UK regulator approves “groundbreaking” gene treatment for sickle cell and β thalassaemia – GWC Mag

UK regulators have approved a “world first” gene treatment for treating sickle cell disease and transfusion dependent β thalassaemia.

Casgevy (exagamglogene autotemcel) is the first medicine using the gene editing tool CRISPR to be licensed for use in patients. The decision to approve the treatment, developed by Vertex Pharmaceuticals (Europe) and CRISPR Therapeutics, was made after a review of the available evidence, said the Medicines and Healthcare Products Regulatory Agency (MHRA).

People eligible for treatment would have stem cells removed from their bone marrow and edited in a laboratory using Casgevy to remove the faulty gene, before the modified cells are returned through an infusion. The approach, which means a hospital stay of as much as a month, has potentially lifelong results.

The National Institute for Health and Care Excellence (NICE) is currently reviewing the treatment for both sickle cell …