leisure time -WSS

leisure time

author：leisure time 2025-09-28 21:11:16 - browse：29

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watchdog said Thursday it is not yet recommending Vertex’s CRISPR-based therapy for sickle cell disease, saying in draft guidance it has questions about the medicine’s lasting benefits for patients.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： Novavax promises a turnaround & Lilly roils the obesity market
Next article： Readout Newsletter: Insmed, Asahi Kasei, Agios, Royalty Pharma

knowledge

explore

WSS

How Brent Saunders' Allergan experiment failed

Adam's Biotech Scorecard: Do Sarepta's exon

Adam's Biotech Scorecard: Do Sarepta's exon

Merus drug and Keytruda shrank tumors in patients with head and neck cancer

Startling documents reveal new facets of Sacklers' opioid empire

iTeos Therapeutics debuts first data on TIGIT

leisure time

leisure time

Wikipedia

Novavax promises a turnaround & Lilly roils the obesity market

iTeos Therapeutics debuts first data on TIGIT

Dobbs anniversary: the lost opportunity of abortion as health care

Stigma and the return of syphilis

Psychedelics group wrestles with new pharma identity

Startling documents reveal new facets of Sacklers' opioid empire

Sports medicine is finally prioritizing gender equality

Addiction doctors spar with methadone clinics over legislation

BioMarin wins approval for gene therapy to treat hemophilia A

iTeos Therapeutics debuts first data on TIGIT

leisure time

EMA weighs against approval for Mirati's lung cancer drug

Readout Newsletter: Insmed, Asahi Kasei, Agios, Royalty Pharma

Stigma and the return of syphilis

Florida babysitter arrested over hot car death, authorities say

Average age of first period drops to 11.9 years, study shows

AI used to predict Covid

knowledge

explore

leisure time

focus