JP6684707B2 - タウ発現を調節するための組成物 - Google Patents

タウ発現を調節するための組成物 Download PDF

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JP6684707B2
JP6684707B2 JP2016527154A JP2016527154A JP6684707B2 JP 6684707 B2 JP6684707 B2 JP 6684707B2 JP 2016527154 A JP2016527154 A JP 2016527154A JP 2016527154 A JP2016527154 A JP 2016527154A JP 6684707 B2 JP6684707 B2 JP 6684707B2
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compound
modified
tau
antisense
certain embodiments
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JP2016528891A (ja
JP2016528891A5 (enExample
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コルダシェヴィッチ，ホリー
スウェイジ，エリック・イー
フレアー，スーザン・エム
ブイ，フィン−ホア
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Ionis Pharmaceuticals Inc
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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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/34—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
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
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  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Steroid Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
  • Thiazole And Isothizaole Compounds (AREA)
  • Pyridine Compounds (AREA)
JP2016527154A 2013-07-19 2014-07-21 タウ発現を調節するための組成物 Active JP6684707B2 (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201361856551P 2013-07-19 2013-07-19
US61/856,551 2013-07-19
US201361879621P 2013-09-18 2013-09-18
US61/879,621 2013-09-18
US201361885371P 2013-10-01 2013-10-01
US61/885,371 2013-10-01
US201462014486P 2014-06-19 2014-06-19
US62/014,486 2014-06-19
PCT/US2014/047486 WO2015010135A2 (en) 2013-07-19 2014-07-21 Compositions for modulating tau expression

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2020057966A Division JP2020141670A (ja) 2013-07-19 2020-03-27 タウ発現を調節するための組成物

Publications (3)

Publication Number Publication Date
JP2016528891A JP2016528891A (ja) 2016-09-23
JP2016528891A5 JP2016528891A5 (enExample) 2017-08-31
JP6684707B2 true JP6684707B2 (ja) 2020-04-22

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JP2016527154A Active JP6684707B2 (ja) 2013-07-19 2014-07-21 タウ発現を調節するための組成物
JP2020057966A Withdrawn JP2020141670A (ja) 2013-07-19 2020-03-27 タウ発現を調節するための組成物
JP2022069532A Active JP7354342B2 (ja) 2013-07-19 2022-04-20 タウ発現を調節するための組成物
JP2023152191A Pending JP2023164642A (ja) 2013-07-19 2023-09-20 タウ発現を調節するための組成物
JP2026009884A Pending JP2026069566A (ja) 2013-07-19 2026-01-23 タウ発現を調節するための組成物

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JP2020057966A Withdrawn JP2020141670A (ja) 2013-07-19 2020-03-27 タウ発現を調節するための組成物
JP2022069532A Active JP7354342B2 (ja) 2013-07-19 2022-04-20 タウ発現を調節するための組成物
JP2023152191A Pending JP2023164642A (ja) 2013-07-19 2023-09-20 タウ発現を調節するための組成物
JP2026009884A Pending JP2026069566A (ja) 2013-07-19 2026-01-23 タウ発現を調節するための組成物

Country Status (25)

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US (4) US9683235B2 (enExample)
EP (2) EP3910060A1 (enExample)
JP (5) JP6684707B2 (enExample)
KR (6) KR102526525B1 (enExample)
CN (3) CN108064227B (enExample)
AU (2) AU2014290395B2 (enExample)
BR (1) BR112016000654B1 (enExample)
CA (3) CA3149282C (enExample)
CY (1) CY1124292T1 (enExample)
DK (1) DK3022217T3 (enExample)
ES (1) ES2871533T3 (enExample)
HR (1) HRP20211051T1 (enExample)
HU (1) HUE055144T2 (enExample)
IL (4) IL302146A (enExample)
LT (1) LT3022217T (enExample)
MX (2) MX379535B (enExample)
NZ (2) NZ755605A (enExample)
PL (1) PL3022217T3 (enExample)
PT (1) PT3022217T (enExample)
RS (1) RS62054B1 (enExample)
RU (1) RU2735551C2 (enExample)
SI (1) SI3022217T1 (enExample)
SM (1) SMT202100371T1 (enExample)
TW (4) TWI702046B (enExample)
WO (1) WO2015010135A2 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
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JP2020141670A (ja) * 2013-07-19 2020-09-10 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． タウ発現を調節するための組成物

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