This project describes a drug discovery plan for Classic Galactosemia, a rare disorder of galactose metabolism where there is currently no disease-transforming therapy. This TEP provides the protein reagents, biophysical assays, and structural information to facilitate two aspects of galactosemia research: (i) to understand the molecular basis of the disease due to defective GALT enzyme, and (ii) to provide chemical starting points to explore metabolic intervention of the upstream enzyme GALK1 aimed at mitigating the GALT defect (substrate reduction).

This document represents version 2 of the TEP datasheet and includes all updates on the project as of June 2019. For more information about TEPs and the TEP Programme, please visit https://thesgc.org/tep