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RU2014150340A - Лечение amd с применением экспессируемого aav sflt-1 - Google Patents

Лечение amd с применением экспессируемого aav sflt-1 Download PDF

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RU2014150340A
RU2014150340A RU2014150340A RU2014150340A RU2014150340A RU 2014150340 A RU2014150340 A RU 2014150340A RU 2014150340 A RU2014150340 A RU 2014150340A RU 2014150340 A RU2014150340 A RU 2014150340A RU 2014150340 A RU2014150340 A RU 2014150340A
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pharmaceutical composition
sequence
human subject
recombinant virus
virus
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RU2014150340A
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Ян Дж. КОНСТЕЙБЛ
Элизабет П. РАКОЧИ
Чоои-май ЛАЙ
Томас В. Мл. ЧАЛБЕРГ
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Эвеланч Острелиа Пти Лтд.
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Abstract

1. Способ лечения или профилактики неоваскуляризации в глазу являющегося человеком субъекта, у которого имеется неоваскуляризация в глазу или который подвержен риску ее развития, при этом способ включает введение в сетчатку являющегося человеком субъекта эффективного количества фармацевтической композиции, включающей рекомбинантный вирус.2. Способ по п. 1, где рекомбинантный вирус включает последовательность нуклеиновой кислоты, кодирующую направленный против VEGF белок.3. Способ по п. 2, где направленный против VEGF белок включает функциональный фрагмент sFLT-1, имеющий по меньшей мере 90% идентичность последовательности с полипептидом, кодируемым SEQ ID NO: 102.4. Способ по п. 1, где рекомбинантным вирусом является AAV.5. Способ по п. 1, где введение осуществляют с частотой, составляющей менее чем 3 раза в год, являющемуся человеком субъекту.6. Способ по п. 1, где введение осуществляют только один раз в по меньшей мере 18 месяцев.7. Способ по п. 1, где введение осуществляют в дозе, составляющей самое большее 1x10векторных геномов.8. Способ по п. 1, где введение осуществляют в дозе, составляющей по меньшей мере 1x10векторных геномов.9. Способ по п. 1, где уменьшение неоваскуляризации обнаруживают с помощью флуоресцентной ангиографии (FA) после введения фармацевтической композиции.10. Способ по п. 1, где острота зрения с максимальной коррекцией (BCVA) являющегося человеком субъекта уменьшается на менее чем 15 букв, как определено по буквам в исследовании раннего лечения диабетической ретинопатии (ETDRS), после введения фармацевтической композиции.11. Способ по п. 1, где острота зрения с максимальной коррекцией (BCVA) являющегося человеком субъекта увеличивается на по меньшей мере 1 строку, как определено по буквам в исследо

Claims (33)

