A01

A01 is a small molecule identified as a Smad ubiquitination regulatory factor 1 (Smurf1)-specific inhibitor. It has been shown to block the degradation of mitochondrial antiviral signaling protein (MAVS), thereby enhancing MAVS-mediated antiviral signaling and suppressing viral replication, as demonstrated in bovine ephemeral fever virus infection. Additionally, A01 has been found to improve pulmonary hemodynamics, reduce vascular remodeling and fibrosis, and restore BMPR2 signaling in models of pulmonary arterial hypertension. Separately, a compound also referred to as A01 was identified as a hit compound in enzyme-level screening for histone lysine demethylase 6B (KDM6B) inhibitors, serving as a scaffold for developing derivatives for various diseases including cancer, inflammatory disorders, and psychiatric conditions. Another investigational anticancer medication is also referred to as A01 in clinical trials for advanced solid tumors and gastrointestinal cancer.