Swiss biotech restores motor function in mice with Parkinson’s

A Swiss biotech has restored motor function in mice with Parkinson's disease, paving the way for human trials later this year. In a presentation at the WORLDSymposium conference, Gain Therapeutics described a small molecule that enhances the activity of the lysosomal enzyme glucocerebrosidase, better known as GCase. The Gain team believes that by bolstering the enzyme's activity, the compound, GT-02287, would boost the lysosome's ability to clear waste. That would in turn improve mitochondrial function and decrease the incidence of cell death in people with Parkinson's linked to mutations in GBA1 , the gene that encodes for GCase. So far, that approach has worked—in rodent models. Gain's preclinical study showed that over 3 weeks, mice who'd been injected with α-synuclein-preformed fibrils and GCase-inhibited rat neurons to mimic GBA1 Parkinson's regained motor function after taking GT-02287. The mice also had less of the biomarker neurofilament light chain (Nfl) in their bloodstreams.