RU2015120645A - Композиции и способы модуляции экспрессии рецептора фактора роста фибробластов 3 типа (fgfr3) - Google Patents
Композиции и способы модуляции экспрессии рецептора фактора роста фибробластов 3 типа (fgfr3) Download PDFInfo
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- RU2015120645A RU2015120645A RU2015120645A RU2015120645A RU2015120645A RU 2015120645 A RU2015120645 A RU 2015120645A RU 2015120645 A RU2015120645 A RU 2015120645A RU 2015120645 A RU2015120645 A RU 2015120645A RU 2015120645 A RU2015120645 A RU 2015120645A
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- 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
- C12N15/1138—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 against receptors or cell surface proteins
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
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- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
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- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
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- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
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- Biochemistry (AREA)
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- Physical Education & Sports Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Epidemiology (AREA)
Claims (15)
1. Антисмысловой олигонуклеотид, длиной 8-25 нуклеотидов, включающий один или более аналогов нуклеотидов, который способен гибридизоваться по участку последовательности SEQ ID NO: 4, включающий положение нуклеотида 1394 последовательности SEQ ID NO: 4.
2. Олигонуклеотид по п. 1, который по меньшей мере на 80% идентичен олигонуклеотиду, выбранному из группы, состоящей из последовательностей SEQ ID NO: 9, SEQ ID NO: 10, SEQ ID NO: 11, SEQ ID NO: 12, SEQ ID NO: 13 и SEQ ID NO: 14.
3. Олигонуклеотид по п. 1 или 2, который имеет длину 14-18 нуклеотидов.
4. Олигонуклеотид по п. 1, который предпочтительно модулирует экспрессию мутированной формы рецептора фактора роста фибробластов FGFR3 по сравнению с экспрессией рецептора FGFR3 дикого типа.
5. Олигонуклеотид по п. 1, выбранный из группы, состоящей из последовательностей SEQ ID NO: 9, SEQ ID NO: 10, SEQ ID NO: 11, SEQ ID NO: 12, SEQ ID NO: 13, SEQ ID NO: 14, SEQ ID NO: 21, SEQ ID NO: 22, SEQ ID NO: 23, SEQ ID NO: 24 и SEQ ID NO: 25.
6. Олигонуклеотид по п. 1, в котором один или более аналогов нуклеотидов выбран из группы, состоящей из: звеньев запертой нуклеиновой кислоты (ЗНК); звеньев 2'-O-алкил-РНК, звеньев 2'-ОМе-РНК, звеньев 2'-амино-ДНК и звеньев 2'-фтор-ДНК.
7. Олигонуклеотид по п. 1, который включает два или более мономерных звеньев ЗНК.
8. Олигонуклеотид по п. 1, выбранный из группы, состоящей из последовательностей SEQ ID NO: 21, SEQ ID NO: 22, SEQ ID NO: 23, SEQ ID NO: 24 и SEQ ID NO: 25.
9. Олигонуклеотид по п. 1, который включает SEQ ID NO: 21.
10. Конъюгат, включающий указанный олигонуклеотид по пп. 1-9 и по меньшей мере один ненуклеотидный фрагмент, ковалентно присоединенный к указанному олигонуклеотиду.
11. Применение олигонуклеотида по любому из пп. 1-9 или конъюгата по п. 10 для лечения состояния, связанного с аномальной экспрессией рецептора FGFR3, например, рака или хондродистрофии.
12. Способ снижения экспрессии мРНК FGFR3, включающий доставку к мРНК FGFR3 олигонуклеотида по любому из пп. 1-9 или конъюгата по п. 10, в условиях, подходящих для гибридизации указанной мРНК FGFR3 с указанным олигонуклеотидом, что приводит к снижению экспрессии мРНК FGFR3.
13. Способ снижения экспрессии in vitro мРНК FGFR3, включающий доставку к мРНК FGFR3 олигонуклеотида по любому из пп. 1-9 или конъюгата по п. 10, в условиях, подходящих для гибридизации указанной мРНК FGFR3 с указанным олигонуклеотидом, что приводит к снижению экспрессии мРНК FGFR3.
14. Способ лечения состояния, связанного с аномальной экспрессией рецептора FGFR3, включающий выбор пациента, у которого диагностировали указанное состояние, и введение такому пациенту олигонуклеотида по любому из пп. 1-9 или конъюгата по п. 10.
