Walter and Eliza Hall Institute-glycogen ubiquitination pathway-small molecule

This small molecule is a preclinical drug candidate developed through a collaboration between the Walter and Eliza Hall Institute (WEHI) and Roche. It is designed to modulate the glycogen ubiquitination pathway, a critical regulatory system for glycogen metabolism. In healthy cells, the E3 ubiquitin ligase Malin (encoded by NHLRC1) and the phosphatase Laforin (encoded by EPM2A) work together to regulate glycogen structure and prevent the formation of insoluble glucose polymers. Dysregulation of this pathway is primarily associated with Lafora disease, a severe, progressive myoclonus epilepsy characterized by the accumulation of toxic Lafora bodies (polyglucosans) in the brain and other tissues. By targeting this pathway, the small molecule aims to restore normal glycogen homeostasis and prevent the formation of these toxic aggregates.

Other names

Glycogen ubiquitination pathway modulator