Contact SCGE




Clinical Trials - Gene Therapy Trial Browser

The Gene Therapy Trial Browser represents a unique publicly accessible, free database for the benefit of users seeking information on gene therapy development. The information within integrates various sources, including clinicaltrials.gov, publications, sponsor press releases, meeting abstracts, and more to give a comprehensive overview of the gene therapy clinical trial landscape.

Showing 1-100 of 392 results in ClinicalTrials
NCT07681778 Not yet recruiting Phase2, Phase1
View Report
Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA), LCA5
Sponsor Opus Genetics, Inc (Industry)
Compound OPGx-RDH12
Therapeutic Modality Gene transfer
Target Gene RDH12
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2026-06-24 Last Modified Date: 1784091600000 Enrollment: 10 United States
NCT07052929 Recruiting Phase2, Phase1
View Report
X-Linked Myotubular Myopathy
Sponsor Astellas Gene Therapies (Industry)
Compound ASP2957
Therapeutic Modality Gene transfer
Target Gene MTM1
Vector Type AAV3.8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-06-30 Last Modified Date: 1784091600000 Enrollment: 9 Canada, United States
NCT07592273 Recruiting Phase3, Phase2
View Report
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
First Submitted: 2026-04-24 Last Modified Date: 1784091600000 Enrollment: 576 Puerto Rico, United States
NCT07226206 Recruiting Phase2, Phase1
View Report
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
First Submitted: 2025-10-28 Last Modified Date: 1784005200000 Enrollment: 5 United States
NCT02852213 Recruiting Phase1
View Report
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor Krzysztof Bankiewicz (Other)
Compound AAV2-hAADC
Therapeutic Modality Gene transfer
Target Gene DDC
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-07-20 Last Modified Date: 1783918800000 Enrollment: 42 United States
NCT07223944 Recruiting Phase3
View Report
Gaucher Disease Type 1
Sponsor Spur Therapeutics (Industry)
Compound FLT201
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type AAVS3
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-10-30 Last Modified Date: 1783918800000 Enrollment: 45 Argentina, Australia, Brazil + 7 more
NCT03818763 Active not recruiting Phase1
View Report
Hemophilia A
Sponsor Medical College of Wisconsin (Other)
Compound Pleightlet
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type VSV-G
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2019-01-15 Last Modified Date: 1783918800000 Enrollment: 5 United States
NCT06164730 Recruiting Phase1
View Report
Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-102
Therapeutic Modality Gene editing
Target Gene PCSK9
Vector Type LDLR + GalNAc
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Fast Track
First Submitted: 2023-12-01 Last Modified Date: 1783918800000 Enrollment: 85 Australia, Canada, Israel + 3 more
NCT04797260 Suspended Phase2, Phase1
View Report
Severe Combined Immunodeficiency Due to RAG1 Deficiency
Sponsor Genewity Holding B.V. (Industry)
Compound Autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector
Therapeutic Modality Gene transfer
Target Gene RAG1
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-03-11 Last Modified Date: 1783918800000 Enrollment: 10 Netherlands, Poland, Spain + 1 more
NCT06325709 Recruiting Phase2, Phase1
View Report
X-Linked Chronic Granulomatous Disease
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound Base-edited autologous CD34+ cells
Therapeutic Modality Gene editing
Target Gene CYBB c.676 C>T
Delivery System Electroporation
Mechanism of Action Mutation correction
First Submitted: 2024-03-21 Last Modified Date: 1783659600000 Enrollment: 10 United States
NCT05040217 Active not recruiting Phase1
View Report
Alzheimer's Disease, Mild Cognitive Impairment
Sponsor Mark Tuszynski (Other)
Compound AAV2-BDNF
Therapeutic Modality Gene transfer
Target Gene BDNF
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2021-06-21 Last Modified Date: 1783659600000 Enrollment: 12 United States
NCT06199531 Active not recruiting Phase3
View Report
NGLY1 Deficiency
Sponsor Grace Science, LLC (Industry)
Compound GS-100
Therapeutic Modality Gene transfer
Target Gene NGLY1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-11-21 Last Modified Date: 1783659600000 Enrollment: 10 United States
NCT05419492 Recruiting Phase2, Phase1
View Report
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
First Submitted: 2022-06-10 Last Modified Date: 1783573200000 Enrollment: 47 Australia, United Kingdom, United States
NCT05616793 Recruiting Phase2, Phase1
View Report
Leber Congenital Amaurosis-Type 5
Sponsor Opus Genetics, Inc (Industry)
Compound OPGx-001
Therapeutic Modality Gene transfer
Target Gene LCA5
Vector Type 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
First Submitted: 2022-11-07 Last Modified Date: 1783486800000 Enrollment: 22 United States
NCT06856577 Active not recruiting Phase3
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Adverum Biotechnologies, Inc. (Industry)
Compound ADVM-022
Therapeutic Modality Gene transfer
Target Gene Codon optimized aflibercept
