knowledge -WSS

knowledge

author：comprehensive 2025-10-02 09:47:23 - browse：217

Peter Marks. -- health coverage from STAT — Peter Marks, Director of the Center for Biologics Evaluation and Research at the Food and Drug Administration. Susan Walsh-Pool/Getty Images

Peter Marks wants drug developers to ask more stupid questions.

It’s part of the top Food and Drug Administration official’s plan to reinvigorate gene therapy, a field that has struggled despite significant technological advances. Some companies are shelving programs or going out of business, even when they have promising data.

The problems are numerous: The diseases are often exceptionally rare, limiting the potential market. Manufacturing at commercial quality is complex and expensive. Proving a drug works can be difficult, because there may be too few patients to run a traditional randomized study.

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

Subscribe Log In

Previous article： Wildfire smoke exposes gaps in outdoor worker protections
Next article： STAT Health News: Apple Watch import ban, Pfizer fumbles, etc

Wikipedia

WSS

STAT Health News: Apple Watch import ban, Pfizer fumbles, etc

Can sheer will power keep patients alive in their dying hours?

Readout Newsletter: Illumina, CRISPR, Regeneron

Medicare Advantage insurers pressure Biden for bigger payments

Keeping primary care docs in field isn't impossible, says AAFP CEO

Ibogaine psychedelic therapy for opioid addiction unlikely, official says

hotspot

knowledge

focus

Virginia high school admissions case could be legal follow

SEC charges former device maker CEO with $41 million fraud

Drug repurposing or repositioning? The language matters

This gene therapy has record $4.25 million price. It's saved children.

How to save PrEP access — and even expand it

Hospitals owned by private equity see complications spike 25%

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

Asbestos ban is immediate, but health effects will linger for decades

Hepatitis C has a cure — but many Americans still lack access to it

Virtual care provider acquires bankrupt Pear's prescription apps

hotspot

What STAT readers think about the FDA and patient advocacy

What STAT readers think about the FDA and patient advocacy

David Ricks of Eli Lilly: best biopharma CEO of 2023

Chinese researcher claims birth of first gene

Humana’s Medicare Advantage lawsuit should be tossed, DOJ says

Feng Zhang suggests Editas didn’t move quickly enough to find product targets

entertainment

hotspot

Wikipedia

Wikipedia