Exonics Therapeutics Email Format

BiotechnologyMassachusetts, United States11-50 Employees

Exonics Therapeutics is a biotechnology company focused on developing gene-editing therapies for genetic neuromuscular diseases, beginning with Duchenne muscular dystrophy. It was founded to advance the work of founder Dr. Eric Olson and his laboratory at the University of Texas Southwestern Medical Center, with the aim of translating that research into treatments that restore dystrophin production in muscle tissue. Headquartered in Cambridge, Massachusetts, the company was established in 2017 to pursue this translational effort.

Its initial program targets the most common mutations that cause Duchenne, and preclinical data describe potential to treat a large proportion of affected boys. Although Duchenne is the primary focus, Exonics plans to expand its therapeutic scope to other related neuromuscular diseases where its gene-editing approach could offer clinical benefit. Operating in the biotechnology research sector, Exonics positions itself as a developer of experimental genetic therapies from its Cambridge base with a relatively small team.

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