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.
Claims39
A method for targeted delivery of a therapeutic agent to a non-liver organ or a non-liver cell therein in a subject in need thereof, the method comprising:
- administering to said subject said therapeutic agent assembled with a selective organ targeting (SORT) lipid composition that comprises:
- an ionizable cationic lipid;
- and
- 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 said SORT lipid composition is characterized by an apparent ionization constant (pKa) outside a range of about 6 to about 7 as determined by a 2-(p-toluidino)-6-naphthalenesulfonic acid titration assay,
- thereby providing a greater amount or activity of said therapeutic agent in said non-liver organ or said non-liver cell therein in said subject as compared to that achieved absent said SORT lipid.
A method for targeted delivery of a therapeutic agent to a non-liver organ or a non-liver cell of a subject, the method comprising:
- administering to said subject said therapeutic agent assembled with a selective organ targeting (SORT) lipid composition that comprises:
- an ionizable cationic lipid, and
- a cationic SORT lipid,
- thereby providing a greater amount or activity of said therapeutic agent in said non-liver organ or said non-liver cell in said subject as compared to that achieved absent said cationic SORT lipid.
Dependencies are inferred from the claim text.