EA202090717A1 - RNAi MEANS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF ANGIOPOETIN-LIKE PROTEIN 3 (ANGPTL3) AND METHODS OF APPLICATION - Google Patents

RNAi MEANS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF ANGIOPOETIN-LIKE PROTEIN 3 (ANGPTL3) AND METHODS OF APPLICATION

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Publication number
EA202090717A1
EA202090717A1 EA202090717A EA202090717A EA202090717A1 EA 202090717 A1 EA202090717 A1 EA 202090717A1 EA 202090717 A EA202090717 A EA 202090717A EA 202090717 A EA202090717 A EA 202090717A EA 202090717 A1 EA202090717 A1 EA 202090717A1
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EA
Eurasian Patent Office
Prior art keywords
angptl3
rnai agents
compositions
methods
rnai
Prior art date
Application number
EA202090717A
Other languages
Russian (ru)
Inventor
Чжэнь Ли
Жуй Чжу
Со Вонг
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Эрроухед Фармасьютикалс, Инк.
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Application filed by Эрроухед Фармасьютикалс, Инк. filed Critical Эрроухед Фармасьютикалс, Инк.
Priority claimed from PCT/US2018/050848 external-priority patent/WO2019055633A1/en
Publication of EA202090717A1 publication Critical patent/EA202090717A1/en

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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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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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
    • C12N2310/35—Nature of the modification
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  • Health & Medical Sciences (AREA)
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  • Life Sciences & Earth Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Настоящее изобретение относится к средствам РНКи, например двухцепочечным средствам РНКи, которые способны ингибировать экспрессию гена ангиопоэтин-подобный 3 (также называемого ANGPTL3, ANGPL3, ангиопоэтин-подобным белком 3), и композициям, которые включают средств РНКи к ANGPTL3. Также раскрыты способы применения средств РНКи к ANGPTL3 и их композиций. Средства РНКи к ANGPTL3, раскрытые в настоящем документе, могут быть конъюгированы с направляющими лигандами для облегчения доставки в клетки, в том числе в гепатоциты. Описаны фармацевтические композиции, которые включают одно или более средств РНКи к ANGPTL3, необязательно с одним или более дополнительными терапевтическими средствами. Доставка средств РНКи к ANGPTL3 in vivo обеспечивает ингибирование экспрессии гена ANGPTL3 и может приводить к снижению уровней триглицеридов и/или холестерина у субъекта. Средства РНКи могут применяться в способах лечения ANGPTL3-ассоциированных заболеваний и нарушений, включая кардиометаболические заболевания, такие как гипертриглицеридемия и гиперлипидемия.The present invention relates to RNAi agents, for example, double-stranded RNAi agents that are capable of inhibiting the expression of an angiopoietin-like 3 gene (also called ANGPTL3, ANGPL3, angiopoietin-like protein 3), and compositions that include ANGPTL3 RNAi agents. Methods of applying RNAi agents to ANGPTL3 and their compositions are also disclosed. ANGPTL3 RNAi agents disclosed herein can be conjugated to guide ligands to facilitate delivery to cells, including hepatocytes. Pharmaceutical compositions are described that include one or more ANGPTL3 RNAs, optionally with one or more additional therapeutic agents. In vivo delivery of RNAi to ANGPTL3 provides inhibition of the expression of the ANGPTL3 gene and can lead to a decrease in the level of triglycerides and / or cholesterol in a subject. RNAi agents can be used in methods of treating ANGPTL3-associated diseases and disorders, including cardiometabolic diseases such as hypertriglyceridemia and hyperlipidemia.

EA202090717A 2018-07-07 2018-09-13 RNAi MEANS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF ANGIOPOETIN-LIKE PROTEIN 3 (ANGPTL3) AND METHODS OF APPLICATION EA202090717A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862694976P 2018-07-07 2018-07-07
PCT/US2018/050848 WO2019055633A1 (en) 2017-09-14 2018-09-13 Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use

Publications (1)

Publication Number Publication Date
EA202090717A1 true EA202090717A1 (en) 2020-06-11

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
EA202090717A EA202090717A1 (en) 2018-07-07 2018-09-13 RNAi MEANS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF ANGIOPOETIN-LIKE PROTEIN 3 (ANGPTL3) AND METHODS OF APPLICATION

Country Status (1)

Country Link
EA (1) EA202090717A1 (en)

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