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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Publication number
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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JPWO2020160163A5 (enExample
JP2022518929A (ja
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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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Ionis Pharmaceuticals Inc
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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
    • C12N15/1138—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 against receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • C—CHEMISTRY; METALLURGY
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    • 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

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  • Health & Medical Sciences (AREA)
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  • 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)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • 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

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JP2022518929A JP2022518929A (ja) 2022-03-17
JPWO2020160163A5 JPWO2020160163A5 (enExample) 2023-02-02
JP7557469B2 true JP7557469B2 (ja) 2024-09-27

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

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019140231A1 (en) 2018-01-12 2019-07-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
AU2019208011B2 (en) 2018-01-12 2025-06-05 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
MY206230A (en) * 2018-12-19 2024-12-05 Alnylam Pharmaceuticals Inc Amyloid precursor protein (app) rnai agent compositions and methods of use thereof
WO2020206115A2 (en) * 2019-04-03 2020-10-08 Bristol-Myers Squibb Company Angptl2 antisense oligonucleotides and uses thereof
JP2022544587A (ja) 2019-08-15 2022-10-19 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 結合修飾オリゴマー化合物及びその使用
UY39344A (es) 2020-07-28 2022-02-25 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de app
EP4284390A4 (en) * 2021-01-29 2025-10-29 Alnylam Pharmaceuticals Inc ARNI COMPOSITIONS AND METHODS FOR SILENCER OF AMYLOID PRECURSOR PROTEIN (APP)
AU2022364838A1 (en) * 2021-10-15 2024-04-11 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
TW202511488A (zh) * 2023-08-31 2025-03-16 大陸商上海拓界生物醫藥科技有限公司 靶向ＡＰＰ的ＲＮＡｉ劑及其醫藥用途
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

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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WO1993013114A1 (en) * 1991-12-24 1993-07-08 Isis Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR MODULATING β-AMYLOID
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
WO2019169243A1 (en) * 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of 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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JP2025000693A (ja) 2025-01-07
JP2022518929A (ja) 2022-03-17
EP3918073A1 (en) 2021-12-08
UY38562A (es) 2020-08-31
TW202043472A (zh) 2020-12-01
EP3918073A4 (en) 2023-11-22
US20220380773A1 (en) 2022-12-01
WO2020160163A1 (en) 2020-08-06

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