Ionizable cationic lipid for RNA delivery
AssigneeArcturus Therapeutics, Inc.
InventorsJoseph E. Payne, Padmanabh Chivukula, Priya Karmali, Steven P. Tanis
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Abstract
Disclosed herein is a compound of Formula I, wherein R1 is a branched chain alkyl consisting of 10 to 31 carbons; R2 is a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons, or a branched chain alkyl consisting of 10 to 31 carbons; L1 and L2 are the same or different, each a linear alkane of 1 to 20 carbons or a linear alkene of 2 to 20 carbons; X1 is S or O; R3 is a linear or branched alkylene consisting of 1 to 6 carbons; and R4 and R5 are the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt or solvate thereof.
Claims20
A pharmaceutical composition comprising a lipid nanoparticle encapsulating a nucleic acid, wherein the lipid nanoparticle comprises a cationic lipid selected from the group consisting of:
A pharmaceutical composition comprising a lipid nanoparticle encapsulating a nucleic acid, wherein the lipid nanoparticle comprises
- i. a cationic lipid in an amount of 30 mol % to 70 mol % of the total lipids present in the lipid nanoparticle, wherein the cationic lipid is selected from the group consisting of:
- ii. cholesterol in an amount of 25 mol % to 45 mol % of the total lipid present in the lipid nanoparticle;
- iii. a helper lipid in an amount of 2 mol % to 30 mol % of the total lipid present in the lipid nanoparticle, wherein the helper lipid is selected from lecithin, phosphatidylethanolamine, lysolecithin, lysophosphatidylethanolamine, phosphatidylserine, phosphatidylinositol, sphingomyelin, egg sphingomyelin (ESM), cephalin, cardiolipin, phosphatidic acid, cerebrosides, dicetylphosphate, distearoylphosphatidylcholine (DSPC), dioleoylphosphatidylcholine (DOPC), dipalmitoylphosphatidylcholine (DPPC), dioleoylphosphatidylglycerol (DOPG), dipalmitoylphosphatidylglycerol (DPPG), dioleoylphosphatidylethanolamine (DOPE), palmitoyloleoyl-phosphatidylcholine (POPC), palmitoyloleoyl-phosphatidylethanolamine (POPE), palmitoyloleyol-phosphatidylglycerol (POPG), dioleoylphosphatidylethanolamine 4-(N-maleimidomethyl)-cyclohexane-1-carboxylate (DOPE-mal), dipalmitoyl-phosphatidylethanolamine (DPPE), dimyristoyl-phosphatidylethanolamine (DMPE), distearoyl-phosphatidylethanolamine (DSPE), monomethyl-phosphatidylethanolamine, dimethyl-phosphatidylethanolamine, dielaidoyl-phosphatidylethanolamine (DEPE), stearoyloleoyl-phosphatidylethanolamine (SOPE), lysophosphatidylcholine, and dilinoleoylphosphatidylcholine;
- and
- iv. a polyethylene glycol lipid conjugate in an amount of 0.1 mol % to 2 mol % of the total lipids present in the lipid nanoparticle.
Dependencies are inferred from the claim text.