The MHRA has approved Casgevy gene therapy for sickle cell anaemia and β-thalassemia treatment, making the UK the first country to approve a gene editing tool as a treatment procedure after
Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is now approved in the United Kingdom for treating the blood disorders sickle cell disease and beta thalassemia. It’s the first regulatory approval in the world for a CRISPR-based therapy.