TW202043472A - 用於減少ａｐｐ表現之化合物及方法 - Google Patents

用於減少ａｐｐ表現之化合物及方法 Download PDF

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TW202043472A
TW202043472A TW109102899A TW109102899A TW202043472A TW 202043472 A TW202043472 A TW 202043472A TW 109102899 A TW109102899 A TW 109102899A TW 109102899 A TW109102899 A TW 109102899A TW 202043472 A TW202043472 A TW 202043472A
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Taiwan
Prior art keywords
modified
oligonucleotide
nucleobases
rnai
nucleosides
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TW109102899A
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English (en)
Chinese (zh)
Inventor
蘇珊 M 弗瑞爾
賀恩 荷亞 比伊
賀利 科達席微茲
艾瑞克 E 史瓦瑟
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美商Ionis製藥公司
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Publication of TW202043472A publication Critical patent/TW202043472A/zh

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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
    • 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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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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
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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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
    • 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

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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)
  • 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)
TW109102899A 2019-01-29 2020-01-30 用於減少ａｐｐ表現之化合物及方法 TW202043472A (zh)

Applications Claiming Priority (6)

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

Publications (1)

Publication Number Publication Date
TW202043472A true TW202043472A (zh) 2020-12-01

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TW109102899A TW202043472A (zh) 2019-01-29 2020-01-30 用於減少ａｐｐ表現之化合物及方法

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

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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

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CA2126451A1 (en) * 1991-12-24 1993-07-08 Brett P. Monia Compositions and methods for modulating .beta.-amyloid
US20030232435A1 (en) * 2002-06-14 2003-12-18 Isis Pharmaceuticals Inc. Antisense modulation of amyloid beta protein precursor expression
GB0324854D0 (en) * 2003-10-24 2003-11-26 Expresson Biosystems Ltd 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
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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
JP7557469B2 (ja) 2024-09-27
US20220380773A1 (en) 2022-12-01
JP2025000693A (ja) 2025-01-07
EP3918073A1 (en) 2021-12-08

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