15. Фармацевтическая композиция, включающая олигонуклеотид по любому из пп. 1-9 или конъюгат по п. 10 и фармацевтически приемлемый разбавитель, носитель, растворитель, соль или адъювант.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261729922P | 2012-11-26 | 2012-11-26 | |
| US61/729,922 | 2012-11-26 | ||
| PCT/EP2013/074552 WO2014080004A1 (en) | 2012-11-26 | 2013-11-25 | Compositions and methods for modulation of fgfr3 expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2015120645A true RU2015120645A (ru) | 2017-01-10 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2015120645A RU2015120645A (ru) | 2012-11-26 | 2013-11-25 | Композиции и способы модуляции экспрессии рецептора фактора роста фибробластов 3 типа (fgfr3) |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US9725723B2 (ru) |
| EP (1) | EP2922955B1 (ru) |
| JP (1) | JP6463687B2 (ru) |
| KR (1) | KR20150088305A (ru) |
| CN (1) | CN104903448B (ru) |
| BR (1) | BR112015012051A2 (ru) |
| CA (1) | CA2889993A1 (ru) |
| DK (1) | DK2922955T3 (ru) |
| HK (1) | HK1208702A1 (ru) |
| MX (1) | MX2015006289A (ru) |
| RU (1) | RU2015120645A (ru) |
| WO (1) | WO2014080004A1 (ru) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
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| RU2015151857A (ru) | 2008-12-02 | 2019-01-15 | Уэйв Лайф Сайенсес Джапан, Инк. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
| AU2010270714B2 (en) | 2009-07-06 | 2015-08-13 | Wave Life Sciences Ltd. | Novel nucleic acid prodrugs and methods use thereof |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| CA3077910A1 (en) | 2010-11-17 | 2012-05-24 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| CA2879023C (en) | 2012-07-13 | 2017-03-28 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| CN104661664B (zh) | 2012-07-13 | 2020-07-03 | 波涛生命科学有限公司 | 手性控制 |
| CN104903448B (zh) | 2012-11-26 | 2017-07-11 | 罗氏创新中心哥本哈根有限公司 | 用于调节fgfr3的表达的组合物和方法 |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| KR20230152178A (ko) | 2014-01-16 | 2023-11-02 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
| EP3424505A4 (en) | 2016-03-04 | 2019-10-16 | Taiho Pharmaceutical Co., Ltd. | PREPARATION AND COMPOSITION FOR TREATING MALIGNANT TUMORS |
| US11883404B2 (en) | 2016-03-04 | 2024-01-30 | Taiho Pharmaceuticals Co., Ltd. | Preparation and composition for treatment of malignant tumors |
| FI3769765T3 (fi) | 2018-03-19 | 2024-05-15 | Taiho Pharmaceutical Co Ltd | Natriumalkyylisulfaattia sisältävä farmaseuttinen koostumus |
| JPWO2021153703A1 (ru) * | 2020-01-31 | 2021-08-05 | ||
| WO2022211095A1 (ja) * | 2021-03-31 | 2022-10-06 | ルクサナバイオテク株式会社 | Ｆｇｆｒ３のアンチセンスオリゴヌクレオチド |
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2013
- 2013-11-25 CN CN201380061132.9A patent/CN104903448B/zh not_active Expired - Fee Related
- 2013-11-25 RU RU2015120645A patent/RU2015120645A/ru not_active Application Discontinuation
- 2013-11-25 BR BR112015012051A patent/BR112015012051A2/pt active Search and Examination
- 2013-11-25 US US14/647,430 patent/US9725723B2/en active Active
- 2013-11-25 EP EP13795243.8A patent/EP2922955B1/en not_active Not-in-force
- 2013-11-25 MX MX2015006289A patent/MX2015006289A/es unknown
- 2013-11-25 KR KR1020157016841A patent/KR20150088305A/ko not_active Application Discontinuation
- 2013-11-25 WO PCT/EP2013/074552 patent/WO2014080004A1/en active Application Filing
- 2013-11-25 JP JP2015543449A patent/JP6463687B2/ja not_active Expired - Fee Related
- 2013-11-25 CA CA2889993A patent/CA2889993A1/en not_active Abandoned
- 2013-11-25 DK DK13795243.8T patent/DK2922955T3/da active
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2015
- 2015-09-21 HK HK15109264.3A patent/HK1208702A1/xx not_active IP Right Cessation
Also Published As
| Publication number | Publication date |
|---|---|
| WO2014080004A1 (en) | 2014-05-30 |
| JP6463687B2 (ja) | 2019-02-06 |
| DK2922955T3 (da) | 2019-05-13 |
| US20150307886A1 (en) | 2015-10-29 |
| HK1208702A1 (en) | 2016-03-11 |
| JP2016501520A (ja) | 2016-01-21 |
| KR20150088305A (ko) | 2015-07-31 |
| CA2889993A1 (en) | 2014-05-30 |
| MX2015006289A (es) | 2015-12-03 |
| US9725723B2 (en) | 2017-08-08 |
| EP2922955A1 (en) | 2015-09-30 |
| CN104903448A (zh) | 2015-09-09 |
| BR112015012051A2 (pt) | 2017-12-12 |
| EP2922955B1 (en) | 2019-03-06 |
| CN104903448B (zh) | 2017-07-11 |
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