JP7776420B2 - Ａｔａｘｉｎ ３発現を調節するための化合物、方法及び医薬組成物 - Google Patents

Ａｔａｘｉｎ ３発現を調節するための化合物、方法及び医薬組成物

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Publication number
JP7776420B2
JP7776420B2 JP2022522177A JP2022522177A JP7776420B2 JP 7776420 B2 JP7776420 B2 JP 7776420B2 JP 2022522177 A JP2022522177 A JP 2022522177A JP 2022522177 A JP2022522177 A JP 2022522177A JP 7776420 B2 JP7776420 B2 JP 7776420B2
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Prior art keywords
seq
modified
sequence
alna
atxn3
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Japanese (ja)
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JPWO2021230286A1 (https=
Inventor
浩昭 澤本
拓也 肥後
俊平 村田
友 荒木
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Tanabe Pharma Corp
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Mitsubishi Tanabe Pharma Corp
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Priority to JP2025193446A priority Critical patent/JP2026027438A/ja
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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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
JP2022522177A 2020-05-12 2021-05-12 Ａｔａｘｉｎ ３発現を調節するための化合物、方法及び医薬組成物 Active JP7776420B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025193446A JP2026027438A (ja) 2020-05-12 2025-11-13 Ａｔａｘｉｎ ３発現を調節するための化合物、方法及び医薬組成物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2020083864 2020-05-12
JP2020083864 2020-05-12
PCT/JP2021/018040 WO2021230286A1 (ja) 2020-05-12 2021-05-12 Ａｔａｘｉｎ ３発現を調節するための化合物、方法及び医薬組成物

Related Child Applications (1)

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Publications (2)

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JPWO2021230286A1 JPWO2021230286A1 (https=) 2021-11-18
JP7776420B2 true JP7776420B2 (ja) 2025-11-26

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JP2025193446A Pending JP2026027438A (ja) 2020-05-12 2025-11-13 Ａｔａｘｉｎ ３発現を調節するための化合物、方法及び医薬組成物

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Country Status (5)

Country Link
US (1) US20230227824A1 (https=)
EP (1) EP4151237A4 (https=)
JP (2) JP7776420B2 (https=)
CN (1) CN115667513A (https=)
WO (1) WO2021230286A1 (https=)

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JP2015511821A (ja) 2012-03-12 2015-04-23 ロシュ・イノベーション・センター・コペンハーゲン・アクティーゼルスカブＲｏｃｈｅ Ｉｎｎｏｖａｔｉｏｎ Ｃｅｎｔｅｒ Ｃｏｐｅｎｈａｇｅｎ Ａ／Ｓ Ａｔｘｎ３発現の調節のための組成物および方法
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JP2019534009A (ja) 2016-11-10 2019-11-28 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． Ａｔｘｎ３発現の低減のための化合物及び方法
WO2020100826A1 (ja) 2018-11-12 2020-05-22 田辺三菱製薬株式会社 架橋型人工核酸ａｌｎａ

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Also Published As

Publication number Publication date
WO2021230286A1 (ja) 2021-11-18
JP2026027438A (ja) 2026-02-18
US20230227824A1 (en) 2023-07-20
JPWO2021230286A1 (https=) 2021-11-18
EP4151237A4 (en) 2024-08-14
CN115667513A (zh) 2023-01-31
EP4151237A1 (en) 2023-03-22

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