Beta thalassaemia: First gene-editing therapy could cure disorder

The National Institute of Health and Care Excellence medicines watchdog has approved the therapy after assessing the costs and benefits of what it calls a “potential cure”.

A deal has been struck which means NHS England is paying less than the official price of £1.6m per patient.

It is estimated that 460 people over the age of 12 would be eligible if they wanted the therapy, and it will be offered at seven specialist centres “within weeks”.

Amanda Pritchard, the NHS chief executive, said: “This is a historic moment for people living with beta thalassaemia with a potential cure for those facing this debilitating disorder now available on the NHS.”

Beta thalassemia mainly affects people of Mediterranean, south Asian, south-east Asian and Middle Eastern backgrounds.

Previously the only alternative to blood transfusions was a stem cell transplant, but this was rare because it required a very close tissue match from a donor.

Casgevy, developed by the company Vertex, is the first approved therapy to use Crispr-technology.

Romaine Maharaj, executive director of the UK Thalassaemia Society, said “we stand on the brink of a revolutionary breakthrough” and “it is a beacon of hope”.

Negotiations are still under way to see if the same therapy can be used on the NHS for another genetic disease affecting haemoglobin – sickle cell anaemia.