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UK's NHS to roll out Vertex/CRISPR gene therapy for rare blood disorder
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UK's NHS to roll out Vertex/CRISPR gene therapy for rare blood disorder
Aug 7, 2024 5:42 PM

Aug 7 (Reuters) - Britain's National Health Service

(NHS) will begin to roll out a gene therapy from Vertex

Pharmaceuticals ( VRTX ) and CRISPR Therapeutics to

treat rare blood disorder beta thalassemia, Vertex said in a

statement on Wednesday.

Casgevy, which requires administration through authorized

treatment centers with experience in stem cell transplantation,

will be made available to eligible patients from Aug. 7, the

company said.

The agreement between Vertex and the NHS comes as the

National Institute for Health and Care Excellence (NICE) issued

positive guidance recommending Casgevy's use, Vertex said.

About 460 patients in England with transfusion-dependent

beta thalassaemia aged 12 and older are potentially eligible for

the treatment, which uses gene-editing technology, the NHS said

in a statement.

The NHS said the therapy will be manufactured in the UK,

and is set to be offered at seven of its specialist centres.

The UK list price for the therapy stands at 1.65 million

pounds ($2.09 million), a spokesperson for Vertex said, adding

that the company cannot comment on a negotiated price.

Britain's medical regulator had authorized Casgevy

CRISPR-based gene-editing therapy last November for the

treatment of sickle-cell disease and another type of inherited

blood disorder for patients aged 12 and over.

The medicine is administered by taking stem cells out of a

patient's bone marrow and editing a gene in the cells in a

laboratory, with the modified cells then infused back into the

patient after conditioning treatment to prepare the bone marrow.

($1 = 0.7890 pounds)

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