RU2013114396A - Антисмысловые нуклеиновые кислоты - Google Patents
Антисмысловые нуклеиновые кислоты Download PDFInfo
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- 150000007523 nucleic acids Chemical class 0.000 title 1
- 102000039446 nucleic acids Human genes 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 title 1
- 230000000692 anti-sense effect Effects 0.000 claims abstract 19
- 239000002773 nucleotide Substances 0.000 claims abstract 13
- 125000003729 nucleotide group Chemical group 0.000 claims abstract 13
- 108091034117 Oligonucleotide Proteins 0.000 claims abstract 10
- 150000001875 compounds Chemical group 0.000 claims abstract 6
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 claims abstract 6
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 claims abstract 4
- PYMYPHUHKUWMLA-LMVFSUKVSA-N Ribose Natural products OC[C@@H](O)[C@@H](O)[C@@H](O)C=O PYMYPHUHKUWMLA-LMVFSUKVSA-N 0.000 claims abstract 2
- 125000000217 alkyl group Chemical group 0.000 claims abstract 2
- 125000002947 alkylene group Chemical group 0.000 claims abstract 2
- HMFHBZSHGGEWLO-UHFFFAOYSA-N alpha-D-Furanose-Ribose Natural products OCC1OC(O)C(O)C1O HMFHBZSHGGEWLO-UHFFFAOYSA-N 0.000 claims abstract 2
- 125000003118 aryl group Chemical group 0.000 claims abstract 2
- 230000000295 complement effect Effects 0.000 claims abstract 2
- 125000000548 ribosyl group Chemical group C1([C@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims abstract 2
- 201000006938 muscular dystrophy Diseases 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 239000000126 substance Substances 0.000 claims 2
- WROTXLSEMHAZEA-UHFFFAOYSA-N 4-diaminophosphoryloxymorpholine Chemical compound NP(N)(=O)ON1CCOCC1 WROTXLSEMHAZEA-UHFFFAOYSA-N 0.000 claims 1
- 239000004480 active ingredient Substances 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- 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
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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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
- C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/32—Chemical structure of the sugar
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Abstract
1. Антисмысловой олигомер, который является причиной пропуска 53-ого экзона в гене дистрофина человека, состоящий из нуклеотидной последовательности, комплементарной любой из последовательностей, состоящей из нуклеотидов с 32-ого по 56-ой или с 36-ого по 56-ой с 5'-конца 53-ого экзона в гене дистрофина человека.2. Антисмысловой олигомер по п. 1, который представляет собой олигонуклеотид.3. Антисмысловой олигомер по п. 2, в случае которого сахарная составляющая и/или область соединения через фосфатную группу по крайней мере одного нуклеотида, составляющего олигонуклеотид, является модифицированной.4. Антисмысловой олигомер по п. 3, в случае которого сахарной составляющей по крайней мере одного нуклеотида, составляющего олигонуклеотид, является рибоза, в которой группа 2'-ОН заменена любой группой, выбираемой из группы, состоящей из OR, R, R'OR, SH, SR, NH, NHR, NR, N, CN, F, Cl, Br и I (где R представляет собой алкил или арил, а R' представляет собой алкилен).5. Антисмысловой олигомер по п. 3, в случае которого областью соединения через фосфатную группу по крайней мере одного нуклеотида, составляющего олигонуклеотид, является любая область, выбираемая из группы, состоящей из фосфоротиоатной связи, фосфородитиоатной связи, алкилфосфонатной связи, фосфорамидатной связи и боранофосфатной связи.6. Антисмысловой олигомер по п. 4, в случае которого областью соединения через фосфатную группу по крайней мере одного нуклеотида, составляющего олигонуклеотид, является любая область, выбираемая из группы, состоящей из фосфоротиоатной связи, фосфородитиоатной связи, алкилфосфонатной связи, фосфорамидатной связи и боранофосфатной связи.7. Антисмысловой олиго
Claims (13)
1. Антисмысловой олигомер, который является причиной пропуска 53-ого экзона в гене дистрофина человека, состоящий из нуклеотидной последовательности, комплементарной любой из последовательностей, состоящей из нуклеотидов с 32-ого по 56-ой или с 36-ого по 56-ой с 5'-конца 53-ого экзона в гене дистрофина человека.
2. Антисмысловой олигомер по п. 1, который представляет собой олигонуклеотид.
3. Антисмысловой олигомер по п. 2, в случае которого сахарная составляющая и/или область соединения через фосфатную группу по крайней мере одного нуклеотида, составляющего олигонуклеотид, является модифицированной.
4. Антисмысловой олигомер по п. 3, в случае которого сахарной составляющей по крайней мере одного нуклеотида, составляющего олигонуклеотид, является рибоза, в которой группа 2'-ОН заменена любой группой, выбираемой 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, а R' представляет собой алкилен).
5. Антисмысловой олигомер по п. 3, в случае которого областью соединения через фосфатную группу по крайней мере одного нуклеотида, составляющего олигонуклеотид, является любая область, выбираемая из группы, состоящей из фосфоротиоатной связи, фосфородитиоатной связи, алкилфосфонатной связи, фосфорамидатной связи и боранофосфатной связи.
6. Антисмысловой олигомер по п. 4, в случае которого областью соединения через фосфатную группу по крайней мере одного нуклеотида, составляющего олигонуклеотид, является любая область, выбираемая из группы, состоящей из фосфоротиоатной связи, фосфородитиоатной связи, алкилфосфонатной связи, фосфорамидатной связи и боранофосфатной связи.
