Wikipedia -WSS

Wikipedia

author：knowledge 2025-09-28 01:30:44 - browse：91446

Two people run on the sugar-phosphate backbone of DNA while holding base pairs — in the lab coverage from STAT — Adobe

LONDON — Widespread genetic testing as well as the development of cutting-edge, customized genetic therapies have opened the door to treating many more inherited conditions than previously possible. A few recent cases, in which bespoke medicines were created for children with exceedingly rare mutations, have raised hopes for furthering the approach.

Now, researchers and government agencies in the U.K. are trying to figure out the nitty-gritty to replicate those early one-off successes for as many patients as possible — what sort of preclinical testing a tailor-made treatment needs to go through before being used, what regulatory thresholds each treatment needs to clear, and how to pay for these therapies.

“A lot of the barriers are no longer scientific — we can make these drugs,” said Matthew Wood, a professor of pediatrics at the University of Oxford. “It’s more about, can we make them at a cost that is reasonable enough? And can we get the regulators to reduce the barriers to doing this so that when a patient shows up, you can actually make a drug quickly enough to benefit that patient, rather than it costing huge amounts of money and taking two or three years to do it?”

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

Subscribe Log In

Previous article： How to save PrEP access — and even expand it
Next article： U.K. not yet recommending Vertex's CRISPR therapy for sickle cell

hotspot

Wikipedia

WSS

Is there really a nursing shortage in the U.S.?

Stop the failed accountable care organization experiment

Readout Newsletter: Geron, Novo Nordisk, WuXi updates

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

Is there really a nursing shortage in the U.S.?

Smaller biotechs seek to piggyback on weight loss drug boom

explore

Wikipedia

hotspot

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

Health tech investors get more selective as pandemic boom fades

The Supreme Court will review a ruling striking down a domestic violence federal gun ban

Health tech investors get more selective as pandemic boom fades

Peter Hotez and the public health issue of online harassment

Physicians and social media: If you see something, say something?

Trump demands the U.S. pay no more for drugs than other countries … again

Why a market frenzy over new weight loss drugs defies logic

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

STAT Summit: Doctor v. ChatGPT showed AI's promise, blind spots

focus

How AI can help patients find reliable information about doctors

Harvard's Jason Buenrostro wins MacArthur grant for genetics work

STAT Readout Newsletter: Biotech news today

STAT Summit: How researchers deal with rejection

UnitedHealth, Optum discontinue NaviHealth name

STAT Summit: How researchers deal with rejection

Wikipedia

hotspot

Wikipedia

hotspot