MHRA authorises uses of Casgevy as a potential cure for sickle cell disease and beta thalassemia

The UK’s medicines regulator has authorised the use of a world-first gene therapy as a potential cure for two inherited blood disorders.

The treatment, Casgevy, for sickle cell disease and beta thalassemia, is the first to be licensed that uses the gene-editing tool known as Crispr, whose inventors were awarded the Nobel prize in 2020.