Lipid nanoparticle compositions and methods for MRNA delivery
AssigneeTranslate Bio, Inc.
InventorsFrank DeRosa, Braydon Charles Guild, Michael Heartlein
Life of this patent
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Abstract
Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.
Claims19
A composition comprising an mRNA encoding a cytokine, encapsulated within a lipid nanoparticle,
- wherein the lipid nanoparticle has a size less than 150 nm and comprises
- (i) a PEG-modified lipid at a molar ratio of greater than 1% of the total lipids in the lipid nanoparticle,
- (ii) a cationic lipid at a molar ratio of greater than 10% of the total lipids in the lipid nanoparticle,
- (iii) a non-cationic lipid at a molar ratio of greater than 5% of total lipids in the lipid nanoparticle, and
- (iv) cholesterol at a molar ratio of greater than 10% of the total lipids in the lipid nanoparticle.
A composition comprising an mRNA encoding a cytokine, encapsulated within a lipid nanoparticle, wherein the lipid nanoparticle has a size less than 150 nm and comprises
- (i) a PEG-modified lipid at a molar ratio of greater than 1% of the total lipids in the lipid nanoparticle,
- (ii) a cationic lipid at a molar ratio of greater than 10% of the total lipids in the lipid nanoparticle,
- (iii) a non-cationic lipid at a molar ratio of greater than 5% of total lipids in the lipid nanoparticle, and
- (iv) cholesterol at a molar ratio greater than 10% of the total lipids in the lipid nanoparticle, and wherein the mRNA comprises
- (i) a 5′-UTR,
- (ii) a 3′-UTR,
- (iii) a 5′ cap structure, and
- (iv) a poly A tail.
A composition comprising an mRNA encoding a cytokine, encapsulated within a lipid nanoparticle, wherein the lipid nanoparticle has a size less than 150 nm and comprises
- (i) a PEG-modified lipid at a molar ratio of greater than 1% of the total lipids in the lipid nanoparticle,
- (ii) a cationic lipid at a molar ratio of greater than 10% of the total lipids in the lipid nanoparticle,
- (iii) a non-cationic lipid at a molar ratio of greater than 5% of total lipids in the lipid nanoparticle, and
- (iv) cholesterol at a molar ratio greater than 10% of the total lipids in the lipid nanoparticle, and
- wherein the mRNA is modified to comprise one or more pseudouridine and the mRNA comprises
- (i) a 5′-UTR,
- (ii) a 3 ‘-UTR,
- (iii) a 5’ cap structure, and
- (iv) a poly A tail.
Dependencies are inferred from the claim text.