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US 12,594,295 B2Granted
CFTR mRNA compositions and related methods and uses
AssigneesTranslate Bio, Inc., ethris GmbH
InventorsMichael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
Life of this patent
Estimating the term…
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.
Claims19
A pharmaceutical composition comprising an mRNA-loaded nanoparticle,
- wherein the mRNA is an in vitro transcribed mRNA, wherein the coding sequence of the mRNA is at least 95% identical to SEQ ID NO:
- 9, and
- wherein the mRNA encodes a human cystic fibrosis transmembrane conductance regulator (CFTR) protein comprising the amino acid sequence of SEQ ID NO:
- 1.
1 more element
Dependencies are inferred from the claim text.