Sarepta Therapeutics Halts Development of Vesleteplirsen, a Next-Generation Exon-Skipping Therapy for Duchenne Muscular Dystrophy

Sarepta Therapeutics, Vesleteplirsen (SRP-5051), Duchenne Muscular Dystrophy (DMD), Exon-Skipping Therapy, FDA Feedback, Therapeutic Landscape

FDA Mandates Enhanced Safety Warnings for CAR-T Therapies Due to Secondary Cancer Risks

CAR-T therapy, secondary cancers, FDA boxed warnings, T-cell malignancies, BCMA-directed, CD19-directed, autologous CAR T-cell immunotherapies.

Vertex’s CASGEVY Marks Historic Milestone with First Commercial Patient Infusions, Paving the Way for 2025 Launches

CASGEVY, Vertex Pharmaceuticals, gene therapy, sickle cell disease, beta thalassemia, CRISPR/Cas9, FDA approval, commercial patient infusions, 2025 launches.