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XYD-A01 is an investigational, orally bioavailable small molecule inhibitor of Ubiquitin-Specific Protease 1 (USP1), developed by Kunshan Xinyunda Biotechnology. USP1 is a deubiquitinating enzyme that plays a pivotal role in the DNA damage response (DDR), specifically within the Fanconi Anemia (FA) pathway and translesion synthesis. By inhibiting USP1, XYD-A01 prevents the deubiquitination of key substrates such as FANCD2 and PCNA, which leads to the accumulation of DNA damage and genomic instability. This mechanism is designed to induce synthetic lethality in tumors with homologous recombination deficiency (HRD), such as those harboring BRCA1/2 mutations. XYD-A01 is primarily being developed for the treatment of advanced solid tumors, including cases that have developed resistance to PARP inhibitors, and is currently undergoing Phase 1 clinical evaluation.
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