VQ-101

VQ-101 is a novel, orally administered, brain-penetrant small molecule that acts as an allosteric activator of the lysosomal enzyme glucocerebrosidase (GCase). Developed by Vanqua Bio, it is designed to increase GCase activity in the brain. In preclinical and early clinical studies, VQ-101 demonstrated robust activation of lysosomal GCase—over 75% in healthy volunteers—which exceeded the target engagement goal informed by human genetics and preclinical models. This activation blocks the accumulation of insoluble alpha-synuclein in patient-derived dopaminergic neurons—a pathological hallmark of Parkinson’s disease (PD). The drug has shown full central nervous system penetration with significant cerebrospinal fluid levels and was well tolerated with only mild or moderate adverse events reported. It is currently being developed for Parkinson’s disease patients both with and without GBA gene mutations[1][2][3][4][5][6][8].

Other names

Glucocerebrosidase activator