World-first gene-editing therapy for blood disorders approved for NHS use

The new treatment offers those who suffer from a life-limiting blood disorder a chance to live without needing regular blood transfusions

Hundreds of people in England with a life-limiting blood disorder are set to benefit from a world-first gene-editing therapy after it was given the green light for NHS use.

Health bosses hailed the decision by the National Institute for Health and Care Excellence (NICE) as a “historic moment” for patients with the condition.

Sold as Casgevy, the treatment has been recommended by NICE to treat patients aged 12 and over the transfusion-dependent beta thalassemia (TDT) which can lead to severe anaemia.

According to the NHS, around 17,000 people live with the disease, with 250 new cases each year. It is more common in people of Black African, Caribbean, Middle Eastern and South Asian heritage.

Those who suffer from the inherited condition rely on regular lifelong blood transfusions to survive.

The only curative treatment currently available for people with sickle cell in the UK is a donor stem cell transplant, so Casgevy is an option for patients when a stem cell transplant is suitable but no donor can be found.

Casgevy works by modifying the faulty gene in a patient’s bone marrow stem cells so the body produces functioning haemoglobin - the protein found in red blood cells that carries oxygen in your body and gives blood its red colour. The edited cells are then infused back into the patient.

It was developed through a partnership between Vertex Pharmaceuticals and Crispr Therapeutics and is licensed using the gene-editing tool Crispr - technology which earned its inventors the Nobel Prize for chemistry in 2020.

According to the Nice guidance, the list price for a course of Casgevy is £1.65 million. However, it is available to the NHS at a discount due to a commercial agreement with the health service. It will be funded immediately and rolled out to up to 460 eligible patients.

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Amanda Pritchard, NHS England chief executive, said: “Ordinarily, patients experience painful side effects and undergo regular transfusions which severely impact their quality of life, but this therapy offers people a life free from that as well as the hope of living longer, which is truly amazing news.

“This is the latest in a series of revolutionary gene therapies to be secured by NHS England over the past five years, bringing significant benefit for patients."

Helen Knight, director of medicines evaluation at NICE, added: “Although there are some uncertainties in the evidence for its long-term benefits, the committee felt [Casgevy] could represent a potential cure for some people with TDT.“

Kirthana Balachandran, 21, from West London, was diagnosed with TDT when she was three months old.

The medical student said: “I constantly worry about the future and what blood transfusions would be like when I’m older and how I will manage my condition. With gene therapy, I wouldn’t have to worry about the side effects of blood transfusions and the reactions I could have, such as iron depositing in my heart and liver.”

Ludovic Fenaux, senior vice president at Vertex International, said: “Through collaboration with NHS England and Nice, we have reached this landmark agreement that recognises the value a one-time treatment can provide to patients, their families and the healthcare system.”

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