Ribonucleic acids with 4′-thio-modified nucleotides and related methods
AssigneeTranslate Bio, Inc.
InventorsFrank DeRosa, Michael Heartlein
Life of this patent
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Abstract
Disclosed are messenger RNA molecules and related compositions incorporating a 4′-thio modification in the furanose ring of at least one nucleotide residue, and methods of using these mRNAs to produce an encoded therapeutic protein in vivo and to treat or prevent diseases or disorders. In certain embodiments, the 4′-thio modified mRNA provides for enhanced stability and/or reduced immunogenicity in in vivo therapies.
Claims21
A method of producing a protein in vivo, comprising administering to a subject a composition comprising:
- at least one full-length mRNA molecule that
- has a coding region and, optionally, one or more non-coding regions,
- encodes a full length protein, and
- is at least 500 nucleotide residues in length, and
- wherein 1%-20% of the total mRNA nucleotide residues of the full-length mRNA
- incorporate a 4′-thio-substituted furanose ring;
- and
- a carrier that is a lipid nanoparticle or a polymer;
- and
- wherein upon administration, the full-length mRNA produces in vivo a protein encoded by the coding region.
A method of treating a patient in need of a therapeutic protein, comprising administering to said patient a composition comprising:
- at least one full-length mRNA molecule that
- has a coding region that encodes said therapeutic protein and, optionally, one or more non-coding regions,
- encodes a full length protein, and
- is at least 500 nucleotide residues in length, and
- wherein 1%-20% of the total mRNA nucleotide residues of the full-length mRNA incorporate a 4′-thio-substituted furanose ring;
- and
- a carrier that is a lipid nanoparticle or a polymer;
- and
- wherein upon administration, the full-length mRNA produces in vivo said therapeutic protein encoded by the coding region.
Dependencies are inferred from the claim text.