Methods and compositions for targeting agents into and across the blood-brain barrier
AssigneeModerna Therapeutics, Inc.
InventorStephane Bancel
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Abstract
This invention relates to modified nucleic acid compositions encoding therapeutic polypeptides and methods of producing the therapeutic polypeptides in cells.
Claims27
A method for in vivo generation of a therapeutic polypeptide encoded by a modified ribonucleic acid (RNA) capable of migration across a microvascular blood-central nervous system (CNS) barrier in a mammalian subject in need thereof, comprising administering to the subject a therapeutic composition comprising a therapeutically effective amount of the modified RNA, the modified RNA comprising a modified nucleotide and a translatable region encoding the therapeutic polypeptide, such that an effective barrier-crossing amount of the modified RNA crosses the microvascular blood-CNS barrier.
A method for ex vivo generation of a therapeutic polypeptide encoded by a modified ribonucleic acid (RNA), comprising introducing into a cell obtainable from a mammalian subject a therapeutic composition comprising a therapeutically effective amount of the modified RNA, the modified RNA comprising a modified nucleotide and a translatable region encoding the therapeutic polypeptide, such that the therapeutic polypeptide is generated in the cell.
A composition comprising i) a synthetic modified ribonucleic acid (RNA) comprising a nucleic acid sequence that encodes a therapeutic polypeptide and ii) a delivery agent for efficient delivery of the modified RNA across the microvascular blood-central nervous system (CNS) barrier.
A delivery system comprising a) a composition comprising i) a synthetic modified ribonucleic acid (RNA) comprising a nucleic acid sequence that encodes a therapeutic polypeptide;
- b) a delivery means for efficient delivery of the modified RNA across the microvascular blood-central nervous system (CNS) barrier;
- and c) a delivery device.
Dependencies are inferred from the claim text.