Project Grant R44NS141531

The National Institute of Neurological Disorders and Stroke (NINDS) awarded Aquinnah Pharmaceuticals, Inc. a $1,499,212 Project Grant (CFDA 93.853) to develop a novel oral brain-penetrant therapeutic for Amyotrophic Lateral Sclerosis (ALS). The funding will support manufacturing, IND-enabling studies, and a PIND meeting with the FDA for this innovative new clinical drug candidate designed to slow and potentially reverse ALS disease progression. The proposed therapeutic targets the key pathological hallmark of ALS, the accumulation of insoluble, aggregated TDP-43 protein, with the goal of restoring its normal RNA splicing function and dramatically changing clinical outcomes for over 90% of ALS patients. This project award is funded through the NINDS Extramural Research Programs in the Neurosciences and Neurological Disorders, which supports diverse scientific investigations into brain function, neurological disease mechanisms, and potential therapeutic interventions.