Aquinnah Pharmaceuticals is focused on an exciting new area of research and development to modulate neurodegenerative stress granules, believed to be important in the pathology of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. Established in 2014 with the goal of developing compounds to treat ALS as its top priority, Aquinnah builds on the groundbreaking work of founder Ben Wolozin, MD, PhD, 2013 recipient of the prestigious Zenith Fellows Award and chairman of the National Institutes of Health Cellular and Molecular Biology Neurodegeneration Study Section. Aquinnah team members also include veteran pharma and biotech executives Glenn Larsen, PhD and Manfred Weigele, PhD.
The medical need for ALS is very high; there are currently no effective treatments for this devastating neurodegenerative disease, which limits nerve cell function of controlling voluntary muscles. ALS progresses rapidly, eventually affecting muscles in the arms, legs, face, diaphragm, and chest. Today, approximately 50% of patients with ALS die within two to three years of diagnosis.
Aquinnah’s drug-screening assays have identified excellent drug candidates with the potential to prevent and possibly reverse the formation of pathological stress granules. In addition to their use in ALS, these compounds show promise as a potential treatment for cognition impairment in patients with Alzheimer’s disease.
Aquinnah is working to better understand and validate the targets of action and plans to develop the most promising compound into an IND drug candidate to treat patients with ALS and change the clinical progression of this devastating orphan disease.
- Glenn Larsen, PhD President and CEO
- Benjamin Wolozin, MD, PhD Chief Scientific Officer
- Manfred Weigele, PhD Chemistry