HB-48

HB-48 is an orally bioavailable, highly selective, and brain-penetrant small molecule inhibitor of Ubiquitin-Specific Protease 1 (USP1). Developed by Guchong Pharmaceutical (Beijing) Co., Ltd., it targets the DNA damage response (DDR) pathway by inhibiting the deubiquitination of key proteins like FANCD2 and PCNA, which are essential for the Fanconi anemia and translesion synthesis pathways. This inhibition leads to the accumulation of DNA damage and subsequent cell death in tumor cells. HB-48 is specifically engineered for high blood-brain barrier permeability, making it a candidate for the treatment of central nervous system malignancies, including recurrent glioblastoma.