Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

The authors conducted an open-label, single-group, Phase III study of exa-cel in patients 12 to 35 years of age with transfusion-dependent β-thalassemia and a β0/β0, β0/β0-like, or non–β0/β0-like genotype.