UA118649C2 - ДВОЛАНЦЮГОВИЙ ЗАСІБ ДЛЯ PHKі ТА ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ТРАНСТИРЕТИНУ (TTR) - Google Patents

ДВОЛАНЦЮГОВИЙ ЗАСІБ ДЛЯ PHKі ТА ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ТРАНСТИРЕТИНУ (TTR)

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UA118649C2
UA118649C2 UAA201406837A UAA201406837A UA118649C2 UA 118649 C2 UA118649 C2 UA 118649C2 UA A201406837 A UAA201406837 A UA A201406837A UA A201406837 A UAA201406837 A UA A201406837A UA 118649 C2 UA118649 C2 UA 118649C2
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ttr
methods
transthyretin
compositions
rnai agents
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UAA201406837A
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English (en)
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Каллантхоттатхіл Дж. Раджив
Трейсі Циммерманн
Мутхіах Манохаран
Мартін Майєр
Сатіянараяна Кучіманчі
Клаус Харіссе
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Елнілем Фармасьютікалз, Інк.
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Application filed by Елнілем Фармасьютікалз, Інк. filed Critical Елнілем Фармасьютікалз, Інк.
Publication of UA118649C2 publication Critical patent/UA118649C2/uk

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Abstract

Винахід стосується дволанцюгового засобу для РНКі, у якого певні нуклеотиди олігонуклеотидної послідовності модифіковані, зокрема містять 2'-O-метилмодифікацію або 2'-фтормодифікацію та смисловий ланцюг кон’югований щонайменше із одним лігандом, де ліганд являє собою GalNAc3. Винахід також стосується фармацевтичної композиції для інгібування експресії транстиретину (TTR), яка містить ефективну кількість вказаного засобу, способу інгібування експресії транстиретину у клітині, способу лікування асоційованого із TTR захворювання у індивідуума та набору для проведення таких способів.
UAA201406837A 2011-11-18 2012-11-16 ДВОЛАНЦЮГОВИЙ ЗАСІБ ДЛЯ PHKі ТА ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ТРАНСТИРЕТИНУ (TTR) UA118649C2 (uk)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201161561710P 2011-11-18 2011-11-18
US201261615618P 2012-03-26 2012-03-26
US201261680098P 2012-08-06 2012-08-06
PCT/US2012/065691 WO2013075035A1 (en) 2011-11-18 2012-11-16 Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases

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UA118649C2 true UA118649C2 (uk) 2019-02-25

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UAA201406837A UA118649C2 (uk) 2011-11-18 2012-11-16 ДВОЛАНЦЮГОВИЙ ЗАСІБ ДЛЯ PHKі ТА ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ТРАНСТИРЕТИНУ (TTR)

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US (5) US9399775B2 (uk)
EP (4) EP3913056A1 (uk)
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