IP Library Granted Patent US 9,790,501
Granted Patent B2
US 9,790,501 · App. 15/589,968 · Granted Oct 17, 2017

Interfering RNA molecules

Inventors: Klaus Giese (Berlin, DE); Jörg Kaufmann (Berlin, DE); Anke Klippel-Giese (Berchtesgaden, DE)
Assignee: Silence Therapeutics GMBH
C12N15/113C12N15/1137C12Y207/11001C12N2310/14C12N2310/321C12N2310/322C12N2310/531C12N2320/51
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Quick Facts
Patent No.
US 9,790,501
App. No.
15/589,968
Granted
Oct 17, 2017
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 (36)

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 is 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 comprises contiguous alternating 2′-O-alkyl ribonucleotides, wherein the 2′-O-alkyl ribonucleotides alternate with unmodified or differently modified ribonucleotides,

wherein the 2′-O-alkyl ribonucleotides of the first strand are shifted by one ribonucleotide relative to the 2′-O-alkyl ribonucleotides of the second strand, and

wherein each of the first stretch and the second stretch consists of at least 17 and fewer than 30 ribonucleotides.

2. The double-stranded siRNA molecule of claim 1 , wherein the double-stranded siRNA molecule is blunt ended on both ends.

3. The double-stranded siRNA molecule of claim 1 , wherein at least one end of the double-stranded siRNA molecule has an overhang.

4. The double-stranded siRNA molecule of claim 3 , wherein the overhang has one to eight nucleotides.

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

6. The double-stranded siRNA molecule of claim 3 , wherein the 5′ end of the first strand has an overhang.

7. The double-stranded siRNA molecule of claim 3 , wherein the 3′ end of the second strand has an overhang.

8. The double-stranded siRNA molecule of claim 3 , wherein the 5′ end of the second strand has an overhang.

9. The double-stranded siRNA molecule of claim 1 , wherein each of the first strand and the second strand consists of at least 18 nucleotides.

10. The double-stranded siRNA molecule of claim 1 , wherein the 2′-O-alkyl ribonucleotides are 2′-O-methyl ribonucleotides.

11. The double-stranded siRNA molecule of claim 10 , wherein the 2′-O-methyl ribonucleotides alternate with the unmodified ribonucleotides.

12. The double-stranded siRNA molecule of claim 1 , wherein the differently modified ribonucleotides have an amino, fluoro, different alkoxy, or alkyl modification at the 2′-position.

13. The double-stranded siRNA molecule of claim 10 , wherein the 2′-O-methyl ribonucleotides alternate with 2′-fluoro ribonucleotides.

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

15. The double-stranded siRNA molecule of claim 14 , wherein the single 2′-O-alkyl ribonucleotides are 2′-O-methyl ribonucleotides.

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

17. The double-stranded siRNA molecule of claim 16 , wherein the single 2′-O-alkyl ribonucleotides are 2′-O-methyl ribonucleotides.

18. The double-stranded siRNA molecule of claim 16 , wherein the differently modified ribonucleotides have an amino, fluoro, different alkoxy, or alkyl modification at the 2′-position.

19. The double-stranded siRNA molecule of claim 16 , wherein the single 2′-O-alkyl ribonucleotides are 2′-O-methyl ribonucleotides, and the differently modified ribonucleotides are 2′-fluoro ribonucleotides.

20. The double-stranded siRNA molecule of claim 1 , wherein the 5′ ribonucleotide of the first stretch is a 2′-O-alkyl modified ribonucleotide.

21. The double-stranded siRNA molecule of claim 11 , wherein the 5′ ribonucleotide of the first stretch is a 2′-O-methyl ribonucleotide.

22. The double-stranded siRNA molecule of claim 13 , wherein the 5′ ribonucleotide of the first stretch is a 2′-O-methyl ribonucleotide.

23. The double-stranded siRNA molecule of claim 14 , wherein the 5′ ribonucleotide of the first stretch is a 2′-O-alkyl modified ribonucleotide that alternates with an unmodified ribonucleotide.

24. The double-stranded siRNA molecule of claim 16 , wherein the 5′ ribonucleotide of the first stretch is a 2′-O-alkyl modified ribonucleotide that alternates with a differently modified ribonucleotide.

25. The double-stranded siRNA molecule of claim 19 , wherein the double-stranded siRNA molecule is blunt on at least one end.

26. The double-stranded siRNA molecule of claim 19 , wherein at least one end of the double-stranded siRNA molecule has an overhang of one to eight nucleotides.

27. The double-stranded siRNA molecule of claim 26 , wherein the 3′ end of the first strand has an overhang.

28. The double-stranded siRNA molecule of claim 26 , wherein the 5′ end of the first strand has an overhang.

29. The double-stranded siRNA molecule of claim 26 , wherein the 3′ end of the second strand has an overhang.

30. The double-stranded siRNA molecule of claim 26 , wherein the 5′ end of the second strand has an overhang.

Assignments (3)
CHANGE OF NAME Recorded Aug 14, 2017
From: ATUGEN AG (AKTIENGESELLSCHAFT)
To: SILENCE THERAPEUTICS AKTIENGESELLSCHAFT (AG)
Reel/Frame 043287/0698 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Aug 14, 2017
From: GIESE, KLAUS; KAUFMANN, JÖRG; KLIPPEL-GIESE, ANKE
To: ATUGEN AG
Reel/Frame 043287/0708 →
CHANGE OF NAME Recorded Aug 14, 2017
From: SILENCE THERAPEUTICS AKTIENGESELLSCHAFT (AG)
To: SILENCE THERAPEUTICS GMBH
Reel/Frame 043546/0453 →
Priority Claims (2)
EP 02017601 · Aug 5, 2002 · regional
EP 03008383 · Apr 10, 2003 · regional
Continuity (8)
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 20170247697A1 · Aug 31, 2017