WO2007089611A3 - Compositions and their uses directed to huntingtin - Google Patents

Compositions and their uses directed to huntingtin Download PDF

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Publication number
WO2007089611A3
WO2007089611A3 PCT/US2007/002215 US2007002215W WO2007089611A3 WO 2007089611 A3 WO2007089611 A3 WO 2007089611A3 US 2007002215 W US2007002215 W US 2007002215W WO 2007089611 A3 WO2007089611 A3 WO 2007089611A3
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WO
WIPO (PCT)
Prior art keywords
huntingtin
compositions
methods
disease
preventing
Prior art date
Application number
PCT/US2007/002215
Other languages
French (fr)
Other versions
WO2007089611A2 (en
Inventor
Susan M Freier
Original Assignee
Isis Pharmaceuticals Inc
Susan M Freier
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 Isis Pharmaceuticals Inc, Susan M Freier filed Critical Isis Pharmaceuticals Inc
Priority to AU2007210038A priority Critical patent/AU2007210038B2/en
Priority to CA2638908A priority patent/CA2638908C/en
Priority to PL17151157T priority patent/PL3210633T3/en
Priority to JP2008552447A priority patent/JP5425474B2/en
Priority to EP17151157.9A priority patent/EP3210633B1/en
Priority to EP07717061A priority patent/EP1991677A2/en
Publication of WO2007089611A2 publication Critical patent/WO2007089611A2/en
Publication of WO2007089611A3 publication Critical patent/WO2007089611A3/en
Priority to AU2013203297A priority patent/AU2013203297B2/en
Priority to AU2017204772A priority patent/AU2017204772B2/en
Priority to AU2019237389A priority patent/AU2019237389A1/en

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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
    • 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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    • C12N2310/00—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
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    • C12N2310/3341—5-Methylcytosine
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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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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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

Abstract

Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.
PCT/US2007/002215 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin WO2007089611A2 (en)

Priority Applications (9)

Application Number Priority Date Filing Date Title
AU2007210038A AU2007210038B2 (en) 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin
CA2638908A CA2638908C (en) 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin
PL17151157T PL3210633T3 (en) 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin
JP2008552447A JP5425474B2 (en) 2006-01-26 2007-01-26 Composition and use thereof for huntingtin
EP17151157.9A EP3210633B1 (en) 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin
EP07717061A EP1991677A2 (en) 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin
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 (4)

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

Publications (2)

Publication Number Publication Date
WO2007089611A2 WO2007089611A2 (en) 2007-08-09
WO2007089611A3 true WO2007089611A3 (en) 2008-01-10

Family

ID=38164262

Family Applications (2)

Application Number Title Priority Date Filing Date
PCT/US2007/002215 WO2007089611A2 (en) 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin
PCT/US2007/002171 WO2007089584A2 (en) 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin

Family Applications After (1)

Application Number Title Priority Date Filing Date
PCT/US2007/002171 WO2007089584A2 (en) 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin

Country Status (12)

Country Link
US (14) US20070299027A1 (en)
EP (5) EP3210633B1 (en)
JP (5) JP5425474B2 (en)
AU (4) AU2007210038B2 (en)
CA (1) CA2638908C (en)
DK (1) DK2161038T3 (en)
ES (2) ES2453380T3 (en)
HK (1) HK1141449A1 (en)
PL (2) PL2161038T3 (en)
PT (1) PT2161038E (en)
SI (1) SI2161038T1 (en)
WO (2) WO2007089611A2 (en)

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US9528109B2 (en) 2007-10-26 2016-12-27 Biomarin Technologies B.V. Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA

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