Exonics Therapeutics closed on a $40 million Series A financing. It was led by The Column Group.

Exonics focuses on developing SingleCut CRISPR technology with the intention of repairing genetic mutations that cause Duchenne muscular dystrophy (DMD) and other neuromuscular diseases. DMD is a muscle-wasting disease that primarily affects young boys, leading to muscular weakness and eventually death, often in their twenties. It is caused by mutations in the DMD gene that prevents the production of dystrophin, a key protein that helps stabilize and protect muscle fibers.

The technology that Exonics is based on originated from the University of Texas Southwestern Medical Center and the laboratory of Eric Olson, the company’s founder and chief scientific advisor.

“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 David Goeddel, managing partner of The Column Group and board member of Exonics, in a statement. “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.”

Also joining Goeddel on the board is JJ Kang, principal with The Column Group.

Much attention has been paid to Sarepta Therapeutics’ Exondys 51 for DMD and its tumultuous journey to approval by the U.S. Food and Drug Administration (FDA) last year. However, Exondys 51, whose efficacy is still in doubt, only applies to about 13 percent of DMD patients. And the DMD gene is so large that it is not currently a candidate for gene-therapy approaches using adeno-associated virus (AAV) as a vector.

Exonics has published preclinical data in mice that suggest its approach had the potential to repair the mutations in the gene. “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, Exonics’ chief executive officer, in a statement. “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, CureDuchenne’s chief executive officer and founder, said in a statement, “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.”