CA3001853A1 - Nucleic acid based tia-1 inhibitors - Google Patents

Nucleic acid based tia-1 inhibitors Download PDF

Info

Publication number
CA3001853A1
CA3001853A1 CA3001853A CA3001853A CA3001853A1 CA 3001853 A1 CA3001853 A1 CA 3001853A1 CA 3001853 A CA3001853 A CA 3001853A CA 3001853 A CA3001853 A CA 3001853A CA 3001853 A1 CA3001853 A1 CA 3001853A1
Authority
CA
Canada
Prior art keywords
nucleic acid
tau
acid based
tia1
tia
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
CA3001853A
Other languages
English (en)
French (fr)
Inventor
Glenn R. Larsen
Benjamin Wolozin
Tara VANDERWEYDE
Daniel J. Apicco
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Boston University
Aquinnah Pharmaceuticals Inc
Original Assignee
Boston University
Aquinnah 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 Boston University, Aquinnah Pharmaceuticals Inc filed Critical Boston University
Publication of CA3001853A1 publication Critical patent/CA3001853A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3001853A 2015-10-14 2016-10-14 Nucleic acid based tia-1 inhibitors Abandoned CA3001853A1 (en)

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 (1)

Publication Number Publication Date
CA3001853A1 true CA3001853A1 (en) 2017-04-20

Family

ID=57209905

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3001853A Abandoned CA3001853A1 (en) 2015-10-14 2016-10-14 Nucleic acid based tia-1 inhibitors

Country Status (5)

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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113196055A (zh) * 2018-10-15 2021-07-30 马克斯·普朗克科学促进协会 用于治疗疾病的化合物及其筛选方法

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210236532A1 (en) * 2018-01-29 2021-08-05 National University Corporation Tokyo Medical And Dental University Therapeutic agent for frontotemporal lobar degeneration, method for screening therapeutic agent for frontotemporal lobar degeneration and method for treating frontotemporal lobar degeneration
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
CN111500694B (zh) * 2019-01-31 2023-02-24 中国科学院脑科学与智能技术卓越创新中心 Baz2b基因作为靶点在缓解衰老中的应用
EP3921650A1 (en) 2019-02-08 2021-12-15 Dewpoint Therapeutics, Inc. Methods of characterizing condensate-associated characteristics of compounds and uses thereof
US20220332689A1 (en) * 2019-09-16 2022-10-20 The Trustees Of Columbia University In The City Of New York Identification of compounds that inhibit stress granule formation and tau aggregation by targeting tiai
KR20220084047A (ko) 2019-09-18 2022-06-21 듀포인트 테라퓨틱스, 인크. 응축물 관련 특이성에 대한 스크리닝 방법 및 이의 용도
CN113358617B (zh) * 2021-06-02 2024-03-01 重庆大学 胞外囊泡富集检测方法
CN119301453A (zh) * 2021-12-27 2025-01-10 Seq生物标志有限公司 用于诊断和/或治疗阿尔茨海默病的方法

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5798245A (en) * 1993-10-07 1998-08-25 Dana-Farber Cancer Institute TIA-1 binding proteins and isolated complementary DNA encoding the same
EP2305813A3 (en) * 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
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

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113196055A (zh) * 2018-10-15 2021-07-30 马克斯·普朗克科学促进协会 用于治疗疾病的化合物及其筛选方法
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

Also Published As

Publication number Publication date
EP3362564A1 (en) 2018-08-22
WO2017066657A1 (en) 2017-04-20
EP3362564B1 (en) 2020-07-01
US20190142860A1 (en) 2019-05-16
JP2018531046A (ja) 2018-10-25

Similar Documents

Publication Publication Date Title
EP3362564B1 (en) Nucleic acid based tia-1 inhibitors
JP7354342B2 (ja) タウ発現を調節するための組成物
US10888600B2 (en) Methods and agents for treating Alzheimer's disease
TWI787163B (zh) 用於減少tau表現之組合物及方法
JP2018531046A6 (ja) 核酸ベースのｔｉａ−１阻害剤
JP2020073480A (ja) 腱治癒を調節するための物質および方法
JP2019515894A (ja) 反復関連非ａｔｇ（ｒａｎ）翻訳を調節するためのｅｉｆ３の操作
TWI769197B (zh) 用於治療多囊腎病之組成物
JP2025016506A (ja) 二本鎖ｒｎａ及びその使用
US20140221464A1 (en) Compositions and Methods for Treating Skeletal Myopathy
JP2024532019A (ja) アルファ－１アンチトリプシン発現を阻害するための組成物及び方法
US12584133B2 (en) Antisense nucleic acid and use thereof
JPWO2018221649A1 (ja) Ａｐｃｓの発現を抑制する核酸
US20240368252A1 (en) Novel compositions for neuroprotection and axon regeneration
CA3175691A1 (en) Compositions and methods for inhibiting tdp-43 and fus aggregation
WO2015138960A2 (en) Molecular re-engineering of excitation-inhibition balance in memory circuits
US20260022377A1 (en) Polynucleotide compositions and methods for treatment of neurodegenerative diseases
JP2016540049A (ja) 肺動脈性高血圧症の治療のための材料及び方法
WO2026009728A1 (ja) アンチセンス核酸及びその使用
WO2025234407A1 (ja) アンチセンス核酸及びその使用
TW202523337A (zh) 靶向α突觸核蛋白表現之抑制性核酸
PARSI SEPIDEH PARSI Doctorat en neurobiologie Philosophiae doctor (Ph. D.)

Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20230104