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DISC-A is an investigational **small molecule** inhibitor of matriptase-2 designed by Disc Medicine for disorders driven by **inappropriately low hepcidin** and abnormal iron homeostasis. Matriptase-2 is a liver-expressed serine protease that suppresses hepcidin signaling through cleavage of hemojuvelin; DISC-A has been reported to inhibit matriptase-2 proteolytic activity, prevent enzyme autocleavage and hemojuvelin cleavage, and increase hepcidin pathway activity in vitro and in vivo. Preclinical reports describe the compound as a potent low-nanomolar inhibitor with favorable pharmacokinetic properties in rat and monkey studies, positioning it as an early research-stage therapy for hematologic diseases associated with low hepcidin.
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