Aquinnah TDP-43 ALS program

Aquinnah Pharmaceuticals is developing a program of oral small molecules for the treatment of Amyotrophic Lateral Sclerosis (ALS) by targeting TDP-43 pathology. The approach utilizes the company's proprietary platform focused on stress granule biology and biomolecular condensate formation to identify compounds that can eliminate or prevent the formation of toxic stress granules in nerve cells. TDP-43 protein aggregation is a hallmark pathology present in approximately 95% of ALS patients. In 2022, Aquinnah entered into a research collaboration with Roche to advance these brain-penetrant small molecules toward the selection of a clinical candidate, after which Roche will assume responsibility for global development and commercialization. The program has received financial backing from major pharmaceutical investors including Pfizer, AbbVie, and Takeda.

Other names

Aquinnah TDP-43 modulatorAquinnah stress granule modulator