Finding Cures for ALS

Our unique and comprehensive approach leverages industry experience and a nonprofit mission - focused 100% on ALS research.

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Our Research Strategy

We are the world's foremost drug discovery center focused solely on ALS. Pioneering the ‘nonprofit biotech’ model, we have placed the person with ALS at the center of every decision made in our labs. Our interdisciplinary, industry-trained team of scientists collaborate daily in our labs and through academic and pharmaceutical company partnerships across the globe to discover cures for ALS.

To date, ALS TDI has conducted a number of clinical trials. We are currently planning a trial of AT-1501, the most effective treatment ever tested at ALS TDI; the first in-human clinical trial will start enrolling in 2018.

In truth, more than one treatment will be needed to bring relief to all those diagnosed with ALS. So, while we must be all in on AT-1501, we also must ensure that we advance other well validated compounds into trials as quickly as possible. That's why we continue to urgently search for other exciting treatments beyond AT-1501 to advance into clinical trials. In fact, ALS TDI screens more potential treatments in its labs each year than anywhere else in the world. We are agnostic to target, as well as therapeutic modality. We will pursue the best ideas for slowing, stopping and ending ALS. Period.

Scientists at ALS TDI are in the process of setting up a translational research program around the next most exciting treatment beyond AT-1501. Continuing to move the best validated ideas from the lab in to the clinic is what our mission is about; the discovery and development of effective treatments for ALS.

Our innovative science and cutting-edge approach have resulted in:

Our Pipeline

ALS is a complex disease with many different parts of a person's body and biology playing a role in either its onset or progression. We believe that this requires us to explore a robust pipeline of potential treatments at the same time in our labs. This pipeline provides a glimpse into the current potential treatments we are working on, why we are looking at them and where they are in the process. Click on an item (ex TDI-111) on the graphic to learn more about individual projects.

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We take our open-door philosophy and commitment to transparency very seriously. And, we love to show off!


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You are needed in the first ever integrated precision medicine effort to end ALS!

This program seeks to gain critical new insight into the discovery and clinical development of treatments for ALS through the integration of genomic, molecular, and patient reported data.

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Favorable Mentions

TDI 208 TDI 209 TDI 168 Unfolded Protein Response TDI 201 TDI 207 TDI 197 TDI 210 TDI 163 TDI 104 TDI 142 SOD1 TDI 200 TDI 186 Neuromuscular Junction TDI 206 TDI 211 Immune Modulation TDI 202 TDI 28 TDI 132 TDI 53 TDI 158