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TJ036 is a small molecule inhibitor of the Werner syndrome ATP-dependent helicase (WRN), currently in preclinical development by Tuojie Biotech (拓界生物医药). It is designed for the treatment of solid tumors, specifically those characterized by a microsatellite instability-high (MSI-H) phenotype. WRN helicase is a member of the RecQ family and has been identified as a synthetic lethal target in MSI-H cancer cells, where its function is essential for maintaining genomic stability. By inhibiting WRN, TJ036 induces profound DNA damage and apoptosis selectively in MSI-H tumor cells while sparing normal cells. This targeted approach aims to address unmet needs in MSI-H colorectal, endometrial, and gastric cancers.
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