Gene editing for patients with severe sickle cell disease is to be made available in the UK, it was announced last week (31st January).
NHS England estimates that around 50 patients a year are expected to receive the treatment, exa-cel (exagamglogene autotemcel, also known as Casgevy).
The National Institute for Health & Care Excellence (NICE) says English patients will be among the first in the world to benefit from the pioneering technology, which uses CRISPR gene editing to alter blood stem cells to produce red blood cells with high levels of foetal haemoglobin.
The treatment will be available to patients over the age of 12 with recurrent vaso-occlusive crises and a βS/βS, βS/β+ or βS/β0 genotype, who are suitable for a blood stem cell transplant but without a matched donor. The treatment is valued at £1.6 million a course, but NHS England struck a deal with the manufacturer to access the treatment at a reduced cost.
NICE said a patient who received the treatment said it made them “healthier, fitter and stronger than at any point in my life before”.
NICE chief executive Dr Samantha Roberts said: “Exa-cel could represent a potential cure for some people with severe sickle cell disease, freeing people from the burden of complications as well as addressing NICE’s aim of reducing health inequalities associated with the condition and getting the best care to patients fast.”
John James, chief executive of the Sickle Cell Society, said: “We are absolutely thrilled to see this ground-breaking gene therapy treatment available on the NHS from today. The significance of this milestone for the sickle cell community cannot be understated – today’s result will give hope to many and is the result of determined campaigning.”
NHS chief executive, Amanda Pritchard, said: “More than a step, this is a leap in the right direction for people with sickle cell disease – which can be an extremely debilitating and painful condition.
“This innovative, gene-editing therapy offers hope of a cure for people facing a severe form of the disease and could be absolutely transformative – it could enable patients to live free from the fear of sickle cell crises hanging over them.”
The decision follows approval for the same treatment for people with β-thalassaemia in August 2024.
Source: NICE/NHS England
https://www.england.nhs.uk/2025/01/revolutionary-gene-editing-therapy-for-sickle-cell/
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