FDA approved CASGEVY™ for the treatment of transfusion-dependent beta thalassemia in patients 12 years and older.
Vertex Pharmaceuticals announced today that FDA has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older. “On the heels of the historic FDA approval of…