JP6174678B2 - 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド - Google Patents
神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド Download PDFInfo
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- JP6174678B2 JP6174678B2 JP2015506930A JP2015506930A JP6174678B2 JP 6174678 B2 JP6174678 B2 JP 6174678B2 JP 2015506930 A JP2015506930 A JP 2015506930A JP 2015506930 A JP2015506930 A JP 2015506930A JP 6174678 B2 JP6174678 B2 JP 6174678B2
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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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- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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- C12N2310/32—Chemical structure of the sugar
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- C12N2310/333—Modified A
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
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- C12N2310/3341—5-Methylcytosine
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/33—Chemical structure of the base
- C12N2310/336—Modified G
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
- C12N2310/3521—Methyl
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- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- General Chemical & Material Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Pain & Pain Management (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261636914P | 2012-04-23 | 2012-04-23 | |
| US61/636,914 | 2012-04-23 | ||
| EP12165139.2 | 2012-04-23 | ||
| EP12165139 | 2012-04-23 | ||
| PCT/NL2013/050306 WO2013162363A1 (en) | 2012-04-23 | 2013-04-23 | RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017133005A Division JP2018027080A (ja) | 2012-04-23 | 2017-07-06 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2015514418A JP2015514418A (ja) | 2015-05-21 |
| JP2015514418A5 JP2015514418A5 (enExample) | 2016-06-16 |
| JP6174678B2 true JP6174678B2 (ja) | 2017-08-02 |
Family
ID=49483552
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015506930A Active JP6174678B2 (ja) | 2012-04-23 | 2013-04-23 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2017133005A Pending JP2018027080A (ja) | 2012-04-23 | 2017-07-06 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2020115868A Active JP7459691B2 (ja) | 2012-04-23 | 2020-07-03 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2022093254A Pending JP2022126711A (ja) | 2012-04-23 | 2022-06-08 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2025061303A Pending JP2025111478A (ja) | 2012-04-23 | 2025-04-02 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017133005A Pending JP2018027080A (ja) | 2012-04-23 | 2017-07-06 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2020115868A Active JP7459691B2 (ja) | 2012-04-23 | 2020-07-03 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2022093254A Pending JP2022126711A (ja) | 2012-04-23 | 2022-06-08 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2025061303A Pending JP2025111478A (ja) | 2012-04-23 | 2025-04-02 | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
Country Status (19)
| Country | Link |
|---|---|
| US (5) | US9745576B2 (enExample) |
| EP (4) | EP3228712A1 (enExample) |
| JP (5) | JP6174678B2 (enExample) |
| CN (2) | CN110025628B (enExample) |
| AU (1) | AU2013253074C1 (enExample) |
| BR (1) | BR112014026285B1 (enExample) |
| CA (2) | CA2870697C (enExample) |
| DK (1) | DK2841578T3 (enExample) |
| ES (2) | ES2639066T3 (enExample) |
| HR (1) | HRP20171254T1 (enExample) |
| HU (1) | HUE033431T2 (enExample) |
| LT (1) | LT2841578T (enExample) |
| NZ (1) | NZ700561A (enExample) |
| PL (2) | PL2841578T3 (enExample) |
| PT (1) | PT2841578T (enExample) |
| RS (1) | RS56319B1 (enExample) |
| SI (1) | SI2841578T1 (enExample) |
| SM (1) | SMT201700339T1 (enExample) |
| WO (1) | WO2013162363A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2020182472A (ja) * | 2012-04-23 | 2020-11-12 | バイオマリン テクノロジーズ ベー.フェー. | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
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| DK2049664T3 (da) | 2006-08-11 | 2012-01-02 | Prosensa Technologies Bv | Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser |
| EP2614827B1 (en) | 2007-10-26 | 2017-06-28 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
| 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 |
| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| WO2011005761A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc | Novel nucleic acid prodrugs and methods use thereof |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| WO2012138289A1 (en) * | 2011-04-08 | 2012-10-11 | Zain-Luqman Rula | Diagnosis and treatment of friedreich's ataxia |
| EP2734208B1 (en) | 2011-07-19 | 2017-03-01 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| 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 |
| AU2013288048A1 (en) | 2012-07-13 | 2015-01-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| WO2014062686A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for modulating c9orf72 expression |
| BR112015008399A8 (pt) * | 2012-10-15 | 2017-10-03 | Ionis Pharmaceuticals Inc | Composto para modular a expressão de c90rf72, seu uso, oligonucleotídeo modificado, composto de fita dupla e composição |
| WO2014062736A1 (en) * | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
| EP3055414A4 (en) | 2013-10-11 | 2017-07-19 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| CN113278617A (zh) | 2014-01-16 | 2021-08-20 | 波涛生命科学有限公司 | 手性设计 |
| US10006027B2 (en) | 2014-03-19 | 2018-06-26 | Ionis Pharmaceuticals, Inc. | Methods for modulating Ataxin 2 expression |
| CA2942340A1 (en) | 2014-03-19 | 2015-09-24 | Ionis Pharmaceuticals, Inc. | Compositions for modulating ataxin 2 expression |
| US10538762B2 (en) | 2014-10-14 | 2020-01-21 | The Board Of Regents Of The University Of Texas System | Allele selective inhibition of mutant C9orf72 foci expression by duplex RNAS targeting the expanded hexanucleotide repeat |
| WO2016168592A2 (en) * | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| WO2017079291A1 (en) | 2015-11-02 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating c90rf72 |
| EP3397288A4 (en) | 2015-12-31 | 2019-09-11 | Ionis Pharmaceuticals, Inc. | PROCESS FOR REDUCING ATAXIN-2 EXPRESSION |
| US20180028554A1 (en) * | 2016-07-05 | 2018-02-01 | Biomarin Technologies B.V. | Oligomers Having Bicyclic Scaffold Moeities |
| CN118416088A (zh) | 2017-03-03 | 2024-08-02 | 加利福尼亚大学董事会 | 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 |
| CA3083582A1 (en) * | 2017-10-23 | 2019-05-02 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
| EA202190299A1 (ru) | 2018-07-25 | 2021-06-11 | Ионис Фармасьютикалз, Инк. | Соединения и способы для снижения экспрессии atxn2 |
| US12370264B1 (en) | 2018-08-02 | 2025-07-29 | Dyne Therapeutics, Inc. | Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject |
| US12097263B2 (en) | 2018-08-02 | 2024-09-24 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| JP7611825B2 (ja) | 2018-08-02 | 2025-01-10 | ダイン セラピューティクス,インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体およびそれらの使用 |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11911484B2 (en) | 2018-08-02 | 2024-02-27 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating myotonic dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| MX2022004388A (es) * | 2019-11-01 | 2022-05-06 | Alnylam Pharmaceuticals Inc | Composiciones de agentes de arni de huntingtina (htt) y metodos de uso de las mismas. |
| CN115038789A (zh) | 2019-12-02 | 2022-09-09 | 塑造治疗公司 | 治疗性编辑 |
| BR112022021333A2 (pt) * | 2020-05-01 | 2022-12-13 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular atxn1 |
| JP2023542889A (ja) | 2020-09-17 | 2023-10-12 | キュー-ステート バイオサイエンシーズ, インコーポレイテッド | 複数の標的を介して疼痛を処置するための治療用組成物 |
| US20240209377A1 (en) * | 2021-04-28 | 2024-06-27 | Q-State Biosciences, Inc. | Therapeutic compositions for treating pain via multiple targets |
| AU2022309028A1 (en) | 2021-07-09 | 2024-01-25 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
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| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| AR127158A1 (es) * | 2021-09-29 | 2023-12-27 | Hoffmann La Roche | Oligonucléotido antisentido para la edición de arn asociado a enfermedades de poliglutamina |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| JP2020182472A (ja) * | 2012-04-23 | 2020-11-12 | バイオマリン テクノロジーズ ベー.フェー. | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP2022126711A (ja) * | 2012-04-23 | 2022-08-30 | ヴィコ セラピューティクス ビー.ヴイ. | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| JP7459691B2 (ja) | 2012-04-23 | 2024-04-02 | ヴィコ セラピューティクス ビー.ヴイ. | 神経筋障害の治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
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