Julie Ullman, PhD, of Maze Therapeutics, discusses a new investigational drug, MZE001, that is a small molecule inhibitor of glycogen synthase 1 (GSY1) for the treatment of Pompe disease during the Adult Polyglucosan Body Disease conference. Preclinical data on this small molecule inhibitor found that there was a dose-dependent, muscle-specific reduction in glycogen in both wild-type and Pompe mice. This substrate reduction therapy has an additive benefit when combined with currently approved enzyme replacement therapy. The promising preclinical data led to an ongoing Phase 1 clinical study and plans for a Phase 2 clinical study.