Head of Lead Discovery Accent Therapeutics, Massachusetts, United States
RNA modifying proteins (RMPs) represent a large class of novel targets for oncology and other disease indications that can be targeted with small molecule inhibitors. The enzyme ADAR1 (Adenosine Deaminases Acting on RNA) catalyzes the majority of A-to-I editing, where it has been demonstrated to effect coding sequence, miRNA function and the detection of repetitive elements by innate immune pathways. Inhibition of ADAR1 has potential as both a monotherapy for tumors with high Type I interferon signaling and in combination with checkpoint inhibitors in relapsed/refractory tumors; target validation and current efforts in drug discovery will be discussed.