Vector Type AAV.7m8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2025-02-26 Last Modified Date: 1783486800000 Enrollment: 311 United States
NCT07267065 Not yet recruiting Phase1
View Report
Parkinson's Disease (PD), Early Onset Parkinson Disease
Sponsor Krzysztof Bankiewicz (Other)
Compound AAV2-hAADC
Therapeutic Modality Gene transfer
Target Gene DDC
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2025-11-24 Last Modified Date: 1783486800000 Enrollment: 9 United States
NCT01306019 Recruiting Phase2, Phase1
View Report
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound VSV-G pseudotyped CL20-i4-EF1α-hγc-OPT vector
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
First Submitted: 2011-02-26 Last Modified Date: 1783486800000 Enrollment: 40 United States
NCT07543991 Recruiting Phase1
View Report
Giant Axonal Neuropathy (GAN)
Sponsor University of Texas Southwestern Medical Center (Other)
Compound ScAAV9/JeT-GAN
Therapeutic Modality Gene transfer
Target Gene GAN
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2026-03-12 Last Modified Date: 1783486800000 Enrollment: 4 United States
NCT07587021 Not yet recruiting Phase2
View Report
Primary Hyperoxaluria Type 1, PH1
Sponsor YolTech Therapeutics Co., Ltd (Industry)
Compound YOLT-203
Therapeutic Modality Gene editing
Target Gene HAO1
Vector Type LNP
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2026-05-01 Last Modified Date: 1783486800000 Enrollment: 36
NCT04418427 Completed Phase2
View Report
Diabetic Macular Edema, Diabetic Retinopathy
Sponsor Adverum Biotechnologies, Inc. (Industry)
Compound Ixoberogene soroparvovec
Therapeutic Modality Gene transfer
Target Gene Codon optimized aflibercept
Vector Type AAV.7m8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2020-05-28 Last Modified Date: 1783486800000 Enrollment: 36 Puerto Rico, United States
NCT06370351 Active not recruiting Phase2, Phase1
View Report
OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders
Sponsor Sensorion (Industry)
Compound SENS-501
Therapeutic Modality Gene transfer
Target Gene OTOF
Vector Type dual AAV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-04-09 Last Modified Date: 1783486800000 Enrollment: 12 Australia, France
NCT05606614 Active not recruiting Phase3
View Report
Rett Syndrome
Sponsor Taysha Gene Therapies, Inc. (Industry)
Compound TSHA-102
Therapeutic Modality Gene transfer
Target Gene MiniMECP2
Vector Type scAAV9
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
First Submitted: 2022-10-28 Last Modified Date: 1783400400000 Enrollment: 17 Canada, United States
NCT06272149 Recruiting Early phase1
View Report
Type II Gaucher Disease
Sponsor Xinhua Hospital, Shanghai Jiao Tong University School of Medicine (Other)
Compound VGN-R08b
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-07-17 Last Modified Date: 1783400400000 Enrollment: 6 China
NCT06432140 Active not recruiting Phase3, Phase2
View Report
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
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 Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-05-17 Last Modified Date: 1783400400000 Enrollment: 13 China
NCT03368742 Active not recruiting Phase2, Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-001
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type AAV9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-12-05 Last Modified Date: 1783400400000 Enrollment: 12 United States
NCT03812263 Completed Phase2, Phase1
View Report
Leukocyte Adhesion Defect - Type I
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound KRESLADI
Therapeutic Modality Gene transfer
Target Gene ITGB2
Vector Type Chim.hCD18-LV
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
First Submitted: 2019-01-18 Last Modified Date: 1783400400000 Enrollment: 9 Spain, United Kingdom, United States
NCT07007065 Recruiting Phase3
View Report
Neovascular Age-related Macular Degeneration
Sponsor AbbVie (Industry)
Compound ABBV-RGX-314
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2025-05-28 Last Modified Date: 1783400400000 Enrollment: 561 Austria, Belgium, Bulgaria + 14 more
NCT05477563 Recruiting Phase3
View Report
Beta-Thalassemia, Sickle Cell Disease
Sponsor Vertex Pharmaceuticals Incorporated (Industry)
Compound CASGEVY
Therapeutic Modality Gene editing
Target Gene BCL11A
Delivery System Electroporation
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-07-26 Last Modified Date: 1783400400000 Enrollment: 26 Germany, Italy, Saudi Arabia + 1 more
NCT03588299 Active not recruiting Phase2, Phase1
View Report
Hemophilia A
Sponsor Bayer (Industry)
Compound DTX201
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type AAVhu37
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-07-06 Last Modified Date: 1783314000000 Enrollment: 11 Bulgaria, France, Germany + 3 more
NCT05248230 Recruiting Phase2
View Report
Cystic Fibrosis
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-710
Therapeutic Modality Gene transfer
Target Gene CFTR
Vector Type AAV A101
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-02-10 Last Modified Date: 1783314000000 Enrollment: 30 United States
