explore -WSS

explore

author：explore 2025-10-02 11:10:38 - browse：844

Cambridge: Biogen — Ruby Wallau for STAT

A plan to use new genetic techniques to treat non-genetic forms of ALS failed in an early study, Ionis and Biogen announced Thursday. The companies said they would discontinue the program.

In the randomized, 99-person trial, the companies tested a drug designed to silence a gene called ATXN2. Over the last decade, numerous drugs have been built to silence genes in ALS patients, but most of them have, fittingly, focused on rare genetic forms of ALS, where neurons die largely because of the work of a single mutated stretch of DNA.

This approach has led to an approved drug for one very small subset of ALS cases, driven by the gene SOD1, and drugs for other genes are in trials. But only perhaps 10% — or at most 20% — of ALS cases are genetic. The hope, based on some genetic data and promising mice experiments, was that lowering ATXN2 would help any patient who develops ALS.

STAT+ Exclusive Story

Already have an account? Log in

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Monthly

$39

Totals $468 per year

$39/month Get Started

Totals $468 per year

Starter

$30

for 3 months, then $39/month

$30 for 3 months Get Started

Then $39/month

Annual

$399

Save 15%

$399/year Get Started

Save 15%

11+ Users

Custom

Savings start at 25%!

Request A Quote Request A Quote

Savings start at 25%!

2-10 Users

$300

Annually per user

$300/year Get Started

$300 Annually per user

View All Plans

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

Subscribe Log In

Previous article： Virginia high school admissions case could be legal follow
Next article： GOP inquiry over gain

focus

WSS

St. Louis police protesters begin picking up checks in $4.9 million settlement

Tuberculosis patients need more than just free medication

Flash flood warnings continue for parts of Missouri, Illinois

The strange history of BMI, the body mass index

NFL doctors' conflicts of interest could endanger players, report says

Biden slams SCOTUS affirmative action decision: 'Discrimination still exists in America'

explore

explore

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

focus

Anesthesiologist group: stop taking Ozempic before surgery

Sage considers ways to reduce costs following FDA rejection

ChatGPT in medicine: STAT answers readers' burning questions

Paxlovid rebound happens. Why and to whom are still a mystery

Affirmative action in medical school literally saved lives

Prince Harry seeks over $550,000 in phone hacking lawsuit against British tabloid publisher

New Eli Lilly Alzheimer’s data poses Medicare coverage conundrum

Why do we get seasonal allergies?

Virginia high school admissions case could be legal follow

Eating disorder hotline's move to chatbot carried cost for volunteers

focus

Judge in Trump's classified documents case questions use of out

Democrats are overperforming in 2023's special elections. Is it a clue for Biden vs. Trump?

It was time to plan for my old age. If only I could stop crying

Kansas bar where gunfire wounded 9 last month closes permanently

Democrats are overperforming in 2023's special elections. Is it a clue for Biden vs. Trump?

Restaurant meals are packed with calories, and we still keep eating them

hotspot

entertainment

focus

hotspot