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CMX001 is an oral antiviral drug candidate for the treatment of smallpox infections and complications resulting from smallpox vaccine. It is a lipid mimic of cidofovir formed by linking a lipid 3-hexadecyloxy-1-propanol, to the phosphonate group of cidofovir. CMX001 is designed to cross cellular membranes by passive diffusion (vida supra, Phospholipid Mimics Designed to Facilitate Uptake in the Small Intestine). Uptake by this mechanism should be more efficient and lead to a more rapid accumulation of cidofovir in the cytoplasm of the target cell. Once it has reached the cytoplasm, CDV is phosphorylated by host cell nucleoside kinases to form the active antiviral agent cidofovir diphosphate.
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