NCT04819841 Recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Kamau Therapeutics (Industry)
Compound KMAU-001
Therapeutic Modality Gene editing
Target Gene HBB
Vector Type AAV6
Delivery System Viral transduction
Mechanism of Action Mutation correction
First Submitted: 2021-03-24 Last Modified Date: 1782968400000 Enrollment: 15 United States
NCT07667387 Not yet recruiting Phase2, Phase1
View Report
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
First Submitted: 2026-06-18 Last Modified Date: 1782882000000 Enrollment: 7 United States
NCT05788536 Recruiting Phase2, Phase1
View Report
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Sponsor Regeneron Pharmaceuticals (Industry)
Compound OTARMENI
Therapeutic Modality Gene transfer
Target Gene OTOF
Vector Type 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
First Submitted: 2023-03-15 Last Modified Date: 1782882000000 Enrollment: 36 Germany, Japan, Spain + 2 more
NCT05693142 Recruiting Phase3, Phase2
View Report
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
First Submitted: 2023-01-04 Last Modified Date: 1782795600000 Enrollment: 65 Canada, United States
NCT04713475 Active not recruiting Phase2, Phase1
View Report
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
First Submitted: 2021-01-04 Last Modified Date: 1782795600000 Enrollment: 26 Brazil, Turkey (Türkiye), United Kingdom + 1 more
NCT06285643 Recruiting Phase2
View Report
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
First Submitted: 2024-02-14 Last Modified Date: 1782709200000 Enrollment: 127 Germany, Poland, United Kingdom + 1 more
NCT05968118 Unknown Phase3
View Report
Peripheral Artery Disease
Sponsor Beijing Northland Biotech. Co., Ltd. (Industry)
Compound NL003
Therapeutic Modality Gene transfer
Target Gene HGF
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2023-01-04 Last Modified Date: 1782450000000 Enrollment: 36 China
NCT03720418 Terminated Phase2, Phase1
View Report
Parkinson's Disease
Sponsor Sio Gene Therapies (Industry)
Compound OXB-102
Therapeutic Modality Gene transfer
Target Gene TH-GCH1.DDC (tricistronic)
Vector Type EIAV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2018-10-16 Last Modified Date: 1782450000000 Enrollment: 6 France, United Kingdom
NCT06063850 Recruiting Phase2, Phase1
View Report
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
First Submitted: 2023-09-01 Last Modified Date: 1782450000000 Enrollment: 12 United States
NCT05878860 Recruiting Phase3
View Report
X-Linked Retinoschisis
Sponsor Atsena Therapeutics Inc. (Industry)
Compound ATSN-201
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type AAV.SPR
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
First Submitted: 2023-05-12 Last Modified Date: 1782450000000 Enrollment: 97 United States
NCT05398029 Completed Phase1
View Report
Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Sponsor Verve Therapeutics, Inc. (Industry)
Compound VERVE-101
Therapeutic Modality Gene editing
Target Gene PCSK9
Vector Type LDLR
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2022-05-19 Last Modified Date: 1782450000000 Enrollment: 13 New Zealand, United Kingdom
NCT01054339 Completed Phase2
View Report
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
First Submitted: 2010-01-21 Last Modified Date: 1782450000000 Enrollment: 9 Ireland, United States
NCT05682144 Recruiting Phase1
View Report
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Sponsor Immusoft of CA, Inc. (Industry)
Compound ISP-001
Therapeutic Modality Gene transfer
Target Gene IDUA
Vector Type Sleeping Beauty transposon
Delivery System Electroporation
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-12-12 Last Modified Date: 1782450000000 Enrollment: 11 United States
NCT06634420 Active not recruiting Phase3
View Report
Hereditary Angioedema
Sponsor Intellia Therapeutics (Industry)
Compound NTLA-2002
Therapeutic Modality Gene editing
Target Gene KLKB1
Vector Type LDLR
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-10-07 Last Modified Date: 1782450000000 Enrollment: 80 Australia, Canada, France + 6 more
NCT03566043 Active not recruiting Phase3, Phase2
View Report
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
First Submitted: 2018-05-01 Last Modified Date: 1782450000000 Enrollment: 48 Brazil, United States
NCT06559176 Enrolling by invitation Phase2, Phase1
View Report
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
First Submitted: 2024-07-11 Last Modified Date: 1782450000000 Enrollment: 12 Canada, United Kingdom, United States
NCT04124042 Completed Phase2
View Report
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
First Submitted: 2019-10-09 Last Modified Date: 1782450000000 Enrollment: 289 Australia, United States
NCT06461702 Unknown Early phase1
View Report
Familial Hypercholesterolemia
Sponsor The First Affiliated Hospital of Bengbu Medical University (Other)
Compound YOLT-101
Therapeutic Modality Gene editing
Target Gene PCSK9/LDLR
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2024-04-16 Last Modified Date: 1782450000000 Enrollment: 13 China
NCT02144610 Terminated Phase3
View Report
