On Aug. 17, 2022, the U.S. Food and Drug Administration approved Zynteglo (betibeglogene autotemcel) developed by bluebird bio, the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

Beta-thalassemia is a type of inherited blood disorder that causes a reduction of normal hemoglobin and red blood cells in the blood, through mutations in the beta-globin subunit, leading to insufficient delivery of oxygen in the body.