WO2004044141A3 - Conjugated oligomeric compounds and their use in gene modulation - Google Patents

Conjugated oligomeric compounds and their use in gene modulation Download PDF

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Publication number
WO2004044141A3
WO2004044141A3 PCT/US2003/035088 US0335088W WO2004044141A3 WO 2004044141 A3 WO2004044141 A3 WO 2004044141A3 US 0335088 W US0335088 W US 0335088W WO 2004044141 A3 WO2004044141 A3 WO 2004044141A3
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WO
WIPO (PCT)
Prior art keywords
oligomeric compounds
gene modulation
conjugated oligomeric
conjugated
gene
Prior art date
Application number
PCT/US2003/035088
Other languages
French (fr)
Other versions
WO2004044141A2 (en
WO2004044141A8 (en
Inventor
Muthiah Manoharan
Brenda Baker
Ann Eldrup
Balkrishen Bhat
Richard H Griffey
Eric E Swayze
Stanley T Crooke
Original Assignee
Isis Pharmaceuticals Inc
Muthiah Manoharan
Brenda Baker
Ann Eldrup
Balkrishen Bhat
Richard H 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, Muthiah Manoharan, Brenda Baker, Ann Eldrup, Balkrishen Bhat, Richard H Griffey, Eric E Swayze, Stanley T Crooke filed Critical Isis Pharmaceuticals Inc
Priority to AU2003295389A priority Critical patent/AU2003295389A1/en
Priority to CA002505090A priority patent/CA2505090A1/en
Publication of WO2004044141A2 publication Critical patent/WO2004044141A2/en
Publication of WO2004044141A8 publication Critical patent/WO2004044141A8/en
Publication of WO2004044141A3 publication Critical patent/WO2004044141A3/en

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    • 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
    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

Abstract

The present invention provides modified oligomeric compounds that modulate gene expression via an RNA interference pathway. The oligomeric compounds of the invention include one or more conjugate moieties that can modify or enhance the pharmacokinetic and pharmacodynamic properties of the attached oligomeric compound.
PCT/US2003/035088 2002-11-05 2003-11-04 Conjugated oligomeric compounds and their use in gene modulation WO2004044141A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2003295389A AU2003295389A1 (en) 2002-11-05 2003-11-04 Conjugated oligomeric compounds and their use in gene modulation
CA002505090A CA2505090A1 (en) 2002-11-05 2003-11-04 Conjugated 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
US61624103A 2003-07-09 2003-07-09
US10/616,241 2003-07-09

Publications (3)

Publication Number Publication Date
WO2004044141A2 WO2004044141A2 (en) 2004-05-27
WO2004044141A8 WO2004044141A8 (en) 2004-08-26
WO2004044141A3 true WO2004044141A3 (en) 2005-05-19

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2003/035088 WO2004044141A2 (en) 2002-11-05 2003-11-04 Conjugated oligomeric compounds and their use in gene modulation

Country Status (3)

