explore -WSS

explore

author：Wikipedia 2025-10-02 17:21:39 - browse：73448

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： Medical records are filled with copy
Next article： Steward Health Care Massachusetts hospitals face fiscal disaster

WSS

President Biden's budget: Here's what you might have missed

Pfizer's RSV vaccine recommended for approval in Europe

Why scientists need input from humanists on sensitive research

Change Healthcare outage fallout shows need for new oversight

Biden White House, tech companies launch new safeguards around emerging AI technology

As AI in medicine takes off, can 'human in the loop' prevent harm?

hotspot

explore

This article is exclusive to STAT+ subscribers

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

leisure time

Activists slam private health insurers over coverage restrictions

Washington Commanders' Dan Snyder ordered to pay NFL $60M following investigation

Medicare details structure of new drug price negotiation program

Reframing the conversation around gain

Heart attack and stroke risk calculators need revising

Why Marc Tessier

Biden outlines 'new path' to provide student loan relief after Supreme Court rejection

Medicare coverage of Wegovy, once blocked, now looks possible

Alkermes shareholders re

FDA advisers back CAR

hotspot

President Biden's budget: Here's what you might have missed

How many hemophilia patients actually want gene therapy?

Study: Misdiagnosis causes 800,000 deaths, serious disabilities a year in U.S.

EMA weighs against approval for Mirati's lung cancer drug

Prosecutors say FTX founder Sam Bankman

What is the Turing Test that determines if computers can think?

hotspot

knowledge

leisure time

entertainment