JP2018531046A - 核酸ベースのｔｉａ−１阻害剤 - Google Patents

核酸ベースのｔｉａ−１阻害剤 Download PDF

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Publication number
JP2018531046A
JP2018531046A JP2018539237A JP2018539237A JP2018531046A JP 2018531046 A JP2018531046 A JP 2018531046A JP 2018539237 A JP2018539237 A JP 2018539237A JP 2018539237 A JP2018539237 A JP 2018539237A JP 2018531046 A JP2018531046 A JP 2018531046A
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JP
Japan
Prior art keywords
tau
nucleic acid
tia1
tia
based inhibitor
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Pending
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JP2018539237A
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English (en)
Japanese (ja)
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JP2018531046A6 (ja
JP2018531046A5 (https=
Inventor
グレン アール． ラーセン，
グレン アール． ラーセン，
ベンジャミン ウォロジン，
ベンジャミン ウォロジン，
タラ バンダーウェイド，
タラ バンダーウェイド，
ダニエル ジェイ． アピッコ，
ダニエル ジェイ． アピッコ，
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Boston University
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Boston University
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Application filed by Boston University filed Critical Boston University
Publication of JP2018531046A publication Critical patent/JP2018531046A/ja
Publication of JP2018531046A6 publication Critical patent/JP2018531046A6/ja
Publication of JP2018531046A5 publication Critical patent/JP2018531046A5/ja
Pending legal-status Critical Current

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    • 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
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2018539237A 2015-10-14 2016-10-14 核酸ベースのｔｉａ−１阻害剤 Pending JP2018531046A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562241535P 2015-10-14 2015-10-14
US62/241,535 2015-10-14
US201662331795P 2016-05-04 2016-05-04
US62/331,795 2016-05-04
PCT/US2016/057164 WO2017066657A1 (en) 2015-10-14 2016-10-14 Nucleic acid based tia-1 inhibitors

Publications (3)

Publication Number Publication Date
JP2018531046A true JP2018531046A (ja) 2018-10-25
JP2018531046A6 JP2018531046A6 (ja) 2018-12-13
JP2018531046A5 JP2018531046A5 (https=) 2019-11-21

Family

ID=57209905

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2018539237A Pending JP2018531046A (ja) 2015-10-14 2016-10-14 核酸ベースのｔｉａ−１阻害剤

Country Status (5)

Country Link
US (1) US20190142860A1 (https=)
EP (1) EP3362564B1 (https=)
JP (1) JP2018531046A (https=)
CA (1) CA3001853A1 (https=)
WO (1) WO2017066657A1 (https=)

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022511317A (ja) * 2018-10-15 2022-01-31 マックスプランク－ゲセルシャフト・ツール・フェーデルング・デル・ヴィッセンシャフテン・エー・ファウ 疾患の治療のための化合物およびこれをスクリーニングする方法
JP2022519122A (ja) * 2019-01-31 2022-03-18 中国科学院脳科学与智能技術卓越創新中心 老化を軽減するための標的としてのｂａｚ２ｂ遺伝子の使用
US12174198B2 (en) 2019-02-08 2024-12-24 Dewpoint Therapeutics, Inc. Methods of characterizing condensate-associated characteristics of compounds and uses thereof
US12422437B2 (en) 2019-09-18 2025-09-23 Dewpoint Therapeutics, Inc. Methods of screening for condensate-associated specificity and uses thereof

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019146805A1 (ja) * 2018-01-29 2019-08-01 国立大学法人東京医科歯科大学 前頭側頭葉変性症治療剤、前頭側頭葉変性症治療剤のスクリーニング方法、及び前頭側頭葉変性症の治療方法
JP2022512877A (ja) 2018-11-01 2022-02-07 エフ．ホフマン－ラ ロシュ アーゲー Ｔｉａ１を標的とするアンチセンスオリゴヌクレオチド
JP2022547606A (ja) * 2019-09-16 2022-11-14 ザ・トラスティーズ・オブ・コランビア・ユニバーシティー・イン・ザ・シティー・オブ・ニューヨーク Ｔｉａ１を標的としたストレス顆粒形成およびタウ凝集を抑制する化合物の同定
CN113358617B (zh) * 2021-06-02 2024-03-01 重庆大学 胞外囊泡富集检测方法
WO2023129531A2 (en) * 2021-12-27 2023-07-06 Seq Biomarque, Llc. Methods for diagnosing and/or treating alzheimer's disease

