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OATD-4828 is a potent, selective, and orally bioavailable small molecule inhibitor of Ubiquitin-Specific Protease 7 (USP7), developed by Molecure S.A. USP7 is a deubiquitinating enzyme that plays a critical role in regulating the stability of key oncogenic and tumor-suppressive proteins. By inhibiting USP7, OATD-4828 promotes the degradation of MDM2, which is the primary E3 ubiquitin ligase for p53. This leads to the stabilization and activation of the tumor suppressor protein p53, inducing p53-dependent cell cycle arrest and apoptosis in various cancer types. Beyond the p53 pathway, USP7 inhibition may also impact other pathways involved in DNA repair and immune evasion. OATD-4828 is currently being investigated in Phase 1 clinical trials for the treatment of advanced solid tumors, particularly those where the p53 pathway is functional but suppressed by MDM2 overactivity.
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