On Feb. 13, 2024, CRISPR Therapeutics announbed that the European Commission had granted conditional marketing authorization to CASGEVYﾙ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene edited therapy.

CASGEVY was approved for the treatment of patients who are 12 years of age and older with severe sickle cell disease (SCD) characterized by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT), for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.