Aquinnah Pharmaceuticals is focused on a new area of research and development to modulate neurodegenerative stress granules, believed to be important in the pathology of amyotrophic lateral sclerosis (ALS), Alzheimer's disease and other neurodegenerative diseases. The medical need for ALS is very high as there are currently no effective treatments for this devastating neurodegenerative disease. ALS progresses rapidly, eventually affecting muscles in the arms, legs, face, diaphragm, and chest. Today, approximately 60% of patients with ALS will not survive beyond three years following diagnosis, with 10% of new patients surviving at least 10 years.
Aquinnah Pharmaceuticals has identified lead molecules advancing to clinical studies which are designed to prevent and potentially reverse the pathological stress granules in affected patients. In addition to their use in ALS, these compounds also show promise as a potential treatment for cognition impairment in patients with Alzheimer’s disease and other neurodegenerative diseases.
New work published in Nature Neuroscience by co-founder Ben Wolozin, M.D.,Ph.D., has implicated stress granule disease progression with tau; which is a neuronal pathologic protein that correlates with Alzheimer's disease progression. Aquinnah is pioneering new approaches to identify small molecule drugs that modulate intracellular tau-stress granule biology, representing a new first in class treatment for Alzheimer's disease and other tauopathy diseases.
