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US 11,304,911 B2Granted
Compositions and methods for organ specific delivery of nucleic acids
AssigneeBoard of Regents, The University of Texas System
InventorsQiang Cheng, Tuo Wei, Daniel J. Siegwart
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Abstract
The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.
Claims30
A method for non-liver targeted delivery of a therapeutic agent, the method comprising:
- administering to a subject in need thereof said therapeutic agent assembled with a selective organ targeting (SORT) lipid composition, which SORT lipid composition comprises:
- (i) an ionizable cationic lipid, and
- (ii) a SORT lipid separate from said ionizable cationic lipid, which SORT lipid is selected from the group consisting of a cationic SORT lipid, a zwitterionic SORT lipid, and an anionic SORT lipid;
- wherein, upon said administering, a surface of said SORT lipid composition interacts with apolipoprotein E (Apo E) to a lesser degree than with an endogenous protein that is not Apo E in said subject, thereby providing a lesser amount or activity of said therapeutic agent in liver or a cell therein in said subject as compared to that achieved absent said SORT lipid.
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Dependencies are inferred from the claim text.