hotspot -WSS

hotspot

author：comprehensive 2025-09-30 11:07:14 - browse：9

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： Walensky prepares to leave CDC — and Congress — behind
Next article： How a billionaire couple greased the skids for Pelosi’s drug pricing bill

WSS

CMS should re

It's time to retire 'poor historian' from clinicians' vocabularies

The woman behind the Dana Farber

The woman behind the Dana Farber

FDA approves world's first CRISPR

Case highlights the risks of experimental stem cell therapy

hotspot

hotspot

leisure time

At least 13 dead in Texas as scorching temps continue

Radiology is caught between AI's potential and concern for patients

What does generative AI mean for health care? We asked experts

In diabetes hearing, Sanders takes on a new foe: food makers

Duchenne breakthrough therapy leaves behind pioneering families

Addiction experts warn against a second 'war on drugs'

After affirmative action ruling, medical educators look to 'holistic review'

It's time to retire 'poor historian' from clinicians' vocabularies

George Santos makes 1st court appearance after pleading not guilty to 13 counts

Wearables generate powerful data but its not useful to doctors, yet

hotspot

Big Tobacco's 'harm reduction': Is it for real, or a sham?

Can we make enough H5N1 bird flu vaccine if there's a pandemic?

The woman behind the Dana Farber

With measles on the rise, a personal story of a devastating disease

Bird flu: Don't repeat Covid's 'health versus the economy' framing

Why sickle cell disease is getting the first CRISPR treatment

focus

explore

focus

hotspot