AVI Research

Sarepta Therapeutics, formerly known as AVI BioPharma (AVI Research), is a commercial-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapeutics and gene therapies for rare diseases. The company is a leader in the treatment of Duchenne muscular dystrophy (DMD), having secured FDA approvals for several antisense oligonucleotide drugs including Exondys 51, Vyondys 53, and Amondys 45. Beyond its RNA platform, Sarepta is advancing a robust pipeline of gene therapy candidates, most notably Elevidys, the first gene therapy approved for DMD. The company utilizes its proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry to develop precision medicines that skip specific exons to restore protein function.