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US 2016/0106772 A1Published application
Cftr mrna compositions and related methods and uses
AssigneesShire Human Genetic Therapies, Inc., ethris GmbH
InventorsMichael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
Life of this patent
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Abstract
Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.
Claims25
1-47. (canceled)
A pharmaceutical composition comprising an mRNA-loaded nanoparticle,
- wherein the mRNA is an in vitro transcribed mRNA and has a coding sequence at least 80% identical to SEQ ID NO:
- 3, and
- wherein the mRNA encodes a human cystic fibrosis transmembrane conductance regulator (CFTR) protein comprising the amino acid sequence of SEQ ID NO:1.
A method of treating cystic fibrosis, the method comprising:
- administering to the lung of a subject in need of treatment a composition comprising an mRNA-loaded nanoparticle, wherein the mRNA is an in vitro transcribed mRNA and has a coding sequence at least 80% identical to SEQ ID NO:
- 3, and
- wherein the mRNA encodes a human cystic fibrosis transmembrane conductance regulator (CFTR) protein comprising the amino acid sequence of SEQ ID NO:1.
Dependencies are inferred from the claim text.