CureDuchenne Ventures, a subsidiary of nonprofit CureDuchenne, a global leader in Duchenne-related research and patient care, has announced $5 million in seed financing from Boston-based Exonics Therapeutics.

The investment will enable Exonics, a newly formed biotechnology company focused on developing gene editing technologies such as CRISPR/Cas9 to permanently correct mutations that cause Duchenne and other neuromuscular diseases, to advance the preclinical research of its founder and chief science advisor, Eric Olson, PhD. Duchenne, a rare X-linked genetic muscle disease, affects nearly fifteen thousand boys in the U.S. and more than three hundred thousand boys worldwide. Children with the disease start missing development milestones at the age of 3 and often lose their ability to walk by the age of 12. All those with Duchenne eventually experience respiratory or cardiac failure, resulting in a reduced life expectancy in the mid-20s. There is currently no cure for the disease.

Led by Olson, Exonics has demonstrated the ability to use adeno-associated virus to deliver a payload based on CRISPR/Cas9 technology that can identify and correct exon mutations that prevent the production of dystrophin, a protein missing in boys with Duchenne that helps stabilize and protect muscle fibers. Preclinical data suggest that the approach has the potential to permanently treat up to 80 percent of children suffering from the disease.

"We are building on a scientific program of exceptional quality from Dr. Olson's laboratory with the support of our funder CureDuchenne Ventures, an organization with deep understanding of this disease, therapeutic landscape, and large unmet need," said Exonics executive board chair Cristina Csimma. "This funding underscores their recognition of the potential of Exonics' gene editing method to develop a novel therapy for Duchenne. Exonics is extremely well-positioned to advance our preclinical program as we follow a rigorous approach to enable translation to clinical trials."