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

点击翻译按钮获取中文摘要

Project highlights: the full compound library was screened and a lead chemotype was identified for development. Medicinal chemistry is ongoing to improve GALK inhibitory activity, and advanced assays are in progress to characterize cellular activity of these compounds. During this period, the NCGC has fostered and maintained over 180 active collaborations with both NIH and extramural investigators, facilitating drug discovery efforts across the entire spectrum of human disease. These efforts have led to over 100 high-throughput screens and nearly 60 medicinal chemistry campaigns, providing our collaborators and the general research community a wealth of publications and promising small molecule leads. In addition, the NCGC has undertaken a number of informatic challenges to make better use of existing drug and disease target information and provide the general public with easily accessible resources, further catalyzing the development of new therapies for human disease.