JP2019535789A5 - - Google Patents
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- JP2019535789A5 JP2019535789A5 JP2019528527A JP2019528527A JP2019535789A5 JP 2019535789 A5 JP2019535789 A5 JP 2019535789A5 JP 2019528527 A JP2019528527 A JP 2019528527A JP 2019528527 A JP2019528527 A JP 2019528527A JP 2019535789 A5 JP2019535789 A5 JP 2019535789A5
- Authority
- JP
- Japan
- Prior art keywords
- alkyl
- amino
- alkoxy
- hydroxy
- heterocyclyl
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
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- RODSCGMZJVUSFO-UHFFFAOYSA-N CC(C)N(C)C=C Chemical compound CC(C)N(C)C=C RODSCGMZJVUSFO-UHFFFAOYSA-N 0.000 description 1
- 0 CC1(C)*CCC1 Chemical compound CC1(C)*CCC1 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022108509A JP2022153413A (ja) | 2016-11-28 | 2022-07-05 | Rnaスプライシングを調節する方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662426619P | 2016-11-28 | 2016-11-28 | |
| US62/426,619 | 2016-11-28 | ||
| PCT/US2017/063323 WO2018098446A1 (en) | 2016-11-28 | 2017-11-27 | Methods for modulating rna splicing |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022108509A Division JP2022153413A (ja) | 2016-11-28 | 2022-07-05 | Rnaスプライシングを調節する方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019535789A JP2019535789A (ja) | 2019-12-12 |
| JP2019535789A5 true JP2019535789A5 (https=) | 2021-06-17 |
Family
ID=62195368
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019528527A Pending JP2019535789A (ja) | 2016-11-28 | 2017-11-27 | Rnaスプライシングを調節する方法 |
| JP2022108509A Pending JP2022153413A (ja) | 2016-11-28 | 2022-07-05 | Rnaスプライシングを調節する方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022108509A Pending JP2022153413A (ja) | 2016-11-28 | 2022-07-05 | Rnaスプライシングを調節する方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11702646B2 (https=) |
| EP (1) | EP3544435A4 (https=) |
| JP (2) | JP2019535789A (https=) |
| CN (1) | CN110352007A (https=) |
| AU (1) | AU2017363369A1 (https=) |
| CA (1) | CA3043755A1 (https=) |
| EA (1) | EA201991309A1 (https=) |
| MX (1) | MX2019005588A (https=) |
| WO (1) | WO2018098446A1 (https=) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10668171B2 (en) | 2015-05-30 | 2020-06-02 | Ptc Therapeutics, Inc. | Methods for modulating RNA splicing |
| JP2019535789A (ja) | 2016-11-28 | 2019-12-12 | ピーティーシー セラピューティクス,インコーポレーテッド | Rnaスプライシングを調節する方法 |
| JP2020523365A (ja) | 2017-06-14 | 2020-08-06 | ピーティーシー セラピューティクス,インコーポレーテッド | Rnaスプライシングを改変する方法 |
| JP7312749B2 (ja) | 2017-08-04 | 2023-07-21 | スカイホーク・セラピューティクス・インコーポレーテッド | スプライシングをモジュレートする方法および組成物 |
| US20210355509A1 (en) * | 2018-08-07 | 2021-11-18 | The Children's Hospital Of Philadelphia | Alternative splicing regulation of gene expression and therapeutic methods |
| CN113166779A (zh) * | 2018-10-09 | 2021-07-23 | 北卡罗来纳大学查佩尔希尔分校 | 调控的基因编辑系统 |
| JP7551629B2 (ja) | 2019-02-05 | 2024-09-17 | スカイホーク・セラピューティクス・インコーポレーテッド | スプライシングを調節するための方法および組成物 |
| WO2020163405A1 (en) | 2019-02-05 | 2020-08-13 | Skyhawk Therapeutics, Inc. | Methods and compositions for modulating splicing |
| JP7603595B2 (ja) | 2019-02-06 | 2024-12-20 | スカイホーク・セラピューティクス・インコーポレーテッド | スプライシングを調節するための方法および組成物 |
