AU2003213090B2 - RNA interference mediated treatment of alzheimer's disease using short interfering nucleic acid ( siNA) - Google Patents

RNA interference mediated treatment of alzheimer's disease using short interfering nucleic acid ( siNA) Download PDF

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Publication number
AU2003213090B2
AU2003213090B2 AU2003213090A AU2003213090A AU2003213090B2 AU 2003213090 B2 AU2003213090 B2 AU 2003213090B2 AU 2003213090 A AU2003213090 A AU 2003213090A AU 2003213090 A AU2003213090 A AU 2003213090A AU 2003213090 B2 AU2003213090 B2 AU 2003213090B2
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sina
nucleotides
sina molecule
molecule
rna
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AU2003213090A
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AU2003213090A1 (en
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Leonid Beigelman
James Mcswiggen
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Sirna Therapeutics Inc
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Sirna Therapeutics Inc
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Assigned to SIRNA THERAPEUTICS, INC. reassignment SIRNA THERAPEUTICS, INC. Amend patent request/document other than specification (104) Assignors: RIBOZYME PHARMACEUTICALS, INC.
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    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/1137—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07H21/02—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 ribosyl as saccharide radical
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/332—Abasic residue
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Neurology (AREA)
  • Hospice & Palliative Care (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2003213090A 2002-02-20 2003-02-18 RNA interference mediated treatment of alzheimer's disease using short interfering nucleic acid ( siNA) Ceased AU2003213090B2 (en)

Applications Claiming Priority (17)

Application Number Priority Date Filing Date Title
US35858002P 2002-02-20 2002-02-20
US60/358,580 2002-02-20
US36312402P 2002-03-11 2002-03-11
US60/363,124 2002-03-11
US38678202P 2002-06-06 2002-06-06
US60/386,782 2002-06-06
US10/205,309 US20030190635A1 (en) 2002-02-20 2002-07-25 RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
US10/205,309 2002-07-25
US40678402P 2002-08-29 2002-08-29
US60/406,784 2002-08-29
US40837802P 2002-09-05 2002-09-05
US60/408,378 2002-09-05
US40929302P 2002-09-09 2002-09-09
US60/409,293 2002-09-09
US44012903P 2003-01-15 2003-01-15
US60/440,129 2003-01-15
PCT/US2003/004710 WO2003070895A2 (en) 2002-02-20 2003-02-18 RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER'S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA)

Publications (2)

Publication Number Publication Date
AU2003213090A1 AU2003213090A1 (en) 2003-09-09
AU2003213090B2 true AU2003213090B2 (en) 2007-06-07

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AU2003213090A Ceased AU2003213090B2 (en) 2002-02-20 2003-02-18 RNA interference mediated treatment of alzheimer's disease using short interfering nucleic acid ( siNA)

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US (1) US20030190635A1 (enExample)
EP (1) EP1423404B1 (enExample)
JP (2) JP2005517432A (enExample)
AT (1) ATE521620T1 (enExample)
AU (1) AU2003213090B2 (enExample)
CA (1) CA2455506A1 (enExample)
GB (1) GB2396155B (enExample)
WO (1) WO2003070895A2 (enExample)

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WO2003070895A2 (en) 2003-08-28
EP1423404A2 (en) 2004-06-02
JP2007300926A (ja) 2007-11-22
US20030190635A1 (en) 2003-10-09
JP2005517432A (ja) 2005-06-16
GB0404907D0 (en) 2004-04-07
GB2396155B (en) 2005-09-14
EP1423404A4 (en) 2004-09-01
EP1423404B1 (en) 2011-08-24
CA2455506A1 (en) 2003-08-28
AU2003213090A1 (en) 2003-09-09
ATE521620T1 (de) 2011-09-15
WO2003070895A3 (en) 2004-01-15

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