The first therapy that uses gene-editing is to be offered on the NHS in a “revolutionary breakthrough” for patients.
It will be used as a potential cure for the blood disorder beta thalassaemia.
Stem cells which make blood will be extracted, reprogrammed to correct the condition and returned to the patient’s body.
It could spare them needing a blood transfusion, every three to five weeks, for life.
People with beta thalassaemia struggle to produce enough haemoglobin, which is the protein in red blood cells that carries oxygen around the body.
It is a genetic disease that is passed down through families and caused by defects in the body’s instructions for manufacturing haemoglobin.
Share this article
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.”
