FDA has approved CASGEVY™, a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises.
Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced today that FDA has approved CASGEVY™ (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).…