Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	Interim results announced June 2026	Preclinical Publications : (Poster) MyPeak-1: A Phase 1b Study to Evaluate Safety and Efficacy of TN-201, an Adeno-Associated Virus Serotype 9 (AAV9) Investigational Gene Therapy, in Adults with MYBPC3-Associated Hypertrophic Cardiomyopathy (HCM) - HCMS 2023 AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy models (Poster) Low Seroprevalence of Neutralizing Antibodies to Adeno-Associated Virus Serotype 9 (AAV-9) in Preparation for MyPeak-1, the First-in-Human Study of TN-201, an Investigational AAV9-Mediated Gene Therapy for Individuals with MYBPC3-Associated Hypertrophic Cardiomyopathy (HCM) - HCMS 2023 News and Press Releases : Tenaya Therapeutics Announces Interim Data from MyPEAK™-1 Showing Treatment of MYBPC3-associated HCM with TN-201 Gene Therapy Resulted in Consistent Signs of Cardiac Remodeling and Reductions in Symptoms Tenaya Therapeutics Reports Promising Early Data from MyPEAK Phase 1b/2 Clinical Trial of TN-201 for Treatment of MYBPC3-Associated Hypertrophic Cardiomyopathy Tenaya Therapeutics Announces 2025 Strategic Priorities and Anticipated Milestones Tenaya Therapeutics Announces Late Breaker Presentation of New Data from MyPEAK™-1 Phase 1b/2 Clinical Trial of TN-201 at American College of Cardiology Annual Meeting Tenaya Therapeutics Announces Updates on TN-201 Gene Therapy Program for MYBPC3-associated HCM Clinical Publications : (Presentation) Early experience optimizing immunosuppressive use and monitoring in MyPEAKTM-1, a first-in-human study of TN-201, an AAV9 gene replacement therapy in MYBPC3-associated hypertrophic cardiomyopathy - European Society of Cardiology Heart Failure Congress 2026 High rate of seroeligibility among MYBPC3-associated hypertrophic cardiomyopathy patients for TN-201, an adeno-associated virus serotype 9 MYBPC3 gene therapy (Presentation) INTERIM DATA FROM MYPEAK-1, A PH1B/2A STUDY OF TN-201 GENE THERAPY FOR MYBPC3-ASSOCIATED HCM - AHA Scientific Sessions 2025 First-in-human study of TN-201, an AAV9 gene replacement therapy in MYBPC3-associated hypertrophic cardiomyopathy: initial safety, pharmacodynamic, and imaging results from MyPEAK-1
NCT01496040	Leber Congenital Amaurosis	RAAV2/4.hRPE65	Nantes University Hospital	Other	RPE65	Gene transfer	in vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2/4		Dose range: 1.22E10 -4.8E10 vg (n=9)	Phase2, Phase1	Completed	Inactive	2011-11-24	2014-08	2015-10-07	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 Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium 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 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
NCT04537377	Wilson Disease	VTX-801	Vivet Therapeutics SAS	Industry	ATP7B-minigene	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	Liver	Viral transduction	AAV8		Undisclosed dose escalation, 3 levels	Phase2, Phase1	Active not recruiting	Inactive	2020-08-19	2029-06-18	2026-01-30	18 Years - 65 Years	4	10	Locations:United States + 3 more Denmark, Germany, United Kingdom, United States		Fast Track, Orphan Drug Designation	Sponsor terminated the trial due to efficacy concerns at the two doses tested, insufficient funding for further dose escalation	Preclinical Publications : Long-term metabolic correction of Wilson's disease in a murine model by gene therapy High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson's disease Liver Expression of a MiniATP7B Gene Results in Long-Term Restoration of Copper Homeostasis in a Wilson Disease Model in Mice News and Press Releases : VTX-801 Receives U.S. FDA Fast Track Designation for the Treatment of Wilson Disease Vivet Therapeutics discontinues VTX-801 in October 2024
NCT01801709	Metachromatic Leukodystrophy	AAVrh.10-CAG/cuARSA	Institut National de la Santé Et de la Recherche Médicale, France	Other gov	ARSA	Gene transfer	in vivo	Functional gene replacement	Intracerebral	Viral vector		Viral transduction	AAVrh.10		Dose 1: 1E12 vg \| Dose 2: 4E12 vg	Phase2, Phase1	Completed	Inactive	2013-01-28	2022-12-20	2026-03-02	6 Months - 5 Years	5	1	France			INSERM is developing a new therapy with a different AAV (PMID: 34093126)	Preclinical Publications : Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice
