Drug intelligence / Profile preview

GW-01

Development stage
Unknown
Lead developer
Groovy Medicine
Modality
Small Molecules
Administration
Oral
01

Overview

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.

02

Targets

ATXN2 (Ataxin-2)

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