ALS Therapy Development Institute

What ALS Therapy Development Institute does

ALS Therapy Development Institute (ALS TDI) is a 501(c)(3) nonprofit biotech founded in 1999 and headquartered at 480 Arsenal Street, Watertown, Massachusetts, operating as the world's largest drug discovery lab focused exclusively on amyotrophic lateral sclerosis (ALS). The organization runs an integrated drug discovery engine that spans preclinical research, translational research, and clinical development, with capabilities built around iPSC-derived cell models (including C9orf72, SOD1, and TDP-43 mutations), animal models of neurodegeneration, and the ALS Research Collaborative (ARC) — a global natural history study that captures movement, lifestyle, medical history, genetics, biomarkers, voice recordings, and patient cell biology, with data shared publicly through the ARC Data Commons and Neuromine Data Portal. Its lead in-house invention, tegoprubart (AT-1501), an anti-CD40L antibody therapeutic, became the first ALS treatment invented by a nonprofit biotech to reach human clinical trials; ALS TDI advanced it through a wholly-owned for-profit subsidiary, Anelixis Therapeutics, which was acquired in 2020 by Novus Therapeutics (now Eledon Pharmaceuticals) in a transaction that raised over $108 million for continued clinical development.

The organization's product and program portfolio includes the ARC Study and ARC Data Commons, the Champion Insights study (enrolling 500 high-performance athletes, military veterans, and first responders), the ALS Trial Navigator patient-platform, the Endpoints Podcast, and the ALS Forum (46,555 registered members). In 2026 it launched the PRISM Initiative with LifeArc and Axol Bioscience to globally distribute patient-derived iPSC models, and announced a landmark expansion of its research programs.

ALS TDI is funded entirely through philanthropy and grants — no commercial products are sold. Revenue streams comprise individual donations and peer-to-peer fundraising (Facebook Fundraisers exceeding $1 million all-time), community fundraising events (Tri-State Trek $10M+ cumulative since 2003, Driving for Life $7M+), and grants from foundations such as Augie's Quest, ALS Association, ALS Finding a Cure, ALS ONE, and the Department of Defense CDMRP. Licensing income flows from the tegoprubart arrangement with Eledon Pharmaceuticals via milestone payments and royalties, which are reinvested in research. The organization states that 87 cents of every dollar raised goes directly to ALS research, holds a Charity Navigator four-star rating, and operates globally through its data commons, iPSC distribution via Axol Bioscience, and online community channels.

Name

ALS Therapy Development Institute

Legal name

ALS Therapy Development Institute

Website

https://als.net

Company type

Private

Founded year

2001

Operating status

Operating

Headcount range

11–50 employees

Short description

ALS Therapy Development Institute is a 501(c)(3) nonprofit biotech in Watertown, Massachusetts that operates the world's largest drug discovery lab dedicated solely to ALS, running an integrated preclinical-to-clinical pipeline supported by the ALS Research Collaborative natural history study and global iPSC distribution partnerships.

Ownership category

akta.pro rank

Product category

Biotechnology Research

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA)

Keywords

• ALS drug discovery
• Nonprofit biotech
• IPSC research models
• Translational research
• ALS clinical research

Headquarters

HQ city

Watertown

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B and B2C

Offering type

Services

Cost components

Personnel, Technology or R&D, Operations, Infrastructure, Marketing or Sales

Revenue model

1. Individual Donations and Fundraising: Primary funding source from individual donors, community fundraising events, peer-to-peer campaigns, Facebook Fundraisers, and direct monetary contributions. Most research funding comes from events attended or organized by supporters.
2. Corporate and Foundation Grants: Funding from organizations including Augie's Quest, ALS Association, ALS Finding a Cure, ALS ONE, and Department of Defense CDMRP. Also receives matching gift contributions from employer programs.
3. Licensing and Milestone Payments: Through licensing agreements with Anelixis Therapeutics (now Eledon Pharmaceuticals), ALS TDI receives milestone payments and royalties if drugs are successful. Financial returns are reinvested in ALS research.

