We believe that the degradation of toxic cellular components, whether they are damaged organelles or aggregated proteins, is a fundamental node of biology. Mutations that impair these clearance pathways are causative in multiple neurodegenerative diseases, many of which are associated with cognitive dysfunction. The company’s targeted approaches leverage genetic data and unique biological understanding to develop small molecules that activate neurons’ clean-up and recycling processes – accelerating clearance of toxic materials and defective cellular components.

New insights in genetics highlight the link between malfunctioning lysosomal processes, that normally maintain order inside neurons, and the risk of subsequent deterioration. Rheostat is a leader in targeting these mechanisms with disease-modifying compounds for patients suffering the devastating effects of neurodegenerative disease. These strategies could also benefit a range of other life-threatening conditions.