FDA AdCom largely supports safety of Vertex/CRISPR's exa-cel

The US Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Committee concluded that Vertex Pharmaceutical (Nasdaq: VRTX) and Swiss gene editing specialist CRISPR Therapeutics’ (Nasdaq: CRSP) exagamglogene autotemcel (exa-cel), intended for the treatment of sickle cell disease, is generally safe for clinical use.

Yesterday’s AdCom meeting followed the FDA’s briefing papers assertion that the drug is effective, helping 29 of 30 patients stay pain-free for 18 months.

By year’s end, exa-cel could become the first FDA-approved drug using CRISPR, opening the door for a medical revolution in gene-edited therapies - and cures.