Posted by Michael Wonder on 17 Aug 2022
FDA approves first cell based gene therapy to treat adult and paediatric patients with beta thalassaemia who require regular blood transfusions
17 August 2022 - Today, the US FDA approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and paediatric patients with beta thalassemia who require regular red blood cell transfusions.
Zynteglo is a one time gene therapy product administered as a single dose. Each dose of Zynteglo is a customised treatment created using the patient’s own cells (bone marrow stem cells) that are genetically modified to produce functional beta-globin (a haemoglobin component).
