Exonics Therapeutics has secured $40 million in financing from The Column Group (TCG) — a science-driven venture capital firm — to develop its lead gene editing program in Duchenne muscular dystrophy (DMD).
Exonics, based in Cambridge, Massachusetts, is working to develop a safe, one-time gene editing treatment that would provide lifelong benefit for patients with Duchenne.
This treatment is based on the company’s SingleCutCRISPR technology, which it says can potentially repair a defective gene in a living person. This includes the most common defects in the dystrophin gene that cause DMD. The technology is licensed from the University of Texas Southwestern Medical Center and stems from research by Dr. Eric Olson, Exonics’ scientific founder and chief science advisor.
Mice with DMD respond to the technology with dystrophin production, as well as improved structure and function of both skeletal muscle and the heart, without any apparent side effects, the company reports.
“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,” TCG Managing Partner David Goeddel said in a press release. “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.”
Both Goeddel and J.J. Kang will be joining the Exonics 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 Exonics CEO John Ripple. “We look forward to working with TCG to translate our science into a meaningful treatment for the many Duchenne patients and their families. We’re grateful to CureDuchenne Ventures for its support in founding the company and providing the seed financing, which has enabled Exonics to establish a strong scientific and corporate foundation to build upon.”
Debra Miller, CEO and founder of CureDuchenne, said her nonprofit group is proud to support Olson’s work, and that it hopes “Exonics’ CRISPR/Cas9 gene editing technology will ultimately offer a significant and impactful treatment for those affected by Duchenne.”