CA2451738C - Methods for increasing in vivo efficacy of oligonucleotides and inhibiting inflammation in mammals using 2-amino-2'-deoxyadenosine - Google Patents

Methods for increasing in vivo efficacy of oligonucleotides and inhibiting inflammation in mammals using 2-amino-2'-deoxyadenosine Download PDF

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Publication number
CA2451738C
CA2451738C CA2451738A CA2451738A CA2451738C CA 2451738 C CA2451738 C CA 2451738C CA 2451738 A CA2451738 A CA 2451738A CA 2451738 A CA2451738 A CA 2451738A CA 2451738 C CA2451738 C CA 2451738C
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Prior art keywords
nucleic acid
oligonucleotide
acid molecule
adenosine
dap
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Expired - Fee Related
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CA2451738A
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English (en)
French (fr)
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CA2451738A1 (en
Inventor
Zoulfia Allakhverdi
Mustapha Allam
Paolo Renzi
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Pharmaxis Ltd
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Topigen Pharmaceuticals Inc
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
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    • A61P1/04—Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/02—Nasal agents, e.g. decongestants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/06—Antiasthmatics
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    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16—Purine radicals
    • C—CHEMISTRY; METALLURGY
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    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/23—Heterocyclic radicals containing two or more heterocyclic rings condensed among themselves or condensed with a common carbocyclic ring system, not provided for in groups C07H19/14 - C07H19/22
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    • 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
    • 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
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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
    • C12N15/1138—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified A

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  • Molecular Biology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Pulmonology (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Communicable Diseases (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Cardiology (AREA)
  • Pain & Pain Management (AREA)
  • Otolaryngology (AREA)
  • Ophthalmology & Optometry (AREA)
CA2451738A 2001-07-06 2002-07-08 Methods for increasing in vivo efficacy of oligonucleotides and inhibiting inflammation in mammals using 2-amino-2'-deoxyadenosine Expired - Fee Related CA2451738C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US30307101P 2001-07-06 2001-07-06
US60/303,071 2001-07-06
PCT/CA2002/001046 WO2003004511A2 (en) 2001-07-06 2002-07-08 Methods for increasing in vivo efficacy of oligonucleotides and inhibiting inflammation in mammals

Publications (2)

Publication Number Publication Date
CA2451738A1 CA2451738A1 (en) 2003-01-16
CA2451738C true CA2451738C (en) 2013-09-17

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CA2451738A Expired - Fee Related CA2451738C (en) 2001-07-06 2002-07-08 Methods for increasing in vivo efficacy of oligonucleotides and inhibiting inflammation in mammals using 2-amino-2'-deoxyadenosine

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US (2) US7745420B2 (enExample)
EP (1) EP1406667B1 (enExample)
JP (1) JP4903984B2 (enExample)
AT (1) ATE386548T1 (enExample)
AU (1) AU2002319033B2 (enExample)
CA (1) CA2451738C (enExample)
DE (1) DE60225141T2 (enExample)
DK (1) DK1406667T3 (enExample)
ES (1) ES2301659T3 (enExample)
MX (1) MXPA04000162A (enExample)
NZ (1) NZ530360A (enExample)
PT (1) PT1406667E (enExample)
WO (1) WO2003004511A2 (enExample)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006045202A1 (en) * 2004-10-29 2006-05-04 Topigen Pharmaceuticals Inc. Antisense oligonucleotides for treating allergy and neoplastic cell proliferation
ES2373246T3 (es) 2006-08-11 2012-02-01 Prosensa Technologies B.V. Oligonucleótidos monocatenarios complementarios de elementos repetitivos para el tratamiento de trastornos genéticos asociados a la inestabilidad de repeticiones de adn.
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
HUE028662T2 (en) 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
EP2285961A4 (en) * 2008-05-15 2012-10-17 Topigen Pharmaceuticals Inc OLIGONUCLEOTIDES FOR THE TREATMENT OF INFLAMMATION AND NEOPLASTIC CELL REPRODUCTION
WO2010048731A1 (en) * 2008-10-31 2010-05-06 Christopher John Ong Aminoacyl trna-synthetase inhibitors as broad-spectrum immunosuppressive agents
US9622909B2 (en) 2009-02-26 2017-04-18 Advanced Cooling Therapy, Inc. Devices and methods for controlling patient temperature
MY183870A (en) * 2009-02-26 2021-03-17 Advanced Cooling Therapy Llc Devices and methods for controlling patient temperature
US9301871B2 (en) 2009-02-26 2016-04-05 Advanced Cooling Therapy, Inc. Devices and methods for controlling patient temperature
US9326890B2 (en) 2009-02-26 2016-05-03 Advanced Cooling Therapy, Inc. Devices and methods for controlling patient temperature
SG192869A1 (en) * 2011-02-24 2013-09-30 Pharmaxis Ltd Oligonucleotide inhibitors with chimeric backbone and 2-amino-2'-deoxyadenosine
CN111893117B (zh) 2012-01-27 2024-06-04 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
US9272016B2 (en) * 2012-04-20 2016-03-01 University Of Iowa Research Foundation Methods to enhance RNAi oligonucleotide delivery to respiratory epithelial cells
HRP20171254T1 (hr) * 2012-04-23 2018-01-12 Biomarin Technologies B.V. Oligonukleotidi koji moduliraju rnk s poboljšanim karakteristikama za liječenje neuromišićnih poremećaja
FR3057774B1 (fr) * 2016-10-21 2025-11-07 Museum Nat Dhistoire Naturelle Derives de la purine pour leur utilisation dans le traitement ou la prevention de maladies dues a une mutation non-sens