Go-to-market motion1 record

Distribution channels3 records

Marketing channels9 records

Core offering

ALS Therapy Development Institute (ALS TDI) is the world's largest nonprofit drug discovery lab focused solely on ALS, operating an integrated drug discovery engine that combines preclinical research, translational research, and clinical development. The organization runs the ALS Research Collaborative (ARC) global natural history study, develops patient-derived iPSC disease models, and invented tegoprubart (AT-1501) — the first potential ALS treatment developed by a nonprofit biotech and advanced into human clinical trials.

Product overview

ALS Therapy Development Institute (ALS TDI) operates as a nonprofit biotech with a unified drug discovery platform focused solely on ALS. The organization's product portfolio centers on its Drug Discovery Engine, which integrates preclinical research, translational research, and clinical programs under one roof. Key offerings include the ALS Research Collaborative (ARC), a global natural history study gathering comprehensive data from people with ALS; the ARC Data Commons, providing researchers worldwide access to ALS datasets; Champion Insights, investigating ALS risk factors in high-risk populations; the ALS Trial Navigator, helping patients find clinical trials; and the Endpoints Podcast for community engagement. ALS TDI also developed tegoprubart (AT-1501), the first drug invented by a nonprofit biotech and advanced to human clinical trials. The organization recently launched the PRISM Initiative in collaboration with LifeArc and Axol Bioscience to expand access to patient-derived stem cell models.

Differentiator

Problem solved

Functional benefit

Products and services

• ALS Research Collaborative (ARC) A global initiative developed to better understand the underlying biology of ALS and significantly accelerate the discovery of treatments. ARC gathers extensive natural history data from people with ALS — including movement, lifestyle, medical history, genetics, biomarkers, voice recordings, and patient cell biology — and layers this with data measuring underlying biological processes.
• ARC Data Commons A data portal that grants researchers access to over eight years of de-identified natural history data contributed by people with ALS, along with tools to filter and visualize data. Made available through the Neuromine Data Portal, integrating Answer ALS and ALS TDI ARC datasets.
• Champion Insights Study A nationwide study investigating the elevated risk of ALS among high-performance athletes, military veterans, and first responders. The research involves enrolling 500 participants to analyze genetic, biological, and environmental factors.
• ALS Trial Navigator An innovative platform that educates people with ALS about current clinical trials and provides personalized recommendations based on eligibility and preferences.
• Drug Discovery Engine ALS TDI's integrated research operation that encompasses all aspects of drug discovery under one roof, including preclinical research, translational research, and clinical programs to move potential ALS treatments forward.
• Tegoprubart (AT-1501) The first potential ALS treatment invented by a nonprofit biotech (ALS TDI) and advanced to human clinical trial. It is an antibody therapeutic that targets the CD40L pathway to block specific immune cell activation and protect nerves against ALS progression. Licensed to Anelixis Therapeutics (later acquired by Eledon Pharmaceuticals) for continued clinical development.
• AQ Translational Research Center A dedicated research center for translational research focused on moving scientific discoveries from the lab toward clinical application.
• PRISM Initiative (Pluripotent Stem Cell Repository for International ALS Modeling) A global collaboration to accelerate ALS treatment discovery by improving access to patient-derived induced pluripotent stem cell (iPSC) models that better reflect the biological diversity of ALS, including sporadic cases.

Quantifiable outcome

• 87 cents of every dollar raised goes directly to ALS research
• +2 more outcomes

Named customers3 records

Segments3 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Twelve partnerships are on record, tiered core and minor.

