Modified polynucleotides for the production of proteins associated with human disease
AssigneeModernaTX, Inc.
InventorsAntonin DE FOUGEROLLES, Justin GUILD
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Abstract
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
Claims21
1-19. (canceled)
A pharmaceutical composition comprising a plurality of lipid nanoparticles, wherein the plurality of lipid nanoparticles has a mean particle size of from 80 nm to 160 nm, wherein the lipid nanoparticles encapsulate an mRNA molecule comprising an open reading frame encoding a glutaryl-CoA dehydrogenase (GCDH) polypeptide.
A method of expressing a GCDH polypeptide in a mammalian cell, the method comprising administering to the cell a pharmaceutical composition comprising a plurality of lipid nanoparticles, wherein the plurality of lipid nanoparticles has a mean particle size of from 80 nm to 160 nm, wherein the lipid nanoparticles encapsulate an mRNA molecule comprising an open reading frame encoding a GCDH polypeptide.
Dependencies are inferred from the claim text.