IP Library › Patent Application 15294339
Patent Application
App. No. 15/294,339

MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF NUCLEAR PROTEINS

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Patent No.
US None
App. No.
15/294,339
Abstract

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.

Claims (19)

1 . An mRNA encoding a polypeptide of interest selected from the group consisting of SEQ ID NOs 3858-7559, wherein said mRNA comprises a coding region selected from the group consisting of SEQ ID NOs: 7560-26153.

2 . The mRNA of claim 1 , wherein the mRNA comprises at least one untranslated region 5′ relative to the coding region and at least one untranslated region 3′ relative to the coding region.

3 . The mRNA of claim 2 , wherein the 5′ untranslated region is heterologous to the coding region of the mRNA.

4 . The mRNA of claim 2 , wherein the 3′ untranslated region is heterologous to the coding region of the mRNA.

5 . The mRNA of claim 2 , wherein the 5′ untranslated region and the 3′ untranslated region are heterologous to the coding region of the mRNA.

6 . The mRNA of claim 2 , wherein the mRNA comprises at least two stop codons.

7 . A pharmaceutical composition comprising the mRNA of claim 1 and a pharmaceutically acceptable excipient.

8 . The pharmaceutical composition of claim 7 , wherein the pharmaceutically acceptable excipient is selected from a solvent, aqueous solvent, non-aqueous solvent, dispersion media, diluent, dispersion, suspension aid, surface active agent, isotonic agent, thickening or emulsifying agent, preservative, lipid, lipidoids liposome, lipid nanoparticle, core-shell nanoparticles, polymer, lipoplex, peptide, protein, cell, hyaluronidase, and mixtures thereof.

9 . The pharmaceutical composition of claim 8 , where the pharmaceutical composition comprises a lipid and wherein said lipid is selected from DLin-DMA, DLin-K-DMA, DLin-KC2-DMA, 98N12-5, C12-200, DLin-MC3-DMA, DODMA, DSDMA, DLenDMA, reLNPs, PLGA and PEGylated lipids and mixtures thereof.

10 . A method of producing a polypeptide of interest in a mammalian cell, tissue or organism comprising contacting said cell, tissue or organism with the mRNA of claim 1 .

11 . The method of claim 10 , wherein the mRNA is formulated.

12 . The method of claim 11 , wherein the formulation comprises a lipid which is selected from the group consisting of DLin-DMA, DLin-K-DMA, DLin-KC2-DMA, 98N12-5, C12-200, DLin-MC3-DMA, DODMA, DSDMA, DLenDMA, reLNPs, PLGA, PEGylated lipids and mixtures or combinations thereof.

13 . The method of claim 10 , wherein the contacting is a route of administration selected from the group consisting of intramuscular, intradermal, intravenous and subcutaneous.

14 . The method of claim 13 , wherein the route of administration is intramuscular.

15 . The method of claim 10 , wherein the mRNA comprises at least one untranslated region 5′ relative to the coding region and at least one untranslated region 3′ relative to the coding region.

16 . The method of claim 15 , wherein the 5′ untranslated region is heterologous to the coding region of the mRNA.

17 . The method of claim 15 , wherein the 3′ untranslated region is heterologous to the coding region of the mRNA.

18 . The method of claim 15 , wherein the 5′ untranslated region and the 3′ untranslated region are heterologous to the coding region of the mRNA.

19 . The method of claim 15 , wherein the mRNA comprises at least two stop codons.