CPC A61K 47/18 (2013.01) [A61K 31/7088 (2013.01); A61K 31/7115 (2013.01); A61K 38/193 (2013.01); A61K 38/4846 (2013.01); A61K 47/22 (2013.01); A61K 47/28 (2013.01); C07K 14/535 (2013.01); C12N 9/644 (2013.01); C12N 15/67 (2013.01); C12N 15/87 (2013.01); A61K 48/00 (2013.01); C12N 2310/335 (2013.01)] | 18 Claims |
1. A method of producing a polypeptide of interest in a cell in a subject in need thereof, comprising administering to the subject a pharmaceutical composition comprising a modified messenger RNA (mmRNA) such that the mmRNA is introduced into the cell, wherein the mmRNA comprises a translatable region encoding the polypeptide of interest and comprises the modified nucleoside 1-methyl-pseudouridine, wherein the pharmaceutical composition comprises an effective amount of the mmRNA providing for increased polypeptide production and substantially reduced innate immune response in the cell, as compared to a composition comprising a corresponding unmodified mRNA, and wherein 100% of uridines in the mmRNA are replaced with 1-methyl-pseudouridine.
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