A Novel Cell-Based Assay to Identify Small Molecules for Galactocerebrosidase (GALC)

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During this period, the collaborative team has worked to validate and characterize hit compounds previously identified in the primary GALC screen. SAR studies are ongoing, alongside the development of a qHTS assay using MS and human krabbe oligodendrocytes (produced by reprogramming patient fibroblast). This assay will allow the quantification of lipid reduction upon treatment with analogs of hit molecules. As a center, the Early Translation Branch (ETB) has fostered and maintained over 110 active collaborations with both NIH and extramural investigators, facilitating drug discovery efforts across the entire spectrum of human disease. These efforts have led to dozens of high-throughput screens and a number of medicinal chemistry campaigns to further improve on screening hits, providing our collaborators and the general research community with publications and a variety of promising small molecule probes and leads. In addition, the ETB has worked to advance a number of informatics initiatives 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.