NCT03041324	Mucopolysaccharidosis Type II (Hunter Syndrome)	SB-913	Sangamo Therapeutics	Industry	IDS	Gene editing	in vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV2/6	ZFN	Dose 1: 5E12 vg/kg (n=2) \| Dose 2: 1E13 vg/kg (n=2) \| Dose 3: 5E13 vg/kg (n=5)	Phase2, Phase1	Terminated	Inactive	2017-01-13	2021-05-07	2022-10-25	5 Years - 65 Years	9	5	United States		Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	Sangamo announced they had stopped development in 2022	Preclinical Publications : Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing In vivo genome editing of the albumin locus as a platform for protein replacement therapy News and Press Releases : Sangamo Announces Interim Results Of Phase 1/2 CHAMPIONS Study Showing Preliminary Evidence Of In Vivo Genome Editing In Patients With MPS II Treated With SB-913 SEC Form 10-K: SANGAMO THERAPEUTICS, INC. FY2022 Sangamo Receives Fast Track Designation From The FDA For SB-318 And SB-913 In Vivo Genome Editing Product Candidates For The Treatment Of MPS I And MPS II Clinical Publications : First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B Protocol : Clinical Trial Protocol Statistical Analysis Plan Related NCTID : Long Term Follow-Up: NCT04628871
NCT03300453	Sanfilippo Syndrome B	AMT-110	UniQure Biopharma B.V.	Industry	NAGLU	Gene transfer	in vivo	Functional gene replacement	Intraparenchymal	Viral vector		Viral transduction	AAV2/5		Dose 1: Total dose (16 sites): 4E12 vg \| Dose 2: Per site: 2.4E11 vg (60ul)	Phase2, Phase1	Completed	Inactive	2016-06-15	2019-11-27	2019-12-02	18 Months - 60 Months	4	1	France			UniQure announced discontinuation in November 2016	Preclinical Publications : Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes News and Press Releases : uniQure Completes Strategic Review to Refocus its Pipeline, Reduce Operating Costs and Deliver Long-Term Shareholder Value Clinical Publications : Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome Intracerebral Gene Therapy in Four Children with Sanfilippo B Syndrome: 5.5-Year Follow-Up Results Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial
NCT06141460	Neovascular (Wet) Age-Related Macular Degeneration (nAMD)	RRG001	Shanghai Refreshgene Technology Co., Ltd.	Industry	Anti-VEGF	Gene transfer	in vivo	Genetic delivery of therapeutic protein	Subretinal	Viral vector		Viral transduction	AAV		Undisclosed dose escalation, 4 levels	Phase2, Phase1	Recruiting	Active	2023-11-10	2030-12-31	2024-11-14	>= 50 Years	48	1	China
NCT06255782	Ornithine Transcarbamylase (OTC) Deficiency	ECUR-506	iECURE, Inc.	Industry	OTC/PCSK9 locus	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	Hepatocyte	Viral transduction	dual AAV8	ARCUS	Dose 1: 1.3E13 GC/kg \| Dose 2: Undisclosed high dose	Phase2, Phase1	Recruiting	Active	2023-12-19	2027-12	2026-04-15	24 Hours - 7 Months	20	12	Locations:United States + 3 more Australia, Spain, United Kingdom, United States		Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Fast Track	Sponsor reports complete clinical response in first infant dosed, more data expected in the first half of 2026; ECUR-506 was selected to participate in the FDA's Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program	Preclinical Publications : A mutation-independent CRISPR-Cas9-mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice (Whitepaper) ARCUS Shows Promise for In Vivo and Ex Vivo Therapeutics News and Press Releases : iECURE Reports Complete Clinical Response in First Infant Dosed with its In Vivo Gene Editing Candidate ECUR-506 in an Ongoing Phase 1/2 Clinical Trial in Ornithine Transcarbamylase (OTC) Deficiency iECURE Receives FDA Fast Track Designation for ECUR-506 for the Treatment of Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency iECURE Announces Presentation of Full Data for the First Infant Dosed with ECUR-506 in OTC-HOPE Phase 1/2 Clinical Trial at the 2025 ACMG Annual Clinical Genetics Meeting iECURE Selected for U.S. FDA Chemistry, Manufacturing, and Controls Development and Readiness Pilot Program to Support Manufacturing Readiness of ECUR-506 Ahead of Planned BLA Submission iECURE Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ECUR-506 for Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency Clinical Publications : (Poster) Initial clinical results from OTC-HOPE, the first in vivo, liver directed, AAV-mediated gene insertion study in neonatal OTC deficiency; complete clinical response observed in first male infant to receive ECUR-506 and complete 24-week Phase 1/2 study - ASGCT 2025 (Poster) Initial clinical results from OTC-HOPE, the first in vivo, liver directed, AAV-mediated gene insertion study in neonatal OTC deficiency; complete clinical response observed in first male infant to receive ECUR-506: 10-month data - ASHG 2025
