ALS Therapy Development Institute


Full Member, United States of America

Region covered:

United States of America and International



Date founded: 1999


  • Robert Goldstein, Director of Communications


300 Technology Square, 4th Floor
Cambridge, MA 02139 USA


+1 617 441 7295


+1 617 441 7299







ALS Therapy Development Institute Activities

The mission of the ALS Therapy Development Institute (ALS TDI) is to discover and develop effective therapeutics that slow or stop amyotrophic lateral sclerosis (ALS, Lou Gehrig’s disease) as soon as possible for patients today.

Focused on meeting this urgent unmet medical need, ALS TDI executes a robust target discovery program, while simultaneously operating the world’s largest efforts to preclinically validate potential therapeutics; including a pipeline of dozens of small molecules, protein biologics, gene therapies, and cell-based constructs.  Its pipeline is available online and updated regularly. In addition to operating the world’s largest preclinical ALS program, the institute has two clinical stage candidates: TDI-132 (Gilenya) in Phase 2a with assistance from Novartis and TDI-846 (aCD40L) moving forward in partnership with Biogen Idec and UCB Pharma.

The world’s first nonprofit biotech institute, ALS TDI has developed an industrial-scale platform, employs 30 professional scientists, and evaluates dozens of potential therapeutics each year. Since 1999, it has screened more than 200 compounds, more than the rest of the field combined.

In 2008, ALS TDI published the first ever optimized study design for the SOD1 mouse model, which has been now accepted as the basis of all grant applications to many nonprofits such as Prize 4 Life and the MDA. In addition, its guidelines have been echoed by two international consensus meetings to date. It continues to lead in animal model characterization for ALS and related disorders (ie: FTD).

ALS TDI collaborates with leaders in both academia and industry to accelerate ALS therapeutic development, including Biogen Idec, UCB, the Gladstone Institutes, to-BBB, Aestus Therapeutics, the MDA, Driskill, and RGK Foundations. In addition, ALS TDI offers CRO services to pharma, biotech, and academics with an interest in drug development for ALS patients.

Finally, the Institute also maintains an extensive website with podcasts, webcasts, and regular interviews with experts from across the globe on advancements in the diagnosis, tracking, and treatment of ALS. Its staff also maintains a global database of clinical trial listings.

ALS TDI is a 501(c)3 registered charitable nonprofit organization. For more information, please visit us online at