On Jan. 16, 2024, the U.S. Food and Drug Administration (FDA) announced it had approved CRISPR Therapeutics’ 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.

DT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and the lifetime health care costs in the U.S. of managing TDT are estimated between $5 and $5.7 million.