Lipid nanoparticle formulations and compositions
AssigneeCapstan Therapeutics, Inc.
InventorsPriya Prakash KARMALI, Steven TANIS, Yanjie BAO
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Abstract
Disclosed are compositions of lipid nanoparticles (LNP) comprising an ionizable cationic lipid, a phospholipid, a sterol, and a PEG-lipid (non-functionalized and optionally functionalized). The functionalized PEG-lipid can be conjugated with a binding moiety to create a targeted LNP (tLNP). The disclosed tLNP preferentially deliver a nucleic acid molecule or other negatively charged payload to cells expressing a cell surface antigen recognized by the binding moiety of the tLNP, and are better tolerated, as compared to LNPs and tLNPs comprising ionizable cationic lipids found in marketed pharmaceuticals comprising LNPs.
Claims54
A lipid nanoparticle (LNP) comprising a lipid composition comprising:
- about 35 to about 65 mol % an ionizable cationic lipid of structure
- wherein R is
- about 0.5 to about 3 mol % PEG-lipid comprising functionalized PEG-lipid and non-functionalized PEG-lipid,
- about 7 to about 13 mol % a phospholipid, and
- about 27 to about 50 mol % a sterol.
Dependencies are inferred from the claim text.