UK becomes first country in the world to authorise a Crispr-based gene therapy

The UK’s drug agency has become the first in the world to authorise a Crispr gene-editing therapy. The new treatment has been approved for sickle-cell disease and transfusion-dependent β-thalassaemia and has the potential to cure patients with these conditions.

Both genetic conditions are caused by errors in the genes for haemoglobin, a protein found in red blood cells which carries oxygen around the body. Casgevy, manufactured by Vertex Pharmaceuticals and Crispr Therapeutics, uses Crispr to alter a specific gene in a patient’s bone marrow stem cells called BCL11A, to enable the production of a functioning haemoglobin.