The Drug Discovery Engine For ALS

The Drug Discovery Engine For ALS

ALS research is advancing rapidly. Turning that progress into real treatments requires more than discovery—it requires the ability to rigorously test, validate, and advance therapies at scale. The ALS Therapy Development Institute (ALS TDI) is built to do exactly that.
ALS TDI is one of the leading organizations advancing ALS research, focused exclusively on discovering and developing treatments through an integrated, end-to-end drug discovery approach.
Andy in the lab

What Does the ALS Therapy Development Institute Do?

The ALS Therapy Development Institute (ALS TDI) is the world's most comprehensive research lab dedicated solely to discovering and advancing treatments for ALS.
ALS TDI operates as an integrated drug discovery engine, combining:
  • Target identification and validation
    Target identification and validation
  • Cellular and animal disease modeling
    Cellular and animal disease modeling
  • Biomarker discovery and development
    Biomarker discovery and development
  • Preclinical testing and translational research
    Preclinical testing and translational research
In practical terms, ALS TDI identifies potential treatments, tests them in the lab, and advances the most promising toward clinical trials.
This end-to-end capability is critical.
It enables ALS TDI to evaluate therapies more rigorously, reduce gaps between discovery stages, and accelerate the most promising approaches toward clinical trials.

Why ALS TDI's Role Is Critical Right Now

ALS research has reached a pivotal moment.
  • More therapeutic ideas are emerging than ever before
  • New tools are enabling deeper understanding of the disease
  • The field is moving faster—but systems to test these ideas have not scaled at the same pace
Without sufficient capacity, promising approaches risk being delayed, under-tested, or left behind.
ALS TDI addresses this challenge by providing:
A dedicated, ALS-focused research environment
A dedicated, ALS-focused research lab
The ability to test multiple therapies in parallel
The ability to test multiple therapies in parallel
The infrastructure to move seamlessly from discovery to clinical trials
The infrastructure to move seamlessly from discovery to clinical trials
This capability directly impacts how quickly potential treatments can reach people with ALS.

A Landmark Expansion to Accelerate ALS Drug Discovery

A first look at the 33,000 sq. ft. lab space ALS TDI is building out to expand research capacity.
The pace of ALS research is accelerating.
The opportunity to develop effective treatments has never been greater.
To meet the growing demands of the field, ALS TDI is:
  • Doubling its research team
  • Building out a new 33,000 sq. ft. laboratory in Watertown, Massachusetts
This expansion is designed to:
  • Test more potential treatments in parallel
  • Expand translational and biomarker capabilities
  • Accelerate the progression of promising approaches toward clinical trials
  • Reduce bottlenecks in preclinical research
  • Improve the ability to identify therapies most likely to benefit specific patient groups
  • Advance more therapies toward clinical trials faster
To read the expansion announcement, click here

Why This Matters for People Living with ALS

ALS is a progressive disease with limited treatment options.
For people living with ALS, progress depends on:
  • How many therapies can be tested
  • How quickly promising approaches can advance
  • How effectively treatments can be matched to disease biology
This expansion increases the likelihood that effective treatments can be identified and delivered sooner.

Powered by the ALS Community

This expansion is made possible by:
  • A major commitment from Augie's Quest to Cure ALS
  • The continued support of the ALS community
Together, this support enables ALS TDI to scale its impact at a critical moment in ALS research.

How You Can Help Accelerate ALS Research

Supporting ALS TDI helps ensure that promising scientific ideas are not left behind—and that the most effective treatments can reach people living with ALS as quickly as possible.

FAQ

The ALS Therapy Development Institute is a nonprofit biotech focused exclusively on discovering and developing treatments for ALS through an integrated, end-to-end drug discovery approach.

ALS research is being advanced by a range of organizations, including universities, nonprofit research institutes, and biotechnology companies. The ALS Therapy Development Institute (ALS TDI) is one of the leading nonprofit organizations focused exclusively on discovering and developing treatments for ALS.

ALS is a complex and heterogeneous disease. Effective treatment development requires testing therapies across different biological subtypes and advancing them through multiple stages of research.

Scientific ideas must be rigorously tested and validated before they can become treatments. Without sufficient infrastructure, promising therapies may be delayed or not fully evaluated.

Advancing effective therapies requires expertise across multiple areas of research, including disease modeling, biomarker development, therapeutic testing, and translational science.

The expansion will increase research capacity, accelerate testing of therapies, and enable more promising approaches to advance toward clinical trials.