Our Science
Advancing the promise of precision genetic medicine
Sarepta is pursuing the development of precision genetic medicine at the forefront of biotechnology for rare diseases.
Disease State
A genetic mutation in DNA causing absent or dysfunctional protein can lead to a neuromuscular disease such as Duchenne muscular dystrophy (DMD) or limb-girdle muscular dystrophy (LGMD).
RNA Technologies
DMD is caused by a genetic mutation in the dystrophin gene. Most commonly, one or more exons (parts of the gene) are missing, causing errors in the instructions for making dystrophin, which results in the body not being able to produce enough—or any—working dystrophin protein. The goal of exon skipping, and that of Sarepta’s proprietary technologies, is to act on the RNA to allow the body to make a shorter form of the dystrophin protein to bypass the mutation.
Gene Therapy
The goal of gene therapy is to add a functional copy (a transgene) of a missing or malfunctioning gene, with the hope of treating the disease.