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US 2021/0275565 A1Published application
Compositions comprising circular polyribonucleotides and uses thereof
AssigneeFlagship Pioneering Innovations VI, LLC
InventorsAlexandra Sophie DE BOER, Avak KAHVEJIAN, Nicholas McCartney PLUGIS, Erica Gabrielle WEINSTEIN, Sebastian TROUSIL, Morag Helen STEWART, Ki Young PAEK, Catherine CIFUENTES-ROJAS
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Abstract
This invention relates generally to pharmaceutical compositions and preparations of circular polyribonucleotides and uses thereof.
Claims30
A method of expressing a polypeptide in a subject, comprising:
- administering to the subject a covalently closed polyribonucleotide that:
- (a) comprises an internal ribosome entry site (IRES) element;
- (b) comprises an expression sequence that encodes the polypeptide and that lacks one or both of a 5′ cap and a poly-A sequence;
- and (c) comprises a termination element;
- and
- translating the polypeptide from the expression sequence of the covalently closed polyribonucleotide in vivo over a period of at least 7 days.
3 more elements
A method of delivering a therapeutic polypeptide to a human, comprising:
- administering to the human a covalently closed polyribonucleotide that:
- (a) comprises an internal ribosome entry site (IRES) element;
- (b) comprises an expression sequence that encodes the therapeutic polypeptide and that lacks one or both of a 5′ cap and a poly-A sequence;
- and (c) comprises a termination element;
- and
- translating the therapeutic polypeptide from the expression sequence of the covalently closed polyribonucleotide in vivo over a period of at least 7 days,
- wherein the therapeutic polypeptide is selected from the group consisting of an antigen, at least a functional portion of a viral envelope protein, a hormone, a cytokine, a ligand, a receptor, an antibody, an enzyme, a secreted protein, an epigenetic modifying agent, a nuclease, a component of a CRISPR system, a pore forming peptide, a cytotoxic peptide, and an anti-microbial peptide.
4 more elements
Dependencies are inferred from the claim text.