The ‘holy grail’ in harnessing biomolecular condensates for drug development is to identify small molecules that can be delivered as a pill and selectively interfere with the formation of pathological biomolecular condensates to prevent neuronal death and dysfunction.

Aquinnah Pharmaceuticals, the first company to realize the potential of stress granules for drug development, has years of experience developing small compounds which selectively target various pathologic biomolecular condensates.

Our platform has proven immensely successful – where we are able to show in vivo efficacy for our compounds in some of the most aggressive animal models of ALS and Alzheimer’s disease. These successes are driving the advancement of our programs towards clinical trials.