Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia

This phase 3 study evaluated exagamglogene autotemcel (exa-cel), a CRISPR Cas9 gene edited autologous cell therapy designed to reactivate fetal hemoglobin production in patients with transfusion dependent β thalassemia. Among 35 patients with sufficient follow up, 91% achieved transfusion independence for at least 12 consecutive months, with sustained increases in total and fetal hemoglobin levels. Fetal hemoglobin showed pancellular distribution, and iron overload markers improved over time. No deaths or cancers occurred, and the safety profile was consistent with myeloablative busulfan conditioning and autologous transplantation.

This phase 3 study evaluated exagamglogene autotemcel (exa-cel), a CRISPR Cas9 gene edited autologous cell therapy designed to reactivate fetal hemoglobin production in patients with transfusion dependent β thalassemia. Among 35 patients with sufficient follow up, 91% achieved transfusion independence for at least 12 consecutive months, with sustained increases in total and fetal hemoglobin levels. Fetal hemoglobin showed pancellular distribution, and iron overload markers improved over time. No deaths or cancers occurred, and the safety profile was consistent with myeloablative busulfan conditioning and autologous transplantation.