Discovery and Characterization of Inhibitors of Ubiquitin Like Modifier Activating Enzyme 5 (UBA5): Targeting the UFMylation Pathway in Cancer and Neurodegenerative Disease
A robust high-throughput screening assay based on AMP-Glo™ and a library of blood–brain barrier–permeable compounds are developed, identifying five inhibitors spanning three distinct scaffolds with low micromolar potency that establish UBA5 as a tractable enzymatic target and provide first-in-class chemical tools to pr...