BioCentury
ARTICLE | Clinical News

Exonics shows CRISPR treats DMD in dogs

September 7, 2018 5:43 PM UTC

Exonics Therapeutics Inc. (Boston, Mass) is one step closer to bringing its SingleCut CRISPR gene editing Duchenne muscular dystrophy treatment to the clinic after preclinical data showed the therapy led to dystrophin restoration of up to 92% in a canine model of the disease. The company published the results in Science along with researchers from University of Texas Southwestern Medical Center, from whom Exonics licensed the gene editing technology.

Exonics CEO John Ripple told BioCentury the paper "is one of the important milestones on our path toward the clinic" but declined to disclose a development timeline...