The US Food and Drug Administration (FDA) has approved Bluebird Bio’s one-time beta-thalassaemia treatment Zynteglo, also known as betibeglogene autotemcel (beti-cel), as the first cell-based gene therapy for the treatment of patients with beta-thalassaemia who require regular red blood cell transfusions.
Each Zynteglo dose is uniquely created by genetically modifying a patient’s own bone marrow stem cells. Specifically, the patient’s cells are modified to produce functional beta-globin, which is a component of haemoglobin.
According to FDA data, 89% of the 41 adult and paediatric patients across two clinical studies achieved transfusion independence after treatment with Zynteglo.
This represents a significant step for the treatment of these patients, particularly the paediatric population.
More information about this news is available here.