explore -WSS

explore

author：Wikipedia 2025-09-29 21:26:40 - browse：2622

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： How one medical school became remarkably diverse
Next article： Gene therapy denied for Afghan refugee whose father aided U.S.

WSS

Rune Labs, whose app tracks Parkinson's symptoms, raises $12M

Fauci's latest grilling in Congress: 9 hours, behind closed doors

A preacher's new calling: Diversifying neuroscience research

Roche drug slashes death risk in early

Roche acquires Telavant from Roivant, adding experimental IBD treatment

STAT Readout: Friday, Oct. 20 biotech updates

focus

explore

Wikipedia

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

Health tech investors get more selective as pandemic boom fades

Peter Hotez and the public health issue of online harassment

Male doctors called my cancer incurable. A female doctor showed it wasn't

Cancer drug shortages should be causing more outrage

Health tech investors get more selective as pandemic boom fades

Cancer drug shortages should be causing more outrage

The problems with dementia villages

Drug repurposing or repositioning? The language matters

JPM pitch from hospitals? Revenue streams outside of patient care

hotspot

Ascension's ugly legal battle over care for poor patients in Texas

Roche drug slashes death risk in early

FDA clears Verve to begin U.S. study of gene

ADCs take center stage as promising cancer treatments

At JPM 2024, hospital systems pitch financial strength to investors

Mayo Clinic partners with supercomputer AI firm Cerebras at JPM

focus

hotspot

entertainment

explore