Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease

This study evaluates the efficacy and safety of exagamglogene autotemcel (exa-cel), a gene-editing cell therapy, in children aged 5 to 11 with transfusion-dependent β-thalassemia or sickle cell disease. The authors aimed to determine whether exa-cel could achieve transfusion independence in β-thalassemia patients and freedom from severe vaso-occlusive crises in sickle cell disease patients over a follow-up period of at least 16 months. Results indicated that all evaluated children achieved the desired clinical outcomes, although all experienced significant adverse events related to the treatment.