Precision BioSciences is developing a novel gene editing approach for individuals with dystrophin mutations in the “hot spot” region of exons 45-55, with the goal of submitting applications later this year to begin clinical trials in the US and/or Canada.
Precision’s technology is a gene editing approach, which aims to permanently alter an individual’s own copy of the dystrophin gene. It uses ARCUS nucleases, delivered by AAV9, to cut out exons 44-55 of the dystrophin gene. This would produce a functional dystrophin protein that, when naturally occurring in humans, has been associated with mild cases of Becker muscular dystrophy. It is estimated that up to 50-60% of individuals with Duchenne contain mutations in this “hot spot” region, and thus could benefit from excising the whole region in order to restore dystrophin protein expression.
This work is still at the preclinical stage, but Precision has shared additional data from Duchenne mice studies, reporting increases over time in dystrophin-positive muscle cells in key skeletal muscle types, as well as heart and diaphragm. Together with previous data reporting increases in dystrophin protein levels and improvements in muscle force, this data indicates that Precision’s approach to gene editing can produce sustained benefits in mice.
Precision plans to present all the data at a future scientific conference, and complete the other research it would need before beginning clinical trials. While it’s still early days, if Precision is successful, it would be the first gene editing program for Duchenne to reach clinical trials.
Link to press release HERE
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