Exa-cel meeting could help set standards for off-target editing

CRISPR gene editing is approaching its biggest milestone yet as FDA reviews the field’s first application, exa-cel from CRISPR Therapeutics and Vertex. The only question that’s still standing in the way is the same one that’s been dogging gene editing developers for 11 years: whether off-target edits are going to be a problem. 

Next Tuesday’s meeting of FDA’s Cellular, Tissue and Gene Therapy Advisory Committee will convene to discuss the application for exagamglogene autotemcel (exa-cel), an ex vivo gene editing therapy to treat sickle cell disease (SCD), from CRISPR Therapeutics Inc. (NASDAQ:CRSP) and Vertex Pharmaceuticals Inc. (NASDAQ:VRTX). ...