JP7557469B2 - Appの発現を低減するための化合物及び方法 - Google Patents

Appの発現を低減するための化合物及び方法 Download PDF

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JP7557469B2
JP7557469B2 JP2021544144A JP2021544144A JP7557469B2 JP 7557469 B2 JP7557469 B2 JP 7557469B2 JP 2021544144 A JP2021544144 A JP 2021544144A JP 2021544144 A JP2021544144 A JP 2021544144A JP 7557469 B2 JP7557469 B2 JP 7557469B2
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modified
oligonucleotide
nucleosides
certain embodiments
rnai
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JP2022518929A5 (https=
JP2022518929A (ja
JPWO2020160163A5 (https=
Inventor
フレアー,スーザン・エム
ブイ,フィン・ホア
コルダシェヴィッチ,ホリー
スウェイジ,エリック・イー
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Ionis Pharmaceuticals Inc
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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 against receptors or cell surface proteins
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/3222'-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/34Spatial arrangement of the modifications
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Wood Science & Technology (AREA)
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  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021544144A 2019-01-29 2020-01-29 Appの発現を低減するための化合物及び方法 Active JP7557469B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024158813A JP2025000693A (ja) 2019-01-29 2024-09-13 Appの発現を低減するための化合物及び方法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962798353P 2019-01-29 2019-01-29
US62/798,353 2019-01-29
US201962841169P 2019-04-30 2019-04-30
US62/841,169 2019-04-30
US201962915764P 2019-10-16 2019-10-16
US62/915,764 2019-10-16
PCT/US2020/015701 WO2020160163A1 (en) 2019-01-29 2020-01-29 Compounds and methods for reducing app expression

Related Child Applications (1)

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JP2024158813A Division JP2025000693A (ja) 2019-01-29 2024-09-13 Appの発現を低減するための化合物及び方法

Publications (4)

Publication Number Publication Date
JP2022518929A JP2022518929A (ja) 2022-03-17
JP2022518929A5 JP2022518929A5 (https=) 2023-02-02
JPWO2020160163A5 JPWO2020160163A5 (https=) 2023-02-02
JP7557469B2 true JP7557469B2 (ja) 2024-09-27

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JP2021544144A Active JP7557469B2 (ja) 2019-01-29 2020-01-29 Appの発現を低減するための化合物及び方法
JP2024158813A Pending JP2025000693A (ja) 2019-01-29 2024-09-13 Appの発現を低減するための化合物及び方法

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US (1) US20220380773A1 (https=)
EP (1) EP3918073A4 (https=)
JP (2) JP7557469B2 (https=)
TW (1) TW202043472A (https=)
UY (1) UY38562A (https=)
WO (1) WO2020160163A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EA202091693A1 (ru) 2018-01-12 2021-04-14 Бристол-Маерс Сквибб Компани Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
BR112020013994A2 (pt) 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso que direcionam alfa-sinucleína e seus usos
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
TW202039844A (zh) * 2018-12-19 2020-11-01 美商阿尼拉製藥公司 類澱粉前驅蛋白(APP)RNAi劑組成物及其使用方法
KR20210149107A (ko) * 2019-04-03 2021-12-08 브리스톨-마이어스 스큅 컴퍼니 Angptl2 안티센스 올리고뉴클레오티드 및 그의 용도
EP4013767A4 (en) 2019-08-15 2023-10-25 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds and uses thereof
AU2021315992A1 (en) 2020-07-28 2023-02-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
EP4284390A4 (en) * 2021-01-29 2025-10-29 Alnylam Pharmaceuticals Inc ARNI COMPOSITIONS AND METHODS FOR SILENCER OF AMYLOID PRECURSOR PROTEIN (APP)
MX2024004437A (es) * 2021-10-15 2024-07-01 Alnylam Pharmaceuticals Inc Composiciones de inhibidor de la angiotensina-receptor neprilisina (arni) de administracion extrahepatica y metodos de uso de las mismas.
WO2025045195A1 (zh) * 2023-08-31 2025-03-06 上海拓界生物医药科技有限公司 靶向APP的RNAi剂及其医药用途
WO2025228429A1 (zh) * 2024-04-30 2025-11-06 北京安龙生物医药有限公司 靶向淀粉样前体蛋白基因的寡核苷酸及其用途
WO2025261396A1 (zh) * 2024-06-18 2025-12-26 艾码生物科技(南京)有限公司 靶向app的rna干扰方法、核酸及其应用

Citations (6)

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WO2005042777A2 (en) 2003-10-24 2005-05-12 Expresson Biosystems Limited App/ena antisense
JP2011514158A (ja) 2008-02-19 2011-05-06 エダン・バイオテクノロジー・インコーポレイテッド アミロイドベータタンパク質発現のアンチセンス調節
JP2016521556A (ja) 2013-06-07 2016-07-25 ラナ セラピューティクス インコーポレイテッド Foxp3発現を調節するための組成物及び方法
JP2016531570A (ja) 2013-08-16 2016-10-13 ラナ セラピューティクス インコーポレイテッド ユークロマチン領域を標的とするオリゴヌクレオチド
JP2016533717A (ja) 2013-10-11 2016-11-04 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. C9orf72発現を調節するための組成物
US20170044526A1 (en) 2014-04-28 2017-02-16 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds

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US20030232435A1 (en) * 2002-06-14 2003-12-18 Isis Pharmaceuticals Inc. Antisense modulation of amyloid beta protein precursor expression
US20100190807A1 (en) * 2008-08-26 2010-07-29 Senex Biotechnology, Inc. CDKI Pathway inhibitors and uses thereof to regulate expression to TAU protein
US20110301052A1 (en) * 2010-04-16 2011-12-08 Mcneel Douglas G Diagnostic evaluation of antibody responses to commonly recognized prostate cancer-associated antigens
EP3759127A4 (en) * 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005042777A2 (en) 2003-10-24 2005-05-12 Expresson Biosystems Limited App/ena antisense
JP2011514158A (ja) 2008-02-19 2011-05-06 エダン・バイオテクノロジー・インコーポレイテッド アミロイドベータタンパク質発現のアンチセンス調節
JP2016521556A (ja) 2013-06-07 2016-07-25 ラナ セラピューティクス インコーポレイテッド Foxp3発現を調節するための組成物及び方法
JP2016531570A (ja) 2013-08-16 2016-10-13 ラナ セラピューティクス インコーポレイテッド ユークロマチン領域を標的とするオリゴヌクレオチド
JP2016533717A (ja) 2013-10-11 2016-11-04 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. C9orf72発現を調節するための組成物
US20170044526A1 (en) 2014-04-28 2017-02-16 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds

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Publication number Publication date
JP2022518929A (ja) 2022-03-17
UY38562A (es) 2020-08-31
WO2020160163A1 (en) 2020-08-06
EP3918073A4 (en) 2023-11-22
TW202043472A (zh) 2020-12-01
US20220380773A1 (en) 2022-12-01
JP2025000693A (ja) 2025-01-07
EP3918073A1 (en) 2021-12-08

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