RO7623066

RO7623066 (also known as KSQ-4279) is an oral, first-in-class, selective small molecule inhibitor of Ubiquitin Specific Peptidase 1 (USP1), a deubiquitinating enzyme involved in the regulation of DNA damage response pathways such as translesion synthesis and the Fanconi anemia pathway. By inhibiting USP1, RO7623066 aims to disrupt DNA repair mechanisms in cancer cells, potentially enhancing their sensitivity to DNA-damaging agents. The drug is being developed for use as a monotherapy and in combination with other agents like PARP inhibitors (e.g., olaparib) or chemotherapy. It is currently under investigation in Phase 1 clinical trials for patients with advanced solid tumors, particularly those enriched for deleterious homologous recombination repair mutations. The compound was originally developed by KSQ Therapeutics and subsequently licensed to Roche for further development and commercialization[2][3][5][7].