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GW-01 (also known as GW01-200) is a first-in-class small molecule inhibitor of Ataxin-2 (ATXN2) that targets the Notch signaling pathway, developed by Groovy Medicine (Gaowei Pharma). Discovered using the company's proprietary AI-driven high-dimensional biology platform, GROOVY, GW-01 is designed to overcome the historical 'undruggable' barriers associated with the Notch pathway by targeting Ataxin-2 to inhibit Notch pathway activation. GW-01 is currently in Phase I clinical trials in both China and the United States for the treatment of advanced solid tumors and hematological malignancies, including esophageal cancer, lung cancer, breast cancer, and leukemia.
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