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 130 results in ClinicalTrials
Remove Filters:
NCT07667387 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: 1787720400000 Enrollment: 7 United States
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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Fast Track
First Submitted: 2025-11-19 Last Modified Date: 1787720400000 Enrollment: 12 Belgium, Netherlands, United Kingdom + 1 more
NCT06884865 Recruiting Phase2
View Report
Osteoarthritis (OA) of the Knee
Sponsor Pacira Pharmaceuticals, Inc (Industry)
Compound PCRX-201
Therapeutic Modality Gene transfer
Target Gene IL1RA
Vector Type Adenovirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-03-13 Last Modified Date: 1787634000000 Enrollment: 135 United States
NCT07429240 Recruiting Phase2, Phase1
View Report
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
Sponsor Precision BioSciences, Inc. (Industry)
Compound PBGENE-DMD
Therapeutic Modality Gene editing
Target Gene DMD del ex45-55
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Exon skipping/splice editor
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2026-02-17 Last Modified Date: 1787634000000 Enrollment: 18 United States
NCT05248230 Recruiting Phase2
View Report
Cystic Fibrosis
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-710
Therapeutic Modality Gene transfer
Target Gene CFTR
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-02-10 Last Modified Date: 1787634000000 Enrollment: 30 United States
NCT07064759 Recruiting Phase3
View Report
Neovascular Age-Related Macular Degeneration (nAMD)
Sponsor 4D Molecular Therapeutics (Industry)
Compound 4D-150
Therapeutic Modality Gene transfer
Target Gene Anti-VEGF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-07-07 Last Modified Date: 1787634000000 Enrollment: 480 Argentina, Australia, Bulgaria + 11 more
NCT06680232 Recruiting Phase1
View Report
Chronic Hepatitis B
Sponsor Precision BioSciences, Inc. (Industry)
Compound PBGENE-HBV
Therapeutic Modality Gene editing
Target Gene HBV cccDNA
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Fast Track
First Submitted: 2024-10-24 Last Modified Date: 1787547600000 Enrollment: 45 France, Hong Kong, Moldova + 3 more
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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
First Submitted: 2023-09-01 Last Modified Date: 1787547600000 Enrollment: 12 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 Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2011-02-26 Last Modified Date: 1787547600000 Enrollment: 40 United States
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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-03-17 Last Modified Date: 1787288400000 Enrollment: 36 Australia, Spain, United States
NCT07643844 Recruiting Phase1
View Report
Propionic Acidemia
Sponsor Mayo Clinic (Other)
Compound AAVrh10-PCCA
Therapeutic Modality Gene transfer
Target Gene PCCA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2026-06-08 Last Modified Date: 1787288400000 Enrollment: 9 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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-12 Last Modified Date: 1787288400000 Enrollment: 97 United Kingdom, 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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-06-10 Last Modified Date: 1787288400000 Enrollment: 47 Australia, United Kingdom, United States
NCT07721025 Recruiting Phase2
View Report
Friedreich Ataxia with Secondary Cardiomyopathy
Sponsor Lexeo Therapeutics (Industry)
Compound LX2006
Therapeutic Modality Gene transfer
Target Gene FXN
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2026-06-24 Last Modified Date: 1787115600000 Enrollment: 26 United States
NCT07218887 Recruiting Phase2, Phase1
View Report
BAG3 Mutation Associated Dilated Cardiomyopathy
Sponsor Alexion Pharmaceuticals, Inc. (Industry)
Compound ALXN2350
Therapeutic Modality Gene transfer
Target Gene BAG3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-10-17 Last Modified Date: 1787115600000 Enrollment: 6 Italy, Spain, United Kingdom + 1 more
NCT06332807 Recruiting Phase2, Phase1
View Report
Phenylketonuria (PKU)
Sponsor NGGT INC. (Industry)
Compound NGGT002
Therapeutic Modality Gene transfer
Target Gene PAH
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-02-07 Last Modified Date: 1787115600000 Enrollment: 12 United States
NCT06831825 Recruiting Phase1
View Report
Heart Failure With Reduced Ejection Fraction
Sponsor Medley Therapeutics (Industry)
Compound YAP101
