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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    • C12N15/09—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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    • C12N15/09—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
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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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    • C12N2310/334—Modified C
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
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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)
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  • 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)
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  • 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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SG11202006528XA (en) 2018-01-12 2020-08-28 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CN120310791A (zh) 2018-01-12 2025-07-15 百时美施贵宝公司 靶向α-突触核蛋白的反义寡核苷酸及其用途
US11732260B2 (en) 2018-03-02 2023-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-β precursor protein
AU2019405783B2 (en) * 2018-12-19 2026-03-26 Alnylam Pharmaceuticals, Inc. Amyloid precursor protein (APP) RNAi agent compositions and methods of use thereof
EP3947680A2 (en) * 2019-04-03 2022-02-09 Bristol-Myers Squibb Company Angptl2 antisense oligonucleotides and uses thereof
KR20220062517A (ko) 2019-08-15 2022-05-17 아이오니스 파마수티컬즈, 인코포레이티드 결합 변형된 올리고머 화합물 및 이의 용도
CA3189922A1 (en) 2020-07-28 2022-02-03 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
US20240117349A1 (en) * 2021-01-29 2024-04-11 Alnylam Pharmaceuticals, Inc. iRNA COMPOSITIONS AND METHODS FOR SILENCING AMYLOID PRECURSOR PROTEIN (APP)
CN118369427A (zh) * 2021-10-15 2024-07-19 阿尔尼拉姆医药品有限公司 肝外递送irna组合物及其使用方法
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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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
GB0324854D0 (en) * 2003-10-24 2003-11-26 Expresson Biosystems Ltd App/ena antisense
WO2009105572A2 (en) 2008-02-19 2009-08-27 Edunn Biotechonology Inc. Antisense modulation of amyloid beta protein expression
US20100190807A1 (en) * 2008-08-26 2010-07-29 Senex Biotechnology, Inc. CDKI Pathway inhibitors and uses thereof to regulate expression to TAU protein
WO2011130742A2 (en) * 2010-04-16 2011-10-20 Wisconsin Alumni Research Foundation Diagnostic evaluation of antibody responses to commonly recognized prostate cancer-associated antigens
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
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Publication number Publication date
EP3918073A1 (en) 2021-12-08
JP7557469B2 (ja) 2024-09-27
JP2022518929A (ja) 2022-03-17
WO2020160163A1 (en) 2020-08-06
US20220380773A1 (en) 2022-12-01
JP2025000693A (ja) 2025-01-07
EP3918073A4 (en) 2023-11-22
UY38562A (es) 2020-08-31

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