• LifeArccoreStrategic or Co-development Partner · 1 April 2026PRISM (Pluripotent Stem Cell Repository for ALS) initiative launched with ALS TDI and Axol Bioscience to expand access to patient-derived induced pluripotent stem cell (iPSC) models for ALS research. The program addresses the critical gap that approximately 85% of ALS cases are sporadic yet most research has relied on models representing only rare genetic subtypes.
• Axol BiosciencecoreTechnology or Integration · 1 April 2026PRISM initiative partner focused on manufacturing and distributing iPSC-derived motor neuron models. Separate supply agreement also makes ALS patient-derived iPSC lines available to researchers globally. Axol manufactures and distributes iPSC-derived cells to academic and industry researchers worldwide.
• Answer ALScoreStrategic or Co-development Partner · 4 February 2026Champion Insights study launched in partnership with Answer ALS and Augie's Quest to Cure ALS. Answer ALS contributes data integration with the Neuromine Data Portal, combining datasets from over 1,100 participants with ALS TDI's ARC study data.
• Augie's Quest to Cure ALScoreStrategic or Co-development Partner · 13 August 2025Partnership for Champion Insights study to investigate elevated ALS risk among high-performance athletes, military veterans, and first responders. Joint effort to uncover critical genetic and metabolic factors.
• Axol Bioscience (iPSC Supply Agreement)coreTechnology or Integration · 24 June 2025Supply agreement making ALS patient-derived iPSC lines available to researchers worldwide. Axol manufactures and distributes iPSC-derived cells including motor neurons, microglia, and astrocytes with key ALS-associated mutations (C9orf72, SOD1, TDP-43).
• Eledon PharmaceuticalscoreStrategic or Co-development Partner · 1 September 2020Eledon Pharmaceuticals (formerly Novus Therapeutics, acquired Anelixis Therapeutics) is advancing tegoprubart through clinical development. Transaction raised over $108 million for future development. ALS TDI receives milestone payments and royalties if successful.
• BiogenminorOthersSponsor of the Endpoints Podcast
• RegeneronminorOthersSponsor of the Endpoints Podcast
• IonisminorOthersSponsor of the Endpoints Podcast
• EledonminorOthersSponsor of the Endpoints Podcast
• ShionogiminorOthersSponsor of the Endpoints Podcast
• Insource Services, Inc.minorOthersSponsor of the Endpoints Podcast

Scale indicators10 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• ALS Association: The largest U.S. ALS-focused nonprofit, funding research, care services, and advocacy. Directly comparable to ALS TDI as a national-scale ALS mission organization, though differentiated by care-services emphasis vs. ALS TDI's drug discovery focus.
• Augie's Quest to Cure ALS: Disease-specific ALS research nonprofit founded by fitness industry figures and an active ALS TDI partner (Champion Insights). Comparable as a mission-aligned, donation-funded ALS research organization with a narrower programmatic footprint than ALS TDI.
• ALS Finding a Cure: ALS-focused research foundation that funds academic and translational ALS programs. Comparable as an ALS-specific nonprofit research funder that has provided grant support to ALS TDI.
• Target ALS: Nonprofit foundation that funds collaborative ALS research and drug discovery consortia. Directly comparable to ALS TDI as an ALS-exclusive research funder with a consortium-style model.
• Answer ALS: ALS research consortium whose data integrates with ALS TDI's ARC through Neuromine. Directly comparable as a patient-data-generating ALS research initiative.
• ALS ONE: Massachusetts-based ALS nonprofit that has provided grant funding to ALS TDI. Comparable as a regional, ALS-focused research and care nonprofit.
• Robert Packard Center for ALS Research: Johns Hopkins-based ALS research center focused on basic and translational ALS science. Comparable as a dedicated ALS research organization, though housed in academia rather than operating as an independent nonprofit biotech like ALS TDI.
• Prize4Life: Nonprofit that incentivizes ALS research through prize-based challenges and translational funding. Comparable as an ALS-specific, donation-supported research organization focused on accelerating translational outcomes.

Broad incumbents

• Muscular Dystrophy Association (MDA): Large neuromuscular disease nonprofit that funds research and care across ALS, muscular dystrophy, and related conditions. Comparable as a mission-driven, donation-funded research nonprofit with ALS as one of several disease verticals.
• Cystic Fibrosis Foundation: Large disease-specific nonprofit that pioneered the venture-philanthropy model of funding drug discovery and licensing out resulting therapeutics (e.g., royalties from Vertex's CFTR modulators). Comparable as a nonprofit that funds end-to-end drug discovery and captures economic returns to reinvest in research — a structural template for ALS TDI's own model.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/alstdi
• YouTubeyoutube.com/alstdi
• Facebookfacebook.com/alstherapydevelopmentinstitute
• LinkedInlinkedin.com/company/als-therapy-development-institute

Revenue estimate

Valuation estimate

Number of profiles

Profiles2 records

Subsidiaries2 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about ALS Therapy Development Institute