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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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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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    • C—CHEMISTRY; METALLURGY
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    • 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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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
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  • Wood Science & Technology (AREA)
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  • Physics & Mathematics (AREA)
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  • Communicable Diseases (AREA)
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  • Neurosurgery (AREA)
  • Neurology (AREA)
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  • Otolaryngology (AREA)
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  • Pain & Pain Management (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (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 (https=)
EP (1) EP1406667B1 (https=)
JP (1) JP4903984B2 (https=)
AT (1) ATE386548T1 (https=)
AU (1) AU2002319033B2 (https=)
CA (1) CA2451738C (https=)
DE (1) DE60225141T2 (https=)
DK (1) DK1406667T3 (https=)
ES (1) ES2301659T3 (https=)
MX (1) MXPA04000162A (https=)
NZ (1) NZ530360A (https=)
PT (1) PT1406667E (https=)
WO (1) WO2003004511A2 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
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NZ554723A (en) * 2004-10-29 2009-12-24 Topigen Pharmaceuticals Inc Antisense oligonucleotides against CCR3 chemokine receptor
AU2007282224B2 (en) 2006-08-11 2013-08-29 Vico Therapeutics B.V. Methods and means for treating DNA repeat instability associated genetic disorders
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting 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
US9301871B2 (en) 2009-02-26 2016-04-05 Advanced Cooling Therapy, Inc. Devices and methods for controlling patient temperature
DK2401023T3 (en) * 2009-02-26 2016-06-06 Advanced Cooling Therapy Inc Devices for controlling patient temperature
US9622909B2 (en) 2009-02-26 2017-04-18 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
EP2678431A4 (en) * 2011-02-24 2015-07-15 Pharmaxis Ltd OLIGONUCLEOTIDHEMMER WITH A CHIMERIC MAIN CHAIN AND 2-AMINO-2'-DEOXYADENOSINE
CN112251436A (zh) 2012-01-27 2021-01-22 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
SMT201700339T1 (it) 2012-04-23 2017-09-07 Biomarin Tech Bv Oligonucleotidi di modulazione dell'rna con caratteristiche migliorate per il trattamento dei disturbi neuromuscolari
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
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US5872242A (en) 1992-10-05 1999-02-16 Isis Pharmaceuticals, Inc. Antisense oligonucleotide inhibition of ras
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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
ATE346918T1 (de) * 1998-06-19 2006-12-15 Univ Mcgill Auf beta-arabinose, und dessen analogen, basierte antisense oligonukleotide
EP1102786A4 (en) * 1998-08-03 2002-03-06 Univ East Carolina AGENT COMPRISING ANTISENSE OLIGONUCLEOTIDES WITH LOW ADENOSINE CONTENT, COMPOSITION, KIT AND TREATMENTS
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WO2000062736A2 (en) 1999-04-06 2000-10-26 East Carolina University 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
EP1406667A2 (en) 2004-04-14
US20100286235A1 (en) 2010-11-11
US20050032723A1 (en) 2005-02-10
JP4903984B2 (ja) 2012-03-28
WO2003004511A3 (en) 2003-07-10
JP2004532900A (ja) 2004-10-28
US7745420B2 (en) 2010-06-29
EP1406667B1 (en) 2008-02-20
ES2301659T3 (es) 2008-07-01
AU2002319033B2 (en) 2008-01-10
DE60225141T2 (de) 2009-03-05
CA2451738A1 (en) 2003-01-16
DK1406667T3 (da) 2008-06-16
NZ530360A (en) 2006-01-27
ATE386548T1 (de) 2008-03-15
MXPA04000162A (es) 2006-05-22
PT1406667E (pt) 2008-05-23
DE60225141D1 (de) 2008-04-03
WO2003004511A2 (en) 2003-01-16

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