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UK health system to offer cutting-edge gene therapy for sickle cell disease
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UK health system to offer cutting-edge gene therapy for sickle cell disease
Jan 30, 2025 4:34 PM

LONDON, Jan 31 (Reuters) - Britain'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 ( VRTX ) and CRISPR

Therapeutics will cost the state-funded healthcare

system around 1.65 million pounds ($2.1 million) per course.

Britain'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.

($1 = 0.8039 pounds)

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