In a world first, the UK approves gene therapy for sickle cell and thalassemia
Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling illness in the U.K. In a statement on Thursday, the Medicines and Healthcare Regulatory Agency said it had approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020
5:55AM ( 5 months ago )