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UK health system to offer cutting-edge gene therapy for sickle cell disease

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The therapy takes stem cells out of a patient’s bone marrow, edits a gene in the cells, then infuses the cells back into the patient.

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Vertex Pharmaceuticals jointly developed the sickle cell therapy with CRISPR Therapeutics, which is expected to treat around 50 British patients a year. (EPA Images pic)

LONDON:
The UK’s national health service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the National Institute for Health and Care Excellence (NICE) said on Friday.

The therapy from Vertex Pharmaceuticals and CRISPR Therapeutics will cost the state-funded healthcare system around £1.65 million (US$2.1 million) per course.

The UK’s MHRA medical regulator in 2023 became the first in the world to approve the therapy, which uses the gene-editing tool CRISPR.

NICE, which assesses whether new medical technologies can be used in the NHS, said the treatment would be suitable for around 50 patients per year.

Sickle cell disease is a serious and lifelong condition caused by errors in the genes for haemoglobin, which is used by red blood cells to carry oxygen around the body.

The therapy involves taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory and then infusing them back into the patient.

The same technology was approved for use in the NHS in August to treat beta thalassemia, another rare blood disorder.

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