Assessing beti-cel for hemoglobin production in TDT
Kwiatkowski JL, Thompson AA, Schneiderman J, et al. Long-Term Efficacy and Safety Results of Betibeglogene Autotemcel Gene Therapy for Transfusion-Dependent ß-Thalassemia. Blood. 2026; (doi: 10.1182/blood.2025029196).
Research findings showcase betibeglogene autotemcel (beti-cel) as a viable and potentially curative intervention for transfusion-dependent ß-thalassemia (TDT). In this setting, hematopoietic stem and progenitor cells used in autologous transplantation are transduced with a modified ß-globin gene to support functional production of adult hemoglobin. Process improvements since Phase I/II studies were conducted have led to better transduction efficiency, which in turn has boosted hemoglobin rates and transfusion independence (TI). In the Phase III study, 90.2% of 41 participants achieved TI status versus 68.2% of 22 patients in the Phase I/II sample population. TI was sustained for as long as a decade after a single administration of the stem cell-directed gene therapy, which investigators say offers long-term safety similar to that observed with myeloablative autologous transplantation. In addition to the TI benefit, beti-cel normalized iron balance and improved quality of life.