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CS24123 is a small molecule inhibitor of ubiquitin-specific-processing protease 1 (USP1) being developed by Chipscreen Biosciences. USP1 is a deubiquitinating enzyme that plays a critical role in the DNA damage response, particularly in the Fanconi anemia pathway and translesion synthesis. By inhibiting USP1, CS24123 prevents the deubiquitination of key substrates like FANCD2 and PCNA, leading to genomic instability and cell death in cancer cells, especially those with homologous recombination deficiencies. The drug is currently in the IND-enabling phase for the treatment of solid tumors.
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