In September 2022, the FDA has approved ZYNTEGLO® (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. 89% achieved transfusion independence with Hb > 8 g/dL. Common side effects: mommy comities, neutropenia, thrombocytopenia, anemia.
