The company's technology platform uses human genetics and patient-derived neuronal cells to identify and validate novel disease pathways associated with lysosomal dysfunction or aberrant activation of the innate immune system. Vanqua Bio focuses on developing small-molecule activators of glucocerebrosidase (GCase) and advanced programs targeting the innate immune system to accelerate the progression of several neurological diseases. Their unique drug-development approach uses proprietary research tools and in vitro modeling of disease, based on patient-derived neuronal cells, to develop transformative therapies that slow or stop the progression of Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and Gaucher disease (GD).