The FDA has approved Bluebird Bio’s Zynteglo as the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.
The drug, betibeglogene autotemcel (also known as beti-cel), is a one-time gene therapy product administered as a single dose. Each dose of Zynteglo is a customized treatment created using the patient’s own bone marrow stem cells that are genetically modified to produce functional beta-globin.