NCT06480461	Parkinson's Disease	VGN-R09b	Shanghai Vitalgen BioPharma Co., Ltd.	Industry	DDC	Gene transfer	in vivo	Overexpression of protective allele/gene	Intraparenchymal	Viral vector	Striatum	Viral transduction	AAV9		Dose 1: 8.0E11 vg \| Dose 2: 1.6E12 vg \| Dose 3: 3.2E12 vg	Phase2, Phase1	Active not recruiting	Active	2024-06-14	2030-09-10	2026-03-31	40 Years - 75 Years	39	1	China		Fast Track	IND cleared in July 2024	News and Press Releases : Vitalgen's Gene Therapy Drug VGN-R09b for Primary Parkinson's Disease Receives FDA Fast Track Designation VGN-R09b, China's First Domestically Developed Gene Therapy Drug for the Treatment of Primary Parkinson's Disease, Approved for Clinical Trials in the United States Clinical Publications : (Abstract) VGN-R09b, an Adeno-associated virus-based Combination Gene Replacement Therapy for Parkinson’s Disease - MDS 2025
NCT04127578	Parkinson's Disease	PR001	Prevail Therapeutics	Industry	GBA1	Gene transfer	in vivo	Overexpression of protective allele/gene	Intracisternal	Viral vector		Viral transduction	AAV9		Undisclosed dose escalation, 2 levels	Phase2, Phase1	Active not recruiting	Active	2019-10-14	2030-12-31	2026-03-13	35 Years - 80 Years	32	15	Locations:United States + 1 more Israel, United States		Fast Track		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
NCT04480567	Phenylketonuria (PKU)	BMN 307	BioMarin Pharmaceutical	Industry	PAH	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV5		Dose escalation with 3 levels, undisclosed concentrations	Phase2, Phase1	Active not recruiting	Inactive	2020-07-08	2027-12	2024-12-12	>= 15 Years	100	3	Locations:United States + 1 more United Kingdom, United States		Fast Track, Orphan Drug Designation	BioMarin (developer of pegvaliase) has made no official announcement of discontinuation. Clinical hold was placed when mice in preclinical studies developed tumors, and Biomarin was sued on allegations that Biomarin had overstated BMN 307's clinical and commercial prospects.	News and Press Releases : SEC Form 10-K: BioMarin Pharmaceutical Inc. FY2022 SEC Form 10-K: BioMarin Pharmaceutical Inc. FY2021 BioMarin Provides Updates on Progress in Gene Therapy Programs SEC Form 10-K: BioMarin Pharmaceutical Inc. FY2020
NCT05641610	Hemophilia B	ZS801	Institute of Hematology & Blood Diseases Hospital, China	Other	F9	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV		Dose 1: 2.0E12 vg/kg \| Dose 2: 5.0E12 vg/kg \| Dose 3: 1.0E13 vg/kg	Phase2, Phase1	Recruiting	Active	2022-11-25	2028-12	2025-02-24	>= 18 Years	21	1	China				Related NCTID : Phase Not Applicable: NCT05630651
NCT03199469	X-Linked Myotubular Myopathy	AT132	Astellas Gene Therapies	Industry	MTM1	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV8		Dose 1: 1.3E14 vg/kg (n=7) \| Dose 2: 3.5E14 vg/kg (n=20)	Phase3, Phase2	Active not recruiting	Inactive	2017-06-21	2030-03-31	2026-04-08	<= 5 Years	27	6	Locations:United States + 3 more Canada, France, Germany, United States		Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	Astellas has restarted their XLMTM program by creating a new drug product ASP2957, which is based on this prior study, with substantial modifications to the capsid to detarget the liver	Grant : R21 AR064503 R01 AR044345 P50 NS040828 R01 HL115001 Preclinical Publications : Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy rAAV-related therapy fully rescues myonuclear and myofilament function in X-linked myotubular myopathy Long-term effects of systemic gene therapy in a canine model of myotubular myopathy Loss of Mtm1 causes cholestatic liver disease in a model of X-linked myotubular myopathy Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs AAV8 gene therapy and dietary insults together precipitate cholestatic liver disease in a mouse