UK's NHS to roll out Vertex/CRISPR gene therapy for rare blood disorder

[August 08, 2024]  (Reuters) -Britain's National Health Service (NHS) will begin to roll out a gene therapy from Vertex Pharmaceuticals 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.

An NHS logo is displayed outside a hospital in London, Britain May 14, 2017. REUTERS/Neil Hall/File Photo

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)

(Reporting by Gursimran Kaur and additional reporting by Surbhi Misra in Bengaluru; Editing by Anil D'Silva and Sherry Jacob-Phillips)

[© 2024 Thomson Reuters. All rights reserved.]This material may not be published, broadcast, rewritten or redistributed.  Thompson Reuters is solely responsible for this content.