Critical Limb Ischemia
Sponsor AnGes USA, Inc. (Industry)
Compound COLLATEGENE
Therapeutic Modality Gene transfer
Target Gene HGF
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2014-05-20 Last Modified Date: 1782450000000 Enrollment: 46 Belgium, Canada, Denmark + 8 more
NCT06851767 Enrolling by invitation Phase2, Phase1
View Report
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
First Submitted: 2025-02-26 Last Modified Date: 1782450000000 Enrollment: 18 United States
NCT03217617 Recruiting Phase2, Phase1
View Report
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor Shenzhen Geno-Immune Medical Institute (Other)
Compound Ivlv-X1 lentiviral vector
Therapeutic Modality Gene transfer
Target Gene IL2RG
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-07-03 Last Modified Date: 1782190800000 Enrollment: 10 China
NCT03645460 Recruiting Na
View Report
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Sponsor Shenzhen Geno-Immune Medical Institute (Other)
Compound ADA lentiviral vector
Therapeutic Modality Gene transfer
Target Gene ADA
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-07-17 Last Modified Date: 1782190800000 Enrollment: 10 China
NCT07287397 Recruiting Phase2, Phase1
View Report
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
First Submitted: 2025-11-19 Last Modified Date: 1782190800000 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 more
NCT07180355 Recruiting Phase1
View Report
Friedreich's Ataxia
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-212
Therapeutic Modality Gene transfer
Target Gene FXN
Vector Type AAVhu68
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-08-22 Last Modified Date: 1782104400000 Enrollment: 10 United States
NCT07113743 Enrolling by invitation Phase2, Phase1
View Report
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
First Submitted: 2025-08-08 Last Modified Date: 1782104400000 Enrollment: 10 United States
NCT00821340 Completed Phase1
View Report
Leber Congenital Amaurosis-Type 2
Sponsor Hadassah Medical Organization (Other)
Compound RAAV2-hRPE65
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2009-01-12 Last Modified Date: 1781758800000 Enrollment: 3 Israel
NCT06246513 Active not recruiting Phase3
View Report
Limb-Girdle Muscular Dystrophy, Type 2E/R4
Sponsor Sarepta Therapeutics, Inc. (Industry)
Compound SRP-9003
Therapeutic Modality Gene transfer
Target Gene SGCB
Vector Type AAVrh74
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-01-30 Last Modified Date: 1781758800000 Enrollment: 17 Belgium, Germany, Italy + 3 more
NCT03041324 Terminated Phase2, Phase1
View Report
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
First Submitted: 2017-01-13 Last Modified Date: 1781758800000 Enrollment: 9 United States
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
First Submitted: 2023-12-25 Last Modified Date: 1781758800000 Enrollment: 50 China
NCT06623279 Not yet recruiting Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)
Compound HG202
Therapeutic Modality Gene editing
Target Gene VEGFA mRNA
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2024-09-30 Last Modified Date: 1781758800000 Enrollment: 15
NCT04480567 Active not recruiting Phase2, Phase1
View Report
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
First Submitted: 2020-07-08 Last Modified Date: 1781758800000 Enrollment: 100 United Kingdom, United States
NCT06292650 Recruiting Early phase1
View Report
Retinitis Pigmentosa
Sponsor Zhongmou Therapeutics (Industry)
Compound ZM-02
Therapeutic Modality Gene transfer
Target Gene Ch2.0
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Orphan Drug Designation
First Submitted: 2024-02-27 Last Modified Date: 1781758800000 Enrollment: 12 China
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
First Submitted: 2020-08-14 Last Modified Date: 1781758800000 Enrollment: 21 United States
NCT06662188 Recruiting Phase2, Phase1
View Report
SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Sponsor Jaguar Gene Therapy, LLC (Industry)
Compound JAG201
Therapeutic Modality Gene transfer
Target Gene SHANK3
Vector Type AAV2/9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Rare Pediatric Disease Designation
First Submitted: 2024-10-16 Last Modified Date: 1781758800000 Enrollment: 6 United States
NCT06942572 Recruiting Phase2, Phase1
View Report
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
First Submitted: 2025-02-18 Last Modified Date: 1781758800000 Enrollment: 57 United States
NCT03199469 Active not recruiting Phase3, Phase2
View Report
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
First Submitted: 2017-06-21 Last Modified Date: 1781758800000 Enrollment: 27 Canada, France, Germany + 1 more
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
First Submitted: 2017-04-12 Last Modified Date: 1781758800000 Enrollment: 9 Italy, Turkey (Türkiye)
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
First Submitted: 2017-10-12 Last Modified Date: 1781758800000 Enrollment: 12 United States
NCT06022744 Not yet recruiting Na
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine (Other)
Compound LX109
Therapeutic Modality Undisclosed
Target Gene Undisclosed
Vector Type undisclosed
Delivery System Viral transduction
Mechanism of Action Undisclosed
First Submitted: 2023-08-28 Last Modified Date: 1781758800000 Enrollment: 12
NCT04581785 Terminated Phase2, Phase1
View Report
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
First Submitted: 2020-10-02 Last Modified Date: 1781758800000 Enrollment: 4 United States
NCT05222178 Terminated Phase1
View Report
Phenylketonuria (PKU)
Sponsor Homology Medicines, Inc (Industry)
Compound HMI-103
Therapeutic Modality Gene transfer
Target Gene PAH
Vector Type AAVHSC15
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track
First Submitted: 2021-12-21 Last Modified Date: 1781758800000 Enrollment: 3 United States
NCT05672121 Recruiting Phase2, Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Chengdu Origen Biotechnology Co., Ltd. (Industry)
Compound KH631
Therapeutic Modality Gene transfer
Target Gene VEGFR1/R2 fusion protein
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2022-12-21 Last Modified Date: 1781758800000 Enrollment: 42 China
NCT03964792 Completed Phase2, Phase1
View Report
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
First Submitted: 2019-05-14 Last Modified Date: 1781758800000 Enrollment: 6 France
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
First Submitted: 2022-06-23 Last Modified Date: 1781758800000 Enrollment: 15 United States
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
First Submitted: 2021-04-16 Last Modified Date: 1781758800000 Enrollment: 45 Canada, United States
NCT06332807 Recruiting Phase2, Phase1
View Report
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
First Submitted: 2024-02-07 Last Modified Date: 1781758800000 Enrollment: 12 United States
NCT02161380 Completed Phase1
View Report
Leber Hereditary Optic Neuropathy (LHON)
Sponsor Byron Lam (Other)
Compound ScAAV2-P1ND4v2
Therapeutic Modality Gene transfer
Target Gene ND4
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2014-06-06 Last Modified Date: 1781758800000 Enrollment: 28 United States
NCT06615206 Recruiting Na
View Report
MECP2 Duplication Syndrome
Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)
Compound HG204
Therapeutic Modality Gene editing
Target Gene MECP2
Vector Type AAV
Delivery System Viral transduction
Mechanism of Action Gene inactivation
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-09-04 Last Modified Date: 1781758800000 Enrollment: 6 China
NCT06736080 Not yet recruiting Phase2, Phase1
View Report
Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3)
Sponsor Assistance Publique - Hôpitaux de Paris (Other)
Compound Autologous Cells Transduced Ex Vivo with LV-UNC13D
Therapeutic Modality Gene transfer
Target Gene UNC13D
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-12-04 Last Modified Date: 1781758800000 Enrollment: 5 France
NCT00749957 Completed Phase2, Phase1
View Report
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
First Submitted: 2008-09-08 Last Modified Date: 1781758800000 Enrollment: 12 United States
NCT06345898 Recruiting Early phase1
View Report
X-Linked Retinoschisis
Sponsor West China Hospital (Other)
Compound JWK002
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-03-28 Last Modified Date: 1781758800000 Enrollment: 12 China
NCT05973630 Active not recruiting Phase1
View Report
Limb-Girdle Muscular Dystrophy, Type 2C/R5
Sponsor Atamyo Therapeutics (Industry)
Compound ATA-200
Therapeutic Modality Gene transfer
Target Gene SGCG
Vector Type AAV2/8
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-07-24 Last Modified Date: 1781758800000 Enrollment: 4 United States
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
First Submitted: 2022-06-04 Last Modified Date: 1781758800000 Enrollment: 20 United States
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
First Submitted: 2021-11-22 Last Modified Date: 1781758800000 Enrollment: 6 United Kingdom, United States
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
First Submitted: 2016-01-04 Last Modified Date: 1781758800000 Enrollment: 9 Canada, Italy, Netherlands + 1 more
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
First Submitted: 2023-04-29 Last Modified Date: 1781758800000 Enrollment: 9 China
NCT06196827 Active not recruiting Phase1
View Report
Inherited Retinal Dystrophy Associated With RPE65 Mutations
Sponsor Innostellar Biotherapeutics Co.,Ltd (Industry)
Compound LX101
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type AAV2
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-12-25 Last Modified Date: 1781758800000 Enrollment: 9 China
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
First Submitted: 2016-04-28 Last Modified Date: 1781758800000 Enrollment: 15 United Kingdom, United States
NCT03315182 Terminated Phase2, Phase1
View Report
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
First Submitted: 2017-10-10 Last Modified Date: 1781758800000 Enrollment: 11 France, Germany, Spain + 1 more
NCT05903794 Completed Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Exegenesis Bio (Industry)
Compound EXG102-031
Therapeutic Modality Gene transfer
Target Gene ABD-VEGFR
Vector Type AAV8
