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CA2505090A1 - Composes oligomeres conjugues et leur utilisation dans la modulation genique - Google Patents

Composes oligomeres conjugues et leur utilisation dans la modulation genique Download PDF

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Publication number
CA2505090A1
CA2505090A1 CA002505090A CA2505090A CA2505090A1 CA 2505090 A1 CA2505090 A1 CA 2505090A1 CA 002505090 A CA002505090 A CA 002505090A CA 2505090 A CA2505090 A CA 2505090A CA 2505090 A1 CA2505090 A1 CA 2505090A1
Authority
CA
Canada
Prior art keywords
composition
oligomeric compound
conjugate moiety
oligomeric
nucleic acid
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.)
Abandoned
Application number
CA002505090A
Other languages
English (en)
Inventor
Muthiah Manoharan
Brenda Baker
Ann B. Eldrup
Balkrishen Bhat
Richard H. 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.)
Individual
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
Publication of CA2505090A1 publication Critical patent/CA2505090A1/fr
Abandoned legal-status Critical Current

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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 nucleic acids [NA]
    • 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

Landscapes

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

Abstract

L'invention concerne des composés oligomères modifiés qui modulent l'expression génique via une voie d'interférence de l'ARN. Ces composés oligomères comprennent une ou plusieurs fractions conjuguées qui peuvent modifier ou renforcer les propriétés pharmacocinétiques et pharmacodynamiques du composé oligomère fixé.
CA002505090A 2002-11-05 2003-11-04 Composes oligomeres conjugues et leur utilisation dans la modulation genique Abandoned CA2505090A1 (fr)

Applications Claiming Priority (5)

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
PCT/US2003/035088 WO2004044141A2 (fr) 2002-11-05 2003-11-04 Composes oligomeres conjugues et leur utilisation dans la modulation genique

Publications (1)

Publication Number Publication Date
CA2505090A1 true CA2505090A1 (fr) 2004-05-27

Family

ID=32314505

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002505090A Abandoned CA2505090A1 (fr) 2002-11-05 2003-11-04 Composes oligomeres conjugues et leur utilisation dans la modulation genique

Country Status (3)

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

Families Citing this family (27)

* 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
US8969543B2 (en) 2003-04-03 2015-03-03 Bioneer Corporation SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
US8324365B2 (en) 2003-04-03 2012-12-04 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
US9045739B2 (en) 2004-01-16 2015-06-02 Fraunhofer-Gesellschaft Zur Forderung Der Angewandten Forschung E.V. Immunokinases
AU2006280600B2 (en) * 2005-08-17 2012-01-19 Bioneer Corporation Sirna-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
EP1800695A1 (fr) * 2005-12-21 2007-06-27 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Immuno-RNA conjugues
JP5825754B2 (ja) 2006-05-05 2015-12-02 アイシス ファーマシューティカルズ， インコーポレーテッド Ａｐｏｂの発現を調節するための化合物および方法
CA2744987C (fr) 2008-12-02 2018-01-16 Chiralgen, Ltd. Procede pour la synthese d'acides nucleiques modifies par des atomes de phosphore
CA2767253A1 (fr) 2009-07-06 2011-01-13 Ontorii, Inc. Nouveaux precurseurs d'acide nucleique et leurs methodes d'utilisation
WO2012039448A1 (fr) 2010-09-24 2012-03-29 株式会社キラルジェン Groupe auxiliaire asymétrique
WO2013012758A1 (fr) 2011-07-19 2013-01-24 Ontorii, Inc. Procédés pour la synthèse d'acides nucléiques fonctionnalisés
CN104684923B (zh) 2012-07-13 2018-09-28 株式会社新日本科学 手性核酸佐剂
JP6268157B2 (ja) 2012-07-13 2018-01-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ 不斉補助基
KR102712879B1 (ko) 2012-07-13 2024-10-04 웨이브 라이프 사이언시스 리미티드 키랄 제어
US20150291958A1 (en) 2012-11-15 2015-10-15 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
WO2014118272A1 (fr) 2013-01-30 2014-08-07 Santaris Pharma A/S Conjugués glucidiques d'oligonucléotides antimir-22
US20150368642A1 (en) 2013-01-30 2015-12-24 Hoffmann-La Roche Inc. Lna oligonucleotide carbohydrate conjugates
AU2014300981B2 (en) 2013-06-27 2017-08-10 Roche Innovation Center Copenhagen A/S Antisense oligomers and conjugates targeting PCSK9
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
EP4137572A1 (fr) 2014-01-16 2023-02-22 Wave Life Sciences Ltd. Conception chirale
WO2015113922A1 (fr) 2014-01-30 2015-08-06 Roche Innovation Center Copenhagen A/S Composé poly-oligomérique à conjugués bioclivables
WO2020150915A1 (fr) * 2019-01-23 2020-07-30 上海交通大学 Conjugué acide nucléique-médicament, système d'administration de médicament, et procédés de préparation associés et application associées
CN120210186A (zh) * 2019-07-08 2025-06-27 加利福尼亚大学董事会 脂质修饰的寡核苷酸及其使用方法
US12472148B2 (en) 2021-04-26 2025-11-18 Celanese Eva Performance Polymers Llc Implantable device for sustained release of a macromolecular drug compound

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19502912A1 (de) * 1995-01-31 1996-08-01 Hoechst Ag G-Cap Stabilisierte Oligonucleotide
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
US20030190635A1 (en) * 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
US6559279B1 (en) * 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds

Also Published As

Publication number Publication date
WO2004044141A2 (fr) 2004-05-27
WO2004044141A3 (fr) 2005-05-19
AU2003295389A1 (en) 2004-06-03
WO2004044141A8 (fr) 2004-08-26

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Legal Events

Date Code Title Description
FZDE Discontinued