Sarepta Therapeutics

What is Sarepta Therapeutics?

Serepta Therepeutics, formerly AVI BioPharma, is a biotechnology company that is headquartered in Cambridge, Massachusetts, and was founded in 1980 by Marwan Fawaz. The company is developing gene therapies and medicines for the treatment of neuromuscular diseases such as Duchenne Muscular Dystrophy (DMD), Limb-Girdle Muscular Dystrophy (LGMD), and Charcot-Marie-Tooth (CMT). Serepta Therapeutics creates their gene therapies and medicines using multiple therapeutic approaches which include RNA, gene therapy, and gene editing.

• Genethon
• Murdoch University
• University of Western Australia

The company has worked with phosphorodiamidate morpholino oligomer chemistries in order to provide therapies for muscular dystrophy. The PMOs that the company works with are synthetic molecules that are modeled after the natural framework of RNA, and has the nucleic acid bases found in RNA.

They are able to hide specific exons, or skip their location if they are missing in order to provide a proper connection and create a functional dystrophin protein.