Betibeglogene Autotemcel Gene Therapy for Transfusion-Dependent β-Thalassemia

Betibeglogene autotemcel (beti-cel) is an FDA-approved lentiviral vector-based gene therapy for transfusion-dependent β-thalassemia (TDT) that introduces functional copies of a modified β-globin gene into hematopoietic stem and progenitor cells (HSPC). A landmark study showed that beti-cel helped patients with nonsevere genotypes achieve transfusion independence (N Engl J Med 2018; 378:1479).

To determine the impact of beti-cel in patients with severe TDT genotypes, including β0/β0 or IVS-I-110 mutations, investigators enrolled 18 patients (median age, 12.5 years; range, 4 –33 years) in an industry-supported, multinational, phase 3 study. Patients underwent HSPC mobilization before CD34+ cells were collected via apheresis, transduced with BB…