Key information

🔎 Fund Overview

Sarepta Therapeutics, Inc. is a commercial-stage biopharmaceutical company. The Company is focused on the discovery and development of ribonucleic acid (RNA)-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. It develops therapeutic candidates for a range of diseases and disorders, including Duchenne, Limb-girdle muscular dystrophies (LGMDs) and other neuromuscular and central nervous system (CNS) related disorders. It commercializes three products, such as EXONDYS 51 (eteplirsen) Injection (EXONDYS 51), VYONDYS 53 (golodirsen) Injection (VYONDYS 53) and AMONDYS 45 (casimersen) Injection (AMONDYS 45). These commercial products are indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51, exon 53 and exon 45 skipping. Its pipeline includes approximately 40 programs at various stages of discovery, pre-clinical and clinical development.