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2023-06-06 Last Modified Date: 1781758800000 Enrollment: 12 United States
NCT01301443 Completed Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration
Sponsor Oxford BioMedica (Industry)
Compound RetinoStat
Therapeutic Modality Gene transfer
Target Gene COL18A1+PLG
Vector Type EIAV
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2011-02-21 Last Modified Date: 1781758800000 Enrollment: 21 United States
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
First Submitted: 2021-06-15 Last Modified Date: 1781758800000 Enrollment: 27 Puerto Rico, United States
NCT05224505 Active not recruiting Phase1
View Report
Limb-Girdle Muscular Dystrophy, Type 2I/R9
Sponsor Atamyo Therapeutics (Industry)
Compound GNT0006
Therapeutic Modality Gene transfer
Target Gene FKRP
Vector Type AAV2/9
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Rare Pediatric Disease Designation
First Submitted: 2021-12-15 Last Modified Date: 1781758800000 Enrollment: 6 Denmark, France, United Kingdom
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
First Submitted: 2021-04-05 Last Modified Date: 1781758800000 Enrollment: 85 Australia, Canada, United Kingdom + 1 more
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
First Submitted: 2023-06-30 Last Modified Date: 1781758800000 Enrollment: 51 United States
NCT06517888 Recruiting Phase2, Phase1
View Report
Vestibular Schwannoma
Sponsor Akouos, Inc. (Industry)
Compound AAVAnc80-antiVEGF
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type AAVAnc80
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-07-19 Last Modified Date: 1781758800000 Enrollment: 27 United States
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
First Submitted: 2024-06-09 Last Modified Date: 1781758800000 Enrollment: 44 China
NCT05241444 Recruiting Phase1
View Report
Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome (IPEX)
Sponsor Bacchetta, Rosa, MD (Other)
Compound CD4^LVFOXP3
Therapeutic Modality Gene transfer
Target Gene FOXP3
Vector Type LV
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-01-12 Last Modified Date: 1781758800000 Enrollment: 30 United States
NCT04211714 Active not recruiting Phase1
View Report
Telomere Shortening, Bone Marrow Failure
Sponsor Elixirgen Therapeutics, Inc. (Industry)
Compound EXG34217
Therapeutic Modality Gene transfer
Target Gene ZSCAN4
Vector Type SeV18/TS15ΔF
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
First Submitted: 2019-12-06 Last Modified Date: 1781758800000 Enrollment: 12 United States
NCT03328130 Terminated Phase2, Phase1
View Report
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
First Submitted: 2017-10-05 Last Modified Date: 1781758800000 Enrollment: 19 France

Clinical Trials Daily Digest

Updates in the last 14 days

NCT07681778
Vector type Previous: AAV8 Current: Adeno-associated virus curator
Browse condition terms Previous: Gene therapy, Ird, Lca, Leber congenital amaurosis... Current: Gene therapy, Ird, Lca, Leber congenital amaurosis... api
Last update post date Previous: 2026-07-06 Current: 2026-07-15 api
Brief title Previous: Safety and tolerability of subretinal opgx-rdh12-1... Current: Safety and tolerability of subretinal opgx-rdh12-1... api
Compound description Previous: Current: AAV8-RK1-hoptRDH12 curator
Indication doid Previous: Current: DOID:0110330 curator
Development status Previous: Current: Active curator
Record status Previous: Draft Current: Active curator
Compound name Previous: Current: OPGx-RDH12 curator
Recent updates Previous: Current: expected to initiate clinical testing in 4Q2026, f... curator
Dose 2 Previous: Current: high dose: 3E11 vg/eye curator
Dose 1 Previous: Current: low dose: 1E11 vg/eye curator
Delivery system Previous: Current: Viral transduction curator
Target tissue Previous: Current: Photoreceptors curator
Drug product type Previous: Current: Viral vector curator
Route of administration Previous: Current: Subretinal curator
Mechanism of action Previous: Current: Functional gene replacement curator
Therapy route Previous: Current: in vivo curator
Therapy type Previous: Current: Gene transfer curator
Target gene Previous: Current: RDH12 curator
News and press releases Previous: null Current: LinkName:SEC Form 10-K: Opus Genetics, Inc. FY2025... curator
News and press releases Previous: null Current: LinkName:Opus Genetics Highlights Five Gene Therap... curator
Preclinical publications Previous: null Current: LinkName:(Poster) Evaluation of Ocular Tolerabilit... curator
1784091600000
Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA), LCA5
Opus Genetics, Inc Not yet recruiting
Expected to initiate clinical testing in 4Q2026, funding is supported by the RDH12 Alliance
NCT07052929
Vector type Previous: AAV3.8 Current: Adeno-associated virus curator
Last update post date Previous: 2026-05-28 Current: 2026-07-15 api
Number of locations Previous: 3 Current: 4 api
Locations Previous: United states Current: Canada, United states api
Record status Previous: Draft Current: Active curator
Target tissue Previous: Current: muscle cells curator
