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
Stem Cell Therapy for Limbal Stem Cell Deficiency Post author: Post published:March 25, 2026 Post category: Continue ReadingStem Cell Therapy for Limbal Stem Cell Deficiency
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
A Study of the Safety and Tolerability of ASP7317 in Senior Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration Post author: Post published:March 25, 2026 Post category: Continue ReadingA Study of the Safety and Tolerability of ASP7317 in Senior Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration
Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector Post author: Post published:March 25, 2026 Post category: Continue ReadingGene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
Gene Therapy for Cardiomyopathy Associated With Friedreich’s Ataxia Post author: Post published:March 25, 2026 Post category: Continue ReadingGene Therapy for Cardiomyopathy Associated With Friedreich’s Ataxia
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
Expanded Access Program (EAP) for Ciltacabtagene Autoleucel (Cilta-Cel) Out-of-Specification (OOS) in Participants With Multiple Myeloma Post author: Post published:March 17, 2026 Post category: Continue ReadingExpanded Access Program (EAP) for Ciltacabtagene Autoleucel (Cilta-Cel) Out-of-Specification (OOS) in Participants With Multiple Myeloma
