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RCZY690 is an orally bioavailable small molecule inhibitor of Ubiquitin-Specific Protease 1 (USP1), developed by RemeGen (Yantai RemeGen Pharmaceutical). USP1 is a deubiquitinating enzyme that plays a critical role in the DNA damage response (DDR) pathway, specifically by regulating translesion synthesis and the Fanconi anemia pathway through the deubiquitination of PCNA and FANCD2. By inhibiting USP1, RCZY690 promotes the accumulation of ubiquitinated substrates, leading to genomic instability and apoptosis in cancer cells, particularly those with existing deficiencies in other DDR pathways (such as BRCA1/2 mutations). The drug is currently being evaluated in Phase I/II clinical trials in China for the treatment of locally advanced unresectable or metastatic malignant solid tumors, where it is being assessed for safety, tolerability, pharmacokinetics, and preliminary antitumor activity.
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