leisure time -WSS

leisure time

author：leisure time 2025-09-02 11:47:19 - browse：247

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： United colonoscopy coverage change 'may cost lives,' doctors say
Next article： Reframing the conversation around gain

hotspot

WSS

Pfizer's RSV vaccine recommended for approval in Europe

Abortion pill case raises question: who can sue the FDA?

Scientists boost immune system in aging mice by making it youthful

Eliminate the waiting period for sterilization covered by Medicaid

Oprah kicked off a national conversation on obesity. Let's have it

Listen: BridgeBio's big week & Lilly's Alzheimer's data

hotspot

leisure time

focus

Guidelines to prevent youth baseball injuries need more muscle

'The View' co

How to save PrEP access — and even expand it

Why the advice to take all your antibiotics may be wrong

Alkermes shareholders re

Washington Commanders' Dan Snyder ordered to pay NFL $60M following investigation

Psychedelics group wrestles with new pharma identity

Steward Health Care has deal to sell doctor network to UnitedHealth

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

What is the Turing Test that determines if computers can think?

hotspot

Listen: BridgeBio's big week & Lilly's Alzheimer's data

EMA weighs against approval for Mirati's lung cancer drug

Readout Newsletter: Cardior, Regeneron, Nuvation, Dyne and more

Donor acceptance rates hint at racial, gender bias in heart transplants: study

Pfizer's RSV vaccine recommended for approval in Europe

Suspects arrested after 3 women attacked on Manhattan's West Side: Police

focus

hotspot

explore

hotspot