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NNC0614-0001 is a monoclonal antibody developed by Novo Nordisk for the treatment of hereditary haemochromatosis type 1. It targets transferrin receptor 2 (TFR2), a protein primarily expressed in the liver that acts as a sensor of systemic iron levels and a positive regulator of hepcidin production. In patients with hereditary haemochromatosis type 1, mutations in the HFE gene lead to insufficient hepcidin levels, resulting in uncontrolled iron absorption and systemic iron overload. NNC0614-0001 is designed to bind to TFR2 and stimulate the signaling pathway that increases hepcidin expression, thereby reducing serum iron levels and preventing tissue damage associated with iron accumulation.
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