The Tough Tech problem we are solving
There are currently no effective therapies for most neurodegenerative diseases including Alzheimer’s Disease and ALS (also known as Lou Gehrig's disease). One of the primary challenges in developing these much-needed treatments has been the limited understanding of the root causes of these diseases. Recent breakthroughs by our founders and other researchers in the field have identified biomolecular condensates as a revolutionary, previously unrecognized molecular mechanism that regulates protein interactions within cells under normal conditions. When disrupted, this mechanism contributes to neuronal dysfunction and cell death, driving the progression of several brain degenerative diseases.
About our solution
Aquinnah Pharmaceuticals leverages our knowledge of biomolecular condensates, phase separation and stress granules to develop first-in-class, oral, brain-penetrate small molecules that treat the underlying cause of neurodegeneration. Using our core technologies, we have identified new therapeutic agents that have the potential to modify disease and deliver benefit to patients suffering from Alzheimer’s Disease, ALS, and other neurological diseases.