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DK2906696T3 - Fremgangsmåder til modulering af c9orf72-ekspression - Google Patents

Fremgangsmåder til modulering af c9orf72-ekspression Download PDF

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Publication number
DK2906696T3
DK2906696T3 DK13846313T DK13846313T DK2906696T3 DK 2906696 T3 DK2906696 T3 DK 2906696T3 DK 13846313 T DK13846313 T DK 13846313T DK 13846313 T DK13846313 T DK 13846313T DK 2906696 T3 DK2906696 T3 DK 2906696T3
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DK
Denmark
Prior art keywords
methods
c9orf72 expression
modulating
modulating c9orf72
expression
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DK13846313T
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English (en)
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DK2906696T4 (da
Inventor
Frank Rigo
Don W Cleveland
Clotilde Lagier-Tourenne
John M Ravits
C Frank Bennett
Susan M Freier
Michael W Baughn
Original Assignee
Ionis Pharmaceuticals Inc
Ludwig Inst For Cancer Res Ltd
Univ California
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Application filed by Ionis Pharmaceuticals Inc, Ludwig Inst For Cancer Res Ltd, Univ California filed Critical Ionis Pharmaceuticals Inc
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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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    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181Peptide nucleic acid, PNA
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
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    • C12N2310/3233Morpholino-type ring
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    • C12N2310/33415-Methylcytosine
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===

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DK13846313.8T 2012-10-15 2013-10-15 Fremgangsmåder til modulering af c9orf72-ekspression DK2906696T4 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261714139P 2012-10-15 2012-10-15
PCT/US2013/065067 WO2014062686A1 (en) 2012-10-15 2013-10-15 Methods for modulating c9orf72 expression

Publications (2)

Publication Number Publication Date
DK2906696T3 true DK2906696T3 (da) 2019-11-25
DK2906696T4 DK2906696T4 (da) 2023-02-27

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DK13846313.8T DK2906696T4 (da) 2012-10-15 2013-10-15 Fremgangsmåder til modulering af c9orf72-ekspression

Country Status (7)

Country Link
US (1) US9963699B2 (da)
EP (1) EP2906696B2 (da)
DK (1) DK2906696T4 (da)
ES (1) ES2762326T5 (da)
FI (1) FI2906696T4 (da)
PT (1) PT2906696T (da)
WO (1) WO2014062686A1 (da)

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ES2762326T3 (es) 2020-05-22
EP2906696B2 (en) 2022-12-14
FI2906696T4 (fi) 2023-03-18
US9963699B2 (en) 2018-05-08
US20150267197A1 (en) 2015-09-24
WO2014062686A1 (en) 2014-04-24
EP2906696A1 (en) 2015-08-19
DK2906696T4 (da) 2023-02-27
PT2906696T (pt) 2019-12-16
ES2762326T5 (es) 2023-04-27
EP2906696A4 (en) 2016-06-08
EP2906696B1 (en) 2019-10-09

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