Advancing gene editing technology to develop life-changing therapies for devastating neuromuscular diseases

At Exonics Therapeutics, our mission is to 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.

Our technology is licensed from the University of Texas Southwestern Medical Center (UTSW) and is based on the research of our scientific founder and chief science advisor, Eric Olson, PhD. Preclinical data suggest that our novel approach has the potential to permanently treat up to 80 percent of children who suffer from Duchenne.

We are dedicated to working closely with all members of the Duchenne community to address the significant unmet need for a curative Duchenne therapy.

In addition to our initial focus on Duchenne, we look forward to exploring our approach to gene editing in other pediatric indications with unmet medical need.

Publications

Long, C., Amoasii, L., Bassel-Duby, R., and Olson, E.N. (2016). Genome Editing of Monogenic Neuromuscular Diseases. JAMA Neurology. 73 :1349-1355. Read more.

Long, C., Amoasii, L., Mireault, A.A., McAnally, J.R., Li, H., Sanchez-Ortiz, E., Bhattacharyya, S., Shelton, J.M., Bassel-Duby, R., and Olson, E.N. (2016). Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science 351, 400–403. Read more.

Long, C., McAnally, J.R., Shelton, J.M., Mireault, A.A., Bassel-Duby, R., and Olson, E.N. (2014). Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA. Science 345, 1184–1188. Read more.