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US3337530A (en) 1965-09-20 1967-08-22 Upjohn Co Dinucleoside 3', 5' -and 2', 5'-phosphates containing one 7-deazapurin riboside moiety
JPH01173818A (ja) * 1987-12-28 1989-07-10 Aisin Aw Co Ltd 車両用ナビゲーション装置
US5523205A (en) * 1988-08-02 1996-06-04 Institut Pasteur DNA probes specific for hemolytic listeria
US5646265A (en) * 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5112961A (en) * 1990-07-18 1992-05-12 Schering Corporation Dna encoding subunits of a high affinity gm-csf receptor
EP0509826A1 (en) * 1991-04-19 1992-10-21 Schering Corporation Subunit of the human interleukin 3 receptor
US5872242A (en) 1992-10-05 1999-02-16 Isis Pharmaceuticals, Inc. Antisense oligonucleotide inhibition of ras
GB9301000D0 (en) * 1993-01-20 1993-03-10 Glaxo Group Ltd Chemical compounds
US6077668A (en) * 1993-04-15 2000-06-20 University Of Rochester Highly sensitive multimeric nucleic acid probes
CA2235420A1 (en) * 1998-06-17 1999-12-17 Paolo Renzi Antisense oligonucleotides for treating or preventing atopic diseases and neoplastic cell proliferation
EP1088066B1 (en) * 1998-06-19 2006-11-29 McGILL UNIVERSITY Antisense oligonucleotide constructs based on beta-arabinofuranose and its analogues
CA2333901A1 (en) * 1998-08-03 2000-02-24 East Carolina University Low adenosine anti-sense oligonucleotide agent, composition, kit and treatments
US6175004B1 (en) * 1998-09-01 2001-01-16 Isis Pharmaceuticals, Inc. Process for the synthesis of oligonucleotides incorporating 2-aminoadenosine
IL140054A0 (en) 1999-04-06 2002-02-10 Univ East Carolina Low adenosine anti-sense oligonucleotide, compositions, kit and method for treatment of airway disorders associated with bronchoconstriction, lung inflammation, allergy (ies) and surfactant depletion

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Publication number Publication date
US7745420B2 (en) 2010-06-29
CA2451738A1 (en) 2003-01-16
MXPA04000162A (es) 2006-05-22
DE60225141D1 (de) 2008-04-03
ATE386548T1 (de) 2008-03-15
JP2004532900A (ja) 2004-10-28
ES2301659T3 (es) 2008-07-01
US20100286235A1 (en) 2010-11-11
DE60225141T2 (de) 2009-03-05
US20050032723A1 (en) 2005-02-10
AU2002319033B2 (en) 2008-01-10
JP4903984B2 (ja) 2012-03-28
DK1406667T3 (da) 2008-06-16
EP1406667A2 (en) 2004-04-14
WO2003004511A2 (en) 2003-01-16
EP1406667B1 (en) 2008-02-20
WO2003004511A3 (en) 2003-07-10
PT1406667E (pt) 2008-05-23
NZ530360A (en) 2006-01-27

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