NEW YORK (GenomeWeb) – CureDuchenne Ventures has provided $5 million in seed financing to Exonics Therapeutics, a new firm exploring the use of CRISPR/Cas9 in the development of new treatments for Duchenne muscular dystrophy (DMD).
Exonics said in a statement that it will use the proceeds to advance preclinical research from the lab of founder Eric Olson using adeno-associated viruses to deliver CRISPR to cells in order to identify and correct exon mutations in DMD patients. Olson is also a professor and researcher at the University of Texas-Southwestern Medical Center whose work on correcting DMD mutations with CRISPR/Cas9 has led to improvement in a live animal model of the disease.
CureDuchenne Ventures is a subsidiary of the non-profit CureDuchenne, which seeks to improve the standard of care and living of boys with DMD, a genetic disease that hampers the production of the muscle fiber protein dystrophin. The group collaborates with biotech firms to develop therapies for DMD.
"Exonics' CRISPR/Cas9 technology has the potential to dramatically improve the lives of those who live with Duchenne," CureDuchenne CEO Debra Miller said in a statement. "We are hopeful that a one-time treatment with gene editing therapy could provide a lifelong benefit to Duchenne patients."