GT-02287, a clinical-stage GCase enhancer, improves activities of daily living and cognitive performance in a preclinical model of GBA1 Parkinson’s disease
Beatriz Calvo-Flores Guzman, Joanne Taylor · FENS Forum 2024
The aim of this study was to investigate the ability of GT-02287 to rescue nest-building behaviour following delayed administration in a mouse model of GBA1-Parkinson's disease (GBA1-PD). Nest building is a non-invasive test to study activities of daily living and cognitive performance in rodent models. GT-02287 is an orally bioavailable, brain penetrant, small-molecule structurally targeted allosteric regulator that binds to glucocerebrosidase (GCase), stabilizes it, and increases its function by chaperoning it to the lysosome. It is currently in Phase 1 clinical development for the treatmen
ePoster · Neuroscience