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WSD5001 is a potent and selective small molecule inhibitor of Ubiquitin-specific protease 1 (USP1), currently under development by Wayshine Biopharm. USP1 is a deubiquitinating enzyme that plays a pivotal role in the DNA damage response (DDR) pathway, specifically by regulating the monoubiquitination levels of PCNA and FANCD2, which are essential for translesion synthesis and the Fanconi anemia DNA repair pathway. By inhibiting USP1, WSD5001 prevents the deubiquitination of these substrates, leading to the accumulation of DNA damage and inducing synthetic lethality in cancer cells with existing DDR deficiencies, such as those with BRCA1/2 mutations. WSD5001 is primarily being investigated for the treatment of advanced solid tumors, including ovarian and breast cancers, and is designed to be used as a monotherapy or in combination with other DDR-targeted agents like PARP inhibitors to overcome resistance.
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