model of X-linked myotubular myopathy Ultrasound assessment of the diaphragm: Preliminary study of a canine model of X-linked myotubular myopathy News and Press Releases : Notice Regarding Impairment Loss for Products under Development Astellas Sees Path Forward for Gene Therapy Despite Four Patient Deaths SEC Form 10-K: Audentes Therapeutics, Inc. FY2018 Clinical Publications : High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathy Hepatobiliary disease in XLMTM: a common comorbidity with potential impact on treatment strategies Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial Intrahepatic Cholestasis Is a Clinically Significant Feature Associated with Natural History of X-Linked Myotubular Myopathy (XLMTM): A Case Series and Biopsy Report Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
NCT06611436	Hemophilia B	BE-101	Be Biopharma	Industry	F9	Gene editing	ex vivo	Functional gene replacement	Intravenous	Autologous cells	B cells	Viral transduction	AAV6	Cas9 RNP	Undisclosed dose escalation, 3 levels	Phase2, Phase1	Terminated	Active	2024-09-18	2026-05-29	2026-06-15	>= 18 Years	5	5	United States		Fast Track, Orphan Drug Designation	First patient dosed July 2025	Preclinical Publications : (Presentation) Development of an Ex Vivo Precision Gene Engineered B Cell Medicine that Produces Active and Sustained Levels of FIX for the Treatment of Hemophilia B - ASH 2023 News and Press Releases : FDA Grants Orphan Drug Designation for BE-101, a Novel Engineered B Cell Medicine, for the Treatment of Hemophilia B Be Bio Announces First Participant Dosed in BeCoMe-9 Trial of BE-101 in Hemophilia B Protocol : (Poster) BeCoMe-9: A Phase 1/2 Dose Escalation and Expansion Study of BE-101 for the Treatment of Adults with Moderately Severe or Severe Hemophilia B - ASH 2024
NCT00598481	Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)	STRIMVELIS	Fondazione Telethon	Other	ADA	Gene transfer	ex vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	MoMLV (RV)		Dose 1: Median dose: 11.6E6 CD34+ cells/kg (19 patients) \| Dose 2: Median dose: 9.2E6 CD34+ cells/kg (22 patients)	Phase2	Completed	Approved	2008-01-10	2019-06-19	2024-01-29	<= 17 Years	12	2	Locations:Israel + 1 more Israel, Italy			Product was initially developed by SR-TIGET, in-licensed by GlaxoSmithKline in 2010. EMA approval in May 2016. GSK sold Strimvelis in March 2018 to Orchard Therapeutics. Orchard discontinued the program in March 2022. Product license was transferred to Fondazione Telethon in July 2023	Preclinical Publications : Good Laboratory Practice Preclinical Safety Studies for GSK2696273 (MLV Vector-Based Ex Vivo Gene Therapy for Adenosine Deaminase Deficiency Severe Combined Immunodeficiency) in NSG Mice An in vivo model of somatic cell gene therapy for human severe combined immunodeficiency Alterations in the adenosine metabolism and CD39/CD73 adenosinergic machinery cause loss of Treg cell function and autoimmunity in ADA-deficient SCID News and Press Releases : SEC Form 10-Q: Orchard Therapeutics PLC 3Q2023 Clinical Publications : Gene therapy for immunodeficiency due to adenosine deaminase deficiency Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients Altered intracellular and extracellular signaling leads to impaired T-cell functions in ADA-SCID patients ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiency Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID Related NCTID : Phase 1: NCT00018018 Phase 1/2: NCT00599781 Long Term Follow-Up: NCT03478670
NCT05353647	Sickle Cell Disease	Autologous CD34+ HSC cells transduced with the lentiviral vector containing BCL11a-targeted shRNA	David Williams	Other	BCL11A	Gene transfer	ex vivo	Overexpression of protective allele/gene	Intravenous	Autologous cells	CD34+ cells	Viral transduction	VSV-G		Transduced CD34+ cells	Phase2	Active not recruiting	Active	2022-04-21	2027-10	2026-05-12	13 Years - 40 Years	25	9	United States				Grant : CLIN2SCD-12031 R01 HL137848 Preclinical Publications : miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy Clinical Publications : Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease Protocol : Clinical Trial Protocol Related NCTID : Phase 1: NCT03282656
