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The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Patent No.:

US 9713626 B1

PDFFull Text
Date of Patent:
Jul. 25, 2017

Filed:

Oct. 06, 2015

Cftr mrna compositions and related methods and uses

Applicants:

Shire Human Genetic Therapies, Inc., Lexington, MA (US);

Ethris Gmbh, Planegg, DE;

Inventors:

Michael Heartlein, Lexington, MA (US);

Braydon Charles Guild, Concord, MA (US);

Frank DeRosa, Lexington, MA (US);

Carsten Rudolph, Krailling, DE;

Christian Plank, Wessling, DE;

Lianne Smith, Lexington, MA (US);

Assignees:

RaNA Therapeutics, Inc., Lexington, MA (US);

Ethris GmbH, Martinsried, DE;

Attorneys:

Fangli Chen

Proskauer Rose LLP

Primary Examiner:

Shin Lin Chen

Int. Cl.
CPC ...
A61K 9/127 (2006.01); C07H 21/04 (2006.01); A61K 31/713 (2006.01); C07K 14/705 (2006.01); A61K 31/7105 (2006.01); A61K 47/48 (2006.01); A61K 31/7115 (2006.01); A61K 9/00 (2006.01); A61K 9/51 (2006.01); C12N 9/14 (2006.01); C12N 15/63 (2006.01); C07H 21/02 (2006.01); A61K 48/00 (2006.01); C07K 14/47 (2006.01); C12N 15/88 (2006.01);
U.S. Cl.
CPC ...
A61K 31/713 (2013.01); A61K 9/0073 (2013.01); A61K 9/0078 (2013.01); A61K 9/1271 (2013.01); A61K 9/5123 (2013.01); A61K 31/7105 (2013.01); A61K 31/7115 (2013.01); A61K 47/48907 (2013.01); A61K 48/005 (2013.01); C07K 14/705 (2013.01); C12N 9/14 (2013.01); A61K 9/127 (2013.01); A61K 48/00 (2013.01); C07H 21/02 (2013.01); C07K 14/4712 (2013.01); C12N 15/63 (2013.01); C12N 15/88 (2013.01); C12Y 306/03049 (2013.01);
Abstract

Pharmaceutical compositions 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 are provided. The present invention is particularly useful for treating cystic fibrosis.

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