Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
AssigneeTranslate Bio, Inc.
InventorsShrirang Karve, Frank DeRosa, Michael Heartlein, Zarna Patel
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Abstract
The present invention provides, among other things, an improved method of treating cystic fibrosis (CF) in a human subject. The method comprises administering a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein at a concentration of 0.5 mg/mL or greater to a human subject via nebulization. The composition is aerosolized using a nebulizer and a nominal dose of the mRNA is administered to the human subject via the nebulizer over a period of time, typically at least 30 minutes, at a suitable nebulization rate, e.g., at least 0.2 mL/minute.
Claims16
A method of treating cystic fibrosis (CF) in a human subject comprising administering a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein at a concentration of 0.6 mg/ml to the human subject, wherein the step of administering comprises the following:
- (i) the composition is aerosolized using a nebulizer wherein median droplet size in the aerosolized composition is between 4 and 6 μm, and (ii) a nominal dose of the mRNA is administered to the human subject via the nebulizer over a period of time at least 30 minutes at a nebulization rate of 0.2 mL/minute to 0.5 mL/minute, wherein the composition comprises trehalose, and wherein the mRNA encoding the CFTR protein comprises SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.