hotspot -WSS

hotspot

author：explore 2025-10-01 17:35:26 - browse：5

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： Virginia high school admissions case could be legal follow
Next article： Indian pharmaceutical industry in denial over drug

explore

WSS

Health and fitness trackers are measuring the wrong things

DeSantis can now import cheaper drugs. But will Biden get credit?

'Stay indoors': Escaped wild animal on the loose in Berlin prompts urgent citywide search

A Q&A with Gavi’s Seth Berkley on vaccines, Covid, and more

Health and fitness trackers are measuring the wrong things

Heart disease and mental health: A look at cardiac psychology

focus

hotspot

entertainment

Supreme Court strikes down use of affirmative action

Affordable Connectivity Program vital to health care, experts say

Hepatitis C has a cure — but many Americans still lack access to it

Mass General totes up its waste in a move to tackle climate change

FDA approval of Sarepta Duchenne genetic therapy gives me hope

Fauci’s Covid grilling leads GOP to weigh foreign research rules

Affirmative action in medical school literally saved lives

Fauci’s Covid grilling leads GOP to weigh foreign research rules

Walensky prepares to leave CDC — and Congress — behind

Judge rejects Trump's request to move Stormy Daniels hush

hotspot

Ending racial health disparities hinges on penalties, political will

JPM 2024: Nvidia claims we're in biological revolution. Are we?

Jury awards Florida girl burned by McDonald's Chicken McNugget $800,000 in damages

DeSantis can now import cheaper drugs. But will Biden get credit?

Study identifies new proteins linked to dementia risk 25 years later

Tocagen's brain cancer gene therapy trial continues. Investors worry

focus

explore

focus

hotspot