Zynteglo® (betibeglogene autotemcel), made by bluebird bio, Inc., now has the first cell-based gene therapy for the treatment of beta-thalassemia in patients who require regular red blood cell transfusions.

• Beta-thalassemia is an inherited blood disorder that affects hemoglobin and red blood cells causing issues such as insufficient delivery of oxygen in the body.
• Under the recommended dosing, Zynteglo is given as a one-time gene therapy customized to the patient.
• Zynteglo will be available on the S. market at a wholesale acquisition cost (WAC) of $2.8 million per treatment.