Zynteglo Approved as Gene Therapy for Rare Blood Disorder

August 17, 2022

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.

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