09713626 cites 656 patents.

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.

Title
CFTR mRNA compositions and related methods and uses
Application Number
14/876071
Publication Number
9713626 (B2)
Application Date
October 6, 2015
Publication Date
July 25, 2017
Inventor
Lianne Smith
Lexington
MA, US
Christian Plank
Wessling
DE
Carsten Rudolph
Krailling
DE
Frank DeRosa
Lexington
MA, US
Braydon Charles Guild
Concord
MA, US
Michael Heartlein
Lexington
MA, US
Agent
Proskauer Rose
Fangli Chen
Assignee
Ethris
DE
RaNA Therapeutics
MA, US
IPC
C12N 15/88
C07K 14/47
A61K 48/00
C07H 21/02
C12N 15/63
C12N 9/14
A61K 9/51
A61K 9/00
A61K 31/7115
A61K 47/48
A61K 31/7105
C07K 14/705
A61K 31/713
C07H 21/04
A61K 9/127
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