A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia Post author: Post published:April 20, 2026 Post category: Continue ReadingA Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
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:April 20, 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
Gene Modified Immune Cells After Conditioning Regimen for the Treatment of Stage IIIC or IV Melanoma or Metastatic Solid Tumors Post author: Post published:April 20, 2026 Post category: Continue ReadingGene Modified Immune Cells After Conditioning Regimen for the Treatment of Stage IIIC or IV Melanoma or Metastatic Solid Tumors
Pembrolizumab and a Vaccine (ATL-DC) for the Treatment of Surgically Accessible Recurrent Glioblastoma Post author: Post published:April 20, 2026 Post category: Continue ReadingPembrolizumab and a Vaccine (ATL-DC) for the Treatment of Surgically Accessible Recurrent Glioblastoma
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) Post author: Post published:April 20, 2026 Post category: Continue ReadingA Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
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