NCT03580083	Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome	RGX-111	REGENXBIO Inc.	Industry	IDUA	Gene transfer	in vivo	Functional gene replacement	Intrathecal	Viral vector		Viral transduction	AAV2/9		Dose 1: Single Patient IND: 1E10 GC/g brain mass (n=1) \| Dose 2: 1E10 GC/g brain mass (n=2) \| Dose 3: 5E10 GC/g brain mass (n=6)	Phase2, Phase1	Suspended	Active	2018-06-18	2029-03-06	2026-03-12	>= 4 Months	21	2	Locations:United States + 1 more Brazil, United States		Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	FDA placed clinical hold in January 2026 following preliminary analysis of a CNS tumor in a five-year-old patient treated in this study four years ago	Preclinical Publications : Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10 Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer News and Press Releases : REGENXBIO Announces Regulatory Update on RGX-121 BLA for MPS II REGENXBIO Announces Regulatory Update on Ultra Rare MPS Programs REGENXBIO Announces Closing of Strategic Partnership with Nippon Shinyaku for MPS Diseases REGENXBIO Announces Completion of Dosing in the Phase I/II Trial of RGX-111 for the Treatment of Severe MPS I Clinical Publications : Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery (Presentation) RGX111 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type I (MPS I): Interim Analysis of the First in Human Study and a Single Patient IND - WORLDSymposium 2023 Related NCTID : Long Term Follow-Up: NCT06103487
NCT04581785	Methylmalonic Acidemia	LB-001	LogicBio Therapeutics, Inc	Industry	MMUT	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector	Hepatocyte	Viral transduction	AAV-LK03		Dose 1: 5E13 vg/kg \| Dose 2: 1E14 vg/kg	Phase2, Phase1	Terminated	Inactive	2020-10-02	2023-01-10	2024-02-23	6 Months - 12 Years	4	4	United States		Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation	Program was terminated 4/25/23 due to low likelihood of clinical benefit	Preclinical Publications : Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing News and Press Releases : LogicBio Therapeutics Reports Second Quarter 2022 Financial Results and Provides Corporate Update SEC Form 10-Q: LogicBio Therapeutics, Inc. 3Q2022 Clinical Publications : First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study Protocol : Statistical Analysis Plan Clinical Trial Protocol Related NCTID : Long Term Follow-Up: NCT05506254
NCT06422351	Pyruvate Kinase Deficiency	RP-L301	Rocket Pharmaceuticals Inc.	Industry	PKLR	Gene transfer	ex vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	LV		Dose range: 2.3E6 - 6.5E6 transduced CD34+ cells/kg	Phase2	Suspended	Inactive	2024-05-15	2029-01	2026-04-16	8 Years - 55 Years	10	3	Locations:United States + 1 more Spain, United States		Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation	Sponsor is no longer allocating internal resources towards RP-L301 and does not plan to initiate enrollment in Phase 2 study, seeking external partnership opportunities	Preclinical Publications : Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency News and Press Releases : Rocket Pharmaceuticals Reports Third Quarter 2024 Financial Results and Highlights Recent Progress SEC Form 10-Q: Rocket Pharmaceuticals, Inc. 3Q2025 Clinical Publications : (Abstract #4) Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RPL301 - ASGCT 2024 Protocol : (Abstract) RP-L301, a Lentiviral-Mediated Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase 2 Clinical Trial Design - ASH 2024 Related NCTID : Phase 1: NCT04105166
NCT06332807	Phenylketonuria (PKU)	NGGT002	NGGT INC.	Industry	PAH	Gene transfer	in vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV8		Dose 1: 6.0E12 vg/kg \| Dose 2: 1.0E13 vg/kg \| Dose 3: 2.0E13 vg/kg	Phase2, Phase1	Recruiting	Active	2024-02-07	2030-12-30	2025-12-02	18 Years - 55 Years	12	5	United States		Orphan Drug Designation	Recieved Orphan Drug Designation from the FDA in Jan 2023	News and Press Releases : Next Generation Gene Therapeutics (NGGT) Announces Positive New Data on NGGT002 for the Treatment of Phenylketonuria (PKU) Clinical Publications : (Abstract #1410) Safety and Efficacy of NGGT002 (rAAV8hPAH) in Adults with Phenylketonuria (PKU): Results from an Investigator Initiated Study - ASGCT 2024 Related NCTID : Early Phase 1: NCT06061614

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