ES2643576T3 - Dúplex oligonucleotídicos que comprenden nucleótidos de tipo ADN y de tipo ARN y usos de los mismos - Google Patents

Dúplex oligonucleotídicos que comprenden nucleótidos de tipo ADN y de tipo ARN y usos de los mismos Download PDF

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Publication number
ES2643576T3
ES2643576T3 ES09757018.8T ES09757018T ES2643576T3 ES 2643576 T3 ES2643576 T3 ES 2643576T3 ES 09757018 T ES09757018 T ES 09757018T ES 2643576 T3 ES2643576 T3 ES 2643576T3
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rna
aan
sirna
antisense
nucleic acid
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ES09757018.8T
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English (en)
Spanish (es)
Inventor
Masad J. Damha
Jonathan K. Watts
Glen Deleavey
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Paladin Labs Inc
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Paladin Labs Inc
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Priority claimed from PCT/CA2008/002259 external-priority patent/WO2009076775A1/en
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • CCHEMISTRY; METALLURGY
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/51Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/53Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
ES09757018.8T 2008-06-05 2009-06-05 Dúplex oligonucleotídicos que comprenden nucleótidos de tipo ADN y de tipo ARN y usos de los mismos Active ES2643576T3 (es)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US5918608P 2008-06-05 2008-06-05
US59186P 2008-06-05
CA002635187A CA2635187A1 (en) 2008-06-05 2008-06-17 Oligonucleotide duplexes and uses thereof
CA2635187 2008-06-17
WOPCT/CA2008/002259 2008-12-19
PCT/CA2008/002259 WO2009076775A1 (en) 2007-12-19 2008-12-19 Immune response modulation and uses thereof
PCT/CA2009/000789 WO2009146556A1 (en) 2008-06-05 2009-06-05 Oligonucleotide duplexes comprising dna-like and rna-like nucleotides and uses thereof

Publications (1)

Publication Number Publication Date
ES2643576T3 true ES2643576T3 (es) 2017-11-23

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ID=41397419

Family Applications (1)

Application Number Title Priority Date Filing Date
ES09757018.8T Active ES2643576T3 (es) 2008-06-05 2009-06-05 Dúplex oligonucleotídicos que comprenden nucleótidos de tipo ADN y de tipo ARN y usos de los mismos

Country Status (6)

Country Link
US (2) US9090649B2 (OSRAM)
EP (1) EP2294195B1 (OSRAM)
JP (1) JP5684116B2 (OSRAM)
CA (2) CA2635187A1 (OSRAM)
ES (1) ES2643576T3 (OSRAM)
WO (1) WO2009146556A1 (OSRAM)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2007283022B2 (en) 2006-08-08 2011-07-28 Rheinische Friedrich-Wilhelms-Universitat Bonn Structure and use of 5' phosphate oligonucleotides
WO2009141146A1 (en) 2008-05-21 2009-11-26 Gunther Hartmann 5' triphosphate oligonucleotide with blunt end and uses thereof
CA2635187A1 (en) 2008-06-05 2009-12-05 The Royal Institution For The Advancement Of Learning/Mcgill University Oligonucleotide duplexes and uses thereof
WO2012065143A1 (en) 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP2508530A1 (en) 2011-03-28 2012-10-10 Rheinische Friedrich-Wilhelms-Universität Bonn Purification of triphosphorylated oligonucleotides using capture tags
DK2756080T3 (da) 2011-09-14 2019-05-20 Translate Bio Ma Inc Multimeriske oligonukleotidforbindelser
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
AU2013262700A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating hemoglobin gene family expression
CN104540947A (zh) 2012-05-16 2015-04-22 Rana医疗有限公司 用于调节smn基因家族表达的组合物和方法
JP2015518713A (ja) 2012-05-16 2015-07-06 ラナ セラピューティクス インコーポレイテッド Utrn発現を調節するための組成物及び方法
CN104583401A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节atp2a2表达的组合物和方法
CA2884608C (en) 2012-09-14 2025-05-20 Translate Bio Ma, Inc. MULTIMERIC OLIGONUCLEOTIDE COMPOUNDS
EP2712870A1 (en) 2012-09-27 2014-04-02 Rheinische Friedrich-Wilhelms-Universität Bonn Novel RIG-I ligands and methods for producing them
JP2016534035A (ja) 2013-10-04 2016-11-04 ラナ セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症を治療するための組成物及び方法
EP3212824A4 (en) 2014-10-30 2018-08-08 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
US10758558B2 (en) 2015-02-13 2020-09-01 Translate Bio Ma, Inc. Hybrid oligonucleotides and uses thereof
EP3271460A4 (en) 2015-03-17 2019-03-13 The General Hospital Corporation RNA INTERACTOM OF POLYCOMB REPRESSIVE COMPLEX 1 (PRC1)
EP4013428A4 (en) * 2019-08-15 2023-08-09 Ionis Pharmaceuticals, Inc. MODIFIED OLIGOMER COMPOUNDS AND USES THEREOF

