US 11,981,910 B2Granted

Minimal UTR sequences

Assigneeethris GmbH

InventorsChristian Plank, Carsten Rudolph, Manish Kumar Aneja, Ludwig Weiss

Life of this patent

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Abstract

Described are DNA molecules which can be transcribed into an mRNA harbouring novel UTR sequences combining the advantages of being extremely short and at the same time allowing for high translation efficiencies of RNA molecules containing them. Further, described are vectors comprising such a DNA molecule and to host cells comprising such a vector. Moreover, described are corresponding RNA molecules containing such UTRs. Further, described is a pharmaceutical composition comprising the described RNA molecule and optionally a pharmaceutically acceptable carrier as well as to the use of the described UTRs for translating a coding region of an RNA molecule into a polypeptide or a protein encoded by said coding region.

Claims16

Dependent claims

A method of treating a disease or disorder in a subject comprising administering to the subject a therapeutically effective amount of a composition comprising an RNA molecule comprising

(a) a coding region, including a start codon at its 5′ end, coding for a polypeptide;
and
(b) directly upstream of said coding region a UTR selected from the group consisting of:
(b1) a UTR of the sequence R2-CGCCACC (SEQ ID NO:1);
and
(b2) a UTR of the sequence R2-CNGCCACC (SEQ ID NO:2), wherein the nucleotide N at position 2 of SEQ ID NO:2 is U, wherein R2 is an RNA sequence corresponding to the part of a promoter region starting with the nucleotide where a DNA-dependent RNA-polymerase initiates RNA synthesis,
wherein R2 is selected from the group consisting of:
(i)
(SEQ ID NO:
7)
GGGAGA;
(ii)
(SEQ ID NO:
8)
GGGAGA;
(iii)
(SEQ ID NO:
9)
GAAG;
and
(iv)
(SEQ ID NO:
10)
GGGA;
and
wherein the RNA molecule comprises a poly-A tail at the 3′ end having a length of at least 120 nucleotides;
wherein the UTR as defined in (b) has a maximal length of 14 nucleotides when R2 is (i) or (ii);
or
wherein the UTR as defined in (b) has a maximal length of 12 nucleotides when R2 is (iii) or (iv).

26 more elements

Dependencies are inferred from the claim text.

Facts

Application: 17/833,858Filed 6 Jun 2022 · continuation
Priority: EP 1616326431 Mar 2016
Published: US 2023/0086606 A123 Mar 2023
Granted: 14 May 2024
Term ends: Not estimatedThe source records don’t support an estimate.

Classification11 codes

C12N 15/85
Vectors or expression systems specially adapted for eukaryotic hosts: for animal cells
C12N 5/0688
Vertebrate cells: cells from the lungs or the respiratory tract
C12N 15/113
DNA or RNA fragments; Modified forms thereof ; Non-coding nucleic acids having a biological activity: non-coding nucleic acids modulating the…
C12N 15/67
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression: general methods for enhancing…

Family

PCT/EP2017/057592Parent of this continuation · filed 30 Mar 2017
US 11,352,638 B2Parent of this continuation · filed 7 Jun 2022
US 2023/0086606 A1Pre-grant publication · published 23 Mar 2023
Application16/090,588Parent of this continuation
US 2019/0144883 A1Published application
US 11,981,910 B2This patent

From the US filing history. Foreign family members aren’t listed.