MT-003

MT-003 is a small molecule drug candidate developed by Mitotherapeutix. It acts as an inhibitor of DNAJC15 and is being investigated for the treatment of sickle cell disease. The mechanism involves increasing the affinity of hemoglobin (Hb) for oxygen, thereby interfering with the polymerization of deoxygenated sickle hemoglobin (deoxyHbS), which is responsible for red blood cell sickling in this disease. By stabilizing the R-state and destabilizing the T-state of Hb, MT-003 reduces hypoxia-induced sickling in vitro and increases oxygen affinity in vivo. As of now, its highest global R&D status is preclinical[1].