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WO2004043978A3 - Composes oligomeres substitues par methoxy en position 2' et compositions a utiliser dans des modulations geniques - Google Patents

Composes oligomeres substitues par methoxy en position 2' et compositions a utiliser dans des modulations geniques Download PDF

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Publication number
WO2004043978A3
WO2004043978A3 PCT/US2003/034906 US0334906W WO2004043978A3 WO 2004043978 A3 WO2004043978 A3 WO 2004043978A3 US 0334906 W US0334906 W US 0334906W WO 2004043978 A3 WO2004043978 A3 WO 2004043978A3
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WO
WIPO (PCT)
Prior art keywords
oligomer
compositions
oligomeric compounds
methoxy substituted
hybridizing
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2003/034906
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English (en)
Other versions
WO2004043978A2 (fr
Inventor
Brenda F Baker
Ann B Eldrup
Muthiah Manoharan
Balkrishen Bhat
Richard Griffey
Eric E Swayze
Stanley T Crooke
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ionis Pharmaceuticals Inc
Original Assignee
Isis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Isis Pharmaceuticals Inc filed Critical Isis Pharmaceuticals Inc
Priority to AU2003291682A priority Critical patent/AU2003291682A1/en
Publication of WO2004043978A2 publication Critical patent/WO2004043978A2/fr
Publication of WO2004043978A3 publication Critical patent/WO2004043978A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1135—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against oncogenes or tumor suppressor genes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

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  • Chemical & Material Sciences (AREA)
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  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
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  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)

Abstract

L'invention concerne des compositions comprenant un premier oligomère et un second oligomère, dans lesquelles au moins une partie du premier oligomère peut être hybridée avec au moins une partie du second oligomère, au moins une partie du premier oligomère étant complémentaire à une hybridation et permettant une hybridation avec un acide nucléique cible sélectionné, et au moins un de ces oligomères comprenant un sucre modifié et/ou une modification de squelette. Dans certains modes de réalisation, la modification est un groupe substituant 2'-OCH3 sur une fraction de sucre. L'invention concerne également des compositions oligomères/protéiniques comprenant également un oligomère complémentaire à une hybridation et permettant une hybridation avec un acide nucléique cible sélectionné, et au moins une protéine comprenant au moins une partie d'un complexe de silençage induit par ARN (RISC), au moins un nucléotide de l'oligomère présentant un sucre modifié et/ou une modification de squelette.
PCT/US2003/034906 2002-11-05 2003-11-04 Composes oligomeres substitues par methoxy en position 2' et compositions a utiliser dans des modulations geniques Ceased WO2004043978A2 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2003291682A AU2003291682A1 (en) 2002-11-05 2003-11-04 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US42376002P 2002-11-05 2002-11-05
US60/423,760 2002-11-05
US50352103P 2003-09-16 2003-09-16
US60/503,521 2003-09-16

Publications (2)

Publication Number Publication Date
WO2004043978A2 WO2004043978A2 (fr) 2004-05-27
WO2004043978A3 true WO2004043978A3 (fr) 2005-04-07

Family

ID=32314503

Family Applications (3)

Application Number Title Priority Date Filing Date
PCT/US2003/034905 Ceased WO2004043977A2 (fr) 2002-11-05 2003-11-04 Composes oligomeres substitues par fluoro en position 2' et compositions a utiliser dans des modulations geniques
PCT/US2003/035087 Ceased WO2004044140A2 (fr) 2002-11-05 2003-11-04 Composes oligomeres 2'-substitues et compositions destinees a etre utilisees dans des modulations genetiques
PCT/US2003/034906 Ceased WO2004043978A2 (fr) 2002-11-05 2003-11-04 Composes oligomeres substitues par methoxy en position 2' et compositions a utiliser dans des modulations geniques

Family Applications Before (2)

Application Number Title Priority Date Filing Date
PCT/US2003/034905 Ceased WO2004043977A2 (fr) 2002-11-05 2003-11-04 Composes oligomeres substitues par fluoro en position 2' et compositions a utiliser dans des modulations geniques
PCT/US2003/035087 Ceased WO2004044140A2 (fr) 2002-11-05 2003-11-04 Composes oligomeres 2'-substitues et compositions destinees a etre utilisees dans des modulations genetiques

Country Status (4)

Country Link
EP (1) EP1563070A4 (fr)
AU (4) AU2003295388A1 (fr)
CA (1) CA2504554A1 (fr)
WO (3) WO2004043977A2 (fr)

Cited By (4)

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US7456155B2 (en) 2002-06-28 2008-11-25 Idenix Pharmaceuticals, Inc. 2′-C-methyl-3′-O-L-valine ester ribofuranosyl cytidine for treatment of flaviviridae infections
US7582618B2 (en) 2002-06-28 2009-09-01 Idenix Pharmaceuticals, Inc. 2′-C-methyl-3′-O-L-valine ester ribofuranosyl cytidine for treatment of flaviviridae infections
US7598373B2 (en) 2002-12-12 2009-10-06 Idenix Pharmaceuticals, Inc. Process for the production of 2-C-methyl-D-ribonolactone
US7608597B2 (en) 2000-05-23 2009-10-27 Idenix Pharmaceuticals, Inc. Methods and compositions for treating hepatitis C virus

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US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
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PL359169A1 (en) 2000-05-26 2004-08-23 Idenix (Cayman) Limited Methods and compositions for treating flaviviruses and pestiviruses
WO2005078097A2 (fr) 2004-02-10 2005-08-25 Sirna Therapeutics, Inc. Inhibition induite par l'interference arn de l'expression genetique, a l'aide d'un acide nucleique interferant court multifonctionnel (sina multifonctionnel)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US20050148530A1 (en) 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7517864B2 (en) 2001-05-18 2009-04-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
GB2413557B (en) * 2002-02-20 2006-08-16 Sirna Therapeutics Inc RNA interference mediated inhibtion of gene expression using chemically modified short interfering nucleic acid (siNA)
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US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
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Citations (4)

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EP1563070A4 (fr) 2008-05-28
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EP1563070A2 (fr) 2005-08-17
WO2004044140A2 (fr) 2004-05-27
WO2004043977A2 (fr) 2004-05-27
AU2003295388A1 (en) 2004-06-03
AU2003291682A8 (en) 2004-06-03
WO2004044140A3 (fr) 2005-04-14
AU2003287464A8 (en) 2004-06-03
CA2504554A1 (fr) 2004-05-27
AU2010201712B2 (en) 2012-05-31
WO2004043978A2 (fr) 2004-05-27
AU2010201712A1 (en) 2010-05-20

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