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.
Claims31
1-85. (canceled)
A method for targeted delivery of a therapeutic agent to an organ or cell in a subject in need thereof, the method comprising:
- (a) providing a selective organ targeting (SORT) lipid composition characterized by an apparent ionization constant (pKa) by adding a SORT lipid to a reference lipid composition characterized by a reference pKa, which SORT lipid is selected from the group consisting of a permanently cationic SORT lipid, a zwitterionic SORT lipid, and an anionic SORT lipid;
- and wherein said first pKa is different from said second pKa;
- and
- (b) administering to said subject said therapeutic agent assembled with said SORT lipid composition, thereby yielding a greater amount or activity of said therapeutic agent in said organ or cell in said subject as compared to that achieved with said reference lipid composition.
A method for targeted delivery of a therapeutic agent to a non-liver organ or cell of a subject, the method comprising:
- (a) providing a selective organ targeting (SORT) lipid composition by including a cationic SORT lipid in a reference lipid composition, which reference lipid composition comprises one or more lipids;
- and
- (b) administering to said subject said therapeutic agent assembled with said SORT lipid composition, thereby yielding a greater amount or activity of said therapeutic agent in said non-liver organ or cell in said subject as compared to that achieved with said reference lipid composition absent said cationic SORT lipid.
The method of claim 109, wherein said permanently cationic SORT lipid comprises a quaternary ammonium ion.
Dependencies are inferred from the claim text.