WO2004044138A3 - Chimeric oligomeric compounds and their use in gene modulation - Google Patents

Chimeric oligomeric compounds and their use in gene modulation Download PDF

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Publication number
WO2004044138A3
WO2004044138A3 PCT/US2003/035074 US0335074W WO2004044138A3 WO 2004044138 A3 WO2004044138 A3 WO 2004044138A3 US 0335074 W US0335074 W US 0335074W WO 2004044138 A3 WO2004044138 A3 WO 2004044138A3
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WIPO (PCT)
Prior art keywords
oligomer
oligomeric compounds
hybridizing
chimeric
chimeric oligomeric
Prior art date
Application number
PCT/US2003/035074
Other languages
French (fr)
Other versions
WO2004044138A2 (en
Inventor
Brenda F Baker
Anne B Eldrup
Muthiah Manoharan
Balkrishen Bhat
Richard Griffey
Eric E Swayze
Stanley T Crooke
Original Assignee
Isis Pharmaceuticals Inc
Brenda F Baker
Anne 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, Anne B Eldrup, Muthiah Manoharan, Balkrishen Bhat, Richard Griffey, Eric E Swayze, Stanley T Crooke filed Critical Isis Pharmaceuticals Inc
Priority to CA2504720A priority Critical patent/CA2504720C/en
Priority to AU2003287505A priority patent/AU2003287505A1/en
Priority to EP03781746A priority patent/EP1560839A4/en
Publication of WO2004044138A2 publication Critical patent/WO2004044138A2/en
Publication of WO2004044138A3 publication Critical patent/WO2004044138A3/en

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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
    • 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
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03048—Protein-tyrosine-phosphatase (3.1.3.48)
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    • 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 N.A.
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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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    • C12N2310/30—Chemical structure
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    • 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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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)

Abstract

Oligomer 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 capble of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes at least one nucleotide comprising a chimeric organic composition. 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 comprising a chimeric organic composition.
PCT/US2003/035074 2002-11-05 2003-11-04 Chimeric oligomeric compounds and their use in gene modulation WO2004044138A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
CA2504720A CA2504720C (en) 2002-11-05 2003-11-04 Chimeric oligomeric compounds and their use in gene modulation
AU2003287505A AU2003287505A1 (en) 2002-11-05 2003-11-04 Chimeric oligomeric compounds and their use in gene modulation
EP03781746A EP1560839A4 (en) 2002-11-05 2003-11-04 Chimeric oligomeric compounds and their use in gene modulation

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US42376002P 2002-11-05 2002-11-05
US60/423,760 2002-11-05
US50327103P 2003-09-15 2003-09-15
US60/503,271 2003-09-15

Publications (2)

Publication Number Publication Date
WO2004044138A2 WO2004044138A2 (en) 2004-05-27
WO2004044138A3 true WO2004044138A3 (en) 2005-03-24

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Application Number Title Priority Date Filing Date
PCT/US2003/035074 WO2004044138A2 (en) 2002-11-05 2003-11-04 Chimeric oligomeric compounds and their use in gene modulation

Country Status (4)

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EP (1) EP1560839A4 (en)
AU (1) AU2003287505A1 (en)
CA (1) CA2504720C (en)
WO (1) WO2004044138A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9598693B2 (en) 2006-01-27 2017-03-21 Ionis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas

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Publication number Priority date Publication date Assignee Title
US20080119427A1 (en) * 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
US7875733B2 (en) * 2003-09-18 2011-01-25 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising 4′-thionucleosides for use in gene modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
WO2005121368A1 (en) * 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Chimeric gapped oligomeric compositions
EP1578765A4 (en) 2002-11-05 2008-04-23 Isis Pharmaceuticals Inc Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US8569474B2 (en) 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
EP1765847A4 (en) * 2004-05-27 2010-10-20 Alnylam Pharmaceuticals Inc Nuclease resistant double-stranded ribonucleic acid
US8394947B2 (en) * 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
CA2628113A1 (en) * 2005-11-04 2007-05-18 Nastech Pharmaceutical Company Inc. Peptide-dicer substrate rna conjugates as delivery vehicles for sirna
US9045754B2 (en) * 2006-05-05 2015-06-02 Isis Pharmaceuticals, Inc. Short antisense compounds with gapmer configuration
JP5876637B2 (en) * 2006-10-18 2016-03-02 マリーナ バイオテック，インコーポレイテッド Nicked or gapped nucleic acid molecules and their use

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