Identification of Inhibitors of human Galactokinase (GALK) for the treatment of Galactosemia

National Center For Advancing Translational Sciences

Investigators

Linked publications & trials

Abstract

This project aims to identify and develop inhibitors of GALK as a potential novel therapy for Classic Galactosemia. During this period, the collaborative team continued with its ongoing medicinal chemistry campaign to (1) improve GALK inhibitory activity on the lead series, (2) improve GALK inhibitory activity on the backup series, (2) improve BBB exposure. Compounds of interest have been advanced into cell-based assays and animal models to further characterize their cellular activity. In addition, a cell-based Gal1-p LC/MS assay has been in development to enable direct measurement of the metabolite in cells and provide a much needed marker of on-target activity.