Modified polynucleotides for the production of proteins associated with blood and lymphatic disorders

Patent metadata

Field	Value
Publication number	US-9233141-B2
Application number	US-201314104591-A
Country	US
Kind code	B2
Filing date	Dec 12, 2013
Priority date	Apr 2, 2012
Publication date	Jan 12, 2016
Grant date	Jan 12, 2016

Abstract

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

First claim

We claim:

1. A composition comprising at least one mRNA, wherein said at least one mRNA comprises
a first region of linked nucleosides, said first region encoding SEQ ID NO 909, and wherein said first region comprises a coding region having at least 80% identity to SEQ ID NO: 9244.

2. The composition of claim 1 , wherein the at least one mRNA comprises a poly-A tail.

3. The composition of claim 2 , wherein the at least one mRNA comprises a 5′ cap structure.

4. The composition of claim 2 , wherein the at least one mRNA is purified.

5. The composition of claim 1 , comprising a pharmaceutically acceptable excipient.

6. The composition of claim 5 , wherein the pharmaceutically acceptable excipient is selected from the group consisting of a solvent, aqueous solvent, nonaqueous 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.

7. The composition of claim 1 , wherein the coding region of the mRNA is selected from the group consisting of SEQ ID NOs: 9244, 9243, 9264, 9280, 9305, 9308, 9316, 9318, and 9327.

8. A method of producing Factor X, in a cell, tissue, or organism comprising contacting said cell, tissue, or organism with the at least one mRNA of claim 1 .

Assignees

• Moderna Therapeutics Inc

Inventors

• Chakraborty Tirtha
• De Fougerolles Antonin

Classifications

• A61P5/00

  Drugs for disorders of the endocrine system · CPC title

• A61P43/00

  Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title

• A61P9/10

  for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis · CPC title

• A61P37/02

  Immunomodulators · CPC title

• A61P7/00

  Drugs for disorders of the blood or the extracellular fluid · CPC title