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‘Much-needed hope’ as CRISPR gene editing therapy recommended for sickle cell disease

‘Much-needed hope’ as CRISPR gene editing therapy recommended for sickle cell disease


Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant access to gene-editing therapy on the NHS.

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Some with severe sickle cell disease in England will now be able to access landmark CRISPR gene editing therapy, according to new health guidelines which were hailed as marking a “significant shift” in treatment for the blood disorder.

The UK’s National Institute for Health and Care Excellence (NICE) said on Friday that it had approved the use of the gene editing therapy exagamglogene autotemcel (exa-cel) by England’s National Health Service (NHS).

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NICE had previously rejected the therapy’s use on the NHS to treat some people with sickle cell disease in draft guidance released in March. It approved the use of exa-cel for the rare blood disorder beta thalassemia later in the year.

The therapy, which the UK’s medicines regulator cleared for use in November 2023, has a list price of £1.6 million (€1.9 million) per course.

Sickle cell disease is a group of inherited blood disorders that affects the shape of red blood cells. People with the condition can experience severe pain, infections, anaemia, and other problems, according to the NHS.

The disease is more common in people from African, Caribbean, Middle Eastern, or South Asian family backgrounds.

There are currently few treatments for symptoms of the disease and the treatments that exist have “intolerable side effects,” NICE said.

The gene editing therapy will be available to some people aged 12 and older with severe complications from the condition and where a stem cell transplant is “suitable” but a donor cannot be found, according to the watchdog.

‘Much-needed hope’

“The approval of exa-cel today marks a significant shift in the treatment landscape of sickle cell disease in the UK,” Funmi Dasaolu, who has sickle cell disease and spoke to the NICE committee, said in a statement.

“It is the beginning of re-addressing the inequalities in care experienced by so many with the condition,” she said, adding that it provides “much-needed hope”.

The discovery of the CRISPR/Cas9 gene editing tool won two researchers, Emmanuelle Charpentier and Jennifer A. Doudna, the Nobel Prize in Chemistry in 2020.

Exa-cel, also called Casgevy, involves taking a person’s blood stem cells, using CRISPR to edit them in a lab, and replacing them in the patient.

“The approval of Exa-cel for NHS use in England is a very exciting moment, not only because this marks the first approval of a CRISPR-based gene therapy for SCD [sickle cell disease] in the NHS, but also because it offers a potentially curative treatment for eligible patients,” said Felicity Gavins, a professor of pharmacology at Brunel University of London.

“However, while Exa-cel is a breakthrough, it is not a cure for all SCD patients, and uncertainties remain about its long-term effectiveness, safety, and accessibility,” Gavins said in a statement.

“It is critical to continue funding research to develop treatment that benefit the broader SCD population and address remaining challenges in care”.

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Data will be collected as patients receive the medicine before NICE re-evaluates it.



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