Therapeutic Modality Gene transfer
Target Gene SAV1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Gene inactivation
First Submitted: 2025-01-23 Last Modified Date: 1786942800000 Enrollment: 24 United States
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 Adeno-associated virus
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: 1786942800000 Enrollment: 10 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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2022-11-07 Last Modified Date: 1786683600000 Enrollment: 22 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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2026-04-24 Last Modified Date: 1786683600000 Enrollment: 576 Puerto Rico, 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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2025-05-28 Last Modified Date: 1786683600000 Enrollment: 561 Austria, Belgium, Bulgaria + 14 more
NCT07223944 Recruiting Phase3
View Report
Gaucher Disease Type 1
Sponsor Spur Therapeutics (Industry)
Compound FLT201
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2025-10-30 Last Modified Date: 1786683600000 Enrollment: 45 Argentina, Australia, Brazil + 7 more
NCT07627971 Recruiting Phase2, Phase1
View Report
GJB2-mediated Hearing Loss, GJB2 Gene Mutation, DFNB1A
Sponsor Skylark Bio Inc. (Industry)
Compound SKY-GJB2
Therapeutic Modality Gene transfer
Target Gene GJB2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2026-05-28 Last Modified Date: 1786683600000 Enrollment: 10 United States
NCT06128629 Recruiting Phase3
View Report
Transthyretin Amyloidosis (ATTR) with Cardiomyopathy
Sponsor Intellia Therapeutics (Industry)
Compound NTLA-2001
Therapeutic Modality Gene editing
Target Gene TTR
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2023-11-08 Last Modified Date: 1786683600000 Enrollment: 1200 Argentina, Australia, Austria + 23 more
NCT06064890 Recruiting Phase2, Phase1
View Report
Frontotemporal Dementia, FTD, FTD-GRN
Sponsor AviadoBio Ltd (Industry)
Compound AVB-101
Therapeutic Modality Gene transfer
Target Gene GRN
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2023-09-12 Last Modified Date: 1786597200000 Enrollment: 18 Belgium, Canada, Italy + 6 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: 1786597200000 Enrollment: 15 United States
NCT07160634 Recruiting Phase3
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-003
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-08-29 Last Modified Date: 1786597200000 Enrollment: 80 Australia, Canada, United States
NCT07587021 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
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: 1786510800000 Enrollment: 36 United States
NCT06255782 Recruiting Phase3
View Report
Ornithine Transcarbamylase (OTC) Deficiency
Sponsor iECURE, Inc. (Industry)
Compound ECUR-506
Therapeutic Modality Gene transfer
Target Gene OTC/PCSK9 locus
Vector Type Adeno-associated virus
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, Orphan Drug Designation
First Submitted: 2023-12-19 Last Modified Date: 1786510800000 Enrollment: 20 Australia, Spain, United Kingdom + 1 more
NCT06272149 Recruiting Early phase1
View Report
Type II Gaucher Disease
Sponsor Xinhua Hospital, Shanghai Jiao Tong University School of Medicine (Other)
Compound VGN-R08b
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-07-17 Last Modified Date: 1786424400000 Enrollment: 6 China
NCT06765980 Recruiting Phase2, Phase1
View Report
Geographic Atrophy Secondary to Age-related Macular Degeneration
Sponsor Kriya Therapeutics, Inc. (Industry)
Compound KRIYA-825
Therapeutic Modality Gene transfer
Target Gene CR2-CR1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2024-12-19 Last Modified Date: 1786424400000 Enrollment: 62 Canada, Israel, New Zealand + 1 more
NCT07148089 Recruiting Phase1
View Report
Catecholaminergic Polymorphic Ventricular Tachycardia
Sponsor Solid Biosciences Inc. (Industry)
Compound SGT-501
Therapeutic Modality Gene transfer
Target Gene CASQ2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-08-22 Last Modified Date: 1786424400000 Enrollment: 18 Canada, 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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-07-19 Last Modified Date: 1786424400000 Enrollment: 27 United States
NCT05821959 Recruiting Phase2, Phase1
View Report
Bilateral Sensorineural Hearing Loss
Sponsor Akouos, Inc. (Industry)
Compound AK-OTOF
Therapeutic Modality Gene transfer
Target Gene OTOF
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-02-08 Last Modified Date: 1786424400000 Enrollment: 22 Canada, Taiwan, United Kingdom + 1 more
