RU2019130513A - Антисмысловые нуклеиновые кислоты - Google Patents
Антисмысловые нуклеиновые кислоты Download PDFInfo
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- RU2019130513A RU2019130513A RU2019130513A RU2019130513A RU2019130513A RU 2019130513 A RU2019130513 A RU 2019130513A RU 2019130513 A RU2019130513 A RU 2019130513A RU 2019130513 A RU2019130513 A RU 2019130513A RU 2019130513 A RU2019130513 A RU 2019130513A
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- muscular dystrophy
- antisense oligomer
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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Claims (14)
1. Антисмысловой олигомер для лечения мышечной дистрофии, обладающий длиной от 20 до 31 нуклеотида, включающий 20 нуклеотидов из нуклеотидной последовательности SEQ ID NO: 1, и обладающий активностью, вызывающей пропуск экзона 51 в гене дистрофина человека.
2. Антисмысловой олигомер для лечения мышечной дистрофии по п.1, который представляет собой олигонуклоетид.
3. Антисмысловой олигомер для лечения мышечной дистрофии по п.2, в котором модифицирован остаток сахара и/или область соединения через фосфатную группу по меньшей мере одного нуклеотида, входящего в состав олигонуклеотида.
4. Антисмысловой олигомер для лечения мышечной дистрофии по п.2, в котором остаток сахара по меньшей мере одного нуклеотида, составляющего олигонуклеотид, представляет собой рибозу, в которой 2'-ОН-группа заменена любой группой, выбранной из группы, состоящей из OR, R, R'OR, SH, SR, NH2, NHR, NR2, N3, CN, F, Cl, Br и I (где R представляет собой алкил или арил и R' обозначает алкилен).
5. Антисмысловой олигомер для лечения мышечной дистрофии по п.2, в котором область соединения через фосфатную группу по меньшей мере одного нуклеотида, входящего в состав олигонуклеотида представляет собой любую область, выбранную из группы, состоящей из тиофосфатной связи, дитиофосфатной связи, алкилфосфонатной связи, фосфорамидатной связи и боранофосфатной связи.
6. Антисмысловой олигомер для лечения мышечной дистрофии по п.1, который представляет собой морфолино-олигомер.
7. Антисмысловой олигомер для лечения мышечной дистрофии по п.6, который представляет собой фосфодиамидатный морфолино-олигомер.
8. Антисмысловой олигомер для лечения мышечной дистрофии по п.6, в котором 5'-конец имеет любую из приведенных ниже химических формул (1)-(3):
9. Фармацевтическая композиция для лечения мышечной дистрофии, содержащая в качестве активного ингредиента антисмысловой олигомер по п. 1, в суточной дозе в виде количества антисмыслового олигомера в интервале от 0,1 мг до 10 г/человека.
10. Фармацевтическая композиция по п.9, содержащая фармацевтически приемлемый носитель.
11. Способ лечения мышечной дистрофии, включающий введение пациенту с мышечной дистрофией антисмыслового олигомера поп. 1.
12. Способ лечения по п.11, где пациентом с мышечной дистрофией является пациент с делециями нуклеотидов внутри экзонов 29-50, 50, 45-50, 48-50, 49-50, 52, 52-63, 13-50, 19-50, 43-50 или 47-50.