| JP7603594B2 (ja) | 2019-02-06 | 2024-12-20 | スカイホーク・セラピューティクス・インコーポレーテッド | スプライシングを調節するための方法および組成物 |
| US11129829B2 (en) | 2019-06-17 | 2021-09-28 | Skyhawk Therapeutics, Inc. | Methods for modulating splicing |
| WO2021014428A1 (en) | 2019-07-25 | 2021-01-28 | Novartis Ag | Regulatable expression systems |
| EP4017979A4 (en) * | 2019-08-19 | 2024-03-27 | Stoke Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR MODULATING PROTEIN SPLICING AND EXPRESSION |
| IL293340A (en) | 2019-12-02 | 2022-07-01 | Storm Therapeutics Ltd | Polyheterocyclic compounds as mettl3 inhibitors |
| CA3167756A1 (en) * | 2020-02-04 | 2021-08-12 | Regeneron Pharmaceuticals, Inc. | Treatment of ophthalmic conditions with son of sevenless 2 (sos2) inhibitors |
| EP4103717A4 (en) * | 2020-02-12 | 2024-02-28 | The Children's Hospital of Philadelphia | COMPOSITIONS AND METHODS FOR INDUCIBLE ALTERNATIVE SPLICING REGULATION OF GENE EXPRESSION |
| CN115485025A (zh) | 2020-02-28 | 2022-12-16 | 雷密克斯医疗公司 | 用于调节剪接的化合物和方法 |
| WO2021174176A1 (en) | 2020-02-28 | 2021-09-02 | Remix Therapeutics Inc. | Pyridazine dervatives for modulating nucleic acid splicing |
| WO2021174174A1 (en) | 2020-02-28 | 2021-09-02 | Remix Therapeutics Inc. | Thiophenyl derivatives useful for modulating nucleic acid splicing |
| MX2022010681A (es) | 2020-02-28 | 2023-03-21 | Remix Therapeutics Inc | Compuestos y metodos para modular el empalme. |
| WO2021207530A1 (en) | 2020-04-08 | 2021-10-14 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| MX2022012678A (es) | 2020-04-08 | 2023-01-11 | Remix Therapeutics Inc | Compuestos y metodos para modular el corte y empalme. |
| CA3182912A1 (en) | 2020-05-13 | 2021-11-18 | Chdi Foundation, Inc. | Htt modulators for treating huntington's disease |
| TW202216710A (zh) | 2020-07-02 | 2022-05-01 | 美商雷密克斯醫療公司 | 調節剪接之化合物及方法 |
| MX2023000167A (es) | 2020-07-02 | 2023-05-03 | Remix Therapeutics Inc | Derivados de 5-[5-(piperidin-4-il)tieno[3,2-c]pirazol-2-il]indazol y compuestos relacionados como moduladores para el corte y empalme de ácidos nucleicos y para el tratamiento de enfermedades proliferativas. |
| WO2022214520A1 (en) * | 2021-04-07 | 2022-10-13 | Janssen Sciences Ireland Unlimited Company | Antibacterial compounds |
| CN113267626B (zh) * | 2021-05-17 | 2023-01-13 | 郑州大学第一附属医院 | 一种用于高危人群贲门腺癌早期筛查的试纸条 |
| CN113604566B (zh) * | 2021-07-13 | 2023-05-16 | 中山大学孙逸仙纪念医院 | lncRNA BCYRN1在膀胱癌预后、治疗中的应用 |
| US20250333397A1 (en) | 2021-08-30 | 2025-10-30 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| CA3230256A1 (en) | 2021-08-30 | 2023-03-09 | Dominic Reynolds | Compounds and methods for modulating splicing |
| US20240368163A1 (en) | 2021-08-30 | 2024-11-07 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| WO2023034812A1 (en) | 2021-08-30 | 2023-03-09 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| US20240400584A1 (en) | 2021-08-30 | 2024-12-05 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| TW202330552A (zh) | 2021-10-13 | 2023-08-01 | 美商雷密克斯醫療公司 | 調節剪接之化合物及方法 |
| IL312078A (en) | 2021-10-13 | 2024-06-01 | Remix Therapeutics Inc | Compounds and methods for modulating nucleic acid splicing |