7. Антисмысловой олигомер по п. 1, который представляет собой морфолино олигомер.
8. Антисмысловой олигомер по п. 7, который представляет собой морфолино-фосфородиамидатный олигомер.
9. Антисмысловой олигомер по п. 7, в случае которого 5'-концом является любая из групп с химическими формулами (1)-(3) ниже:
10. Антисмысловой олигомер по п. 8, в случае которого 5'-концом является любая из групп с химическими формулами (1)-(3) ниже:
11. Антисмысловой олигомер по любому из пп. 1-10, состоящий из нуклеотидной последовательности, представленной SEQ ID NO: 11 или 35.
12. Фармацевтическая композиция для лечения мышечной дистрофии, включающая в качестве активного ингредиента антисмысловой олигомер по любому из пп. 1-11 или его фармацевтически приемлемую соль или гидрат.
13. Фармацевтическая композиция для лечения мышечной дистрофии по п. 12, которую вводят для лечения мышечной дистрофии Дюшенна.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2010196032 | 2010-09-01 | ||
| JP2010-196032 | 2010-09-01 | ||
| PCT/JP2011/070318 WO2012029986A1 (ja) | 2010-09-01 | 2011-08-31 | アンチセンス核酸 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| RU2013114396A true RU2013114396A (ru) | 2014-10-10 |
| RU2567664C2 RU2567664C2 (ru) | 2015-11-10 |
Family
ID=45773054
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2013114396/10A RU2567664C2 (ru) | 2010-09-01 | 2011-08-31 | Антисмысловые нуклеиновые кислоты |
Country Status (21)
| Country | Link |
|---|---|
| US (15) | US9079934B2 (ru) |
| EP (6) | EP4403632A3 (ru) |
| JP (11) | JP5363655B2 (ru) |
| KR (1) | KR101310569B1 (ru) |
| CN (1) | CN103154245B (ru) |
| AU (1) | AU2011296882B2 (ru) |
| CA (1) | CA2809637C (ru) |
| CY (2) | CY1117367T1 (ru) |
| DK (2) | DK2612917T3 (ru) |
| ES (2) | ES2567411T3 (ru) |
| HR (2) | HRP20160336T1 (ru) |
| HU (2) | HUE046364T2 (ru) |
| LT (1) | LT3018211T (ru) |
| PL (2) | PL2612917T3 (ru) |
| PT (1) | PT3018211T (ru) |
| RS (2) | RS54649B1 (ru) |
| RU (1) | RU2567664C2 (ru) |
| SI (2) | SI2612917T1 (ru) |
| SM (1) | SMT201600111B (ru) |
| TW (1) | TWI541024B (ru) |
| WO (1) | WO2012029986A1 (ru) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2730681C2 (ru) * | 2014-03-12 | 2020-08-24 | Ниппон Синяку Ко., Лтд. | Антисмысловые нуклеиновые кислоты |
Families Citing this family (61)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| EP2206781B1 (en) | 2004-06-28 | 2015-12-02 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| WO2006086667A2 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| CN101896186A (zh) | 2007-10-26 | 2010-11-24 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| US8084601B2 (en) | 2008-09-11 | 2011-12-27 | Royal Holloway And Bedford New College Royal Holloway, University Of London | Oligomers |
| SI3133160T1 (sl) | 2008-10-24 | 2019-05-31 | Sarepta Therapeutics, Inc. | Sestavki, ki preskakujejo ekson za DMD |
| ES2693459T3 (es) | 2009-11-12 | 2018-12-11 | The University Of Western Australia | Moléculas antisentido y métodos para el tratamiento de patologías |
| TWI541024B (zh) * | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| EP4043039A1 (en) | 2012-01-27 | 2022-08-17 | BioMarin Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| EP3885439A1 (en) * | 2012-12-20 | 2021-09-29 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| MX366274B (es) * | 2013-03-14 | 2019-07-04 | Sarepta Therapeutics Inc | Composiciones para el salto del exón para el tratamiento de distrofia muscular. |
| EP3633035A1 (en) | 2013-03-14 | 2020-04-08 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| BR112015022998A2 (pt) | 2013-03-15 | 2017-11-14 | Sarepta Therapeutics Inc | composições melhoradas para o tratamento de distrofia muscular |
| EP3146970B1 (en) * | 2014-05-19 | 2022-11-02 | KNC Laboratories Co., Ltd. | Nucleic acid drug for inducing skipping of variant exon of cd44 gene and increasing expression of normal type cd44 mrna |
| MX2016016526A (es) * | 2014-06-17 | 2017-04-04 | Nippon Shinyaku Co Ltd | Acidos nucleicos antisentido. |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| WO2017047707A1 (ja) | 2015-09-15 | 2017-03-23 | 日本新薬株式会社 | アンチセンス核酸 |
| US10563199B2 (en) * | 2015-09-16 | 2020-02-18 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid for treating amyotrophy |
| AU2016334232B2 (en) | 2015-10-09 | 2022-05-26 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| CN108699555A (zh) | 2015-10-09 | 2018-10-23 | 萨勒普塔医疗公司 | 用于治疗杜兴肌营养不良和相关病症的组合物和方法 |
| FR3044926B1 (fr) | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
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