CA2635187A1 - Oligonucleotide duplexes and uses thereof - Google Patents

Oligonucleotide duplexes and uses thereof Download PDF

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Publication number
CA2635187A1
CA2635187A1 CA002635187A CA2635187A CA2635187A1 CA 2635187 A1 CA2635187 A1 CA 2635187A1 CA 002635187 A CA002635187 A CA 002635187A CA 2635187 A CA2635187 A CA 2635187A CA 2635187 A1 CA2635187 A1 CA 2635187A1
Authority
CA
Canada
Prior art keywords
sirna
rna
oligonucleotide
oligonucleotides
nucleotides
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
CA002635187A
Other languages
English (en)
French (fr)
Inventor
Masad J. Damha
Jonathan K. Watts
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.)
Paladin Labs Inc
Original Assignee
Royal Institution for the Advancement of Learning
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 Royal Institution for the Advancement of Learning filed Critical Royal Institution for the Advancement of Learning
Priority to ES09757018.8T priority Critical patent/ES2643576T3/es
Priority to PCT/CA2009/000789 priority patent/WO2009146556A1/en
Priority to EP09757018.8A priority patent/EP2294195B1/en
Priority to US12/996,362 priority patent/US9090649B2/en
Priority to JP2011511949A priority patent/JP5684116B2/ja
Priority to CA2764456A priority patent/CA2764456C/en
Publication of CA2635187A1 publication Critical patent/CA2635187A1/en
Priority to US14/810,148 priority patent/US9719091B2/en
Abandoned legal-status Critical Current

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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
    • 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
    • C12N15/111General 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
    • 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
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • 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
    • 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
    • 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
    • C12N2320/53Methods 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)
CA002635187A 2008-06-05 2008-06-17 Oligonucleotide duplexes and uses thereof Abandoned CA2635187A1 (en)

Priority Applications (7)

Application Number Priority Date Filing Date Title
ES09757018.8T 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
PCT/CA2009/000789 WO2009146556A1 (en) 2008-06-05 2009-06-05 Oligonucleotide duplexes comprising dna-like and rna-like nucleotides and uses thereof
EP09757018.8A EP2294195B1 (en) 2008-06-05 2009-06-05 Oligonucleotide duplexes comprising dna-like and rna-like nucleotides and uses thereof
US12/996,362 US9090649B2 (en) 2008-06-05 2009-06-05 Oligonucleotide duplexes comprising DNA-like and RNA-like nucleotides and uses thereof
JP2011511949A JP5684116B2 (ja) 2008-06-05 2009-06-05 Dna様ヌクレオチドおよびrna様ヌクレオチドを含むオリゴヌクレオチド二重鎖およびその使用
CA2764456A CA2764456C (en) 2008-06-05 2009-06-05 Oligonucleotide duplexes comprising dna-like and rna-like nucleotides and uses thereof
US14/810,148 US9719091B2 (en) 2008-06-05 2015-07-27 Oligonucleotide duplexes comprising DNA-like and RNA-like nucleotides and uses thereof

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US5918608P 2008-06-05 2008-06-05
US61/059,186 2008-06-05

Publications (1)

Publication Number Publication Date
CA2635187A1 true CA2635187A1 (en) 2009-12-05

Family

ID=41397419

Family Applications (2)

Application Number Title Priority Date Filing Date
CA002635187A Abandoned CA2635187A1 (en) 2008-06-05 2008-06-17 Oligonucleotide duplexes and uses thereof
CA2764456A Active CA2764456C (en) 2008-06-05 2009-06-05 Oligonucleotide duplexes comprising dna-like and rna-like nucleotides and uses thereof

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA2764456A Active CA2764456C (en) 2008-06-05 2009-06-05 Oligonucleotide duplexes comprising dna-like and rna-like nucleotides and uses thereof

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)

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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

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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
ES2643576T3 (es) 2017-11-23
CA2764456A1 (en) 2009-12-10
JP2011521652A (ja) 2011-07-28

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Effective date: 20140617