JP2019535789A5 - - Google Patents

Download PDF

Info

Publication number
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
Application number
JP2019528527A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019535789A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2017/063323 external-priority patent/WO2018098446A1/en
Publication of JP2019535789A publication Critical patent/JP2019535789A/ja
Publication of JP2019535789A5 publication Critical patent/JP2019535789A5/ja
Priority to JP2022108509A priority Critical patent/JP2022153413A/ja
Pending legal-status Critical Current

Links

JP2019528527A 2016-11-28 2017-11-27 Rnaスプライシングを調節する方法 Pending JP2019535789A (ja)

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 (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
EA201991309A1 (ru) 2016-11-28 2019-11-29 Способы модуляции сплайсинга рнк
KR20200017476A (ko) 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. Rna 스플라이싱의 변경 방법
WO2019028440A1 (en) 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICING
US20210355509A1 (en) * 2018-08-07 2021-11-18 The Children's Hospital Of Philadelphia Alternative splicing regulation of gene expression and therapeutic methods
EP3864161A4 (en) * 2018-10-09 2022-11-23 The University of North Carolina at Chapel Hill Regulated gene editing system
WO2020163248A1 (en) 2019-02-04 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN114126613A (zh) 2019-02-05 2022-03-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
EP3920915A4 (en) 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
WO2020163544A1 (en) 2019-02-06 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
CN113661162A (zh) 2019-02-06 2021-11-16 斯基霍克疗法公司 用于调节剪接的方法和组合物
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
EP4004213A1 (en) 2019-07-25 2022-06-01 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
CN115151540A (zh) 2019-12-02 2022-10-04 风暴治疗有限公司 作为mettl3抑制剂的多杂环化合物
CA3167756A1 (en) * 2020-02-04 2021-08-12 Regeneron Pharmaceuticals, Inc. Treatment of ophthalmic conditions with son of sevenless 2 (sos2) inhibitors
CN115605596A (zh) * 2020-02-12 2023-01-13 费城儿童医院(Us) 用于基因表达的诱导型可变剪接调节的组合物和方法
WO2021174167A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CA3169697A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Thiophenyl derivatives useful for modulating nucleic acid splicing
JP7736700B2 (ja) 2020-02-28 2025-09-09 リミックス セラピューティクス インコーポレイテッド 複素環アミド及びスプライシングを調節するためのその使用
MX2022010634A (es) 2020-02-28 2023-01-19 Remix Therapeutics Inc Derivados de piridazina para modular el empalme de acidos nucleicos.
JP2023520916A (ja) 2020-04-08 2023-05-22 リミックス セラピューティクス インコーポレイテッド スプライシングを調整するための化合物及び方法
KR20230005210A (ko) 2020-04-08 2023-01-09 레믹스 테라퓨틱스 인크. 스플라이싱을 조절하기 위한 화합물 및 방법
BR112022023025A2 (pt) 2020-05-13 2023-03-28 Chdi Foundation Inc Moduladores de htt para tratar doença de huntington
EP4175956A1 (en) 2020-07-02 2023-05-10 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
EP4178963A1 (en) 2020-07-02 2023-05-17 Remix Therapeutics Inc. 5-[5-(piperidin-4-yl)thieno[3,2-c]pyrazol-2-yl]indazole derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
WO2022214520A1 (en) * 2021-04-07 2022-10-13 Janssen Sciences Ireland Unlimited Company Antibacterial compounds
CN116087519B (zh) * 2021-05-17 2025-03-14 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标志物及其应用
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
WO2023034836A1 (en) 2021-08-30 2023-03-09 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
US20240368163A1 (en) 2021-08-30 2024-11-07 Remix Therapeutics Inc. Compounds and methods for modulating splicing
IL311132A (en) 2021-08-30 2024-04-01 Remix Therapeutics Inc Splicing Modulation Compounds and Methods
CA3233973A1 (en) 2021-10-13 2023-04-20 Dominic Reynolds Compounds and methods for modulating nucleic acid splicing
WO2023064879A1 (en) 2021-10-13 2023-04-20 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
US12281116B2 (en) 2021-11-17 2025-04-22 Chdi Foundation, Inc. HTT modulators for treating Huntington's disease
US20250326748A1 (en) 2022-01-05 2025-10-23 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US20250109140A1 (en) 2022-01-05 2025-04-03 Remix Theraputics Inc. 5-[5-(piperidin-4-yl)thieno[3,2-c]pyrazol-2-yl]indazole derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
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
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)

