Exonics Therapeutics, a biotechnology company focused on developing SingleCut CRISPR technology to repair mutations causing Duchenne muscular dystrophy and other neuromuscular diseases, today announced it has secured a $40 million Series A financing to advance its lead gene editing program in Duchenne from The Column Group (TCG), a science-driven venture capital firm. Exonics also announced that TCG's David Goeddel, Ph.D., managing partner, and JJ Kang, Ph.D., principal, will be joining the company's board of directors.

Exonics 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, a devastating muscle disease in children for which there is no cure.

"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 Dr. 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."

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, Ph.D., Exonics' founder and chief science advisor.

"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. "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."

"As a leading research organization focused on Duchenne muscular dystrophy, CureDuchenne, through CureDuchenne Ventures, is proud to support the work of Dr. Eric Olson, and we are hopeful that Exonics' CRISPR/Cas9 gene editing technology will ultimately offer a significant and impactful treatment for those affected by Duchenne," said Debra Miller, chief executive officer and founder of CureDuchenne.

About Exonics Therapeutics

Exonics Therapeutics has developed SingleCut CRISPR, a gene repair technology that has the potential to effectively halt the progression of certain genetic neuromuscular diseases. In multiple Duchenne muscular dystrophy preclinical models, Exonics has used SingleCut CRISPR to genetically repair and restore dystrophin, the key protein missing in children with Duchenne. Exonics is initially focused on correcting mutations that cause Duchenne in order to develop a therapy to treat many children with the devastating disease, for which there is no cure. Exonics' technology is licensed from the University of Texas Southwestern Medical Center and is based on the research of Eric Olson, Ph.D., Exonics' founder and chief science advisor. Exonics is located in Cambridge, Mass. For more information, please visit www.exonicstx.com.

About The Column Group

The Column Group (TCG) is a leading venture capital firm based in San Francisco that seeks to partner with exceptional scientific founders, entrepreneurs, executives, and investment organizations with a shared vision for building the next generation of drug discovery and development companies. TCG invests in disease-focused drug discovery companies with the potential to become leaders in their respective fields. These companies are strongly supported by the unique and complementary skill sets of TCG's team, which includes prominent authorities in the scientific, operational, and financial arenas. For more information, please visit www.thecolumngroup.com.

About CureDuchenne

CureDuchenne was founded in 2003 with a focus on saving the lives of those with Duchenne muscular dystrophy, a disease that affects more than 300,000 boys worldwide. With support from CureDuchenne, nine research projects have advanced to human clinical trials. CureDuchenne is also the innovator in bringing physical therapy and standard of care to local communities around the country through CureDuchenne Cares. For more information, please visit www.CureDuchenne.org and follow us on Facebook, Twitter, Instagram and YouTube.