On Dec. 8, 2023, Vertex Pharmaceuticals and CRISPR Therapeutics announced that the U.S. Food and Drug Administration (FDA) had approved CASGEVYﾙ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

This approval meant that for the first time, approximately 16,000 patients with SCD may be eligible for a durable one-time therapy that offers the potential of a functional cure for their disease by eliminating severe VOCs and hospitalizations caused by severe VOCs.