UK authorizes first gene therapy for treating sickle cell disease

In a landmark decision, the UK's Medicines and Healthcare products Agency (MHRA) approved the use of a gene-editing therapy called Casgevy for patients with sickle cell disease and beta thalassemia -- both of which are hereditary disorders related to genetic mutations of the red blood cells. The treatment, manufactured by Vertex , is the first-ever approved therapy that utilizes CRISPR-based gene editing technology to treat eligible patients. The UK approval of the novel therapy is informed by t...