RU2017115477A - Полинуклеотиды aadc для лечения болезни паркинсона - Google Patents
Полинуклеотиды aadc для лечения болезни паркинсона Download PDFInfo
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- 102100038238 Aromatic-L-amino-acid decarboxylase Human genes 0.000 title claims 4
- 101710151768 Aromatic-L-amino-acid decarboxylase Proteins 0.000 title claims 4
- 208000018737 Parkinson disease Diseases 0.000 title claims 4
- 102000040430 polynucleotide Human genes 0.000 title 1
- 108091033319 polynucleotide Proteins 0.000 title 1
- 239000002157 polynucleotide Substances 0.000 title 1
- 239000002773 nucleotide Substances 0.000 claims 23
- 125000003729 nucleotide group Chemical group 0.000 claims 23
- 239000013607 AAV vector Substances 0.000 claims 22
- 101000899111 Homo sapiens Hemoglobin subunit beta Proteins 0.000 claims 12
- 241000702421 Dependoparvovirus Species 0.000 claims 9
- 239000008194 pharmaceutical composition Substances 0.000 claims 8
- 108090000623 proteins and genes Proteins 0.000 claims 6
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 4
- 108091034057 RNA (poly(A)) Proteins 0.000 claims 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 3
- 208000012661 Dyskinesia Diseases 0.000 claims 2
- 102000002265 Human Growth Hormone Human genes 0.000 claims 2
- 108010000521 Human Growth Hormone Proteins 0.000 claims 2
- 239000000854 Human Growth Hormone Substances 0.000 claims 2
- 210000000234 capsid Anatomy 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 230000036385 rapid eye movement (rem) sleep Effects 0.000 claims 2
- 230000007958 sleep Effects 0.000 claims 2
- 208000019116 sleep disease Diseases 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 1
- 241000701022 Cytomegalovirus Species 0.000 claims 1
- 101150089187 IE1 gene Proteins 0.000 claims 1
- 206010030312 On and off phenomenon Diseases 0.000 claims 1
- 208000013738 Sleep Initiation and Maintenance disease Diseases 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 230000004424 eye movement Effects 0.000 claims 1
- 101150072154 hb gene Proteins 0.000 claims 1
- 206010022437 insomnia Diseases 0.000 claims 1
- 208000022925 sleep disturbance Diseases 0.000 claims 1
- 230000008454 sleep-wake cycle Effects 0.000 claims 1
- 208000020685 sleep-wake disease Diseases 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
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- C12Y401/01—Carboxy-lyases (4.1.1)
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Claims (45)
1. AAV-векторный геном, содержащий в следующем порядке:
а) 5ʹ-инвертированный концевой повтор (ITR), где указанный 5ʹ-ITR имеет длину не более 141 нуклеотида,
b) участок последовательности цитомегаловируса (CMV), где указанный участок последовательности CMV получен из гена CMV и в направлении от 5ʹ- к 3ʹ-концу содержит энхансерный участок и промоторный участок, при этом указанный участок последовательности CMV имеет размер по меньшей мере 500 нуклеотидов,
c) участок последовательности предраннего 1 (IE1) гена, полученный из IE1 гена,
d) участок последовательности бета глобина (HB) человека, полученный из HB гена,
e) участок последовательности AADC, где указанный участок последовательности AADC содержит нуклеотидную последовательность, кодирующую SEQ ID NO: 1,
f) участок последовательности сигнала поли(A), полученный из гена гормона роста человека (hGH), и
g) 3ʹ-ITR, где указанный 3ʹ-ITR имеет длину не более 141 нуклеотида.
2. AAV-векторный геном по п.1, где 5ʹ-ITR и 3ʹ-ITR получены из AAV2.
3. AAV-векторный геном по п.2, где участок последовательности CMV имеет длину 507 нуклеотидов.
4. AAV-векторный геном по п.2, где участок последовательности IE1 содержит нуклеотидную последовательность из IE1 экзона 1.
5. AAV-векторный геном по п.4, где участок последовательности IE1 дополнительно содержит нуклеотидную последовательность из IE1 интрона 1 или ее фрагмент.
6. AAV-векторный геном по п.5, где участок последовательности IE1 имеет длину по меньшей мере 160 нуклеотидов.
7. AAV-векторный геном по п.5, где участок последовательности IE1 имеет длину 166 нуклеотидов.
8. AAV-векторный геном по п.2, где участок последовательности HB содержит нуклеотидную последовательность из HB интрона 2.
9. AAV-векторный геном по п.8, где участок последовательности HB дополнительно содержит нуклеотидную последовательность из HB экзона 3.
10. AAV-векторный геном по п.9, где участок последовательности HB имеет длину примерно 400 нуклеотидов.
11. AAV-векторный геном по п.2, где участок последовательности сигнала поли(A) имеет длину по меньшей мере 450 нуклеотидов.
12. AAV-векторный геном по п.11, где участок последовательности сигнала поли(A) имеет длину 477 нуклеотидов.
13. AAV-векторный геном по п.2, где
i) участок последовательности IE1 содержит нуклеотидную последовательность из IE1 экзона 1,
ii) участок последовательности IE1 содержит нуклеотидную последовательность из IE1 интрона 1 или ее фрагмент,
iii) участок последовательности HB содержит нуклеотидную последовательность из HB интрона 2, и
iv) участок последовательности HB содержит нуклеотидную последовательность из HB экзона 3.
