WO2004043978A3 - 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations - Google Patents

2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations 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
Application number
PCT/US2003/034906
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French (fr)
Other versions
WO2004043978A2 (en
Inventor
Brenda F Baker
Ann B Eldrup
Muthiah Manoharan
Balkrishen Bhat
Richard Griffey
Eric E Swayze
Stanley T Crooke
Original Assignee
Isis Pharmaceuticals Inc
Brenda F Baker
Ann B Eldrup
Muthiah Manoharan
Balkrishen Bhat
Richard Griffey
Eric E Swayze
Stanley T Crooke
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, Brenda F Baker, Ann B Eldrup, Muthiah Manoharan, Balkrishen Bhat, Richard Griffey, Eric E Swayze, Stanley T Crooke filed Critical Isis Pharmaceuticals Inc
Priority to AU2003291682A priority Critical patent/AU2003291682A1/en
Publication of WO2004043978A2 publication Critical patent/WO2004043978A2/en
Publication of WO2004043978A3 publication Critical patent/WO2004043978A3/en

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    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/321—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
    • C—CHEMISTRY; METALLURGY
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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 ===---===
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C12N2310/34—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
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

Abstract

Compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes a modified sugar and/or backbone modification. In some embodiments the modification is a 2'-OCH3 substituent group on a sugar moiety. Oligomer/protein compositions are also provided comprising an oligomer complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein at least one nucleotide of the oligomer has a modified sugar and/or backbone modification.
PCT/US2003/034906 2002-11-05 2003-11-04 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations WO2004043978A2 (en)

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 (en) 2004-05-27
WO2004043978A3 true WO2004043978A3 (en) 2005-04-07

Family

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Family Applications (3)

Application Number Title Priority Date Filing Date
PCT/US2003/034906 WO2004043978A2 (en) 2002-11-05 2003-11-04 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations
PCT/US2003/035087 WO2004044140A2 (en) 2002-11-05 2003-11-04 2’-substituted oligomeric compounds and compositions for use in gene modulations
PCT/US2003/034905 WO2004043977A2 (en) 2002-11-05 2003-11-04 2’-fluoro substituted oligomeric compounds and compositions for use in gene modulations

Family Applications After (2)

Application Number Title Priority Date Filing Date
PCT/US2003/035087 WO2004044140A2 (en) 2002-11-05 2003-11-04 2’-substituted oligomeric compounds and compositions for use in gene modulations
PCT/US2003/034905 WO2004043977A2 (en) 2002-11-05 2003-11-04 2’-fluoro substituted oligomeric compounds and compositions for use in gene modulations

Country Status (4)

Country Link
EP (1) EP1563070A4 (en)
AU (4) AU2003291682A1 (en)
CA (1) CA2504554A1 (en)
WO (3) WO2004043978A2 (en)

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US8299038B2 (en) 2000-05-23 2012-10-30 Idenix Pharmaceuticals, Inc. Methods and compositions for treating hepatitis C virus

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US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
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AU2010201712B2 (en) 2012-05-31
WO2004043978A2 (en) 2004-05-27
AU2003291682A1 (en) 2004-06-03
WO2004043977A3 (en) 2005-01-20
AU2003287464A8 (en) 2004-06-03
EP1563070A4 (en) 2008-05-28
AU2003287464A1 (en) 2004-06-03
EP1563070A2 (en) 2005-08-17
AU2010201712A1 (en) 2010-05-20
CA2504554A1 (en) 2004-05-27
WO2004043977A2 (en) 2004-05-27
AU2003291682A8 (en) 2004-06-03
WO2004044140A3 (en) 2005-04-14
AU2003295388A1 (en) 2004-06-03
WO2004044140A2 (en) 2004-05-27

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