PL3210633T3 - Kompozycje i ich zastosowania ukierunkowane na huntingtynę - Google Patents

Kompozycje i ich zastosowania ukierunkowane na huntingtynę

Info

Publication number
PL3210633T3
PL3210633T3 PL17151157T PL17151157T PL3210633T3 PL 3210633 T3 PL3210633 T3 PL 3210633T3 PL 17151157 T PL17151157 T PL 17151157T PL 17151157 T PL17151157 T PL 17151157T PL 3210633 T3 PL3210633 T3 PL 3210633T3
Authority
PL
Poland
Prior art keywords
compositions
targeting huntingtin
uses targeting
huntingtin
targeting
Prior art date
Application number
PL17151157T
Other languages
English (en)
Polish (pl)
Inventor
Susan M. Freier
Original Assignee
Ionis Pharmaceuticals, Inc.
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 Ionis Pharmaceuticals, Inc. filed Critical Ionis Pharmaceuticals, Inc.
Publication of PL3210633T3 publication Critical patent/PL3210633T3/pl

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    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/30—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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • 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)
PL17151157T 2006-01-26 2007-01-26 Kompozycje i ich zastosowania ukierunkowane na huntingtynę PL3210633T3 (pl)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US76295406P 2006-01-26 2006-01-26
US83629006P 2006-08-07 2006-08-07
EP10193707.6A EP2422819B1 (en) 2006-01-26 2007-01-26 Compositions and their uses directed to 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
PCT/US2007/002215 WO2007089611A2 (en) 2006-01-26 2007-01-26 Compositions and their uses directed to huntingtin

Publications (1)

Publication Number Publication Date
PL3210633T3 true PL3210633T3 (pl) 2019-12-31

Family

ID=38164262

Family Applications (2)

Application Number Title Priority Date Filing Date
PL17151157T PL3210633T3 (pl) 2006-01-26 2007-01-26 Kompozycje i ich zastosowania ukierunkowane na huntingtynę
PL09075505T PL2161038T3 (pl) 2006-01-26 2007-01-26 Kompozycje i ich zastosowanie ukierunkowane na huntingtynę

Family Applications After (1)

Application Number Title Priority Date Filing Date
PL09075505T PL2161038T3 (pl) 2006-01-26 2007-01-26 Kompozycje i ich zastosowanie ukierunkowane na huntingtynę

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) PL3210633T3 (enExample)
PT (1) PT2161038E (enExample)
SI (1) SI2161038T1 (enExample)
WO (2) WO2007089611A2 (enExample)

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US12509681B2 (en) 2025-12-30
US8952145B2 (en) 2015-02-10
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AU2017204772A1 (en) 2017-07-27
JP2020096624A (ja) 2020-06-25
JP2009524431A (ja) 2009-07-02
ES2744098T3 (es) 2020-02-21
AU2007210038A1 (en) 2007-08-09
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JP5425474B2 (ja) 2014-02-26
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US20100069472A1 (en) 2010-03-18
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US20080039418A1 (en) 2008-02-14
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EP1991677A2 (en) 2008-11-19
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US11761000B2 (en) 2023-09-19
PL2161038T3 (pl) 2014-06-30
EP2422819A2 (en) 2012-02-29
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AU2017204772B2 (en) 2019-07-11
US20210017522A1 (en) 2021-01-21
WO2007089611A2 (en) 2007-08-09
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US20130281684A1 (en) 2013-10-24
EP2422819A3 (en) 2012-06-27
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US20130189782A1 (en) 2013-07-25
SI2161038T1 (sl) 2014-05-30
EP3210633A3 (en) 2017-11-01
WO2007089584A3 (en) 2007-11-29
US11072794B2 (en) 2021-07-27
CA2638908A1 (en) 2007-08-09
US20180273945A1 (en) 2018-09-27
US20220042013A1 (en) 2022-02-10
AU2019237389A1 (en) 2019-10-17
PT2161038E (pt) 2014-03-10
US20070299027A1 (en) 2007-12-27

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