MZE001

MZE001 is an investigational oral small molecule designed as a substrate reduction therapy for Pompe disease. It acts as a potent and selective inhibitor of muscle glycogen synthase (GYS1), the enzyme chiefly responsible for producing glycogen in muscle cells. By inhibiting GYS1, MZE001 aims to reduce the synthesis and subsequent toxic accumulation of glycogen in skeletal and respiratory muscles—a hallmark of Pompe disease that is often resistant to current standard-of-care enzyme replacement therapies. Developed by Maze Therapeutics using their Compass platform, which leverages human genetic data and advanced data science, MZE001 has demonstrated promising preclinical results and was well-tolerated in Phase 1 clinical trials, showing exposure-dependent reductions in blood cell glycogen levels. The drug has received orphan drug designation from the FDA for Pompe disease and is advancing into Phase 2 clinical trials[1][2][4][5][6][7][8].

Other names

MazeMaze Pompe program