| MX2024005933A (es) | 2021-11-17 | 2024-08-06 | Chdi Foundation Inc | Moduladores de htt pata tratar la enfermedad de huntington. |
| WO2023133217A1 (en) | 2022-01-05 | 2023-07-13 | Remix Therapeutics Inc. | 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases |
| TW202346305A (zh) | 2022-01-05 | 2023-12-01 | 美商雷密克斯醫療公司 | 用於調節剪切之化合物及方法 |
| WO2023133229A2 (en) | 2022-01-05 | 2023-07-13 | Remix Therapeutics Inc. | Compounds and methods for modulating splicing |
| CN118647405A (zh) * | 2022-01-25 | 2024-09-13 | 上海魁特迪生物科技有限公司 | 改善认知障碍的方法 |
| WO2024036143A2 (en) * | 2022-08-09 | 2024-02-15 | Ptc Therapeutics, Inc. | Methods for modulating rna splicing |
Family Cites Families (60)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3558618A (en) | 1968-04-01 | 1971-01-26 | Dow Chemical Co | Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones |
| US4122274A (en) | 1977-05-25 | 1978-10-24 | Bristol-Myers Company | 3-Tetrazolo-5,6,7,8-substituted-pyrido[1,2-a]pyrimidin-4-ones |
| US4342870A (en) | 1980-03-28 | 1982-08-03 | Janssen Pharmaceutica N.V. | Novel 3-(1-piperidinylalkyl)-4H-pyrido[1,2-a]pyrimidin-4-one derivatives |
| JPS56150091A (en) | 1980-03-28 | 1981-11-20 | Janssen Pharmaceutica Nv | 3-(1-piperidinylalkyl)-4h-pyrido(1,2-a)pyrimidine- 4-one derivative and its manufacture |
| US5089633A (en) | 1987-04-28 | 1992-02-18 | Georgia Tech Research Corporation | Substituted isocoumarins |
| US5726182A (en) | 1990-05-02 | 1998-03-10 | Abbott Laboratories | Quinolizinone type compounds |
| AU4231293A (en) | 1992-05-13 | 1993-12-13 | E.I. Du Pont De Nemours And Company | Substituted pyrido(1,2-A)pyrimidinone derivatives as fungicides |
| CA2222322A1 (en) | 1995-06-06 | 1996-12-12 | Abbott Laboratories | Quinolizinone type compounds |
| US5869500A (en) | 1996-12-13 | 1999-02-09 | Hoffmann-La Roche Inc. | Pyridone compounds useful in treating Alzheimer's disease |
| US6630488B1 (en) | 1998-09-21 | 2003-10-07 | Biochem Pharma, Inc. | Quinolizinones as integrin inhibitors |
| AU7961100A (en) | 1999-10-28 | 2001-05-08 | Daiichi Pharmaceutical Co., Ltd. | Drug discharge pump inhibitors |
| EP1857443B1 (en) | 2000-01-24 | 2012-03-28 | AstraZeneca AB | Therapeutic morpholino-substituted compounds |
| WO2002053576A1 (en) | 2001-01-05 | 2002-07-11 | The General Hospital Corporation | Viral delivery system for infectious transfer of large genomic dna inserts |
| JPWO2002087589A1 (ja) | 2001-04-26 | 2004-08-12 | 第一製薬株式会社 | 薬剤排出ポンプ阻害薬 |
| DE60236322D1 (de) | 2001-12-07 | 2010-06-17 | Vertex Pharma | Verbindungen auf pyrimidin-basis als gsk-3-hemmer |
| GB0205281D0 (en) | 2002-03-06 | 2002-04-17 | Novartis Ag | Organic compounds |
| AU2003256755A1 (en) | 2002-07-24 | 2004-02-09 | Ptc Therapeutics, Inc. | Ureido substituted benzoic acid compounds, their use for nonsense suppression and the treatment of diseases caused by such mutations |
| US9068234B2 (en) | 2003-01-21 | 2015-06-30 | Ptc Therapeutics, Inc. | Methods and agents for screening for compounds capable of modulating gene expression |
| WO2004113335A2 (en) | 2003-06-20 | 2004-12-29 | Chiron Corporation | Pyridino[1,2-a]pyrimidin-4-one compounds as anticancer agents |
