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1. WO2011031520 - COMPOSITION FOR INHIBITING GENE EXPRESSION AND USES THEREOF

Publication Number WO/2011/031520
Publication Date 17.03.2011
International Application No. PCT/US2010/046781
International Filing Date 26.08.2010
IPC
C12N 15/113 2010.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
CPC
A61P 11/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
11Drugs for disorders of the respiratory system
A61P 11/06
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
11Drugs for disorders of the respiratory system
06Antiasthmatics
A61P 17/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
17Drugs for dermatological disorders
A61P 29/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
29Non-central analgesic, antipyretic or anti-inflammatory agents, e.g antirheumatic agents; Non-steroidal anti-inflammatory drugs (NSAIDs)
A61P 31/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
A61P 31/04
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
04Antibacterial agents
Applicants
  • IDERA PHARMACEUTICALS, INC. [US]/[US] (AllExceptUS)
  • AGRAWAL, Sudhir [US]/[US] (UsOnly)
  • KANDIMALLA, Ekambar [US]/[US] (UsOnly)
  • PUTTA, Millikarjuna [IN]/[US] (UsOnly)
  • LAN, Tao [CN]/[US] (UsOnly)
  • BHAGAT, Lakshmi [IN]/[US] (UsOnly)
  • WANG, Daqing [US]/[US] (UsOnly)
  • YU, Dong [CN]/[US] (UsOnly)
Inventors
  • AGRAWAL, Sudhir
  • KANDIMALLA, Ekambar
  • PUTTA, Millikarjuna
  • LAN, Tao
  • BHAGAT, Lakshmi
  • WANG, Daqing
  • YU, Dong
Agents
  • WAYNE, A., Keown
Priority Data
61/240,55308.09.2009US
61/275,25227.08.2009US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITION FOR INHIBITING GENE EXPRESSION AND USES THEREOF
(FR) COMPOSITION POUR INHIBER L'EXPRESSION GÉNIQUE ET SES UTILISATIONS
Abstract
(EN)
The inventors have examined the means for providing more efficacious gene expression blocking compounds. The inventors have discovered new structural features that surprisingly improve the efficacy of gene expression blocking molecules. These features include the presence of multiple 3' ends and a linker at the 5' ends. Surprisingly, these features improve the efficacy of the gene expression blocking compounds in a manner that decreases the compound's biologic instability. Even more surprisingly, this effect has been found to be applicable to both DNA and RNA oligonucleotide -based compounds and to have application in traditional antisense and RNAi technologies.
(FR)
Les inventeurs ont examiné les moyens pour fournir des composés de blocage d'expression génique plus efficaces. Les inventeurs ont découvert de nouvelles caractéristiques structurales qui améliorent de façon surprenante l'efficacité de molécules de blocage d'expression génique. Ces caractéristiques comprennent la présence de multiples extrémités 3' et d'un liant aux extrémités 3'. De façon surprenante, ces caractéristiques améliorent l'efficacité des composés de blocage d'expression génique d'une manière qui diminue l'instabilité biologique des composés. De façon encore plus surprenante, il a été démontré que cet effet est applicable à des composés à base d'oligonucléotides à la fois d'ADN et d'ARN et a une application dans des technologies antisens et d'ARNi traditionnel.
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