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
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2024-08-01 Last Modified Date: 1786424400000 Enrollment: 31 China
NCT06217861 Recruiting Phase1
View Report
Glutaric Acidemia Type I
Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound VGM-R02b
Therapeutic Modality Gene transfer
Target Gene GCDH
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-12-12 Last Modified Date: 1786424400000 Enrollment: 12 China
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 Adeno-associated virus
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: 1786424400000 Enrollment: 12 China
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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-06-30 Last Modified Date: 1786078800000 Enrollment: 9 Canada, 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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-03-15 Last Modified Date: 1785906000000 Enrollment: 36 Germany, Japan, Spain + 2 more
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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-10-28 Last Modified Date: 1785819600000 Enrollment: 5 United States
NCT07195825 Recruiting Phase1
View Report
Parkinson's Disease
Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound BBM-P002
Therapeutic Modality Gene transfer
Target Gene TH90del-AADC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2025-09-10 Last Modified Date: 1785819600000 Enrollment: 18 China
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
Delivery System Endocytosis
Mechanism of Action Gene inactivation
FDA Designations Fast Track
First Submitted: 2023-12-01 Last Modified Date: 1785733200000 Enrollment: 85 Australia, Canada, Israel + 3 more
NCT06910813 Recruiting Phase2, Phase1
View Report
Nephropathic Cystinosis
Sponsor Novartis Pharmaceuticals (Industry)
Compound DFT383
Therapeutic Modality Gene transfer
Target Gene CTNS
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-03-17 Last Modified Date: 1785474000000 Enrollment: 30 United States
NCT06817382 Recruiting Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Insmed Gene Therapy LLC (Industry)
Compound INS1201
Therapeutic Modality Gene transfer
Target Gene Micro-dystrophin
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-02-04 Last Modified Date: 1785214800000 Enrollment: 12 United States
NCT06185673 Recruiting Phase2, Phase1
View Report
Oculopharyngeal Muscular Dystrophy
Sponsor Benitec Biopharma, Inc. (Industry)
Compound BB-301
Therapeutic Modality Gene transfer
Target Gene PABPN1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement, Gene inactivation
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2023-12-15 Last Modified Date: 1784869200000 Enrollment: 30 United States
NCT07491172 Recruiting Phase1
View Report
Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Uncontrolled Hypercholesterolemia, Mixed Dyslipidemia
Sponsor CRISPR Therapeutics AG (Industry)
Compound CTX310
Therapeutic Modality Gene editing
Target Gene ANGPTL3
Delivery System Endocytosis
Mechanism of Action Gene inactivation
First Submitted: 2026-03-18 Last Modified Date: 1784869200000 Enrollment: 90 Australia, New Zealand, United Kingdom + 1 more
NCT05791864 Recruiting Phase2, Phase1
View Report
Neuronal Ceroid Lipofuscinosis Type 2
Sponsor Tern Therapeutics, LLC (Industry)
Compound TTX-381
Therapeutic Modality Gene transfer
Target Gene TPP1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-03-17 Last Modified Date: 1784869200000 Enrollment: 16 Germany, United Kingdom
NCT07185256 Recruiting Phase2, Phase1
View Report
ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
Sponsor Opus Genetics, Inc (Industry)
Compound OPGx-BEST1
Therapeutic Modality Gene transfer
Target Gene BEST1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-07-17 Last Modified Date: 1784782800000 Enrollment: 10 United States
NCT03952637 Recruiting Phase2, Phase1
View Report
GM1 Gangliosidosis
Sponsor National Human Genome Research Institute (NHGRI) (NIH)
Compound AXO-AAV-GM1
Therapeutic Modality Gene transfer
Target Gene GLB1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-05-15 Last Modified Date: 1784782800000 Enrollment: 54 United States
NCT06389877 Recruiting Phase2, Phase1
View Report
Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor Beam Therapeutics Inc. (Industry)
Compound BEAM-302
Therapeutic Modality Gene editing
Target Gene SERPINA1 (p.Glu366Lys)
Delivery System Endocytosis
Mechanism of Action Mutation correction
