Base editing therapy shows durable remission in sickle cell disease and beta-thalassemia patients from diverse backgrounds
A study published in Cell Stem Cell on September 7, 2026, shows that CS-101/CS-206, a base-editing therapy, achieved consistent efficacy and safety in patients with beta-hemoglobinopathies from different genetic backgrounds. The study included four patients from Nigeria, Laos, Malaysia, and Pakistan, all of whom achieved transfusion independence or freedom from vaso-occlusive crises.