hotspot -WSS

hotspot

author：fashion 2025-10-02 22:57:59 - browse：1

Vertex Pharmaceuticals - sunset — Bill Sikes/AP

LONDON — Regulators in the U.K. on Thursday approved a CRISPR-based medicine to treat both sickle cell disease and beta thalassemia, making it the world’s first therapy built on the revolutionary gene-editing technology and ushering in a new era of genetic medicine.

The authorization of the therapy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is itself not a surprise. Clinical trials have shown the one-time treatment enabled many sickle cell patients to live free of debilitating pain crises and relieved thalassemia patients of needing regular blood transfusions, raising hopes that they have been effectively cured. But it also stands as a major achievement, coming just over a decade after the first and seminal lab experiments that showed CRISPR’s potential as a gene-editing tool were published.

In granting conditional approval to the therapy, which will have the brand name Casgevy and is also known as exa-cel, the U.K.’s Medicines and Healthcare products Regulatory Agency jumped ahead of its counterparts in the U.S. and Europe, which are also reviewing the medicine, in greenlighting the globe’s first CRISPR treatment. The MHRA’s authorization is for patients who are at least 12 years old.

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

Subscribe Log In

Previous article： How one medical school became remarkably diverse
Next article： Study: Adult vaccinations pay for themselves in societal benefits

leisure time

WSS

Families look for replacement for GSK asthma drug Flovent

The public health thinking behind the CDC’s new Covid guidance

Readout Newsletter: BridgeBio, Akero, and human embryo models

Readout Newsletter: Sanofi, Novavax, Maze x Shionogi news

Change Healthcare: What is it, why does its cyberattack matter?

Steward Health Care files for bankruptcy

focus

hotspot

focus

Trump demands the U.S. pay no more for drugs than other countries … again

Fetal cell organoids grown from amniotic fluid, study finds

Anesthesiologist group: stop taking Ozempic before surgery

Software glitch causes Tandem insulin pump to shutdown, FDA warns

Indiana Supreme Court upholds abortion ban, says state constitution gives only limited protections

It shouldn't be easy to buy synthetic DNA to recreate a deadly virus

Malaria cases in Florida and Texas: Here’s what you need to know

Steward Health Care files for bankruptcy

Duchenne breakthrough therapy leaves behind pioneering families

Florida health officials providing scant details on measles cases

leisure time

Bird flu outbreak in cows is latest avian flu curveball

Researchers on aging grapple with how to calculate biological age

Readout Newsletter: Sanofi, Novavax, Maze x Shionogi news

CDC wastewater surveillance dashboard to track bird flu hotspots

Change Healthcare cyberattack affects 25% of CVS/Aetna insurance claims

Harvard's David Sinclair gets blowback over aging

knowledge

leisure time

leisure time

leisure time