explore -WSS

explore

author：focus 2025-09-30 23:39:29 - browse：2

illustration of one blue dna strand — Adobe

The Food and Drug Administration is convening a meeting of outside experts on Tuesday to review exa-cel, a CRISPR-based treatment for sickle cell disease made by Vertex Pharmaceuticals and CRISPR Therapeutics.

Tuesday’s meeting is set up a bit differently than most FDA advisory panels. The agency has not raised any concerns about exa-cel’s efficacy or safety, and there will not be a typical vote at the end of the day on whether the data from exa-cel’s pivotal clinical trial support an approval as a treatment for sickle cell disease.

Instead, the FDA is seeking input about the methods used by Vertex and CRISPR Therapeutics to evaluate the risk of inadvertently making unwanted changes to patients’ DNA — so-called off-target editing — when exa-cel is administered.

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

Subscribe Log In

Previous article： FDA approval of Sarepta Duchenne genetic therapy gives me hope
Next article： Fatty liver disease was alleviated by Lilly’s ‘triple

entertainment

WSS

Canadians with cystic fibrosis outlive Americans by a decade

Obesity drug from Roche shows promise in early study

Canadians with cystic fibrosis outlive Americans by a decade

Use doctors trained outside the U.S. to ease the physician shortage

STAT wants to know your pick for a great summer read or listen

CVS is willing to dump 10% of its Medicare Advantage members

focus

explore

Wikipedia

23andMe had bad news about my health. I wish a person had delivered it

CVS is willing to dump 10% of its Medicare Advantage members

Heart attack and stroke risk calculators need revising

Penis shrinkage, a side effect of prostate cancer surgery, is temporary

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

Canadians with cystic fibrosis outlive Americans by a decade

Hollywood unions extend contract negotiations for actors

HHS suspends federal funding for EcoHealth Alliance

How one medical school became remarkably diverse

Why H5N1 bird flu is keeping the CDC's top flu scientist awake

Wikipedia

To spot an incipient stroke, tiny brain probe acts ‘like a microscope’

Vertex cell therapy lets patients with type 1 diabetes stop insulin

ALS treatment from Biogen, Ionis fails in study

UpToDate has a racism problem: Dr. Stanley Goldfarb

ALS treatment from Biogen, Ionis fails in study

WuXi Biologics beefs up its lobbying operation

entertainment

focus

leisure time

focus