13. Способ лечения по п.11, в котором пациентом является человек.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2014-048897 | 2014-03-12 | ||
| JP2014048897 | 2014-03-12 |
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| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2016139108A Division RU2702424C2 (ru) | 2014-03-12 | 2015-03-11 | Антисмысловые нуклеиновые кислоты |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| RU2019130513A true RU2019130513A (ru) | 2019-10-10 |
| RU2019130513A3 RU2019130513A3 (ru) | 2020-03-13 |
| RU2730681C2 RU2730681C2 (ru) | 2020-08-24 |
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| RU2019130513A RU2730681C2 (ru) | 2014-03-12 | 2015-03-11 | Антисмысловые нуклеиновые кислоты |
| RU2016139108A RU2702424C2 (ru) | 2014-03-12 | 2015-03-11 | Антисмысловые нуклеиновые кислоты |
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| RU2016139108A RU2702424C2 (ru) | 2014-03-12 | 2015-03-11 | Антисмысловые нуклеиновые кислоты |
Country Status (30)
| Country | Link |
|---|---|
| US (4) | US9988629B2 (ru) |
| EP (2) | EP3118311B1 (ru) |
| JP (1) | JP6606488B2 (ru) |
| KR (1) | KR102335801B1 (ru) |
| CN (2) | CN106459955B (ru) |
| AU (2) | AU2015227733B2 (ru) |
| BR (1) | BR112016020618B1 (ru) |
| CA (1) | CA2939948A1 (ru) |
| CY (1) | CY1121322T1 (ru) |
| DK (1) | DK3118311T3 (ru) |
| ES (1) | ES2710802T3 (ru) |
| HR (1) | HRP20190235T1 (ru) |
| HU (1) | HUE042283T2 (ru) |
| IL (1) | IL247663B (ru) |
| LT (1) | LT3118311T (ru) |
| MX (1) | MX2016011538A (ru) |
| MY (1) | MY193708A (ru) |
| NZ (1) | NZ724836A (ru) |
| PH (1) | PH12016501761B1 (ru) |
| PL (1) | PL3118311T3 (ru) |
| PT (1) | PT3118311T (ru) |
| RS (1) | RS58573B1 (ru) |
| RU (2) | RU2730681C2 (ru) |
| SG (1) | SG11201607095RA (ru) |
| SI (1) | SI3118311T1 (ru) |
| TR (1) | TR201901939T4 (ru) |
| TW (2) | TWI672314B (ru) |
| UA (1) | UA120849C2 (ru) |
| WO (1) | WO2015137409A1 (ru) |
| ZA (1) | ZA201605864B (ru) |
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| RU2730681C2 (ru) * | 2014-03-12 | 2020-08-24 | Ниппон Синяку Ко., Лтд. | Антисмысловые нуклеиновые кислоты |
| RS60493B1 (sr) | 2015-09-15 | 2020-08-31 | Nippon Shinyaku Co Ltd | Antismisaona nukleinska kiselina |
| CR20180233A (es) | 2015-10-09 | 2018-05-25 | Wave Life Sciences Ltd | Composiciones oligonucleotídicas y sus métodos |
| JP2019525742A (ja) | 2016-06-30 | 2019-09-12 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマー |
| EP3485015A4 (en) * | 2016-07-15 | 2020-07-29 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS OF MODULATION OF DYSTROPHINE TRANSCRIPT |
| AU2017382773A1 (en) | 2016-12-19 | 2019-08-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| BR112019012647A2 (pt) * | 2016-12-19 | 2019-11-19 | Sarepta Therapeutics Inc | conjugados de oligômero de salto de éxon para distrofia muscular |
| WO2018118627A1 (en) | 2016-12-19 | 2018-06-28 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| MX2019008199A (es) * | 2017-01-06 | 2019-11-25 | Avidity Biosciences Llc | Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon. |
| GB201711809D0 (en) * | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| EP3672601B1 (en) * | 2017-09-25 | 2023-09-13 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers via fast-flow synthesis |
| JP2020536060A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| WO2020028864A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CN113286887A (zh) * | 2018-11-02 | 2021-08-20 | 比奥马林技术公司 | 用于肌营养不良蛋白外显子跳跃的双特异性反义低聚核苷酸 |
| CN113383078A (zh) * | 2018-12-06 | 2021-09-10 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| JPWO2020158792A1 (ja) * | 2019-01-30 | 2021-12-02 | 国立研究開発法人国立精神・神経医療研究センター | 核酸送達複合体 |
| JPWO2021172498A1 (ru) | 2020-02-28 | 2021-09-02 | ||
| EP4330395A1 (en) | 2021-04-30 | 2024-03-06 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2023178230A1 (en) | 2022-03-17 | 2023-09-21 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
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