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WO2004045543A2 (en) * 2002-11-14 2004-06-03 Dharmacon, Inc. Functional and hyperfunctional sirna

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US5798245A (en) * 1993-10-07 1998-08-25 Dana-Farber Cancer Institute TIA-1 binding proteins and isolated complementary DNA encoding the same
US7217807B2 (en) * 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
JP2014501097A (ja) * 2009-07-06 2014-01-20 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 生物由来物質の産生を高めるための組成物及び方法
CA2752947A1 (en) * 2011-06-30 2012-12-30 Benjamin J. Blencowe Foxp1 splice variants and methods and uses thereof

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2004045543A2 (en) * 2002-11-14 2004-06-03 Dharmacon, Inc. Functional and hyperfunctional sirna

Non-Patent Citations (5)

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Title
A. ALBORNOZ: "THE STRESS GRANULE COMPONENT TIA-1 BINDS TICK-BORNE ENCEPHALITIS VIRUS RNA AND IS RECRUITED 以下備考", JOURNAL OF VIROLOGY, vol. VOL: 88, NR: 12, JPN5018007298, 15 June 2014 (2014-06-15), US, pages 6611 - 6622, ISSN: 0004560585 *
ALZHEIMER'S AND DEMENTIA, vol. vol.11, no.7, Supp.1, JPN6020041510, July 2015 (2015-07-01), pages 300 - 301, ISSN: 0004560589 *
BRUNELLO, C.A: "ADPD5-0748 - REGULATION OF TAU LOCALIZATION TO STRESS GRANULES", NEURODEGENERATIVE DISEASES, vol. 15, no. 1, JPN5018007300, 19 March 2015 (2015-03-19), pages 1508, ISSN: 0004560587 *
OSCAR. APARICIO: "ADENOVIRUS VA RNA-DERIVED MIRNAS TARGET CELLULAR GENES INVOLVED IN CELL GROWTH, GENE 以下備考", NUCLEIC ACIDS RESEARCH, vol. VOL: 38, NR: 3, JPN5018007299, 2010, pages 750 - 763, ISSN: 0004560586 *
VANDERWEYDE TARA: "ROLE OF STRESS GRANULES AND RNA-BINDING PROTEINS IN NEURODEGENERATION: A MINI-REVIEW", GERONTOLOGY, vol. 59, no. 6, JPN5018007301, 2013, pages 524 - 533, ISSN: 0004560588 *

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022511317A (ja) * 2018-10-15 2022-01-31 マックスプランク－ゲセルシャフト・ツール・フェーデルング・デル・ヴィッセンシャフテン・エー・ファウ 疾患の治療のための化合物およびこれをスクリーニングする方法
JP7654540B2 (ja) 2018-10-15 2025-04-01 マックスプランク－ゲセルシャフト・ツール・フェーデルング・デル・ヴィッセンシャフテン・エー・ファウ 疾患の治療のための化合物およびこれをスクリーニングする方法
US12422427B2 (en) 2018-10-15 2025-09-23 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Compounds for treatment of diseases and methods of screening therefor
JP2022519122A (ja) * 2019-01-31 2022-03-18 中国科学院脳科学与智能技術卓越創新中心 老化を軽減するための標的としてのｂａｚ２ｂ遺伝子の使用
US12174198B2 (en) 2019-02-08 2024-12-24 Dewpoint Therapeutics, Inc. Methods of characterizing condensate-associated characteristics of compounds and uses thereof
US12422437B2 (en) 2019-09-18 2025-09-23 Dewpoint Therapeutics, Inc. Methods of screening for condensate-associated specificity and uses thereof

Also Published As

Publication number Publication date
EP3362564A1 (en) 2018-08-22
WO2017066657A1 (en) 2017-04-20
CA3001853A1 (en) 2017-04-20
EP3362564B1 (en) 2020-07-01
US20190142860A1 (en) 2019-05-16

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