RU2017116576A - Композиции и способы лечения бокового амиотрофического склероза (als) - Google Patents
Композиции и способы лечения бокового амиотрофического склероза (als) Download PDFInfo
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- 239000000203 mixture Substances 0.000 title claims 4
- 208000034189 Sclerosis Diseases 0.000 title 1
- 239000002773 nucleotide Substances 0.000 claims 13
- 125000003729 nucleotide group Chemical group 0.000 claims 13
- 210000004027 cell Anatomy 0.000 claims 8
- 102000008221 Superoxide Dismutase-1 Human genes 0.000 claims 7
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 claims 7
- 230000000692 anti-sense effect Effects 0.000 claims 7
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 6
- 230000014509 gene expression Effects 0.000 claims 6
- 210000004962 mammalian cell Anatomy 0.000 claims 6
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 5
- 108091081021 Sense strand Proteins 0.000 claims 5
- 108020004459 Small interfering RNA Proteins 0.000 claims 5
- 150000007523 nucleic acids Chemical group 0.000 claims 5
- 101150062190 sod1 gene Proteins 0.000 claims 5
- 230000002401 inhibitory effect Effects 0.000 claims 4
- 230000035772 mutation Effects 0.000 claims 4
- 210000001130 astrocyte Anatomy 0.000 claims 2
- 210000002161 motor neuron Anatomy 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 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
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
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Claims (25)
1. Вектор на основе аденоассоциированного вируса (AAV), содержащий последовательность нуклеиновой кислоты, расположенную между двумя инвертированными концевыми повторами (ITR), причем указанная последовательность нуклеиновой кислоты при экспрессии ингибирует или подавляет экспрессию гена SOD1 в клетке, при этом указанная последовательность нуклеиновой кислоты содержит последовательность смысловой цепи и последовательность антисмысловой цепи, причем последовательность смысловой цепи содержит по меньшей мере 15 смежных нуклеотидов, отличающихся не более чем 3 нуклеотидами от нуклеотидной последовательности для последовательностей, перечисленных в таблице 3, таблице 11 или таблице 14, и последовательность антисмысловой цепи содержит по меньшей мере 15 смежных нуклеотидов, отличающихся не более чем 3 нуклеотидами от нуклеотидной последовательности для последовательностей, перечисленных в таблице 3, таблице 11 или таблице 14, и при этом указанная последовательность смысловой цепи и последовательность антисмысловой цепи имеют общий участок комплементарности длиной по меньшей мере четыре нуклеотида.
2. Вектор на основе AAV по п. 1, где последовательность нуклеиновой кислоты содержит последовательность смысловой цепи и последовательность антисмысловой цепи дуплекса siRNA.
3. Вектор на основе AAV по п. 2, где дуплекс siRNA выбран из группы, состоящей из дуплекса siRNA с ID № от D-2741 до D-2985.
4. Вектор на основе AAV по п. 2, где дуплекс siRNA выбран из группы, состоящей из последовательности нуклеиновой кислоты siRNA с ID: D-2757, D-2806, D-2860, D-2861, D-2875, D-2871, D-2758, D-2759, D-2866, D-2870, D-2823 и D-2858.
5. Вектор на основе AAV по п. 1, где участок комплементарности имеет длину по меньшей мере 17 нуклеотидов.
6. Вектор на основе AAV по п. 5, где участок комплементарности имеет длину от 19 до 21 нуклеотида.
7. Вектор на основе AAV по п. 6, где участок комплементарности имеет длину 19 нуклеотидов.
8. Вектор на основе AAV по п. 1, где последовательность смысловой цепи и последовательность антисмысловой цепи составляют, независимо, 30 нуклеотидов или менее.
9. Вектор на основе AAV по п. 1, где по меньшей мере одна из последовательности смысловой цепи и последовательности антисмысловой цепи содержит 3ʹ выступающий конец из по меньшей мере 1 нуклеотида.
10. Вектор на основе AAV по п. 9, где по меньшей мере одна из последовательности смысловой цепи и последовательности антисмысловой цепи содержит 3ʹ выступающий конец из по меньшей мере 2 нуклеотидов.
11. Вектор на основе AAV по п. 1, где вектор на основе AAV содержит капсид серотипа, выбранного из группы, состоящей из AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV9.47, AAV9(hul4), AAV10, AAV11, AAV 12, AAVrh8, AAVrhl0, AAV-DJ8 и AAV-DJ, а также их вариантов.
12. Способ ингибирования экспрессии гена SOD1 в клетке, включающий введение в клетку композиции, содержащей вектор на основе AAV по любому из пп. 1-11.
