Compositions and methods for delivery of agents
AssigneeModernaTX, Inc.
InventorsGilles Besin, Stephen Hoge, Joseph Senn, Kerry Benenato, Staci Sabnis
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Abstract
This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.
Claims31
A pharmaceutical composition formulated for repeat administration to a subject in need of a therapeutic protein, the composition comprising lipid nanoparticles (LNPs) comprising an mRNA coding for the protein, wherein the LNPs comprise an ionizable lipid, a helper lipid, a structural lipid, and a PEG-lipid, wherein the PEG-lipid is a compound of Formula (V-OH):
- or a pharmaceutically acceptable salt thereof, wherein:
- r is an integer between 1 and 100, inclusive;
- and
- R5 is optionally substituted C10-40 alkyl;
- wherein the mRNA comprises at least one miR binding site;
- and
- wherein said LNPs result in a reduced anti-PEG IgM response in the subject as compared to LNPs lacking a PEG-lipid of Formula (V-OH).
Dependencies are inferred from the claim text.