The AI-powered drug discovery platform Magellan paved the way for GT-02287, a potential disease-modifying treatment for Parkinson’s disease. [Image: Gain Therapeutics]
The Bethesda biotech Gain Therapeutics, armed with supercomputer firepower, aims to disrupt Parkinson’s disease treatment. Their GCase regulator, named GT-02287, completely restored motor function in a recent animal study.GCase, short for glucocerebrosidase, targets both lysosomal and mitochondrial dysfunction. In Parkinson’s, the failure of cellular powerhouses (mitochondria) and recycling centers (lysosomes) disrupts essential functions. This breakdown leads to a toxic buildup of glycosphingolipids (fatty substances within cells) and damaging clumps of alpha-synuclein (a misfolded protein), hallmarks of Parkinson’s disease and the cause of Lewy bodies. For a visual explanation of this process, see the diagram several par…