IP Library Granted Patent US 10,266,829
Granted Patent B2
US 10,266,829 · App. 16/013,776 · Granted Apr 23, 2019

Interfering RNA molecules

Inventors: Klaus Giese (Berlin, DE); Jörg Kaufmann (Berlin, DE); Anke Klippel-Giese (Berchtesgaden, DE)
Assignee: Silence Therapeutics GMBH
C12N15/1137A61K31/713C12N5/10C12N15/111C12N15/113C12Q1/68C12Y207/11001A61K38/00C12N2310/14C12N2310/3183C12N2310/32C12N2310/321C12N2310/322C12N2310/343C12N2310/346C12N2310/3521C12N2310/3525C12N2310/53C12N2310/531C12N2320/51
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Quick Facts
Patent No.
US 10,266,829
App. No.
16/013,776
Granted
Apr 23, 2019
Kind
B2
Abstract

The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double-stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended.

Claims (25)

1. A double stranded siRNA molecule against a target nucleic acid, wherein the double stranded siRNA molecule comprises a first strand and a second strand, wherein the first strand comprises a first stretch that is complementary to the target nucleic acid, wherein the second strand comprises a second stretch that has the same length as the first stretch and is complementary to the first stretch, wherein the first strand and the second strand form a double-stranded structure comprising the first stretch and the second stretch,

wherein the first stretch and the second stretch each has the length of 17-21 nucleotides,

wherein the first stretch and the second stretch each comprises a plurality of groups of 2′-O-methyl ribonucleotides flanked on one or both sides by a flanking group of 2′-fluororibonucleotides, wherein at least one end of the double stranded siRNA molecule is blunt ended.

2. The double stranded siRNA molecule of claim 1 , wherein the first stretch and the second stretch each has the length of 21 nucleotides.

3. The double stranded siRNA molecule of claim 1 , wherein the number of 2′-O′methyl ribonucleotides in each of the plurality of groups of 2′-O-methyl ribonucleotides is one to ten.

4. The double stranded siRNA molecule of claim 1 , wherein the number of 2′-fluororibonucleotides in each of the flanking groups is one to ten.

5. The double stranded siRNA molecule of claim 1 , wherein the number of 2′-fluororibonucleotides in each of the flanking groups is five.

6. The double stranded siRNA molecule of claim 1 , wherein the number of 2′-fluororibonucleotides in each of the flanking groups is six.

7. The double stranded siRNA molecule of claim 1 , wherein each 2′-fluoro ribonucleotide on the first stretch corresponds to a 2′-O-methyl ribonucleotide on the second stretch.

8. The double stranded siRNA molecule of claim 7 , wherein the number of 2′-O-methyl ribonucleotides on the first stretch is less than the number of 2′-O-methyl ribonucleotides on the second stretch.

9. The double stranded siRNA molecule of claim 1 , wherein each 2′-fluoro ribonucleotide on the second stretch corresponds to a 2′-O-methyl ribonucleotide on the first stretch.

10. The double stranded siRNA molecule of claim 9 , wherein the number of 2′-O-methyl ribonucleotides on the first stretch is less than the number of 2′-O-methyl ribonucleotides on the second stretch.

11. The double-stranded siRNA molecule of claim 1 , wherein the number of 2′-O-methyl ribonucleotides on the first stretch is less than the number of 2′-O-methyl ribonucleotides on the second stretch.

12. The double-stranded siRNA molecule of claim 1 , wherein the first stretch and the second stretch each comprises contiguous alternating single 2′-O-methyl ribonucleotides, and wherein the single 2′-O-methyl ribonucleotides alternate with the 2′-fluororibonucleotides.

13. The double-stranded siRNA molecule of claim 12 , wherein each 2′-fluoro ribonucleotide on the second stretch corresponds to a 2′-O-methyl ribonucleotide on the first stretch.

14. The double-stranded siRNA molecule of claim 1 , wherein the first strand, the second strand or both the first strand and the second strand has a partial phosphorothioate backbone.

15. The double stranded siRNA molecule of claim 1 , wherein the 3′-end of the first strand has an overhang.

16. The double stranded siRNA molecule of claim 15 , wherein the overhang has one to eight nucleotides.

17. The double stranded siRNA molecule of claim 16 , wherein the overhang has two nucleotides.

18. The double stranded siRNA molecule of claim 1 , wherein the double stranded siRNA molecule is blunt ended at the 5′-end of the first strand and 3′-end of the second strand.

19. The double stranded siRNA molecule of claim 1 , wherein the 3′-end of the second strand has an end modification.

20. The double stranded siRNA molecule of claim 1 , wherein the target nucleic acid is selected from the group comprising structural genes, housekeeping genes, transcription factors, motility factors, cell cycle factors, cell cycle inhibitors, enzymes, growth factors, cytokines, and tumor suppressors.

21. A composition comprising the double stranded siRNA molecule of claim 1 .

22. The composition of claim 21 , wherein the composition is a pharmaceutical composition.

23. The double stranded siRNA molecule of claim 1 , wherein both ends of the double stranded siRNA molecule are blunt ended.

Assignments (3)
CHANGE OF NAME Recorded Aug 23, 2018
From: SILENCE THERAPEUTICS AG
To: SILENCE THERAPEUTICS GMBH
Reel/Frame 046922/0677 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 23, 2018
From: GIESE, KLAUS; KAUFMANN, JÖRG; KLIPPEL-GIESE, ANKE
To: ATUGEN AG
Reel/Frame 046689/0147 →
CHANGE OF NAME Recorded Aug 23, 2018
From: ATUGEN AG (AKTIENGESELLSCHAFT)
To: SILENCE THERAPEUTICS AKTIENGESELLSCHAFT (AG)
Reel/Frame 046689/0168 →
Priority Claims (2)
EP 02017601 · Aug 5, 2002 · regional
EP 03008383 · Apr 10, 2003 · regional
Continuity (10)
Continuation 15678024 · Aug 15, 2017
Continuation 15589968 · May 8, 2017
Continuation 14977710 · Dec 22, 2015
Continuation 14578636 · Dec 22, 2014
Continuation 13692178 · Dec 3, 2012
Continuation 12986389 · Jan 7, 2011
Continuation 12200296 · Aug 28, 2008
Continuation 10633630 · Aug 5, 2003
Provisional Application 60402541 · Aug 12, 2002
Related Publication 20180291381A1 · Oct 11, 2018