RU2020121752A - Средство для лечения синдрома ангельмана на основе антисмысловой нуклеиновой кислоты - Google Patents
Средство для лечения синдрома ангельмана на основе антисмысловой нуклеиновой кислоты Download PDFInfo
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- RU2020121752A RU2020121752A RU2020121752A RU2020121752A RU2020121752A RU 2020121752 A RU2020121752 A RU 2020121752A RU 2020121752 A RU2020121752 A RU 2020121752A RU 2020121752 A RU2020121752 A RU 2020121752A RU 2020121752 A RU2020121752 A RU 2020121752A
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- oligonucleotide
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- 208000009575 Angelman syndrome Diseases 0.000 title claims 3
- 150000007523 nucleic acids Chemical class 0.000 title claims 3
- 230000000692 anti-sense effect Effects 0.000 title 1
- 102000039446 nucleic acids Human genes 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 title 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 17
- 125000003835 nucleoside group Chemical group 0.000 claims 4
- 239000002777 nucleoside Substances 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 102100034343 Integrase Human genes 0.000 claims 1
- 101710203526 Integrase Proteins 0.000 claims 1
- -1 LNA nucleoside Chemical class 0.000 claims 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 239000002671 adjuvant Substances 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 239000002904 solvent Substances 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
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Claims (13)
1. Антисмысловой олигонуклеотид, содержащий непрерывную нуклеотидную последовательность длиной 10-30 нуклеотидов по меньшей мере с 98% комплементарностью непрерывной части последовательности нуклеиновой кислоты с SEQ ID NO: 1, SEQ ID NO:2, SEQ ID NO: 3, SEQ ID NO: 4 или SEQ ID NO: 5.
2. Олигонуклеотид по п. 1, причем олигонуклеотид содержит последовательность, выбранную из группы, состоящей из SEQ ID NO: 6, 7, 8, 9, 10 или 11.
3. Олигонуклеотид по п. 1 или 2, содержащий один или несколько модифицированных нуклеозидов.
4. Олигонуклеотид по п. 3, причем один или несколько модифицированных нуклеозидов представляют собой модифицированный по 2'-атому сахара нуклеозид.
5. Олигонуклеотид по п. 4, причем один или несколько модифицированных по 2'-атому сахара нуклеозидов независимо выбраны из группы, состоящей из 2'-O-алкил-РНК, 2'-O-метил-РНК, 2'-алкокси-РНК, 2'-O-метоксиэтил-РНК, 2'-амино-ДНК, 2'-фтор-ДНК, арабинонуклеиновой кислоты (ANA), 2'-фтор-ANA и нуклеозидов LNA.
6. Олигонуклеотид по п. 5, причем один или несколько модифицированных нуклеозидов представляют собой нуклеозид LNA.
7. Олигонуклеотид по п. 1, причем олигонуклеотид содержит по меньшей мере один модифицированный межнуклеозидный мостик.
8. Олигонуклеотид по п. 7, причем межнуклеозидные мостики в пределах непрерывной нуклеотидной последовательности представляют собой фосфоротиоатные межнуклеозидные мостики.
9. Олигонуклеотид по п. 1, причем олигонуклеотид способен привлекать РНКазу Н.
10. Олигонуклеотид по п. 9, причем олигонуклеотид представляет собой гэпмер.
11. Олигонуклеотид по п. 10, причем олигонуклеотид имеет последовательность нуклеиновой кислоты, выбранную из группы, состоящей из SEQ ID NO: 362-392.
12. Фармацевтическая композиция, содержащая один или несколько из олигонуклеотидов по любому из пп. 1-11 и фармацевтически приемлемый разбавитель, растворитель, носитель, соль и/или вспомогательное средство.
13. Способ лечения или предупреждения синдрома Ангельмана у субъекта, предусматривающий введение терапевтически или профилактически эффективного количества композиции по п. 12 субъекту, страдающему от синдрома Ангельмана или чувствительному к нему.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762593431P | 2017-12-01 | 2017-12-01 | |
| US62/593,431 | 2017-12-01 | ||
| US201862676034P | 2018-05-24 | 2018-05-24 | |
| US62/676,034 | 2018-05-24 | ||
| PCT/US2018/063416 WO2019109001A1 (en) | 2017-12-01 | 2018-11-30 | Angelman syndrome antisense treatment |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2020121752A true RU2020121752A (ru) | 2021-12-30 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
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| RU2020121752A RU2020121752A (ru) | 2017-12-01 | 2018-11-30 | Средство для лечения синдрома ангельмана на основе антисмысловой нуклеиновой кислоты |
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| Country | Link |
|---|---|
| US (5) | US11198869B2 (ru) |
| EP (2) | EP4328306A3 (ru) |
| JP (3) | JP7297320B2 (ru) |
| KR (2) | KR102723989B1 (ru) |
| CN (2) | CN111433361B (ru) |
| AU (1) | AU2018375807B2 (ru) |
| BR (1) | BR112020009431A2 (ru) |
| CA (1) | CA3079755A1 (ru) |
| CL (1) | CL2020001344A1 (ru) |
| CO (1) | CO2020006361A2 (ru) |
| DK (1) | DK3717646T3 (ru) |
| ES (1) | ES3032315T3 (ru) |
| FI (1) | FI3717646T3 (ru) |
| IL (1) | IL274146B2 (ru) |
| LT (1) | LT3717646T (ru) |
| MX (1) | MX2020005680A (ru) |
| PE (1) | PE20211238A1 (ru) |
| RU (1) | RU2020121752A (ru) |
| SG (1) | SG11202003596WA (ru) |
| WO (1) | WO2019109001A1 (ru) |
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| US20210054370A1 (en) * | 2018-02-27 | 2021-02-25 | The University Of North Carolina At Chapel Hill | Methods and compositions for treating angelman syndrome |
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| KR20240155345A (ko) * | 2022-03-07 | 2024-10-28 | 유니버시티 오브 코네티컷 | 안젤만 증후군에서 부계 UBE3A 유전자 발현을 회복하기 위한 SNORD115 위치를 표적으로 하는 shRNA |
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