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
US6336859B2 (en) * 1993-03-31 2002-01-08 Progressive Games, Inc. Method for progressive jackpot gaming
US7084125B2 (en) * 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US6639059B1 (en) * 1999-03-24 2003-10-28 Exiqon A/S Synthesis of [2.2.1]bicyclo nucleosides
US6734291B2 (en) * 1999-03-24 2004-05-11 Exiqon A/S Synthesis of [2.2.1]bicyclo nucleosides
AU776362B2 (en) * 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
US6083482A (en) * 1999-05-11 2000-07-04 Icn Pharmaceuticals, Inc. Conformationally locked nucleosides and oligonucleotides
US6338859B1 (en) 2000-06-29 2002-01-15 Labopharm Inc. Polymeric micelle compositions
US20020132788A1 (en) * 2000-11-06 2002-09-19 David Lewis Inhibition of gene expression by delivery of small interfering RNA to post-embryonic animal cells in vivo
US20020173478A1 (en) * 2000-11-14 2002-11-21 The Trustees Of The University Of Pennsylvania Post-transcriptional gene silencing by RNAi in mammalian cells
EP1386004A4 (en) * 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
US6780428B2 (en) * 2001-06-08 2004-08-24 Labopharm, Inc. Unimolecular polymeric micelles with an ionizable inner core
US7094810B2 (en) * 2001-06-08 2006-08-22 Labopharm, Inc. pH-sensitive block copolymers for pharmaceutical compositions
US6939564B2 (en) * 2001-06-08 2005-09-06 Labopharm, Inc. Water-soluble stabilized self-assembled polyelectrolytes
US7018655B2 (en) * 2002-03-18 2006-03-28 Labopharm, Inc. Amphiphilic diblock, triblock and star-block copolymers and their pharmaceutical compositions
US6780324B2 (en) * 2002-03-18 2004-08-24 Labopharm, Inc. Preparation of sterile stabilized nanodispersions
IL166546A0 (en) * 2002-08-05 2006-01-15 Atugen Ag Further novel forms of interfering ran molecules
ES2332178T5 (es) 2003-03-21 2014-02-04 Santaris Pharma A/S Análogos de ARN pequeños de interferencia (ARNpi)
US7262253B2 (en) * 2003-12-02 2007-08-28 Labopharm, Inc. Process for the preparation of amphiphilic poly (N-vinyl-2-pyrrolidone) block copolymers
US7468431B2 (en) * 2004-01-22 2008-12-23 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 expression
US20060198891A1 (en) * 2004-11-29 2006-09-07 Francois Ravenelle Solid formulations of liquid biologically active agents
US20090306177A1 (en) * 2005-09-16 2009-12-10 Coley Pharmaceutical Gmbh Modulation of Immunostimulatory Properties of Short Interfering Ribonucleic Acid (Sirna) by Nucleotide Modification
EP1945267A2 (en) * 2005-10-28 2008-07-23 Topigen Pharmaceuticals Inc. Small interfering oligonucleotides comprising arabinose modified nucleotides
US20090069263A1 (en) * 2005-12-16 2009-03-12 Damha Masad J 4'-thioarabinonucleotide-containing oligonucleotides, compounds and methods for their preparation and uses thereof
CN102124107A (zh) 2006-07-17 2011-07-13 瑟纳治疗公司 使用短干扰核酸(siNA)的RNA干扰介导的前蛋白转化酶枯草杆菌蛋白酶Kexin9(PCSK9)基因表达的抑制
CA2699184A1 (en) * 2006-09-22 2008-03-27 Labopharm Inc. Compositions and methods for ph targeted drug delivery
CA2635187A1 (en) 2008-06-05 2009-12-05 The Royal Institution For The Advancement Of Learning/Mcgill University Oligonucleotide duplexes and uses thereof

Also Published As

Publication number Publication date
WO2009146556A1 (en) 2009-12-10
US9090649B2 (en) 2015-07-28
EP2294195B1 (en) 2017-07-19
US20110077286A1 (en) 2011-03-31
US9719091B2 (en) 2017-08-01
CA2764456C (en) 2020-09-15
EP2294195A1 (en) 2011-03-16
EP2294195A4 (en) 2012-12-19
JP5684116B2 (ja) 2015-03-11
US20160145613A1 (en) 2016-05-26
CA2764456A1 (en) 2009-12-10
CA2635187A1 (en) 2009-12-05
JP2011521652A (ja) 2011-07-28

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