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation
First Submitted: 2024-04-22 Last Modified Date: 1784782800000 Enrollment: 106 Australia, Ireland, Netherlands + 3 more
NCT04676048 Recruiting Phase2, Phase1
View Report
Hemophilia A
Sponsor ASC Therapeutics (Industry)
Compound ASC618
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2020-12-15 Last Modified Date: 1784782800000 Enrollment: 12 United States
NCT07058662 Recruiting Phase2, Phase1
View Report
Duchenne Muscular Dystrophy (DMD)
Sponsor Belief BioMed (Beijing) Co., Ltd (Industry)
Compound BBM-D101
Therapeutic Modality Gene transfer
Target Gene Undisclosed
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Undisclosed
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2025-07-01 Last Modified Date: 1784782800000 Enrollment: 9 China
NCT06833983 Recruiting Phase3
View Report
Hemophilia A
Sponsor Gritgen Therapeutics Co., Ltd. (Industry)
Compound GS1191-0445
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-01-28 Last Modified Date: 1784696400000 Enrollment: 50 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 Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-01-12 Last Modified Date: 1784696400000 Enrollment: 30 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 Adeno-associated virus
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: 1784696400000 Enrollment: 6 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 Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2016-07-20 Last Modified Date: 1784696400000 Enrollment: 42 United States
NCT06291935 Recruiting Phase1
View Report
Retinitis Pigmentosa
Sponsor VeonGen Therapeutics GmbH (Industry)
Compound VG901
Therapeutic Modality Gene transfer
Target Gene CNGA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-02-01 Last Modified Date: 1784696400000 Enrollment: 6 Germany
NCT06519552 Recruiting Phase1
View Report
Mucopolysaccharidosis Type I (Hurler Syndrome)
Sponsor West China Hospital (Other)
Compound JWK-008
Therapeutic Modality Gene transfer
Target Gene IDUA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-06-23 Last Modified Date: 1784696400000 Enrollment: 6 China
NCT06465550 Recruiting Phase1
View Report
Beta-Thalassemia Major
Sponsor Shanghai BDgene Co., Ltd. (Industry)
Compound BD211
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-06-12 Last Modified Date: 1784696400000 Enrollment: 9 China
NCT03538899 Recruiting Phase2, Phase1
View Report
Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency
Sponsor University of California, San Francisco (Other)
Compound AProArt
Therapeutic Modality Gene transfer
Target Gene DCLRE1C
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-05-03 Last Modified Date: 1784696400000 Enrollment: 24 United States
NCT05885412 Recruiting Phase1
View Report
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-A601
Therapeutic Modality Gene transfer
Target Gene PKP2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2023-05-22 Last Modified Date: 1784696400000 Enrollment: 9 United States
NCT05906953 Recruiting Phase2, Phase1
View Report
Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)
Compound HG004
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-09 Last Modified Date: 1784696400000 Enrollment: 20 China, United States
NCT06364774 Recruiting Phase2, Phase1
View Report
Beta-Thalassemia Major
Sponsor Children's Hospital of Philadelphia (Other)
Compound CHOP-ALS20
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-03-26 Last Modified Date: 1784696400000 Enrollment: 12 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 Adeno-associated virus
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: 1784696400000 Enrollment: 57 United States
NCT06641154 Recruiting Phase2, Phase1
View Report
Crigler-Najjar Syndrome Type I
Sponsor Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare (Other gov)
Compound GT-UGT1A1-AAV8-02
Therapeutic Modality Gene transfer
Target Gene UGT1A1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-10-11 Last Modified Date: 1784696400000 Enrollment: 5 Russia
NCT06399107 Recruiting Phase2, Phase1
View Report
Sickle Cell Disease, Sickle-Cell Disease With Crisis
Sponsor Essen Biotech (Other)
Compound BAH243
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
First Submitted: 2024-04-29 Last Modified Date: 1784696400000 Enrollment: 85 China
NCT05762510 Recruiting Early phase1
View Report
Beta-Thalassemia Major
Sponsor First Affiliated Hospital of Guangxi Medical University (Other)
Compound GMCN-508B
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2022-01-06 Last Modified Date: 1784696400000 Enrollment: 5 China
