LAE122

LAE122 is a novel, potent, and selective small molecule inhibitor of the Werner syndrome ATP-dependent helicase (WRN), developed by Laekna Therapeutics. It is designed as a synthetic lethal therapy for the treatment of microsatellite instability-high (MSI-H) cancers, including colorectal, gastric, endometrial, and ovarian malignancies. LAE122 demonstrates high potency with single-digit nanomolar IC50 values in MSI-H cell lines while sparing microsatellite stable (MSS) cells, suggesting a wide therapeutic window. As of early 2025, the candidate has completed Preclinical Candidate (PCC) declaration and is undergoing IND-enabling studies.