Novel Lipid Nanoparticles and Novel Components for Delivery of Nucleic Acids
The instant invention provides for novel lipid nanoparticles and novel lipid nanoparticle components (specifically cationic lipids) that are useful for the delivery of nucleic acids, specifically siRNA, for therapeutic purposes.
1 . A cationic lipid which is selected from:
2-({8-[(3β)-cholest-5-en-3-yloxy]octyl}oxy)-N,N-dimethyl-3-[(9Z,12Z)-octadeca-9,12-dien-1-yloxy]propan-1-amine (Octyl-CLinDMA);
(2R)-2-({8- [(3β)-cholest-5-en-3-yloxy]octyl}oxy)-N,N-dimethyl-3-[(9Z,12Z)-octadeca-9,12-dien-1-yloxy]propan-1-amine (Octyl-CLinDMA (2R)); and
(2S)-2-({8-[(3β)-cholest-5-en-3-yloxy]octyl}oxy)-N,N-dimethyl-3- [(9Z,12Z)-octadeca-9,12-dien-1-yloxy]propan-1-amine (Octyl-CLinDMA (2S)).
2 . A lipid nanoparticle composition comprising one or more biologically active molecules, cationic lipid selected from Octyl-CLinDMA, Octyl-CLinDMA (2R) and Octyl-CLinDMA (2S) or combinations thereof, neutral lipid which is (PEG-DMG), and cholesterol.
3 . A lipid nanoparticle composition comprising one or more siRNA molecules, cationic lipid selected from Octyl-CLinDMA, Octyl-CLinDMA (2R) and Octyl-CLinDMA (2S) or combinations thereof, neutral lipid which is (PEG-DMG), and cholesterol.
4 . A lipid nanoparticle composition of claim 3 comprising siRNA molecules, Octyl-CLinDMA, PEG-DMG, and cholesterol.
5 . A lipid nanoparticle composition of claim 3 comprising siRNA molecules, Octyl-CLinDMA (2R), PEG-DMG, and cholesterol.
6 . A lipid nanoparticle composition of claim 3 comprising siRNA molecules, Octyl-CLinDMA (2S), PEG-DMG, and cholesterol.
7 . A lipid nanoparticle composition of claim 3 , wherein said Octyl-CLinDMA, PEG-DMG, and cholesterol have a molar ratio of 60/38/2.
8 . A lipid nanoparticle composition of claim 3 , wherein said Octyl-CLinDMA (2R), PEG-DMG, and cholesterol have a molar ratio of 60/38/2.
9 . A lipid nanoparticle composition of claim 3 , wherein said Octyl-CLinDMA (2S), PEG-DMG, and cholesterol have a molar ratio of 60/38/2.