GNE-781 is a potent, selective, and brain-penetrant inhibitor of EZH2 (enhancer of zeste homolog 2), a histone methyltransferase involved in epigenetic regulation. It is primarily used in neuroscience research to study neurological and psychiatric disorders, such as Alzheimer's disease, schizophrenia, Huntington's disease, and other neurodegenerative conditions. By inhibiting EZH2, GNE-781 helps investigate pathways related to cognitive, behavioral, and neuronal processes, including the reduction of abnormal protein accumulation and improvement of neuronal function. Researchers employ it to explore potential therapeutic effects, understand disease mechanisms, and develop targeted treatments. Additionally, it is utilized in preclinical studies to evaluate efficacy, safety, pharmacokinetics, and brain penetration, advancing novel drug candidates for CNS disorders.