| ATE373659T1 (de) | 2004-05-04 | 2007-10-15 | Warner Lambert Co | Pyrrolylsubstituierte pyrido(2,3-d)pyrimidin-7- one und derivate davon als therapeutische mittel |
| CA2595477A1 (en) | 2005-01-21 | 2006-07-27 | Janssen Pharmaceutica N.V. | Novel heterocyclic benzo[c]chromene derivatives useful as modulators of the estrogen receptors |
| JP2008539698A (ja) * | 2005-04-29 | 2008-11-20 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 転写後レベルでの核酸発現調節のための方法および組成物 |
| US7563601B1 (en) | 2005-06-01 | 2009-07-21 | City Of Hope | Artificial riboswitch for controlling pre-mRNA splicing |
| WO2006138696A2 (en) | 2005-06-17 | 2006-12-28 | Genizon Biosciences, Inc. | Genemap of the human genes associated with longevity |
| JP2009524406A (ja) | 2005-10-13 | 2009-07-02 | ビーシー キャンサー エージェンシー | 合成生物学および代謝工学のためのモジュラー型ゲノム |
| AR059339A1 (es) | 2006-02-09 | 2008-03-26 | Chugai Pharmaceutical Co Ltd | Derivados de la cumarina para trastornos proliferativos de celulas, composicion farmaceutica y agente terapeutico que los contiene |
| US8110681B2 (en) | 2006-03-17 | 2012-02-07 | The United States Of America As Represented By The Secretary, Department Of Health And Human Services | Compounds for the treatment of spinal muscular atrophy and other uses |
| WO2008013997A2 (en) | 2006-07-27 | 2008-01-31 | The Trustees Of Columbia University In The City Of New York | Fluorescent substrates for monoamine transporters as optical false neurotransmitters |
| KR20090097194A (ko) | 2006-12-22 | 2009-09-15 | 아벡사 리미티드 | 바이사이클 피리미디논 및 그 용도 |
| WO2009151546A2 (en) | 2008-05-27 | 2009-12-17 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
| EP2138493A1 (en) | 2008-06-26 | 2009-12-30 | Sanofi-Aventis | Substituted pyrimidone derivatives |
| CN102143962B (zh) | 2008-07-02 | 2015-03-11 | 爱维艾珂瑟有限公司 | 具有抗病毒特性的化合物 |
| US8932818B2 (en) | 2008-08-13 | 2015-01-13 | Ptc Therapeutics, Inc. | Screening assays for compounds that modulate programmed ribosomal frameshifting |
| CA2750379A1 (en) * | 2009-01-14 | 2010-07-22 | Gordon J. Lutz | Modulation of pre-mrna using splice modulating oligonucleotides as therapeutic agents in the treatment of disease |
| US20100303776A1 (en) | 2009-04-16 | 2010-12-02 | The University Of North Carolina At Chapel Hill | Methods and compositions for regulated expression of multiple nucleic acids |
| WO2011050245A1 (en) | 2009-10-23 | 2011-04-28 | Yangbo Feng | Bicyclic heteroaryls as kinase inhibitors |
| EP2501231B1 (en) | 2009-11-20 | 2016-12-21 | Merck Sharp & Dohme Corp. | Quinolizidinone carboxamide m1 receptor positive allosteric modulators |
| CN102812023A (zh) | 2010-01-13 | 2012-12-05 | 韩国巴斯德研究所 | 抗感染吡啶并(1,2-a)嘧啶类 |
| EP2621491B1 (en) | 2010-09-27 | 2018-08-29 | Emergent Product Development Gaithersburg Inc. | 2-pyridone antimicrobial compositions |
| WO2013059606A1 (en) | 2011-10-21 | 2013-04-25 | Tufts Medical Center, Inc. | Compounds and methods for the treatment of muscular disease, and related screening methods |
| MX352861B (es) | 2011-12-30 | 2017-12-13 | Ptc Therapeutics Inc | Compuestos para tratar la atrofia muscular espinal. |
