Exonics Therapeutics
Biotechnology ResearchMassachusetts, United States11-50 Employees
Starting with Duchenne muscular dystrophy, Exonics is translating SingleCut CRISPR gene editing technology into therapies for genetic neuromuscular diseases with significant unmet need. Exonics Therapeutics was launched in February 2017 to advance the research of our founder, Dr. Eric Olson and his laboratory at the University of Texas Southwestern Medical Center (UTSW) to develop treatments for patients with neuromuscular diseases. Dr. Olson is one of the world’s leading experts in the study of muscle cells and the application of gene editing to treat these types of diseases. In particular, Dr. Olson’s laboratory has used adeno-associated virus (AAV) to deliver a CRISPR/Cas9 technology that can identify and repair exon mutations to restore the production of dystrophin, a protein that helps stabilize and protect muscle fibers. Dystrophin is the protein missing in boys with Duchenne. The loss of dystrophin causes Duchenne. Initial Program in Duchenne Exonics is initially focused on repairing the most frequent mutations that cause Duchenne muscular dystrophy, for which there is currently no cure. Preclinical data suggest Exonics’ novel SingleCut CRISPR gene editing has the potential to treat a large proportion of boys who suffer from Duchenne. This is achieved by repairing errors in the DNA sequence that causes mutations of dystrophin, the protein missing in boys with Duchenne. Though our primary focus is on Duchenne, we plan to expand our therapeutic focus to include other related neuromuscular diseases where our proprietary SingleCut CRISPR gene editing may provide a significant clinical benefit.
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