Advance CRISPR/Cas9 Therapy for Duchenne
Exonics Therapeutics is focused on developing gene editing technologies like CRISPR/Cas9 to correct a majority of mutations causing Duchenne muscular dystrophy and other neuromuscular diseases. Dr. Olson’s laboratory has demonstrated the ability to use adeno-associated virus (AAV) to deliver a payload based on CRISPR/Cas9 technology that can identify and correct exon mutations that prevent the production of dystrophin, a protein that helps stabilize and protect muscle fibers. Dystrophin is the key protein missing in boys with Duchenne, and published preclinical data suggest that this approach has the potential to treat up to 80 percent of children suffering from the disease. Funding will allow Exonics to advance the preclinical research of its scientific founder and chief science advisor Eric Olson, PhD.