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1. (US20190194669) UTRs INCREASING THE TRANSLATION EFFICIENCY OF RNA MOLECULES

Office : United States of America
Application Number: 15739147 Application Date: 30.06.2016
Publication Number: 20190194669 Publication Date: 27.06.2019
Publication Kind : A1
Prior PCT appl.: Application Number:PCTEP1665297 ; Publication Number: Click to see the data
IPC:
C12N 15/67
C07K 14/47
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
67
General methods for enhancing the expression
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
14
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435
from animals; from humans
46
from vertebrates
47
from mammals
CPC:
A61K 38/00
C12N 15/67
C07K 14/47
Applicants: Ethris GmbH
Inventors: Carsten Rudolph
Manish Kumar Aneja
Mehrije Ferizi
Johannes Geiger
Priority Data: 15174683.1 30.06.2015 EP
Title: (EN) UTRs INCREASING THE TRANSLATION EFFICIENCY OF RNA MOLECULES
Abstract: front page image
(EN)

Described is an RNA molecule comprising (a) a coding region coding for a polypeptide; and (b) upstream of said coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:1 or a sequence which shows 1 to 4 substitutions in comparison to SEQ ID NO:1 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:1; and/or (c) downstream of said coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:2 or a sequence which shows 1 to 7 substitutions in comparison to SEQ ID NO:2 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:2; wherein said polypeptide encoded by said coding region is not a cytochrome b-245 alpha polypeptide (CYBA). Moreover, described is a nucleic acid molecule encoding the RNA molecule according to the present invention. Further, described is a vector comprising the nucleic acid molecule according to the present invention and to a host cell comprising the vector according to the present invention. Further, described is a pharmaceutical composition comprising the RNA molecule according to the present invention and optionally a pharmaceutically acceptable carrier. Moreover, described is a kit comprising the RNA molecule according to the present invention. Finally, described is the use of one or more UTR(s) as defined in (b) and/or one or more UTR(s) as defined in (c) for increasing the efficiency of translating a coding region of an RNA molecule into a polypeptide or a protein encoded by said coding region.


Also published as:
EP3112469AU2016287436CA2990881IN201717045893KR1020180035789EP3317415
DK3112469ES2667644CN107849574JP2018519830BR112017028205WO/2017/001554