As R&D setbacks multiply, a pioneering CRISPR/Cas9 program for Duchenne MD continues to impress in new dog study
While one drug after the next seems to get regularly shot down in Duchenne muscular dystrophy, as we saw today with Pfizer’s failed Phase II, a preclinical CRISPR gene editing program has been making some impressive progress in animal studies.
Thursday afternoon, Exonics provided some intriguing details to show how their gene-editing approach works in dogs. Dispatching a Cas9 scalpel with guide RNA inside an AAV delivery vehicle straight to muscle cells, the researchers say that their approach was able to restore dystrophin production that reached up to 92% of normal in a canine model for the disease.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.