EA202090687A1 - РНКи-АГЕНТЫ И КОМПОЗИЦИИ ДЛЯ ИНГИБИРОВАНИЯ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (APOC3) - Google Patents

РНКи-АГЕНТЫ И КОМПОЗИЦИИ ДЛЯ ИНГИБИРОВАНИЯ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (APOC3)

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EA202090687A1
EA202090687A1 EA202090687A EA202090687A EA202090687A1 EA 202090687 A1 EA202090687 A1 EA 202090687A1 EA 202090687 A EA202090687 A EA 202090687A EA 202090687 A EA202090687 A EA 202090687A EA 202090687 A1 EA202090687 A1 EA 202090687A1
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Prior art keywords
apoc3
rnai agents
iii
agents
compositions
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EA202090687A
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English (en)
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Чжэнь Ли
Жуй Чжу
Тао Пэй
Стивен Каннер
Со Вонг
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Эрроухед Фармасьютикалс, Инк.
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Priority claimed from PCT/US2018/050248 external-priority patent/WO2019051402A1/en
Publication of EA202090687A1 publication Critical patent/EA202090687A1/ru

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Abstract

Настоящее изобретение относится к РНКи-агентам, например агентам на основе интерференций с двухцепочечной РНК, способным ингибировать экспрессию генов аполипопротеина C-III (также называемого APOC3, apoC-III, APOC-III и APO C-III), и композициям, которые включают APOC3 РНКи-агенты. APOC3 РНКи-агенты, описанные в настоящем документе, могут быть конъюгированы со специфическими лигандами, включая лиганды, которые включают N-ацетилгалактозамин, для облегчения доставки в клетки, включая гепатоциты. Также описаны фармацевтические композиции, которые включают один или более APOC3 РНКи-агентов, необязательно с одним или более дополнительными терапевтическими средствами. Доставка APOC3 РНКи-агентов in vivo обеспечивает ингибирование экспрессии гена APOC3 и может привести к снижению уровня триглицеридов и/или холестерина у субъекта. APOC3 РНКи-агенты могут применяться в способах лечения заболеваний и расстройств, связанных с APOC3, включая гипертриглицеридемию, сердечно-сосудистые заболевания и другие метаболические расстройства и заболевания.
EA202090687A 2018-08-21 2018-09-10 РНКи-АГЕНТЫ И КОМПОЗИЦИИ ДЛЯ ИНГИБИРОВАНИЯ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (APOC3) EA202090687A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862720434P 2018-08-21 2018-08-21
PCT/US2018/050248 WO2019051402A1 (en) 2017-09-11 2018-09-10 RNA INTERFERENCE AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF APOLIPOPROTEIN C-III (APOC3)

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EA202090687A1 true EA202090687A1 (ru) 2020-06-09

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EA202090687A EA202090687A1 (ru) 2018-08-21 2018-09-10 РНКи-АГЕНТЫ И КОМПОЗИЦИИ ДЛЯ ИНГИБИРОВАНИЯ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (APOC3)

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