GNE-617 is a potent and selective small molecule inhibitor primarily investigated for its role in cancer research, specifically targeting tumors with specific genetic mutations. It functions by inhibiting mutant forms of the isocitrate dehydrogenase 1 (IDH1) enzyme, which are commonly found in certain gliomas and acute myeloid leukemia (AML). By blocking the abnormal activity of mutant IDH1, GNE-617 helps reduce the production of the oncometabolite 2-hydroxyglutarate (2-HG), which is known to interfere with cellular differentiation and promote tumor growth. This restoration of normal cellular differentiation makes GNE-617 a valuable tool in preclinical studies aimed at understanding targeted therapy mechanisms in IDH1-mutant cancers. Its application is mainly focused on oncology drug development, serving as a lead compound for designing therapies that can cross the blood-brain barrier, making it particularly relevant for treating brain cancers.