NCT03466463 Recruiting Na
View Report
Crigler-Najjar Syndrome
Sponsor Genethon (Other)
Compound GNT0003
Therapeutic Modality Gene transfer
Target Gene UGT1A1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-02-01 Last Modified Date: 1784696400000 Enrollment: 17 France, Italy, Netherlands
NCT06692712 Recruiting Phase3
View Report
Hereditary Spastic Paraplegia Type 50
Sponsor Elpida Therapeutics SPC (Industry)
Compound MELPIDA
Therapeutic Modality Gene transfer
Target Gene AP4M1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-11-14 Last Modified Date: 1784696400000 Enrollment: 24 Spain, United States
NCT06280378 Recruiting Phase2, Phase1
View Report
Beta-Thalassemia Major
Sponsor Kanglin Biotechnology (Hangzhou) Co., Ltd. (Industry)
Compound KL003
Therapeutic Modality Gene transfer
Target Gene HBB
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-02-20 Last Modified Date: 1784696400000 Enrollment: 41 China
NCT05761899 Recruiting Phase2, Phase1
View Report
Hereditary Pulmonary Alveolar Proteinosis
Sponsor Children's Hospital Medical Center, Cincinnati (Other)
Compound Gene-Corrected Macrophages
Therapeutic Modality Gene transfer
Target Gene CSF2RA
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-02-28 Last Modified Date: 1784696400000 Enrollment: 3 United States
NCT05824169 Recruiting Phase2, Phase1
View Report
Spinal Muscular Atrophy
Sponsor GeneCradle Inc (Industry)
Compound GC101
Therapeutic Modality Gene transfer
Target Gene SMN1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2023-04-10 Last Modified Date: 1784696400000 Enrollment: 18 China
NCT06345898 Recruiting Early phase1
View Report
X-Linked Retinoschisis
Sponsor West China Hospital (Other)
Compound JWK002
Therapeutic Modality Gene transfer
Target Gene RS1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-03-28 Last Modified Date: 1784696400000 Enrollment: 12 China
NCT06492876 Recruiting Phase2, Phase1
View Report
Diabetic Macular Edema
Sponsor Frontera Therapeutics (Industry)
Compound FT-003
Therapeutic Modality Gene transfer
Target Gene Aflibercept
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2024-07-01 Last Modified Date: 1784696400000 Enrollment: 78 China
NCT05858983 Recruiting Phase2, Phase1
View Report
Biallelic RPE65 Mutation-associated Retinal Dystrophy
Sponsor Frontera Therapeutics (Industry)
Compound FT-001
Therapeutic Modality Gene transfer
Target Gene RPE65
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2023-04-29 Last Modified Date: 1784696400000 Enrollment: 9 China
NCT05860569 Recruiting Phase1
View Report
Hypertriglyceridemia
Sponsor GeneCradle Inc (Industry)
Compound GC304
Therapeutic Modality Gene transfer
Target Gene GPIHBP1 or LPL
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement, Overexpression of protective allele/gene
First Submitted: 2023-04-23 Last Modified Date: 1784696400000 Enrollment: 7 China
NCT05672121 Recruiting Phase2, Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Chengdu Origen Biotechnology Co., Ltd. (Industry)
Compound KH631
Therapeutic Modality Gene transfer
Target Gene VEGFR1/R2 fusion protein
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2022-12-21 Last Modified Date: 1784696400000 Enrollment: 42 China
NCT04819841 Recruiting Phase2, Phase1
View Report
Sickle Cell Disease
Sponsor Kamau Therapeutics (Industry)
Compound KMAU-001
Therapeutic Modality Gene editing
Target Gene HBB
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Mutation correction
First Submitted: 2021-03-24 Last Modified Date: 1784696400000 Enrollment: 15 United States
NCT06818838 Recruiting Phase2, Phase1
View Report
Gaucher Disease Type 1
Sponsor Lingyi Biotech Co., Ltd. (Industry)
Compound LY-M001
Therapeutic Modality Gene transfer
Target Gene GBA1
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2025-01-24 Last Modified Date: 1784696400000 Enrollment: 12 China
NCT07137338 Recruiting Phase1
View Report
BAG3-Associated Dilated Cardiomyopathy (DCM)
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-A701
Therapeutic Modality Gene transfer
Target Gene BAG3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track
First Submitted: 2025-07-25 Last Modified Date: 1784696400000 Enrollment: 8 United States
NCT04998396 Recruiting Phase2, Phase1
View Report
Canavan Disease
Sponsor Aspa Therapeutics (Industry)
Compound BBP-812
Therapeutic Modality Gene transfer
Target Gene ASPA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-08-05 Last Modified Date: 1784696400000 Enrollment: 26 United States
NCT06092034 Recruiting Phase2