13. Способ по п. 12, где клетка представляет собой клетку млекопитающего.
14. Способ по п. 13, где клетка млекопитающего представляет собой двигательный нейрон.
15. Способ по п. 13, где клетка млекопитающего представляет собой астроцит.
16. Способ лечения и/или ослабления бокового амиотрофического склероза (ALS) у субъекта, нуждающегося в лечении, причем способ включает введение субъекту терапевтически эффективного количества композиции, содержащей вектор на основе AAV по любому из пп. 1-11.
17. Способ по п. 16, где экспрессию SOD1 ингибируют или подавляют.
18. Способ по п. 17, где SOD1 представляет собой SOD1 дикого типа, мутированную SOD1 по меньшей мере с одной мутацией или как SOD1 дикого типа, так и мутированную SOD1 по меньшей мере с одной мутацией.
19. Способ по п. 16, где экспрессию SOD1 ингибируют или подавляют на величину от приблизительно 20% до приблизительно 100%.
20. Способ по п. 16, где ALS представляет собой семейный ALS с идентифицированной мутацией в гене SOD1.
21. Способ по п. 16, где ALS представляет собой спорадический ALS.
22. Способ ингибирования экспрессии гена SOD1 в клетке, включающий введение в клетку композиции, содержащей вектор на основе AAV по любому из пп. 1-11, при этом ген SOD1 включает в себя мутацию, которая вызывает эффект приобретения функции внутри клетки.
23. Способ по п. 22, где клетка представляет собой клетку млекопитающего.
24. Способ по п. 23, где клетка млекопитающего представляет собой двигательный нейрон.
25. Способ по п. 23, где клетка млекопитающего представляет собой астроцит.
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462079588P | 2014-11-14 | 2014-11-14 | |
| US62/079,588 | 2014-11-14 | ||
| US201562211992P | 2015-08-31 | 2015-08-31 | |
| US62/211,992 | 2015-08-31 | ||
| US201562234466P | 2015-09-29 | 2015-09-29 | |
| US62/234,466 | 2015-09-29 | ||
| PCT/US2015/060562 WO2016077687A1 (en) | 2014-11-14 | 2015-11-13 | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
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| RU2020108189A Division RU2020108189A (ru) | 2014-11-14 | 2015-11-13 | Композиции и способы лечения бокового амиотрофического склероза (als) |
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| Publication Number | Publication Date |
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| RU2017116576A true RU2017116576A (ru) | 2018-11-14 |
| RU2017116576A3 RU2017116576A3 (ru) | 2019-04-16 |
| RU2716422C2 RU2716422C2 (ru) | 2020-03-11 |
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| Application Number | Title | Priority Date | Filing Date |
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| RU2017116576A RU2716422C2 (ru) | 2014-11-14 | 2015-11-13 | Композиции и способы лечения бокового амиотрофического склероза (als) |
| RU2020108189A RU2020108189A (ru) | 2014-11-14 | 2015-11-13 | Композиции и способы лечения бокового амиотрофического склероза (als) |
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| Application Number | Title | Priority Date | Filing Date |
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| RU2020108189A RU2020108189A (ru) | 2014-11-14 | 2015-11-13 | Композиции и способы лечения бокового амиотрофического склероза (als) |
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| Country | Link |
|---|---|
| US (5) | US10597660B2 (ru) |
| EP (1) | EP3218484A4 (ru) |
| JP (4) | JP2017535266A (ru) |
| KR (2) | KR20230169197A (ru) |
| CN (2) | CN114717264A (ru) |
| AU (2) | AU2015346162B2 (ru) |
| CA (2) | CA2967367C (ru) |
| HK (1) | HK1244299A1 (ru) |
| IL (3) | IL292999A (ru) |
| MX (2) | MX2017006216A (ru) |
| RU (2) | RU2716422C2 (ru) |
| SG (1) | SG11201703281RA (ru) |
| WO (1) | WO2016077687A1 (ru) |
| ZA (1) | ZA201702991B (ru) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MY192634A (en) | 2014-04-01 | 2022-08-29 | Biogen Ma Inc | Compositions for modulating sod-1 expression |
| KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| GB201609597D0 (en) | 2016-06-01 | 2016-07-13 | Univ Sheffield | Therapy |
| EP3619310A4 (en) * | 2017-05-05 | 2021-01-27 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| WO2018204803A1 (en) * | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| AU2018261790B2 (en) * | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| US11434502B2 (en) * | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| EP4454654A3 (en) * | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| BR112020008033A2 (pt) * | 2017-10-23 | 2020-10-27 | Prevail Therapeutics, Inc. | terapias gênicas para doença neurodegenerativa |
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