Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency Post author: Post published:March 25, 2026 Post category: Continue ReadingClinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations Post author: Post published:March 25, 2026 Post category: Continue ReadingA Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia Post author: Post published:March 25, 2026 Post category: Continue ReadingOpen-Label Study of mRNA-3927 in Participants With Propionic Acidemia
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency Post author: Post published:March 25, 2026 Post category: Continue ReadingAn Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) Post author: Post published:March 25, 2026 Post category: Continue ReadingPhase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease Post author: Post published:March 25, 2026 Post category: Continue ReadingA Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
A Study to Evaluate the Efficacy and Safety of Sefaxersen (RO7434656) in Participants With Primary Immunoglobulin A (IgA) Nephropathy at High Risk of Progression Post author: Post published:March 25, 2026 Post category: Continue ReadingA Study to Evaluate the Efficacy and Safety of Sefaxersen (RO7434656) in Participants With Primary Immunoglobulin A (IgA) Nephropathy at High Risk of Progression
A Phase 1/2 Trial of ADI-270 in ccRCC Post author: Post published:March 17, 2026 Post category: Continue ReadingA Phase 1/2 Trial of ADI-270 in ccRCC
T-Cell Therapy (ECT204) in Adults With Advanced HCC Post author: Post published:March 17, 2026 Post category: Continue ReadingT-Cell Therapy (ECT204) in Adults With Advanced HCC
Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein Post author: Post published:March 1, 2026 Post category: Continue ReadingStudy to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
