GNE-317 is an investigational small molecule inhibitor primarily studied for its potential in cancer therapy. It targets the PI3K/AKT/mTOR signaling pathway, which is frequently dysregulated in various tumors. By inhibiting key kinases in this pathway, GNE-317 helps suppress tumor cell proliferation and survival. It has shown activity against brain-penetrant tumor models, making it particularly relevant for the treatment of central nervous system (CNS) malignancies, including glioblastoma and brain metastases. Due to its ability to cross the blood-brain barrier, GNE-317 is being explored in preclinical and early clinical settings as a promising agent for hard-to-treat neurological cancers. Its development supports efforts to create more effective, targeted therapies with improved CNS exposure.