View Report
Danon Disease
Sponsor Rocket Pharmaceuticals Inc. (Industry)
Compound RP-A501
Therapeutic Modality Gene transfer
Target Gene LAMP2B
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-10-11 Last Modified Date: 1784696400000 Enrollment: 14 Germany, Italy, United Kingdom + 1 more
NCT07543991 Recruiting Phase1
View Report
Giant Axonal Neuropathy (GAN)
Sponsor University of Texas Southwestern Medical Center (Other)
Compound ScAAV9/JeT-GAN
Therapeutic Modality Gene transfer
Target Gene GAN
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2026-03-12 Last Modified Date: 1784696400000 Enrollment: 4 United States
NCT07476183 Recruiting Phase1
View Report
RPS19 Deficient Diamond-Blackfan Anemia
Sponsor Apriligen, Inc. (Industry)
Compound APR-2020
Therapeutic Modality Gene transfer
Target Gene RPS19
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2026-03-12 Last Modified Date: 1784696400000 Enrollment: 4 United States
NCT07447557 Recruiting Phase2, Phase1
View Report
Charcot-Marie-Tooth Disease Type 4J
Sponsor Elpida Therapeutics SPC (Industry)
Compound ELP-02
Therapeutic Modality Gene transfer
Target Gene FIG4
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2026-01-20 Last Modified Date: 1784696400000 Enrollment: 8 United States
NCT06270316 Recruiting Phase2, Phase1
View Report
Fabry Disease
Sponsor UniQure Biopharma B.V. (Industry)
Compound AMT-191
Therapeutic Modality Gene transfer
Target Gene GLA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation
First Submitted: 2023-12-19 Last Modified Date: 1784696400000 Enrollment: 12 United States
NCT05092685 Recruiting Phase2, Phase1
View Report
Ornithine Transcarbamylase (OTC) Deficiency
Sponsor University College, London (Other)
Compound BGT-OTCD
Therapeutic Modality Gene transfer
Target Gene OTC
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2021-09-28 Last Modified Date: 1784696400000 Enrollment: 12 United Kingdom
NCT06238908 Recruiting Early phase1
View Report
Hemophilia A
Sponsor Institute of Hematology & Blood Diseases Hospital, China (Other)
Compound NGGT003
Therapeutic Modality Gene transfer
Target Gene F8
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-01-26 Last Modified Date: 1784696400000 Enrollment: 6 China
NCT03311503 Recruiting Phase2, Phase1
View Report
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor David Williams (Other)
Compound G2SCID lentiviral vector transduced CD34+ cells
Therapeutic Modality Gene transfer
Target Gene IL2RG
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2017-10-12 Last Modified Date: 1784696400000 Enrollment: 12 United States
NCT06474442 Recruiting Phase2
View Report
Herpes Simplex Virus Type I Stromal Keratitis
Sponsor Shanghai BDgene Co., Ltd. (Industry)
Compound BD111
Therapeutic Modality Gene editing
Target Gene HSV genes
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Gene excision
FDA Designations Orphan Drug Designation
First Submitted: 2024-06-13 Last Modified Date: 1784696400000 Enrollment: 40 China
NCT06492863 Recruiting Phase2, Phase1
View Report
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor Frontera Therapeutics (Industry)
Compound FT-003
Therapeutic Modality Gene transfer
Target Gene Aflibercept
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Genetic delivery of therapeutic protein
First Submitted: 2024-07-01 Last Modified Date: 1784696400000 Enrollment: 78 China
NCT06650319 Recruiting Early phase1
View Report
Wilson Disease
Sponsor Chaohui Yu (Other)
Compound LY-M003
Therapeutic Modality Gene transfer
Target Gene ATP7B
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-10-15 Last Modified Date: 1784696400000 Enrollment: 18 China
NCT04278131 Recruiting Phase2, Phase1
View Report
Retinitis Pigmentosa
Sponsor Bionic Sight LLC (Industry)
Compound BS01
Therapeutic Modality Gene transfer
Target Gene ChronosFP
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Overexpression of protective allele/gene
FDA Designations Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2020-02-13 Last Modified Date: 1784696400000 Enrollment: 20 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
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: 1784696400000 Enrollment: 11 United States
NCT05071222 Recruiting Phase2, Phase1
View Report
Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency
Sponsor Assistance Publique - Hôpitaux de Paris (Other)
Compound ARTEGENE
Therapeutic Modality Gene transfer
Target Gene DCLRE1C
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2021-08-27 Last Modified Date: 1784696400000 Enrollment: 7 France
