JP2024539596A - タウ発現を低減するための化合物及び方法 - Google Patents

タウ発現を低減するための化合物及び方法 Download PDF

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Publication number
JP2024539596A
JP2024539596A JP2024520945A JP2024520945A JP2024539596A JP 2024539596 A JP2024539596 A JP 2024539596A JP 2024520945 A JP2024520945 A JP 2024520945A JP 2024520945 A JP2024520945 A JP 2024520945A JP 2024539596 A JP2024539596 A JP 2024539596A
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JP
Japan
Prior art keywords
oligomeric
modified
modified oligonucleotide
certain embodiments
oligomeric compound
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Pending
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JP2024520945A
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English (en)
Japanese (ja)
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JP2024539596A5 (enExample
Inventor
フレアー，スーザン・エム
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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Publication of JP2024539596A publication Critical patent/JP2024539596A/ja
Publication of JP2024539596A5 publication Critical patent/JP2024539596A5/ja
Pending legal-status Critical Current

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    • 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
    • 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
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/32—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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/32—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
    • 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
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
JP2024520945A 2021-10-07 2022-10-07 タウ発現を低減するための化合物及び方法 Pending JP2024539596A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163253516P 2021-10-07 2021-10-07
US63/253,516 2021-10-07
US202263317743P 2022-03-08 2022-03-08
US63/317,743 2022-03-08
PCT/US2022/077740 WO2023064707A1 (en) 2021-10-07 2022-10-07 Compounds and methods for reducing tau expression

Publications (2)

Publication Number Publication Date
JP2024539596A true JP2024539596A (ja) 2024-10-29
JP2024539596A5 JP2024539596A5 (enExample) 2025-10-14

Family

ID=85988949

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024520945A Pending JP2024539596A (ja) 2021-10-07 2022-10-07 タウ発現を低減するための化合物及び方法

Country Status (4)

Country Link
US (1) US20250340872A1 (enExample)
EP (1) EP4413138A4 (enExample)
JP (1) JP2024539596A (enExample)
WO (1) WO2023064707A1 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2024007056A (es) 2021-12-13 2024-06-24 Lilly Co Eli Agentes de interferencia de arn de mapt.
EP4754256A1 (en) * 2023-08-02 2026-06-10 Consejo Nacional de Investigaciones Científicas Y Técnicas (CONICET) Micrornas designed against the microtubule-associated protein tau for the treatment of tauopathies
AU2024321134A1 (en) 2023-08-10 2026-02-19 Harness Therapeutics Limited Functional nucleic acid
GB202401412D0 (en) 2024-02-02 2024-03-20 Harness Therapeutics Ltd Functional nucleic acid
WO2025212467A1 (en) 2024-03-31 2025-10-09 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of microtubule associated protein tau (mapt), compositions thereof, and methods of use
WO2026002277A1 (zh) * 2024-06-28 2026-01-02 艾码生物科技(南京)有限公司 靶向Tau的RNA干扰方法、核酸及其应用
WO2026010722A1 (en) * 2024-07-03 2026-01-08 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2026021537A1 (en) * 2024-07-26 2026-01-29 Qilu Pharmaceutical Co., Ltd. iRNA COMPOSITION AND METHOD OF USING SAME
CN118979036B (zh) * 2024-08-01 2025-08-19 北京尧景基因技术有限公司 抑制APP基因表达的siRNA及其缀合物和应用
WO2026026982A1 (zh) * 2024-08-02 2026-02-05 成都先衍生物技术有限公司 一种靶向MAPT基因表达的siRNA及其缀合物和用途
CN121780524A (zh) * 2024-08-02 2026-04-03 北京尧景基因技术有限公司 抑制MAPT基因表达的siRNA及其缀合物和应用
WO2026067655A1 (zh) * 2024-09-26 2026-04-02 大睿生物医药科技(上海)有限公司 调控TAU表达的dsRNA分子
WO2026154123A1 (en) * 2025-01-17 2026-07-23 Aviadobio Limited Compositions and methods for treating neurological diseases

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040241854A1 (en) * 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
TWI702046B (zh) * 2013-07-19 2020-08-21 美商Ionis製藥公司 用於調節τ蛋白表現之組合物
CA2975525A1 (en) * 2015-02-04 2016-08-11 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
MD3519572T2 (ro) * 2016-09-29 2024-03-31 Biogen Ma Inc Compuși și procedee de reducere a expresiei Tau
US11827880B2 (en) * 2019-12-02 2023-11-28 Shape Therapeutics Inc. Therapeutic editing
JP2023518439A (ja) * 2020-03-18 2023-05-01 ユニバーシティー オブ マサチューセッツ Ｍａｐｔ調節のためのオリゴヌクレオチド
JP2023521604A (ja) * 2020-03-30 2023-05-25 アルナイラム ファーマシューティカルズ， インコーポレイテッド 微小管関連タンパク質タウ（ＭＡＰＴ）ｉＲＮＡ剤組成物およびその使用方法

Also Published As

Publication number Publication date
EP4413138A1 (en) 2024-08-14
WO2023064707A1 (en) 2023-04-20
EP4413138A4 (en) 2026-04-01
US20250340872A1 (en) 2025-11-06

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