comprehensive -WSS

comprehensive

author：comprehensive 2025-09-27 07:46:24 - browse：2

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： Supreme Court strikes down use of affirmative action
Next article： Drastic changes should bring profitability, startup health insurers say

focus

WSS

Hawaii wildfires fueled by Hurricane Dora's winds prompt evacuations

Wegovy’s successful heart trial raises vital question about weight loss

Ebola vaccine can save some who are already sick, per new study

STAT Boddities: What happens to the brain when we get scared?

Amylyx and the next ALS drug test, plus a chat with CRISPR's CEO

Ebola vaccine can save some who are already sick, per new study

hotspot

comprehensive

focus

Food as medicine: CMS rules hamper 'prescribing' of fruits, veggies

How to use technology to prevent future drug shortages

ChatGPT in medicine: STAT answers readers' burning questions

Tome Biosciences co

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

Wegovy’s successful heart trial raises vital question about weight loss

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

Amylyx's ALS drug launch starts strong. It could be trouble for FDA

Alkermes shareholders re

Resistance to opioid

focus

A biotech analyst on neuroscience, placebo effect, not over

For Cytokinetics, FDA rejection of heart drug would help, not hinder

Methadone doses haven’t kept up in the age of fentanyl

Anavex keeps shifting the goalposts in its clinical trials

FDA panel issues narrow recommendation for Covid vaccine booster doses

Eli Lilly reports tirzepatide MASH success during Q4 earnings

hotspot

entertainment

focus

Wikipedia