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CS31864 is a preclinical-stage small molecule inhibitor targeting the Werner syndrome ATP-dependent helicase (WRN), developed by Chipscreen Biosciences. The drug is designed to exploit the concept of synthetic lethality in cancer cells, particularly those exhibiting high microsatellite instability (MSI-H). WRN helicase is essential for the survival of MSI-H cancer cells, which are characterized by defects in DNA mismatch repair. By inhibiting WRN, CS31864 aims to induce DNA damage and cell death specifically in MSI-H solid tumors while sparing normal cells.
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