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
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
Stem Cell Gene Therapy for Sickle Cell Disease Post author: Post published:March 25, 2026 Post category: Continue ReadingStem Cell Gene Therapy for Sickle Cell Disease
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
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
CCL21-Gene Modified Dendritic Cell Vaccine and Pembrolizumab in Treating Patients With Stage IV Non-small Cell Lung Cancer Post author: Post published:March 25, 2026 Post category: Continue ReadingCCL21-Gene Modified Dendritic Cell Vaccine and Pembrolizumab in Treating Patients With Stage IV Non-small Cell Lung Cancer
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
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)
