Exonic Therapeutics

At Exonics Therapeutics, our mission is to permanently correct genetic mutations resulting in neuromuscular diseases like Duchenne muscular dystrophy. Duchenne is a devastating muscle disease in children, primarily boys, caused by the absence of dystrophin. Our approach utilizes gene editing technologies such as CRISPR/Cas9 to correct a majority of the genetic mutations in Duchenne, and enable dystrophin production.