Recent updates Previous: Astellas has restarted their XLMTM program by crea... Current: Astellas has restarted their XLMTM program by crea... curator
Dose 2 Previous: Current: 2E12 vg/kg curator
Dose 1 Previous: Current: 1E12 vg/kg curator
Related nctid Previous: null Current: LinkName:Phase 2/3: NCT03199469 (prior drug produc... curator
Related nctid Previous: null Current: LinkName:Epidemiological study: NCT06581146;LinkUr... curator
News and press releases Previous: null Current: LinkName:Astellas Takes Aim at XLMTM Again, Now wi... curator
News and press releases Previous: null Current: LinkName:VALOR Clinical Trial Patient Information;... curator
Protocol Previous: null Current: LinkName:(Abstract) VALOR study design: a phase 1/... curator
Protocol Previous: null Current: LinkName:(Abstract 74P) Design of a phase 1/2 stud... curator
Preclinical publications Previous: null Current: LinkName:(Abstract) Efficacy, Biodistribution, and... curator
Compound description Previous: Current: MyoAAV3.8-MHCK7-hMTM1 curator
Indication doid Previous: Current: DOID:0111225 curator
Development status Previous: Current: Active curator
Compound name Previous: Current: ASP2957 curator
Delivery system Previous: Current: viral transduction curator
Drug product type Previous: Current: viral vector curator
Route of administration Previous: Current: intravenous curator
Mechanism of action Previous: Current: functional gene replacement curator
Therapy route Previous: Current: in-vivo curator
Therapy type Previous: Current: gene transfer curator
Target gene Previous: Current: MTM1 curator
Alias type Previous: null Current: proprietary name curator
Alias value Previous: null Current: KT430 curator
Clinical publications Previous: null Current: LinkName:Intrahepatic Cholestasis Is a Clinically ... curator
Clinical publications Previous: null Current: LinkName:Hepatobiliary disease in XLMTM: a common ... curator
News and press releases Previous: null Current: LinkName:Astellas Sees Path Forward for Gene Thera... curator
Eligibility criteria Previous: null Current: Inclusion criteria: * participant is projected to... api
1784091600000
X-Linked Myotubular Myopathy
Astellas Gene Therapies Recruiting
Astellas has restarted their XLMTM program by creating a new drug product ASP2957, with substantial modifications to the capsid to detarget the liver,...
NCT07592273
Vector type Previous: AAV8 Current: Adeno-associated virus curator
Last update post date Previous: 2026-07-15 Current: 2026-07-22 api
Number of locations Previous: 14 Current: 15 api
Recent updates Previous: First patient has been dosed in the Phase IIb/III ... Current: First patient has been dosed in the Phase IIb/III ... curator
Dose 1 Previous: Phase 2 doses: 2.5E11 GC/eye, 5.0E11 GC/eye,1.0E12... Current: Phase 2 doses: 2.5E11 GC/eye, 5.0E11 GC/eye, 1.0E1... curator
Clinical publications Previous: null Current: LinkName:(Presentation) Suprachoroidal Delivery of... curator
News and press releases Previous: null Current: LinkName:REGENXBIO Presents Positive Long-Term Dat... curator
Related nctid Previous: LinkName:Phase 2: NCT06942520 (uses subretinal inj... Current: LinkName:Phase 2: NCT06942520 (using subretinal in... curator
News and press releases Previous: null Current: LinkName:REGENXBIO Announces First Patient Dosed i... curator
Locations Previous: United states Current: Puerto rico, United states api
Indication doid Previous: DOID:9191, DOID:8947 Current: DOID:8947 curator
Record status Previous: Draft Current: Active curator
Dose 3 Previous: 1.0E12 GC/eye Current: curator
Dose 2 Previous: 5.0E11 GC/eye Current: Phase 3 dose: 1.0E12 GC/eye curator
News and press releases Previous: null Current: LinkName:REGENXBIO Announces Pivotal Program for S... curator
Development status Previous: Current: Active curator
Compound name Previous: Current: Surabgene lomparvovec curator
Delivery system Previous: Current: Viral transduction curator
Drug product type Previous: Current: Viral vector curator
Route of administration Previous: Current: Suprachoroidal curator
Mechanism of action Previous: Current: Genetic delivery of therapeutic protein curator
Therapy route Previous: Current: In-vivo curator
Therapy type Previous: Current: Gene transfer curator
Target gene Previous: Current: Anti-VEGF curator
Alias type Previous: null Current: proprietary name curator
Alias value Previous: null Current: ABBV-RGX-314 curator
Related nctid Previous: null Current: LinkName:Long Term Follow-Up: NCT05296447;LinkUrl:... curator
Related nctid Previous: null Current: LinkName:Phase 2: NCT04567550;LinkUrl:https://clin... curator
News and press releases Previous: null Current: LinkName:REGENXBIO Reports Third Quarter 2025 Fina... curator
Clinical publications Previous: null Current: LinkName:(Presentation) Suprachoroidal Surabgene L... curator
Estimated completion date Previous: 2036-02 Current: 2036-01 api
Overall status Previous: Not yet recruiting Current: Recruiting api
1784091600000
Diabetic Retinopathy
AbbVie Recruiting
First patient has been dosed in the Phase IIb/III trial using suprachoroidal delivery. Phase IIb data will be presented at the ASRS annual meeting in ...
