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DXO-1801 is a fully chemically stabilized, liver-targeted, and highly specific GalNAC-conjugated small interfering RNA (siRNA). It functions by silencing the expression of a key gene within the hepcidin-regulatory pathway in hepatocytes. This mechanism is designed to attenuate inflammation-driven iron restriction, thereby enhancing iron availability and improving erythropoiesis. The drug is being developed as a potential therapeutic for anemia of inflammation, a complication associated with various chronic conditions such as inflammatory bowel and autoimmune diseases, chronic kidney disease, advanced solid tumors, and hematological malignancies.
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