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Modified polynucleotides

  • US 10,501,512 B2
  • Filed: 03/09/2013
  • Issued: 12/10/2019
  • Est. Priority Date: 04/02/2012
  • Status: Active Grant
First Claim
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1. A composition comprising:

  • a plurality of lipid nanoparticles comprising a cationic lipid, a PEGylated lipid, cholesterol, and a phospholipid, wherein the mean particle size of the plurality of lipid nanoparticles is 80 nm to 160 nm and the lipid nanoparticles encapsulate a polynucleotide,wherein the polynucleotide comprises;

    (a) a first region of linked nucleosides, said first region encoding factor IX, wherein said first region of linked nucleosides consists of nucleotides selected from 1-methyl-pseudouridine, cytidine, adenosine, and guanosine;

    (b) a first flanking region located at the 5′

    -terminus of said first region comprising;

    (i) a 5′

    -untranslated region (5′

    -UTR); and

    (ii) 5′

    terminal cap;

    (c) a second flanking region located at the 3′

    -terminus of said first region comprising;

    (i′

    ) a 3′

    -untranslated region (3′

    -UTR); and

    (ii′

    ) a 3′

    -tailing sequence of linked nucleosides.

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