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 |
|---|---|---|---|
| RU2020121752A RU2020121752A (ru) | 2017-12-01 | 2018-11-30 | Средство для лечения синдрома ангельмана на основе антисмысловой нуклеиновой кислоты |
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| EP (2) | EP4328306A3 (ru) |
| JP (3) | JP7297320B2 (ru) |
| KR (2) | KR20240161203A (ru) |
| CN (2) | CN111433361B (ru) |
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| HUE067905T2 (hu) | 2015-11-12 | 2024-11-28 | Hoffmann La Roche | Oligonukleotidok paternális UBE3A expresszió indukálására |
| CN111770758A (zh) * | 2018-02-27 | 2020-10-13 | 北卡罗来纳-查佩尔山大学 | 用于治疗Angelman综合征的方法和组合物 |
| FI3947684T3 (fi) | 2019-03-29 | 2025-05-27 | Ionis Pharmaceuticals Inc | Yhdisteitä ja menetelmiä ube3a-ats:n moduloimiseksi |
| BR112022024206A2 (pt) * | 2020-06-29 | 2023-01-03 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular plp1 |
| US20230416851A1 (en) * | 2020-10-06 | 2023-12-28 | University Of Maryland, Baltimore | Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides |
| IL315359A (en) * | 2022-03-07 | 2024-11-01 | Univ Connecticut | shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME |
| WO2024162126A1 (ja) | 2023-01-31 | 2024-08-08 | 富士フイルム株式会社 | 硬化性組成物、硬化物、光学材料、回折光学素子及び化合物 |
| KR20250068540A (ko) * | 2023-11-08 | 2025-05-16 | 한국과학기술원 | Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도 |
| TW202548015A (zh) * | 2024-04-11 | 2025-12-16 | 美商奧崔基尼克斯製藥公司 | 天使症候群反義治療的給藥方案 |
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| WO1993007883A1 (en) | 1991-10-24 | 1993-04-29 | Isis Pharmaceuticals, Inc. | Derivatized oligonucleotides having improved uptake and other properties |
| US20040259247A1 (en) * | 2000-12-01 | 2004-12-23 | Thomas Tuschl | Rna interference mediating small rna molecules |
| EP2397563A3 (en) | 2004-09-17 | 2012-07-18 | Isis Pharmaceuticals, Inc. | Enhanced antisense oligonucleotides |
| EP1676834A1 (en) | 2004-12-30 | 2006-07-05 | Sanofi-Aventis Deutschland GmbH | Fused bicyclic carboxamide derivates for use as CXCR2 inhibitors in the treatment of inflammation |
| WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
| US8178503B2 (en) * | 2006-03-03 | 2012-05-15 | International Business Machines Corporation | Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes |
| US20080299659A1 (en) * | 2007-03-02 | 2008-12-04 | Nastech Pharmaceutical Company Inc. | Nucleic acid compounds for inhibiting apob gene expression and uses thereof |
| WO2008113832A2 (en) | 2007-03-22 | 2008-09-25 | Santaris Pharma A/S | SHORT RNA ANTAGONIST COMPOUNDS FOR THE MODULATION OF TARGET mRNA |
| DK2285819T3 (da) | 2008-04-04 | 2013-12-02 | Isis Pharmaceuticals Inc | Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider |
| RU2572826C2 (ru) | 2008-12-02 | 2016-01-20 | Чиралджен, Лтд. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
| EP2462153B1 (en) | 2009-08-06 | 2015-07-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| US20120282176A1 (en) | 2011-04-20 | 2012-11-08 | Roche Glycart Ag | Method and Constructs for the pH Dependent Passage of the Blood-brain-barrier |
| EP3453761A1 (en) | 2011-08-29 | 2019-03-13 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| EP2850092B1 (en) | 2012-04-09 | 2017-03-01 | Ionis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| US20150141320A1 (en) * | 2012-05-16 | 2015-05-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
| BR112014028634A2 (pt) * | 2012-05-16 | 2017-06-27 | Rana Therapeutics Inc | composições e métodos para modulação da expressão de utrn |
| ES2809199T3 (es) | 2012-06-25 | 2021-03-03 | Ionis Pharmaceuticals Inc | Modulación de la expresión de UBE3A-ATS |
| JP6453212B2 (ja) | 2012-07-13 | 2019-01-16 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | キラル制御 |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| JP2017536366A (ja) | 2014-11-19 | 2017-12-07 | ロシュ イノベーション センター コペンハーゲン エーエス | Ｌｎａキラルホスホロチオエート |
| WO2016086104A1 (en) | 2014-11-25 | 2016-06-02 | Ionis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| CN107208092B (zh) | 2014-12-16 | 2021-09-10 | 罗氏创新中心哥本哈根有限公司 | 手性毒性筛选方法 |
| AU2016215155A1 (en) * | 2015-02-04 | 2017-08-17 | F. Hoffmann-La Roche Ag | Tau antisense oligomers and uses thereof |
| EP3286318A2 (en) * | 2015-04-22 | 2018-02-28 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| HUE067905T2 (hu) | 2015-11-12 | 2024-11-28 | Hoffmann La Roche | Oligonukleotidok paternális UBE3A expresszió indukálására |
| CN109311925B (zh) | 2016-05-12 | 2022-06-03 | 罗氏创新中心哥本哈根有限公司 | 立体限定的氧杂氮杂磷杂环戊烷亚磷酰胺单体与核苷或寡核苷酸的增强的偶联 |
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| JP7164540B2 (ja) | 2017-03-29 | 2022-11-01 | ロシュ イノベーション センター コペンハーゲン エーエス | 立体的に規定されたホスホロチオエートオリゴヌクレオチドの調製のための直交保護基 |
| JP2020524718A (ja) | 2017-06-22 | 2020-08-20 | キュラデブ・ファーマ・リミテッドＣｕｒａｄｅｖ Ｐｈａｒｍａ Ｌｉｍｉｔｅｄ | ヒトｓｔｉｎｇの複素環式小分子調節因子 |
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