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Medivir's USP7 inhibitor is a preclinical-stage small molecule program targeting Ubiquitin-Specific Protease 7 (USP7), a deubiquitinating enzyme also known as HAUSP. USP7 plays a pivotal role in the regulation of protein stability within the cell, particularly the p53-MDM2 pathway. By inhibiting USP7, the drug prevents the deubiquitination of MDM2, an E3 ubiquitin ligase that targets the tumor suppressor p53 for degradation. The resulting decrease in MDM2 levels leads to the stabilization and activation of p53, which induces cell cycle arrest and apoptosis in cancer cells. Beyond p53 stabilization, USP7 inhibition is also thought to modulate the immune system by reducing the suppressive activity of regulatory T cells (Tregs). Originally developed under a research collaboration with Bristol Myers Squibb, the program was returned to Medivir in 2018 for further internal development.
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