Country Link
AU (1) AU2003295389A1 (en)
CA (1) CA2505090A1 (en)
WO (1) WO2004044141A2 (en)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9827263B2 (en) 2002-11-05 2017-11-28 Ionis Pharmaceuticals, Inc. 2′-methoxy substituted oligomeric compounds and compositions for use in gene modulations
US9150605B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
WO2004087931A1 (en) 2003-04-03 2004-10-14 Korea Advanced Institute Of Science And Technology Conjugate for gene transfer comprising oligonucleotide and hydrophilic polymer, polyelectrolyte complex micelles formed from the conjugate, and methods for preparation thereof
US8969543B2 (en) 2003-04-03 2015-03-03 Bioneer Corporation SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
ES2384940T3 (en) 2004-01-16 2012-07-16 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Immunokinase
AU2006280600B2 (en) * 2005-08-17 2012-01-19 Bioneer Corporation Sirna-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
EP1800695A1 (en) * 2005-12-21 2007-06-27 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Immuno-RNA-constructs
EP2023939B1 (en) 2006-05-05 2012-06-27 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of pcsk9
BRPI0923225A2 (en) 2008-12-02 2016-10-04 Chiralgen Ltd Phosphorus-modified nucleic acid synthesis method
MX342945B (en) 2009-07-06 2016-10-18 Ontorii Inc * Novel nucleic acid prodrugs and methods use thereof.
JP5868324B2 (en) 2010-09-24 2016-02-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ Asymmetric auxiliary group
WO2013012758A1 (en) 2011-07-19 2013-01-24 Ontorii, Inc. Methods for the synthesis of functionalized nucleic acids
CN104684893B (en) 2012-07-13 2016-10-26 日本波涛生命科学公司 Asymmetric auxiliary group
RU2677639C2 (en) 2012-07-13 2019-01-18 Шин Ниппон Биомедикал Лэбораториз, Лтд. Chiral nucleic acid adjuvant
KR102450907B1 (en) 2012-07-13 2022-10-04 웨이브 라이프 사이언시스 리미티드 Chiral control
RU2015119411A (en) 2012-11-15 2017-01-10 Рош Инновейшен Сентер Копенгаген А/С ANTI-SENSE COMPOUNDS CONJUGATES AIMED AT APOLIPOPROTEIN IN
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
MY177989A (en) 2013-01-30 2020-09-28 Hoffmann La Roche Lna oligonucleotide carbohydrate conjugates
BR112015032432B1 (en) 2013-06-27 2023-02-07 Roche Innovation Center Copenhagen A/S ANTI-SENSE OLIGOMER, ANTI-SENSE OLIGONUCLEOTIDE CONJUGATES, PHARMACEUTICAL COMPOSITION, USE THEREOF FOR THE TREATMENT OF HYPERCHOLESTEROLEMIA OR RELATED DISORDERS, AND IN VITRO METHOD FOR REDUCING THE EXPRESSION LEVELS AND/OR THE ACTIVITY OF PCSK9 IN A CELL
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
JPWO2015108047A1 (en) 2014-01-15 2017-03-23 株式会社新日本科学 Chiral nucleic acid adjuvant having immunity induction activity and immunity induction activator
US10149905B2 (en) 2014-01-15 2018-12-11 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
KR20230152178A (en) 2014-01-16 2023-11-02 웨이브 라이프 사이언시스 리미티드 Chiral design
KR102287532B1 (en) 2014-01-30 2021-08-11 에프. 호프만-라 로슈 아게 Poly oligomer compound with biocleavable conjugates
CN114127306A (en) * 2019-07-08 2022-03-01 加利福尼亚大学董事会 Lipid-modified oligonucleotides and methods of use thereof

Citations (5)

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US5998588A (en) * 1995-09-01 1999-12-07 University Of Washington Interactive molecular conjugates
US20020102267A1 (en) * 1999-10-21 2002-08-01 Lu Peter S. CLASP-5 transmembrane protein
US20020151512A1 (en) * 1995-01-31 2002-10-17 Anuschirwan Peyman G cap-stabilized oligonucleotides
US20020156235A1 (en) * 2000-09-08 2002-10-24 Muthiah Manoharan Process for preparing peptide derivatized oligomeric compounds
US20030190635A1 (en) * 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA

Patent Citations (5)

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US20020151512A1 (en) * 1995-01-31 2002-10-17 Anuschirwan Peyman G cap-stabilized oligonucleotides
US5998588A (en) * 1995-09-01 1999-12-07 University Of Washington Interactive molecular conjugates
US20020102267A1 (en) * 1999-10-21 2002-08-01 Lu Peter S. CLASP-5 transmembrane protein
US20020156235A1 (en) * 2000-09-08 2002-10-24 Muthiah Manoharan Process for preparing peptide derivatized oligomeric compounds
US20030190635A1 (en) * 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA

Non-Patent Citations (4)

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Title
ASTRIAB-FISHER ET AL: "Conjugates of antisense oligonucleotides with the Tat and antennapedia cell-penetrating peptides: effects on cellular uptake, binding to target sequences, and biologic actions.", PHARMACEUTICAL RESEARCH, vol. 19, no. 6, June 2002 (2002-06-01), pages 744 - 754, XP002259727 *
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MANOHARAN M. ET AL: "Oligonucleotide conjugates as potential antisense drugs with improved uptake, biodistribution, targeted delivery, and mechanism of action.", ANTISENSE & NUCLEIC ACID DRUG DEVELOPMENT, vol. 12, 2002, pages 103 - 128, XP002294027 *
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Also Published As

Publication number Publication date
AU2003295389A1 (en) 2004-06-03
WO2004044141A2 (en) 2004-05-27
WO2004044141A8 (en) 2004-08-26
CA2505090A1 (en) 2004-05-27

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