14 Mar 2024

Sickle cell patients ‘let down’ by decision to not approve new gene therapy on NHS


Sickle cell patients and campaign groups have called a decision not to approve a new type of gene therapy for NHS use a “shocking letdown”.

There are more than 17,000 people living with sickle cell disease in the UK, and the medicine watchdog’s approval of the new treatment in November was widely welcomed. It works by editing faulty genes to help the body produce functioning haemoglobin.

But the National Institute for Health and Care Excellence said today there is not enough data for the NHS to fund it.

We’re joined by Yasmin Sheikh from the Anthony Nolan stem cell charity, and Iggie Aikhomu, who had a stem cell transplant for the disease.