Modified polynucleotides for the production of proteins associated with blood and lymphatic disorders
View Patent ↗The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
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 .