NCT07226206
Vector type Previous: LK03 Current: Adeno-associated virus curator
Last update post date Previous: 2026-06-23 Current: 2026-07-14 api
Editor type Previous: none Current: curator
Overall status Previous: Not yet recruiting Current: Recruiting api
Number of locations Previous: 1 Current: 2 api
Compound description Previous: AAV LK03 capsid + TTRmut-hFVIII-X07 Current: AAV LK03 capsid + TTRmut + APC-resistant FVIII var... curator
Preclinical publications Previous: null Current: LinkName:Activated protein C has a regulatory role... curator
Indication doid Previous: null Current: DOID:12134 curator
Development status Previous: null Current: Active curator
Record status Previous: Provisional Current: Active curator
Compound name Previous: null Current: SPK-8011QQ curator
Recent updates Previous: null Current: Study was withdrawn by Sponsor (no participants we... curator
Dose 4 Previous: null Current: 2E12 vg/kg curator
Dose 3 Previous: null Current: 1.5E12 vg/kg curator
Dose 2 Previous: null Current: 1E12 vg/kg curator
Dose 1 Previous: null Current: 5E11 vg/kg curator
Delivery system Previous: null Current: Viral transduction curator
Drug product type Previous: null Current: Viral vector curator
Route of administration Previous: null Current: Intravenous curator
Mechanism of action Previous: null Current: Functional gene replacement curator
Therapy route Previous: null Current: In-vivo curator
Therapy type Previous: null Current: Gene transfer curator
Target gene Previous: null Current: F8 curator
Alias type Previous: null Current: proper name curator
Alias value Previous: null Current: Dirloctogene samoparvovec curator
Related nctid Previous: null Current: LinkName:Phase 3: NCT06297486 (Withdrawn);LinkUrl:... curator
Related nctid Previous: null Current: LinkName:Phase 1/2: NCT03003533;LinkUrl:https://ww... curator
Related nctid Previous: null Current: LinkName:Long Term Follow-Up: NCT03432520;LinkUrl:... curator
Preclinical publications Previous: null Current: LinkName:Preclinical assessment of an optimized AA... curator
Preclinical publications Previous: null Current: LinkName:(Abstract) Preclinical evaluation of SPK-... curator
News and press releases Previous: null Current: LinkName:Roche mothballs another hemophilia A gene... curator
News and press releases Previous: null Current: LinkName:Integration of Spark into Roche Group Set... curator
Clinical publications Previous: null Current: LinkName:Multiyear Factor VIII Expression after AA... curator
Eligibility criteria Previous: null Current: Inclusion criteria: * signed informed consent for... api
Locations Previous: Current: United states api
1784005200000
Hemophilia A
Hoffmann-La Roche Recruiting
Study was withdrawn by Sponsor (no participants were enrolled), Sponsor is extensively modifying the product before clinical trials resume
NCT02852213
Vector type Previous: AAV2 Current: Adeno-associated virus curator
Last update post date Previous: 2025-10-21 Current: 2026-07-13 api
Estimated completion date Previous: 2031-07 Current: 2037-03 api
Recent updates Previous: Current: Product is also under evaluation for the treatment... curator
Related nctid Previous: null Current: LinkName:Phase 1: NCT07267065 (Parkinson's disease... curator
Editor type Previous: none Current: curator
Route of administration Previous: CED Current: Intracerebral curator
Eligibility criteria Previous: null Current: Inclusion criteria 1. definite diagnosis of aadc ... api
1783918800000
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Krzysztof Bankiewicz Recruiting
Product is also under evaluation for the treatment of Parkinson's disease
7 more update(s) recently — View All
Disclaimer: The information on this dashboard has been collected for the convenience of patients and researchers. The SCGE team are not medical doctors and cannot provide medical advice. Please discuss with your provider the risks/benefits of participating in a clinical trial, and do not send us your personal medical information. The information contained within this table does not make use of any confidential or privileged information-all data is collected from publicly available sources. The SCGE makes no comment as to the efficacy and safety of the items listed, as these are not known at the time of publication. For the most up to date information, or to inquire about enrollment, please refer to clinicaltrials.gov or the Sponsor's website for contact information.