CFTR MRNA compositions and related methods and uses
AssigneeTranslate Bio, Inc.
InventorsMichael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
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Abstract
The present invention is particularly useful for treating cystic fibrosis. The disclosure concerns method and compositions for treating cystic fibrosis. The method comprises inducing CFTR expression in epithelial cells in a lung of a mammal by contacting the epithelial cells in the lung of the mammal with a composition, where the composition is a pharmaceutical composition comprising in vitro transcribed mRNA.
Claims13
A method of producing cystic fibrosis transmembrane conductance regulator (CFTR) protein in a lung of a mammal, the method comprising:
- administering to the lung of the mammal by aerosolization a composition comprising an mRNA-loaded nanoparticle, wherein the nanoparticle is a liposome, wherein the mRNA is an in vitro transcribed mRNA and has a coding sequence at least 80% identical to SEQ ID NO:
- 3, wherein the mRNA encodes a human CFTR protein comprising the amino acid sequence of SEQ ID NO:1, and wherein the mRNA is unmodified.
Dependencies are inferred from the claim text.