Exonics Therapeutics, Inc., a Cambridge, Mass.-based biotechnology company, secured $40m in Series A financing.
The Column Group (TCG), a science-driven venture capital firm, made the investment. In conjunction with the funding, TCG’s David Goeddel, Ph.D., managing partner, and JJ Kang, Ph.D., principal, will be joining the company’s board of directors.
The company intends to use the funds to advance its lead gene editing program in Duchenne.
Led by Eric Olson, Ph.D., founder and chief science advisor, and John Ripple, chief executive officer, Exonics Therapeutics focuses on developing SingleCut CRISPR technology to repair mutations causing Duchenne muscular dystrophy and other neuromuscular diseases.
The company is generating data to support the development of a safe and efficacious one-time gene editing treatment leveraging SingleCut CRISPR to potentially provide lifelong benefit for patients with Duchenne.
Published preclinical data in mice suggest the therapy has the potential to identify and repair exon mutations that prevent the production of dystrophin, the key protein missing in children with Duchenne that helps stabilize and protect muscle fibers. The technology underlying Exonics’ gene editing approach is licensed from the University of Texas Southwestern Medical Center and is based on research from the laboratory of Eric Olson.