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

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).

NICE had previously rejected the therapy’s use on the NHS to treat some people with sickle cell disease in draft guidance released in March.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.

"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