explore -WSS

explore

author：focus 2025-09-02 22:07:46 - browse：22994

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watchdog said Thursday it is not yet recommending Vertex’s CRISPR-based therapy for sickle cell disease, saying in draft guidance it has questions about the medicine’s lasting benefits for patients.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

Get unlimited access to award-winning journalism and exclusive events.

Subscribe Log In

Previous article： Wildfire smoke exposes gaps in outdoor worker protections
Next article： IVF, abortion rights issues kick off Biden’s State of the Union

knowledge

WSS

In his State of the Union, Biden takes clear aim at 'Big Pharma'

What could happen if Russia blows up the Zaporizhzhia Nuclear Power Plant?

Submersible on Titanic tour reported missing off Newfoundland with 5 people aboard

Headspace, Meta partner to launch VR meditation app for Quest

A deep dive into Applied Therapeutics’ data, and questions on Cassava

Readout LOUD podcast: Venture capital turmoil, obesity drug competition

hotspot

explore

focus

Medical leaders decry Supreme Court decision on affirmative action

RSV shot was 90% effective at preventing hospitalizations in kids

Medical leaders decry Supreme Court decision on affirmative action

UPS workers' union leader explains what's at stake as strike edges closer

Biden outlines 'new path' to provide student loan relief after Supreme Court rejection

IVF, abortion rights issues kick off Biden’s State of the Union

Peter Hotez and the public health issue of online harassment

Novo Nordisk gets FDA approval to tout heart benefits of Wegovy

How to save PrEP access — and even expand it

RSV shot was 90% effective at preventing hospitalizations in kids

entertainment

FDA approves Leqembi, a milestone in Alzheimer’s treatment

US intelligence assessment says Iran not currently developing nuclear weapons

How corporate America is slashing DEI workers amid backlash to diversity programs

Florida group launches initiative to get abortion constitutional amendment on 2024 ballot

The problem with charging patients to message their doctors

Masked men burn a market in a Mexican city plagued by gang violence, killing 9 people

focus

leisure time

knowledge

explore