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LG00313112 is an investigational small molecule designed to selectively target and stabilize the Y220C mutant form of the p53 tumor suppressor protein. The TP53 Y220C mutation is a common oncogenic driver that creates a surface pocket in the p53 DNA-binding domain, leading to protein destabilization and loss of function. LG00313112 acts as a molecular chaperone, binding to this specific pocket to restore the wild-type conformation and transcriptional activity of p53. By reactivating p53, the drug aims to trigger tumor-suppressive pathways, including cell cycle arrest and apoptosis, in cancer cells harboring this specific mutation. It is currently being evaluated in Phase 1/2 clinical trials for patients with advanced solid malignancies.
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