Exa-cel review sets 2023 up to be a milestone year for CRISPR gene editing

There’s a new timeline for an FDA decision on exa-cel from CRISPR Therapeutics and Vertex that confirms 2023 is the year that could see the first approval of a CRISPR-based therapy, and new data from Editas suggest multiple gene editing success stories could be in store from sickle cell and β-thalassemia patients.

CRISPR Therapeutics Inc. (NASDAQ:CRSP) and Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) announced on Friday that FDA granted priority review to exagamglogene autotemcel (exa-cel) for the severe sickle cell disease indication, leading to a Dec. 8 PDUFA date. It also accepted the BLA for transfusion-dependent β-thalassemia, which will have a standard review and a March 30, 2024, PDUFA date...