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.

Endpoints News

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.