Exonics Raises $40 Million to Advance CRISPR Technology to Treat DMD

Rare Daily Staff

Exonics Therapeutics said it completed a series A financing to advance development of its CRISPR technology to repair mutations causing neuromuscular diseases including Duchenne muscular dystrophy, a progressive and fatal condition.

The venture capital firm The Column Group provided the funding. The Column Group’s Managing Partner David Goeddel and Principal JJ Kang, will be joining the company’s board of directors.

“This funding from a leading healthcare venture capital firm further validates the potential for Exonics’ novel gene editing technology to help correct many of the mutations that cause Duchenne and other neuromuscular diseases,” said John Ripple, chief executive officer of Exonics.

Exonics is conducting preclinical development of a one-time gene editing treatment leveraging SingleCut CRISPR to potentially provide lifelong benefit for patients with Duchenne. SingleCut CRISPR is an approach to make minimal and precise genetic modifications and to use the cell’s natural mechanisms to repair the faulty gene in DMD and induce normal expression of dystrophin. Dystrophin is the key protein missing in children with Duchenne that helps stabilize and protect muscle fibers.

Published preclinical data in mice suggest the therapy has the potential to identify and repair exon mutations that prevent the production of dystrophin. Exonics is initially focusing DMD patients with an exon 51 mutation, which represents the largest group of DMD patients. It plans to pursue of DMD mutations later.

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, Exonics scientific founder and chief science advisor. CureDuchenne provide funding for the research and CureDuchenne Ventures provided seed financing for the company.

Exonics is not alone in seeking to use CRISPR technology to treat DMD. GenEdit is using an approach developed at the University of California, Berkeley. Sarepta Therapeutics at the end of last month announced a collaboration with Duke University, which gave it an option to an exclusive license to intellectual property and technology related to CRISPR/Cas9 technology to restore dystrophin expression in DMD patients. 

“Exonics’ transformative gene repair technology presents a promising opportunity to create novel therapies that address the significant unmet need in the treatment of serious genetic neuromuscular diseases,” said The Column Group’s Goeddel. “The company has generated compelling early data, and we are pleased to support Exonics as it advances its preclinical development program in Duchenne closer to the clinic.”

November 9, 2017