Interfering RNA molecules

Patent metadata

Field	Value
Publication number	US-9222092-B2
Application number	US-201414578636-A
Country	US
Kind code	B2
Filing date	Dec 22, 2014
Priority date	Aug 5, 2002
Publication date	Dec 29, 2015
Grant date	Dec 29, 2015

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.

First claim

We claim:

1. 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 and whereby said second stretch is at least partially identical to a target nucleic acid, whereby the double stranded structure is blunt ended or has an overhang and contains a group of ribonucleic acids on the first or second stretch/strand that has a modification at the 2′-position selected from an amino, fluoro, methoxy, alkoxy or alkyl modification and said group of modified ribonucleic acids on the first or second stretch/strand is flanked on one or both sides by a group of flanking ribonucleic acids having a modification at the 2′-position selected from an amino, fluoro, methoxy, alkoxy or alkyl modification that is different from the modification on said group of ribonucleic acids modified at the 2′-position and the number of ribonucleic acids in the group of modified ribonucleic acids is selected from one to ten ribonucleic acids and the number of ribonucleic acids in the group of flanking ribonucleic acids is selected from one to ten ribonucleic acids.

2. The ribonucleic acid according to claim 1 , wherein the 2′ modification is selected from 2′-O-methyl and 2′-fluoro.

3. The ribonucleic acid according to claim 1 , wherein the double stranded structure is blunt ended on the double stranded structure which is defined by the 3′-end of the first strand and the 5′-end of the second strand.

4. The ribonucleic acid according to claim 1 , wherein at least one of the strands has an overhang of at least one nucleotide at the 3′-end consisting of a ribonucleotide or deoxyribonucleotide.

5. The ribonucleic acid according to claim 1 , wherein the double stranded structure has a length of 17 to 21 nucleotides.

6. The ribonucleic acid according to claim 1 , wherein the ribonucleic acid has an overhang at the 3′-end of the second strand.

7. The ribonucleic acid according to claim 1 , wherein said ribonucleic acid has a double stranded structure that is 15 to 23 nucleotides in length.

8. The ribonucleic acid according to claim 1 , wherein the modifications to the groups of modified ribonucleic acids and the groups of flanking ribonucleic acids are made on internal ribonucleic acids.

9. The ribonucleic acid according to claim 1 , wherein a group of modified ribonucleic acids on the first or second stretch/strand is flanked on both sides by a group of flanking ribonucleic acids having a modification at the 2′-position selected from an amino, fluoro, methoxy, alkoxy or alkyl modification that is different from the modification on said group of modified ribonucleic acids.

10. The ribonucleic acid according to claim 1 , wherein the 2′ modification is selected from 2′-O-methyl and 2′-fluoro.

Assignees

• Silence Therapeutics Ag Ag
• Silence Therapeutics Gmbh

Inventors

• Giese Klaus
• Kaufmann Jörg
• Klippel-Giese Anke

Classifications

• A61P35/00

  Antineoplastic agents · CPC title

• A61P35/02

  specific for leukemia · CPC title

• A61P3/04

  Anorexiants; Antiobesity agents · CPC title

• A61P43/00

  Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title

• A61P9/00

  Drugs for disorders of the cardiovascular system · CPC title