14. AAV-векторный геном по п.13, где участок последовательности CMV имеет длину 507 нуклеотидов, участок последовательности IE1 имеет длину 166 нуклеотидов, участок последовательности HB имеет длину 400 нуклеотидов, участок последовательности сигнала поли(A) имеет длину 477 нуклеотидов,
15. AAV-векторный геном по п.1, где AAV-векторный геном содержит последовательность, выбранную из группы, состоящей из: SEQ ID NO: 1-13, 15 и 16 или вариантов, имеющих с ними по меньшей мере 95% идентичность.
16. AAV-векторный геном по п.2, где AAV-векторный геном содержит кодон-оптимизированную нуклеотидную последовательность, кодирующую SEQ ID NO: 1.
17. AAV-векторный геном по п.16, где AAV-векторный геном содержит последовательность, выбранную из группы, состоящей из: SEQ ID NO: 7 и 12 или вариантов, имеющих с ними по меньшей мере 95% идентичность.
18. Рекомбинантный AAV-вирус, содержащий AAV-векторный геном по любому из пп.1-17.
19. Рекомбинантный AAV-вирус по п.18, содержащий капсид серотипа, выбранного из группы, состоящей из: AAV2, AAV1, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV9.47, AAV9(hu14), AAV10, AAV11, AAV12, AAVrh8, AAVrh10, AAV-DJ и AAV-DJ8.
20. Рекомбинантный AAV-вирус по п. 19, где капсид серотипа представляет собой AAV2.
21. Фармацевтическая композиция, содержащая рекомбинантный AAV-вирус по любому из пп.18-20.
22. Фармацевтическая композиция по п. 21, где по меньшей мере 70% векторов на основе рекомбинантного AAV содержат AAV-векторный геном.
23. Способ уменьшения периодов дискинезии у нуждающегося в этом субъекта, включающий введение указанному субъекту фармацевтической композиции, содержащей рекомбинантный AAV-вирус по любому из пп.18-20.
24. Способ по п.23, где уменьшения периодов дискинезии видно по улучшению показателей периода «выключения» и двигательных флуктуаций по меньшей мере на 30%.
25. Способ по п. 24, где улучшение длится по меньшей мере 6 часов.
26. Способ по любому из пп.23-25, где субъект страдает болезнью Паркинсона.
27. Способ улучшения цикла сна-бодрствования у нуждающегося в этом субъекта, включающий введение указанному субъекту фармацевтической композиции, содержащей рекомбинантный AAV-вирус по любому из пп.18-20.
28. Способ по п.27, где интервал сна с быстрым движением глаз (REM) у субъекта снижается по сравнению с интервалом REM-сна у субъекта до введения фармацевтической композиции.
29. Способ по п.27, где интервал не-REM (NREM) сна у субъекта увеличивается по сравнению с интервалом REM-сна у субъекта до введения фармацевтической композиции.
30. Способ по любому из пп.27-29, где субъект страдает болезнью Паркинсона.
31. Способ лечения нарушения сна у нуждающегося в этом субъекта, включающий введение указанному субъекту фармацевтической композиции, содержащей рекомбинантный AAV-вирус по любому из пп.18-20.
32. Способ по п. 31, где нарушение сна представляет собой инсомнию.
33. Способ по любому из пп.31-32, где субъект страдает болезнью Паркинсона.
34. Способ увеличения уровня белка AADC у нуждающегося в этом субъекта, включающий введение указанному субъекту фармацевтической композиции, содержащей рекомбинантный AAV-вирус по любому из пп.18-20.
Applications Claiming Priority (9)
Application Number | Priority Date | Filing Date | Title |
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US201462075298P | 2014-11-05 | 2014-11-05 | |
US62/075,298 | 2014-11-05 | ||
US201562155692P | 2015-05-01 | 2015-05-01 | |
US62/155,692 | 2015-05-01 | ||
US201562199578P | 2015-07-31 | 2015-07-31 | |
US62/199,578 | 2015-07-31 | ||
US201562243537P | 2015-10-19 | 2015-10-19 | |
US62/243,537 | 2015-10-19 | ||
PCT/US2015/059201 WO2016073693A2 (en) | 2014-11-05 | 2015-11-05 | Aadc polynucleotides for the treatment of parkinson's disease |
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RU2020109343A Division RU2020109343A (ru) | 2014-11-05 | 2015-11-05 | Полинуклеотиды aadc для лечения болезни паркинсона |
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RU2017115477A true RU2017115477A (ru) | 2018-11-09 |
RU2017115477A3 RU2017115477A3 (ru) | 2019-03-26 |
RU2716991C2 RU2716991C2 (ru) | 2020-03-17 |
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RU2017115477A RU2716991C2 (ru) | 2014-11-05 | 2015-11-05 | Полинуклеотиды aadc для лечения болезни паркинсона |
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US (6) | US10335466B2 (ru) |
EP (1) | EP3215191A4 (ru) |
JP (4) | JP6401871B2 (ru) |
KR (1) | KR20170096998A (ru) |
CN (2) | CN107106689A (ru) |
AU (2) | AU2015343037B2 (ru) |
BR (1) | BR112017009497A2 (ru) |
CA (1) | CA2966620A1 (ru) |
HK (1) | HK1243922A1 (ru) |
IL (2) | IL251787B (ru) |
MX (1) | MX2017005834A (ru) |
RU (2) | RU2020109343A (ru) |
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BR112017009497A2 (pt) | 2014-11-05 | 2018-02-06 | Voyager Therapeutics, Inc. | polinucleotídeos de aadc para o tratamento da doença de parkinson |
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