AU2007210038B2 - Compositions and their uses directed to huntingtin - Google Patents

Compositions and their uses directed to huntingtin Download PDF

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Publication number
AU2007210038B2
AU2007210038B2 AU2007210038A AU2007210038A AU2007210038B2 AU 2007210038 B2 AU2007210038 B2 AU 2007210038B2 AU 2007210038 A AU2007210038 A AU 2007210038A AU 2007210038 A AU2007210038 A AU 2007210038A AU 2007210038 B2 AU2007210038 B2 AU 2007210038B2
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Australia
Prior art keywords
huntingtin
antisense
antisense oligonucleotide
seq
oligonucleotide
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AU2007210038A
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AU2007210038A1 (en
Inventor
Susan M. Freier
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of AU2007210038A1 publication Critical patent/AU2007210038A1/en
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Publication of AU2007210038B2 publication Critical patent/AU2007210038B2/en
Priority to AU2013203297A priority Critical patent/AU2013203297B2/en
Priority to AU2017204772A priority patent/AU2017204772B2/en
Assigned to IONIS PHARMACEUTICALS, INC. reassignment IONIS PHARMACEUTICALS, INC. Request to Amend Deed and Register Assignors: ISIS PHARMACEUTICALS, INC.
Priority to AU2019237389A priority patent/AU2019237389A1/en
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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
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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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
AU2007210038A 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin Active AU2007210038B2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
AU2013203297A AU2013203297B2 (en) 2006-01-26 2013-04-10 Compositions and their uses directed to huntingtin
AU2017204772A AU2017204772B2 (en) 2006-01-26 2017-07-12 Compositions and their uses directed to huntingtin
AU2019237389A AU2019237389A1 (en) 2006-01-26 2019-09-30 Compositions and their uses directed to huntingtin

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US76295406P 2006-01-26 2006-01-26
US60/762,954 2006-01-26
US83629006P 2006-08-07 2006-08-07
US60/836,290 2006-08-07
PCT/US2007/002215 WO2007089611A2 (en) 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2013203297A Division AU2013203297B2 (en) 2006-01-26 2013-04-10 Compositions and their uses directed to huntingtin

Publications (2)

Publication Number Publication Date
AU2007210038A1 AU2007210038A1 (en) 2007-08-09
AU2007210038B2 true AU2007210038B2 (en) 2013-01-10

Family

ID=38164262

Family Applications (4)

Application Number Title Priority Date Filing Date
AU2007210038A Active AU2007210038B2 (en) 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin
AU2013203297A Active AU2013203297B2 (en) 2006-01-26 2013-04-10 Compositions and their uses directed to huntingtin
AU2017204772A Active AU2017204772B2 (en) 2006-01-26 2017-07-12 Compositions and their uses directed to huntingtin
AU2019237389A Abandoned AU2019237389A1 (en) 2006-01-26 2019-09-30 Compositions and their uses directed to huntingtin

Family Applications After (3)

Application Number Title Priority Date Filing Date
AU2013203297A Active AU2013203297B2 (en) 2006-01-26 2013-04-10 Compositions and their uses directed to huntingtin
AU2017204772A Active AU2017204772B2 (en) 2006-01-26 2017-07-12 Compositions and their uses directed to huntingtin
AU2019237389A Abandoned AU2019237389A1 (en) 2006-01-26 2019-09-30 Compositions and their uses directed to huntingtin

Country Status (11)

Country Link
US (14) US20070299027A1 (enExample)
EP (5) EP2161038B1 (enExample)
JP (5) JP5425474B2 (enExample)
AU (4) AU2007210038B2 (enExample)
CA (1) CA2638908C (enExample)
DK (1) DK2161038T3 (enExample)
ES (2) ES2453380T3 (enExample)
PL (2) PL2161038T3 (enExample)
PT (1) PT2161038E (enExample)
SI (1) SI2161038T1 (enExample)
WO (2) WO2007089611A2 (enExample)

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