NCT06285643 Recruiting Phase2
View Report
Parkinson's Disease
Sponsor AskBio Inc (Industry)
Compound AB-1005
Therapeutic Modality Gene transfer
Target Gene GDNF
Vector Type Adeno-associated virus
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: 1784696400000 Enrollment: 127 Germany, Poland, United Kingdom + 1 more
NCT06228924 Recruiting Phase1
View Report
Arrhythmogenic Right Ventricular Cardiomyopathy
Sponsor Tenaya Therapeutics (Industry)
Compound TN-401
Therapeutic Modality Gene transfer
Target Gene PKP2
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2024-01-18 Last Modified Date: 1784696400000 Enrollment: 15 United States
NCT06391736 Recruiting Phase2, Phase1
View Report
Pompe Disease (Late-onset)
Sponsor GeneCradle Inc (Industry)
Compound GC301
Therapeutic Modality Gene transfer
Target Gene GAA
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Orphan Drug Designation
First Submitted: 2024-04-25 Last Modified Date: 1784696400000 Enrollment: 33 China
NCT05836259 Recruiting Phase2, Phase1
View Report
Hypertrophic Cardiomyopathy
Sponsor Tenaya Therapeutics (Industry)
Compound TN-201
Therapeutic Modality Gene transfer
Target Gene MYBPC3
Vector Type Adeno-associated virus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-04-18 Last Modified Date: 1784696400000 Enrollment: 30 United States
NCT03727555 Recruiting Na
View Report
X-linked Adrenoleukodystrophy
Sponsor Shenzhen Geno-Immune Medical Institute (Other)
Compound (LV) TYF-ABCD1
Therapeutic Modality Gene transfer
Target Gene ABCD1
Vector Type Lentivirus
Delivery System Viral transduction
Mechanism of Action Functional gene replacement
First Submitted: 2018-09-25 Last Modified Date: 1784696400000 Enrollment: 30 China

Clinical Trials Daily Digest

Updates in the last 14 days

NCT05394064
With has results Previous: False Current: True api
Last update post date Previous: 2025-10-31 Current: 2026-08-26 api
Enrollment count Previous: 8 Current: 9 api
Intervention description Previous: Sbt101 treatmentprocedure that mimics sbt101 infus... Current: Sbt101 treatment api
Intervention name Previous: Sbt101, Imitation procedure Current: Sbt101 treatment api
Vector type Previous: AAV9 Current: Adeno-associated virus curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: Inclusion criteria: 1. diagnosed with x-linked ad... api
1787720400000
Adrenomyeloneuropathy (AMN)
SwanBio Therapeutics, Inc. Terminated
Study was terminated for business/strategic reasons, not due to safety concerns
NCT07667387
Last update post date Previous: 2026-08-11 Current: 2026-08-26 api
Estimated completion date Previous: 2028-07 Current: 2028-09 api
Overall status Previous: Not yet recruiting Current: Recruiting api
Brief title Previous: A phase i/ii open-label safety and efficacy study ... Current: Study of lnp.ucd.abe in patients with urea cycle d... api
Eligibility criteria Previous: Inclusion criteria: 1. diagnosis of a severe urea... Current: Inclusion criteria: 1. diagnosis of a severe urea... api
Record status Previous: Draft Current: Active curator
Compound description Previous: Current: personalized variant-specific LNP.UCD.ABE curator
Indication doid Previous: Current: DOID:9267 curator
Development status Previous: Current: Active curator
Compound name Previous: Current: LNP.UCD.ABE curator
Recent updates Previous: Current: This clinical trial protocol is a master protocol ... curator
Dose 1 Previous: Current: undisclosed dose based on body weight with potenti... curator
Editor type Previous: Current: ABE mRNA curator
Delivery system Previous: Current: Endocytosis curator
Drug product type Previous: Current: Encapsulated mRNA curator
Route of administration Previous: Current: Intravenous curator
Mechanism of action Previous: Current: Mutation correction curator
Therapy route Previous: Current: in vivo curator
Therapy type Previous: Current: Gene editing curator
Target gene Previous: Current: CPS1,OTC,ASS1,ASL,ARG,NAGS,SLC25A15 curator
Preclinical publications Previous: null Current: LinkName:Pre-IND Official Responses/Meeting Summar... curator
Preclinical publications Previous: null Current: LinkName:Pre-IND Briefing Book;LinkUrl:https://scg... curator
Preclinical publications Previous: null Current: LinkName:Patient-Specific In Vivo Gene Editing to ... curator
Preclinical publications Previous: null Current: LinkName:How to create personalized gene editing p... curator
1787720400000
Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
Rebecca Ahrens-Nicklas Recruiting
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. Participant...
