Modified polynucleotides encoding SIAH E3 ubiquitin protein ligase 1

Patent metadata

Field	Value
Publication number	US-9255129-B2
Application number	US-201314107105-A
Country	US
Kind code	B2
Filing date	Dec 16, 2013
Priority date	Apr 2, 2012
Publication date	Feb 9, 2016
Grant date	Feb 9, 2016

Abstract

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

First claim

We claim:

1. An mRNA encoding amino acid 17 to 298 of SEQ ID NO: 8363, wherein said mRNA nucleic acid comprises a coding region, said coding region having at least 80% identity to SEQ ID NO: 33695.

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 nucleic acid 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. The mRNA of claim 1 , wherein the coding region is at least 99% identical to SEQ ID NO: 33695.

8. The mRNA of claim 1 , wherein the coding region consists of SEQ ID NO: 33695.

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

10. The pharmaceutical composition of claim 9 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.

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

12. The method 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.

13. The method of claim 12 , wherein the 5′ untranslated region is heterologous to the coding region of the mRNA.

14. The method of claim 12 , wherein the 3′ untranslated region is heterologous to the coding region of the mRNA.

15. The method of claim 12 , wherein the 5′ untranslated region and the 3′ untranslated region are heterologous to the coding region of the mRNA.

16. The method of claim 12 , wherein the mRNA comprises at least two stop codons.

Assignees

• Moderna Therapeutics Inc

Inventors

• Chakraborty Tirtha
• De Fougerolles Antonin

Classifications

• C12N9/6451

  Coagulation factor XIIa (3.4.21.38) · CPC title

• C12N9/93

  Ligases (6) · CPC title

• C07K14/4713

  Autoimmune diseases, e.g. Insulin-dependent diabetes mellitus, multiple sclerosis, rheumathoid arthritis, systemic lupus erythematosus; Autoantigens · CPC title

• A61K31/7115

  Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine · CPC title

• A61K48/005

  characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered · CPC title