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    • 13. 发明公开
    • AMINO ACID LIPIDS AND USES THEREOF
    • EP3434259A1
    • 2019-01-30
    • EP18178105.5
    • 2008-05-02
    • Marina Biotech, Inc.
    • QUAY, StevenHOUSTON, MichaelHARVIE, PierrotADAMI, RogerFAM, RenataPRIEVE, MaryFOSNAUGH, KathySETH, Shaguna
    • A61K9/127
    • The present invention provides a compound comprising the structure shown in Formula I for use in delivering a therapeutic nucleic acid, one or more drug agents, or a biologically active agent to a cell,

               R 3 -(C=O)-Xaa-Z-R 4      Formula I

      wherein
      Xaa is histidine, 1-methylhistidine, pyridylalanine, lysine, arginine, homoarginine, nor-norarginine, ornithine, homolysine, asparagine, N ethylasparagine, glutamine, 4-aminophenylalanine, and nor-arginine, or an N-methylated version thereof;
      R 3 is independently a lipophilic tail derived from a naturally-occurring or synthetic lipid, phospholipid, glycolipid, triacylglycerol, glycerophospholipid, sphingolipid, ceramide, sphingomyelin, cerebroside, or ganglioside, wherein the tail may contain a steroid; or a substituted or unsubstituted C(3-22)alkyl, C(6-12)cycloalkyl, C(6-12)cycloalkyl-C(3-22)alkyl, C(3-22)alkenyl, C(3-22)alkynyl, C(3-22)alkoxy, or C(6-12)alkoxy-C(3-22)alkyl;
      R 4 is independently a lipophilic tail derived from a naturally-occurring or synthetic lipid, phospholipid, glycolipid, triacylglycerol, glycerophospholipid, sphingolipid, ceramide, sphingomyelin, cerebroside, or ganglioside, wherein the tail may contain a steroid; or a substituted or unsubstituted C(3-22)alkyl, C(6-12)cycloalkyl, C(6-12)cycloalkyl-C(3-22)alkyl, C(3-22)alkenyl, C(3-22)alkynyl, C(3-22)alkoxy, or C(6-12)alkoxy-C(3-22)alkyl;
      wherein both R 3 and R 4 are lipophilic tails; and
      Z is NH, O, S, -CH 2 S-, -CH 2 S(O)-, or an organic linker consisting of 1-40 atoms selected from hydrogen, carbon, oxygen, nitrogen, and sulfur atoms;
      and salts thereof.