UCT-01-097

UCT-01-097 is an orally bioavailable, selective small molecule inhibitor of **Ubiquitin Specific Peptidase 1 (USP1)**, currently under development by **1200 Pharma** (Twelve Hundred Pharma) for the treatment of advanced solid tumors. USP1 is a critical deubiquitinating enzyme (DUB) that regulates the DNA damage response (DDR) by removing monoubiquitin tags from Proliferating Cell Nuclear Antigen (PCNA) and FANCD2, proteins essential for translesion synthesis and the Fanconi Anemia pathway, respectively. By inhibiting USP1, UCT-01-097 promotes the accumulation of ubiquitinated substrates, leading to genomic instability and cell death, particularly in cancers with homologous recombination deficiency (HRD) or BRCA mutations. This mechanism offers a synthetic lethal approach that may overcome resistance to PARP inhibitors.

Other names

USP1 inhibitorUSP-1 inhibitorUSP 1 inhibitor