Exagamglogene Autotemcel for Severe Sickle Cell Disease

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 severe sickle cell disease. Among 44 treated patients, 97% were free from vaso-occlusive crises for at least 12 consecutive months, and 100% were free from hospitalization for such crises over the same period. The therapy led to sustained increases in total and fetal hemoglobin, normalization of hemolysis markers, and improved quality of life. No cases of cancer or graft failure were reported.

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 severe sickle cell disease. Among 44 treated patients, 97% were free from vaso-occlusive crises for at least 12 consecutive months, and 100% were free from hospitalization for such crises over the same period. The therapy led to sustained increases in total and fetal hemoglobin, normalization of hemolysis markers, and improved quality of life. No cases of cancer or graft failure were reported.