knowledge -WSS

knowledge

author：knowledge 2025-09-29 20:00:37 - browse：9

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： Walensky prepares to leave CDC — and Congress — behind
Next article： Paxlovid study fails to answer questions over benefit for broader groups

WSS

Cloning humans is technically possible. It's curious no one has tried

STAT's guide to the next generation of CAR

ALS treatment from Biogen, Ionis fails in study

FDA grants conditional OK to Sarepta’s gene therapy for Duchenne

The strategy behind pharma's lawsuits over drug pricing reform

CVS is willing to dump 10% of its Medicare Advantage members

entertainment

knowledge

Wikipedia

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

The battle over the Brookhaven Landfill on Long Island

In age of alternative facts, a scholarly course on calling out crap

Adam's Biotech Scorecard: PDS Biotech's survival benefit is overstated

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

New guidelines on severe brain injury complicate already difficult decisions

BioMarin wins approval for gene therapy to treat hemophilia A

The strategy behind pharma's lawsuits over drug pricing reform

Malaria cases in Florida and Texas: Here’s what you need to know

STAT series 'Denied by AI' wins major journalism prizes

hotspot

Gene therapy has helped boys walk. Can scientists keep it that way?

STAT series 'Denied by AI' wins major journalism prizes

Death of patient in Alzheimer’s trial raises question of possible risks

As H5N1 spreads in cows, experts warn against drinking raw milk

Fertility clinic egg and embryo storage is a disaster

WuXi Biologics execs register to lobby as Congress comes for the company

leisure time

hotspot

hotspot

hotspot