UK to roll out gene-editing therapy for rare blood disorder

LONDON — UK'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 thalassaemia, Vertex said in a statement on Aug 7.

Casgevy, which requires administration through authorised treatment centres 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-dependant 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 (S$2.78 million), a spokesperson for Vertex said, adding that the company cannot comment on a negotiated price.

Britain's medical regulator had authorised 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.

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