
SRPT
Sarepta Therapeutics develops treatments using proprietary technology platforms including phosphorodiamidate morpholino oligomers (PMO), gene therapy, and small interfering RNA (siRNA) to address a broad range of diseases. The company has marketed products for muscular dystrophy and other conditions, while maintaining a pipeline of clinical and development-stage candidates supported by manufacturing partnerships. Sarepta faced a clinical hold on gene therapy trials for limb-girdle muscular dystrophy in July 2025 and is conducting a confirmatory trial for two of its marketed products.
Pipeline
Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping.