Gene-edited therapy cleared


United Kingdom

The world’s first regulatory approval of a gene-edited therapy, Casgevy (exagamglogene autotemcel), was issued by the UK Medicines and Healthcare products Regulatory Authority (MHRA) on 16 November, paving the way for the launch in the UK of a new treatment for  sickle cell disease and transfusion-dependent beta thalassaemia. Both diseases are inherited blood disorders that affect red blood cells which are essential for carrying oxygen to all organs and tissues in the body.