Cell-Based Gene Therapy for ?-Thalassemia

ABSTRACT

The United States Food and Drug Administration (FDA) approved betibeglogene autotemcel (beti-cel), the first cell-based gene therapy for adult and pediatric patients with ?-thalassemia in August, 2022. This update details this and other novel therapies that have emerged in the treatment of ?-thalassemia, apart from transfusion and iron chelation, with particular focus on newly approved gene therapy