NCT03645460
Last update post date Previous: 2026-06-23 Current: 2026-08-26 api
Estimated completion date Previous: 2027-12-31 Current: 2028-12-31 api
Overall status Previous: Recruiting Current: Not yet recruiting api
Vector type Previous: LV Current: Lentivirus curator
Number of locations Previous: 2 Current: 1 curator
Eligibility criteria Previous: null Current: Inclusion criteria: * diagnosis of classical ada-... api
1787720400000
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Shenzhen Geno-Immune Medical Institute Not yet recruiting
NCT07287397
Last update post date Previous: 2026-06-23 Current: 2026-08-26 api
Vector type Previous: AAV5.2 Current: Adeno-associated virus curator
Dose 2 Previous: Undisclosed high dose Current: curator
Dose 1 Previous: Undisclosed low dose Current: Undisclosed dose escalation, 2 levels curator
Intervention description Previous: An investigational gene therapy targeting a specif... Current: An investigational gene therapy targeting a specif... api
Intervention name Previous: Vtx-002, Optional rescue medication - corticostero... Current: Vtx-002, Preventative (prophylactic) medication - ... api
Eligibility criteria Previous: null Current: Key inclusion criteria: 1. capable of, And willin... api
Record status Previous: Draft Current: Active curator
Preclinical publications Previous: null Current: LinkName:(Poster) AAV-mediated intrabody therapy t... curator
Preclinical publications Previous: null Current: LinkName:(Poster) VecTabs® and advanced human in-v... curator
Preclinical publications Previous: null Current: LinkName:(Poster) AAV-mediated intrabody therapy t... curator
Preclinical publications Previous: null Current: LinkName:(Poster) Non-clinical development and Pha... curator
Indication doid Previous: null Current: DOID:332 curator
Development status Previous: null Current: Active curator
Compound name Previous: null Current: VTx-002 curator
Recent updates Previous: null Current: FDA cleared IND 12/25 curator
Delivery system Previous: null Current: viral transduction curator
Drug product type Previous: null Current: viral vector curator
Route of administration Previous: null Current: intracisternal curator
Mechanism of action Previous: null Current: genetic delivery of therapeutic protein curator
Therapy route Previous: null Current: in-vivo curator
Therapy type Previous: null Current: gene transfer curator
Target gene Previous: null Current: antibody to TDP-43 curator
Fda designation Previous: Current: Fast Track curator
News and press releases Previous: null Current: LinkName:VectorY Therapeutics Receives FDA Clearan... curator
News and press releases Previous: null Current: LinkName:VectorY Therapeutics Receives FDA Fast Tr... curator
1787720400000
Amyotrophic Lateral Sclerosis
Vector Y Therapeutics Recruiting
FDA cleared IND 12/25
NCT05230459
Last update post date Previous: 2026-02-20 Current: 2026-08-26 api
Overall status Previous: Recruiting Current: Active not recruiting api
Enrollment type Previous: Estimated Current: Actual api
Enrollment count Previous: 10 Current: 9 api
Vector type Previous: AAV9 Current: Adeno-associated virus curator
Dose 2 Previous: Undisclosed dose 2 Current: curator
Dose 1 Previous: Undisclosed dose 1 Current: Undisclosed dose escalation, 2 levels curator
Editor type Previous: none Current: curator
Eligibility criteria Previous: null Current: Inclusion criteria: 1. male and female subjects a... api
1787720400000
Limb-Girdle Muscular Dystrophy, Type 2I/R9
AskBio Inc Active not recruiting
First patient dosed (8/3/23)
27 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.