Exonics Therapeutics is developing gene editing therapies to treat patients with Duchenne muscular dystrophy and other severe genetic neuromuscular diseases. In multiple Duchenne preclinical models, Exonics has used SingleCut CRISPR to genetically repair and restore dystrophin, the key protein missing in children with Duchenne. Exonics is initially focused on repairing mutations that cause Duchenne in order to develop a therapy to treat many children with the devastating disease, for which there is no cure. Exonics’ technology is licensed from UT Southwestern Medical Center and is based on the research of Eric Olson, Ph.D., Exonics’ founder and chief science advisor.
