RU2019101298A - Антисмысловые олигонуклеотиды для модулирования экспрессии htra1 - Google Patents

Антисмысловые олигонуклеотиды для модулирования экспрессии htra1 Download PDF

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RU2019101298A
RU2019101298A RU2019101298A RU2019101298A RU2019101298A RU 2019101298 A RU2019101298 A RU 2019101298A RU 2019101298 A RU2019101298 A RU 2019101298A RU 2019101298 A RU2019101298 A RU 2019101298A RU 2019101298 A RU2019101298 A RU 2019101298A
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antisense oligonucleotide
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Роберто ЯКОНЕ
Петер ХАГЕДОРН
Сюзанн КАММЛЕР
Сёрен Оттосен
Синдри ТРАУСТАСОН
Хайди ХУДЛБУШ
Люкке ПЕДЕРСЕН
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Ф. Хоффманн-Ля Рош Аг
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Claims (19)

1. Антисмысловой олигонуклеотид формулы: TsAsTststsascscstsgsgstsTsGsTsT (SEQ ID NO 143), где заглавная буква представляет нуклеозидное звено LNA, строчная буква представляет нуклеозидное звено ДНК, подстрочный символ s представляет фосфоротиоатную межнуклеозидную связь.
2. Антисмысловой олигонуклеотид по п. 1, где все нуклеозиды LNA представляют собой бета-D-окси нуклеозиды LNA.
3. Антисмысловой олигонуклеотид по п. 1, где антисмысловой олигонуклеотид имеет формулу:
Figure 00000001
4. Фармацевтически приемлемая соль антисмыслового олигонуклотида по любому из пп. 1-3.
5. Фармацевтически приемлемая соль по п. 4, где данная соль представляет собой натриевую соль.
6. Фармацевтически приемлемая соль по п. 4, где данная соль представляет собой калиевую соль.
7. Фармацевтическая композиция, содержащая антисмысловой олигонуклеотид по любому из пп. 1-3; и фармацевтически приемлемый разбавитель, носитель, соль и/или адъювант.
8. Фармацевтическая композиция по п. 7, где данная фармацевтическая композиция содержит фармацевтически приемлемый разбавитель.
9. Фармацевтическая композиция по п. 8, где фармацевтически приемлемый разбавитель представляет собой фосфатно-солевой буферный раствор.
10. Фармацевтическая композиция по любому из пп. 7-9, где олигонуклеотид находится в форме фармацевтически приемлемой соли.
11. Фармацевтическая композиция, содержащая фармацевтически приемлемую соль по любому из пп. 4-6; и фармацевтически приемлемый разбавитель, носитель, соль и/или адъювант.
12. Фармацевтическая композиция по любому из пп. 7-11, в которой фармацевтически приемлемая соль представляет собой натриевую соль или калиевую соль.
13. Антисмысловой олигонуклеотид по любому из пп. 1-3 или фармацевтически приемлемая соль по любому из пп. 4-6, фармацевтическая композиция по любому из пп. 7-12 для применения в медицине.
14. Антисмысловой олигонуклеотид по любому из пп. 1-3 или фармацевтически приемлемая соль по любому из пп. 4-6, или фармацевтическая композиция по любому из пп. 7-12 для применения в лечении макулодистрофии.
15. Применение антисмыслового олигонуклеотида по любому из пп. 1-3 или фармацевтически приемлемой соли по любому из пп. 4-6, или фармацевтической композиции по любому из пп. 7-12 для получения лекарственного средства для лечения или предупреждения макулодистрофии.
16. Способ лечения или предупреждения макулодистрофии у субъекта, страдающего от макулодистрофии, включающий введение субъекту терапевтически эффективного количества антисмыслового олигонуклеотида по любому из пп. 1-3 или фармацевтически приемлемой соли по любому из пп. 4-6, или фармацевтической композиции по любому из пп. 7-12.
17. Антисмысловой олигонуклеотид, фармацевтически приемлемая соль, фармацевтическая композиция по любому из пп. 13-14, применение по пп. 15 или способ по п. 16, где заболевание выбрано из влажной AMD (возрастная макулодистрофия), сухой AMD, географической атрофии и промежуточной dAMD.
18. Способ in vivo или in vitro модулирования экспрессии HTRA1 в клетке-мишени, которая экспрессирует HTRA1, причем указанный способ включает введение в указанную клетку антисмыслового олигонуклеотида, фармацевтически приемлемой соли или фармацевтической композиции в эффективном количестве.
RU2019101298A 2016-07-01 2017-06-28 Антисмысловые олигонуклеотиды для модулирования экспрессии htra1 RU2019101298A (ru)

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