DLIN-KC2-DMA lipid nanoparticle delivery of modified polynucleotides
View Patent ↗The present disclosure provides, inter alia, formulation compositions comprising modified nucleic acid molecules which may encode a protein, a protein precursor, or a partially or fully processed form of the protein or a protein precursor. The formulation composition may further include a modified nucleic acid molecule and a delivery agent. The present invention further provides nucleic acids useful for encoding polypeptides capable of modulating a cell's function and/or activity.
1. A method for producing a protein of interest in a mammal comprising contacting said mammal with a modified mRNA comprising SEQ ID NO: 10 formulated in a lipid nanoparticle.
2. The method of claim 1 wherein the lipid nanoparticle has a final total molar ratio of 50 mol % cationic lipid, 10 mol % DSPC, 38.5 mol % Cholesterol and 1.5 mol % of PEG lipid.
3. The method of claim 2 , wherein the lipid nanoparticle has a 20:1 weight ratio of total lipid to the modified RNA.
4. The method of claim 2 , wherein the cationic lipid is DLin-KC2-DMA.
5. The method of claim 4 , wherein the PEG lipid is PEG-c-DOMG.
6. The method of claim 5 , and wherein said protein of interest is detectable 8 hours after contacting said mammal.
7. The method of claim 1 , wherein the modified mRNA comprises at least one nucleoside modification.