| MX357834B (es) | 2012-01-26 | 2018-07-26 | Ptc Therapeutics Inc | Compuestos de tipo isocumarina sustituidos para tratar la atrofia muscular espinal. |
| EP2812004B1 (en) * | 2012-02-10 | 2018-06-27 | PTC Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| CA2865957C (en) | 2012-03-01 | 2020-02-11 | F. Hoffmann-La Roche Ag | Compounds for treating spinal muscular atrophy |
| CA2868026C (en) | 2012-03-23 | 2021-02-16 | Ptc Therapeutics, Inc. | Compounds for treating spinal muscular atrophy |
| US9212209B2 (en) | 2012-07-13 | 2015-12-15 | Indiana University Research And Technology Corporation | Screening methods for spinal muscular atrophy |
| US9040712B2 (en) | 2013-01-23 | 2015-05-26 | Novartis Ag | Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions |
| EP3035935B1 (en) | 2013-08-19 | 2020-03-11 | F. Hoffmann-La Roche AG | Compounds for use in the prophylaxis and treatment of cancer |
| WO2015095449A1 (en) | 2013-12-19 | 2015-06-25 | Ptc Therapeutics, Inc. | Methods for modulating the amount rna transcripts |
| US10195202B2 (en) | 2013-12-19 | 2019-02-05 | Ptc Therapeutics, Inc. | Methods for modulating the amount of RNA transcripts |
| WO2015095446A1 (en) | 2013-12-19 | 2015-06-25 | Ptc Therapeutics, Inc. | Methods for modulating the amount of rna transcripts |
| ES2761423T3 (es) * | 2014-05-15 | 2020-05-19 | Hoffmann La Roche | Compuestos para tratar la atrofia muscular espinal |
| WO2016042015A1 (en) | 2014-09-16 | 2016-03-24 | Centre National De La Recherche Scientifique (Cnrs) | Method for evaluating developmental competence of an oocyte |
| EP3256126B1 (en) * | 2015-02-09 | 2024-03-27 | F. Hoffmann-La Roche AG | Compounds for the treatment of cancer |
| CN107635999B (zh) | 2015-05-20 | 2020-09-25 | 豪夫迈·罗氏有限公司 | 用于治疗脊髓性肌萎缩的化合物 |
| US10668171B2 (en) | 2015-05-30 | 2020-06-02 | Ptc Therapeutics, Inc. | Methods for modulating RNA splicing |
| JP2019535789A (ja) | 2016-11-28 | 2019-12-12 | ピーティーシー セラピューティクス,インコーポレーテッド | Rnaスプライシングを調節する方法 |
| JP2020523365A (ja) | 2017-06-14 | 2020-08-06 | ピーティーシー セラピューティクス,インコーポレーテッド | Rnaスプライシングを改変する方法 |
| JP7312749B2 (ja) | 2017-08-04 | 2023-07-21 | スカイホーク・セラピューティクス・インコーポレーテッド | スプライシングをモジュレートする方法および組成物 |
| US20230152257A1 (en) | 2017-09-25 | 2023-05-18 | Skyhawk Therapeutics, Inc. | Methods and compositions for screening and identification of splicing |
-
2017
- 2017-11-27 JP JP2019528527A patent/JP2019535789A/ja active Pending
- 2017-11-27 WO PCT/US2017/063323 patent/WO2018098446A1/en not_active Ceased
- 2017-11-27 MX MX2019005588A patent/MX2019005588A/es unknown
- 2017-11-27 CN CN201780084649.8A patent/CN110352007A/zh active Pending
- 2017-11-27 EP EP17873550.2A patent/EP3544435A4/en not_active Withdrawn
- 2017-11-27 AU AU2017363369A patent/AU2017363369A1/en not_active Abandoned
- 2017-11-27 CA CA3043755A patent/CA3043755A1/en active Pending
- 2017-11-27 US US16/463,775 patent/US11702646B2/en active Active
- 2017-11-27 EA EA201991309A patent/EA201991309A1/ru unknown
-
2022
- 2022-07-05 JP JP2022108509A patent/JP2022153413A/ja active Pending
-
2023
- 2023-05-12 US US18/316,871 patent/US20230272367A1/en not_active Abandoned
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