1. Способ лечения или профилактики неоваскуляризации в глазу являющегося человеком субъекта, у которого имеется неоваскуляризация в глазу или который подвержен риску ее развития, при этом способ включает введение в сетчатку являющегося человеком субъекта эффективного количества фармацевтической композиции, включающей рекомбинантный вирус.
2. Способ по п. 1, где рекомбинантный вирус включает последовательность нуклеиновой кислоты, кодирующую направленный против VEGF белок.
3. Способ по п. 2, где направленный против VEGF белок включает функциональный фрагмент sFLT-1, имеющий по меньшей мере 90% идентичность последовательности с полипептидом, кодируемым SEQ ID NO: 102.
4. Способ по п. 1, где рекомбинантным вирусом является AAV.
5. Способ по п. 1, где введение осуществляют с частотой, составляющей менее чем 3 раза в год, являющемуся человеком субъекту.
6. Способ по п. 1, где введение осуществляют только один раз в по меньшей мере 18 месяцев.
7. Способ по п. 1, где введение осуществляют в дозе, составляющей самое большее 1x1011 векторных геномов.
8. Способ по п. 1, где введение осуществляют в дозе, составляющей по меньшей мере 1x108 векторных геномов.
9. Способ по п. 1, где уменьшение неоваскуляризации обнаруживают с помощью флуоресцентной ангиографии (FA) после введения фармацевтической композиции.
10. Способ по п. 1, где острота зрения с максимальной коррекцией (BCVA) являющегося человеком субъекта уменьшается на менее чем 15 букв, как определено по буквам в исследовании раннего лечения диабетической ретинопатии (ETDRS), после введения фармацевтической композиции.
11. Способ по п. 1, где острота зрения с максимальной коррекцией (BCVA) являющегося человеком субъекта увеличивается на по меньшей мере 1 строку, как определено по буквам в исследовании раннего лечения диабетической ретинопатии (ETDRS), после введения фармацевтической композиции.
12. Способ по п. 1, где введение включает субретинальную инъекцию.
13. Способ по п. 12, где субретинальная инъекция осуществляется в пределах центральной части сетчатки.
14. Способ по п. 12, дополнительно включающий удаление стекловидного тела до введения фармацевтической композиции.
15. Способ по п. 1, где неоваскуляризация в глазу связана с заболеванием, выбираемым из группы, состоящей из возрастной дегенерации желтого пятна (AMD), неоваскуляризации в сетчатке, неоваскуляризации в сосудистой оболочке глаза, диабетической ретинопатии, окклюзии вены сетчатки, отека желтого пятна на фоне диабета, ишемии сетчатки на фоне диабета, ишемической ретинопатии и отека сетчатки на фоне диабета.
16. Способ по п. 15, где заболеванием является AMD.
17. Фармацевтическая композиция, включающая последовательность нуклеиновой кислоты, кодирующую направленный против VEGF белок, функционально связанную с последовательностью промотора.
18. Фармацевтическая композиция по п. 17, где направленный против VEGF белок включает функциональный фрагмент sFLT-1 человека.
19. Фармацевтическая композиция по п. 17, где функциональный фрагмент имеет по меньшей мере 90% идентичность последовательности с полипептидом, кодируемым SEQ ID NO: 102.
20. Фармацевтическая композиция по п. 18, где функциональный фрагмент включает лиганд-связывающий домен sFLT-1.
21. Фармацевтическая композиция по п. 17, где промоторная последовательность и последовательность нуклеиновой кислоты, кодирующая направленный против VEGF белок, разделены последовательностью, которая составляет 300 пар оснований или более.
22. Фармацевтическая композиция по п. 17, где промоторная последовательность и последовательность нуклеиновой кислоты, кодирующая направленный против VEGF белок, разделены последовательностью UTR.
23. Фармацевтическая композиция по п. 17, где фармацевтическая композиция не включает прокариотическую регуляторную последовательность.
24. Фармацевтическая композиция по п. 17, где нуклеиновая кислота не включает последовательность устойчивости к антибиотику.
25. Фармацевтическая композиция по п. 17, где рекомбинантный вирус включает последовательность нуклеиновой кислоты, кодирующую направленный против VEGF белок, функционально связанную с последовательностью промотора.
26. Фармацевтическая композиция по п. 25, где вирус выбирают из группы, состоящей из аденоассоциированного вируса (AAV), аденовируса, зависимого от помощника аденовируса, ретровируса, вируса простого герпеса, лентивируса, поксвируса, вируса Сендай (HVJ), вируса лейкоза у мышей Молони и вируса на основе ВИЧ.
27. Фармацевтическая композиция по п. 25, где вирусом является AAV.
28. Фармацевтическая композиция по п. 25, где геном рекомбинантного вируса включает элементы нуклеиновой кислоты в следующем порядке:
a) последовательность первого ITR;
b) промоторную последовательность;
c) последовательность интрона;
d) первую UTR-последовательность;
e) последовательность, кодирующую направленный против VEGF белок;
f) вторую UTR-последовательность;
g) последовательность поли(А); и
h) последовательность второго ITR.
29. Фармацевтическая композиция по п. 25, где фармацевтическая композиция включает самое большее 1x1013 векторных геномов рекомбинантного вируса.
30. Фармацевтическая композиция по п. 25, где фармацевтическая композиция включает самое большее 1x1011 векторных геномов рекомбинантного вируса.
31. Фармацевтическая композиция по п. 25, где фармацевтическая композиция включает по меньшей мере 1x108 векторных геномов рекомбинантного вируса.
32. Фармацевтическая композиция по п. 25, где фармацевтическую композицию вводят в объеме от 0,1 до 0,5 мл.
33. Фармацевтическая композиция по п. 25, где фармацевтическую композицию вводят в объеме 100 мкл.
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