explore -WSS

explore

author：comprehensive 2025-09-02 17:51:08 - browse：1931

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： In age of alternative facts, a scholarly course on calling out crap
Next article： Juul spent big to court Black leaders to promote its e

WSS

How to make Best Hospitals ratings actually useful for patients

Some dementia cases could be undiagnosed liver disease

Vertex non

Time to name cancers by genetics, not organ of origin, expert says

Quicker fix for pulse oximeters urged during FDA public meeting

Departing Stanford president retracts two papers

leisure time

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

Peter Hotez and the public health issue of online harassment

Radiation, mainstay of cancer treatment, begins a fade

Affirmative action in medical school literally saved lives

New Ultima Genomics DNA sequencer can read genome for $100

In Memoriam: Notable people who died in 2023

Unicorn down: Following layoffs, Biofourmis CEO quietly departs

Medical records are filled with copy

What AAP's gender

Tori Bowie and the unacceptable rates of Black maternal death

Apple Vision Pro: A first look at health apps on the new VR headset

focus

Departing Stanford president retracts two papers

Appeals court upholds limits to abortion pill

How to make Best Hospitals ratings actually useful for patients

Appeals court upholds limits to abortion pill

How to make AI work for medicine

Some dementia cases could be undiagnosed liver disease

entertainment

hotspot

focus

focus