Treatment of cystic fibrosis by delivery of codon-optimized mRNA encoding CFTR
AssigneeTranslate Bio, Inc.
InventorsMichael Heartlein, Frank DeRosa, Alan Kimura, Jonathan Abysalh, Anusha Dias, Shrirang Karve, Zarna Patel
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Abstract
The present invention provides, among other things, methods of treating cystic fibrosis, comprising a step of administering to a subject in need of treatment a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA encoding the CFTR protein comprises a polynucleotide sequence at least 80% identical to SEQ ID NO: 1, wherein the mRNA is at a concentration of at least 0.4 mg/mL, and wherein the step of administering comprises inhalation.
Claims20
A pharmaceutical composition comprising a lipid nanoparticle comprising one or more cholesterol-based lipid(s) at a molar ratio of greater than 30% of total lipid content and a PEG-modified lipid, wherein the lipid nanoparticle encapsulates an mRNA comprising an open reading frame encoding a human Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA is at a concentration of at least 0.6 mg/ml, wherein the composition further comprises one or more diluents selected from the group consisting of DMSO, ethylene glycol, glycerol, 2-Methyl-2,4-pentanediol (MPD), propylene glycol, sucrose, and trehalose, and wherein said composition is formulated for inhalation.
A pharmaceutical composition comprising a lipid nanoparticle comprising one or more cholesterol-based lipid(s) at a molar ratio of greater than 30% of total lipid content, a PEG-modified lipid, one or more cationic lipid(s), and one or more non-cationic lipid(s), wherein the lipid nanoparticle encapsulates an mRNA comprising an open reading frame encoding a human Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the one or more cholesterol-based lipid(s) is cholesterol, wherein the PEG-modified lipid is DMG-PEG 2000, wherein the one or more non-cationic lipid(s) is DSPC, wherein the mRNA is at a concentration of at least 0.5 mg/mL, wherein the composition further comprises a diluent, wherein the diluent is sucrose, and wherein said composition is formulated for inhalation.
A pharmaceutical composition comprising a lipid nanoparticle comprising one or more cholesterol-based lipid(s) at a molar ratio of greater than 30% of total lipid content, a PEG-modified lipid, one or more cationic lipid(s), and one or more non-cationic lipid(s), wherein the lipid nanoparticle encapsulates an mRNA comprising an open reading frame encoding a human Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the one or more cholesterol-based lipid(s) is cholesterol, wherein the PEG-modified lipid is DMG-PEG 2000, wherein the one or more non-cationic lipid(s) is DSPC, wherein the one or more cationic lipid(s) is DOTAP, wherein the mRNA is at a concentration of at least 3 mg/mL, wherein the composition further comprises a diluent, wherein the diluent is sucrose, and wherein said composition is formulated for inhalation.
Dependencies are inferred from the claim text.