focus -WSS

focus

author：leisure time 2025-10-02 00:28:29 - browse：49

The Food and Drug Administration on Friday approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder.

The new medicine, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations — ushering in a new era of genetic medicines for inherited diseases.

In a clinical trial, Casgevy was shown to eliminate recurrent episodes of debilitating pain caused by sickle cell, which afflicts approximately 100,000 people in the U.S., a vast majority of whom are Black. The therapy, whose scientific name is exa-cel, is described as a potential cure because the genetic fix enabled by CRISPR is designed to last a lifetime, although confirmation will require years of follow-up.

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

Subscribe Log In

Previous article： Tori Bowie and the unacceptable rates of Black maternal death
Next article： What do new weight loss drugs mean for bariatric surgery?

focus

explore

WSS

Medicare explains how it will choose drugs for negotiation

Complete sequence of Y chromosome published for first time

Neuroscience startup Neumora files for IPO

Reanimated hearts work just as well for transplants, study finds

Reanimated hearts work just as well for transplants, study finds

Radiation, mainstay of cancer treatment, begins a fade

hotspot

focus

hotspot

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

A public health expert passes on football's full body collisions for youths

Medical records are filled with copy

Living organ donors deserve priority status for transplants

Activists slam private health insurers over coverage restrictions

Australia’s approach to dialysis celebrates life

Continuity nursing helped my family in our darkest PICU moments

Neuroscience startup Neumora files for IPO

Sports medicine is finally prioritizing gender equality

As pharma fights Medicare drug negotiation, hospitals, docs silent

hotspot

Departing Stanford president retracts two papers

Timeline: Key moments in fight for gay rights

In science, is the line between industry and academia shifting?

Patients, doctors are clashing about side effects of hormonal birth control

FDA panel gives mixed vote on blood pressure devices

Senate panel passes drug pricing bills, but drops one PBM reform

focus

leisure time

explore

hotspot