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author：knowledge 2025-09-30 14:58:13 - browse：63619

JOSEPH PREZIOSO/AFP via Getty Images

LONDON — The U.K.’s cost-effectiveness watchdog said Thursday it is not yet recommending Vertex’s CRISPR-based therapy for sickle cell disease, saying in draft guidance it has questions about the medicine’s lasting benefits for patients.

U.K. regulators approved the therapy, known as Casgevy or exa-cel, last year, granting it the world’s first authorization for any CRISPR-based medicine. But observers have been watching to see how the U.K.’s National Institute for Health and Care Excellence, as well as similar agencies in European countries, would view the pricey, one-time therapy. NICE needs to recommend the therapy for it to be offered in the National Health Service.

In the U.S., Casgevy has a list price of $2.2 million. It was developed by Vertex in partnership with CRISPR Therapeutics.

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