The first cell-based gene therapy for the treatment of patients with beta thalassaemia, an inherited blood disorder, was approved by the US Food and Drug Administration on 17 August. Zynteglo (betibeglogene autotemcel) is a one-time treatment administered as an ex vivo lentiviral vector gene therapy. A functional beta globin gene is added into a patient’s cells outside of the body and then infused back into the patient. The treatment process is comprised of several steps and will be administered at qualified treatment centres in the US.
