GW01-200

GW01-200 is an orally bioavailable small molecule inhibitor of Ubiquitin-Specific Protease 1 (USP1), developed by Groovy Medicine (Hangzhou) Ltd. for the treatment of advanced malignancies. USP1 is a deubiquitinating enzyme that plays a pivotal role in the DNA damage response (DDR) by regulating the ubiquitination status of proteins such as PCNA and FANCD2, which are essential for translesion synthesis and the Fanconi Anemia pathway. By inhibiting USP1, GW01-200 disrupts these repair mechanisms, leading to the accumulation of DNA damage and inducing apoptosis, particularly in tumors with homologous recombination deficiencies. The drug is currently being evaluated in a Phase 1 first-in-human study to assess its safety, tolerability, and preliminary efficacy in patients with advanced solid tumors and hematological malignancies.