* Cited by examiner, † Cited by third party
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
US5599816A (en) 1990-05-02 1997-02-04 Abbott Laboratories Quinolizinone type compounds
EP0640083A1 (en) 1992-05-13 1995-03-01 E.I. Du Pont De Nemours And Company Substituted pyrido 1,2-a]pyrimidinone derivatives as fungicides
KR19990022524A (ko) 1995-06-06 1999-03-25 웨인스톡 스티븐 에프. 퀴놀리지논형화합물
US5869500A (en) 1996-12-13 1999-02-09 Hoffmann-La Roche Inc. Pyridone compounds useful in treating Alzheimer's disease
AU5991699A (en) 1998-09-21 2000-04-10 Biochem Pharma Inc. Quinolizinones as integrin inhibitors
WO2001030757A1 (en) 1999-10-28 2001-05-03 Microcide Pharmaceuticals, Inc. Drug discharge pump inhibitors
CN1891693A (zh) 2000-01-24 2007-01-10 基纳西亚股份有限公司 用于治疗的吗啉代基取代的化合物
US7214515B2 (en) 2001-01-05 2007-05-08 The General Hospital Corporation Viral delivery system for infectious transfer of large genomic DNA inserts
US20050009843A1 (en) 2001-04-26 2005-01-13 Kiyoshi Nakayama Medicine for inhibiting drug elimination pump
ATE466581T1 (de) 2001-12-07 2010-05-15 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
CN1809563A (zh) 2003-06-20 2006-07-26 希龙公司 吡啶并[1,2-a]嘧啶-4-酮化合物用作抗癌药
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
AU2006206446A1 (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
WO2007110695A2 (en) 2005-10-13 2007-10-04 Bc Cancer Agency Modular genomes for synthetic biology and metabolic engineering
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
US8337941B2 (en) 2006-07-27 2012-12-25 The Trustees Of Columbia University In The City Of New York Fluorescent substrates for monoamine transporters as optical false neurotransmitters
JP5208962B2 (ja) 2006-12-22 2013-06-12 アベキサ・リミテッド 二環式ピリミジノンおよびその使用
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
PL2212324T3 (pl) 2008-07-02 2014-11-28 Avexa Ltd Związki posiadające właściwości przeciwwirusowe
WO2010019243A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc. Methods for treating viral infections
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
WO2012047487A1 (en) 2010-09-27 2012-04-12 Evolva Sa 2-pyridone antimicrobial compositions
US9662314B2 (en) 2011-10-21 2017-05-30 Tufts Medical Center, Inc. Compounds and methods for the treatment of muscular disease, and related screening methods
KR102057351B1 (ko) 2011-12-30 2019-12-18 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
PL2809322T3 (pl) 2012-01-26 2019-11-29 Ptc Therapeutics Inc Związki do leczenia zaniku mięśni pochodzenia rdzeniowego
DK2812004T3 (en) * 2012-02-10 2018-10-15 Ptc Therapeutics Inc COMPOUNDS FOR TREATMENT OF SPINAL MUSCLE DROPHY
CN104302181B (zh) 2012-03-01 2017-09-15 Ptc医疗公司 用于治疗脊髓性肌萎缩的化合物
KR102109992B1 (ko) 2012-03-23 2020-05-13 피티씨 테라퓨틱스, 인크. 척수성 근위축증을 치료하기 위한 화합물
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
WO2015024876A2 (en) 2013-08-19 2015-02-26 F. Hoffmann-La Roche Ag Screening method
WO2015095446A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
WO2015095449A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount rna transcripts
EP3082820B1 (en) 2013-12-19 2022-07-20 PTC Therapeutics, Inc. Methods for modulating the amount of rna transcripts
SI3143025T1 (sl) 2014-05-15 2020-01-31 F. Hoffmann-La Roche Ag Spojine za zdravljenje spinalne mišične atrofije
WO2016042015A1 (en) * 2014-09-16 2016-03-24 Centre National De La Recherche Scientifique (Cnrs) Method for evaluating developmental competence of an oocyte
WO2016128343A1 (en) 2015-02-09 2016-08-18 F. Hoffmann-La Roche Ag Compounds for the treatment of cancer
EP3298017B1 (en) 2015-05-20 2019-08-14 H. Hoffnabb-La Roche Ag Compounds for treating spinal muscular atrophy
EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
EA201991309A1 (ru) 2016-11-28 2019-11-29 Способы модуляции сплайсинга рнк
KR20200017476A (ko) 2017-06-14 2020-02-18 피티씨 테라퓨틱스, 인크. Rna 스플라이싱의 변경 방법
WO2019028440A1 (en) 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICING
KR20200057071A (ko) 2017-09-25 2020-05-25 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱 조절제의 스크리닝 및 확인을 위한 방법 및 조성물

Similar Documents

Publication Publication Date Title
JP2019535789A5 (https=)
JP2022153413A5 (https=)
JP6906591B2 (ja) 筋特異的核酸調節エレメント並びにその方法及び使用
JP5823969B2 (ja) 心臓特異的核酸調節因子ならびにこの方法および使用
JP2023093487A (ja) 横隔膜特異的核酸調節エレメントならびにその方法および使用
JP2013509168A5 (https=)
US10053709B2 (en) Insulator to improve gene transfer vectors
PT1501931E (pt) Moléculas de arnnp (snrna) quiméricas portadoras de sequências anti-sentido contra as junções de splicing do gene da distrofina e suas aplicações terapêuticas
JP2015517803A5 (https=)
US10801027B2 (en) Inhibitors of SRSF1 to treat neurodegenerative disorders
JP2020535845A5 (https=)
Shih et al. Exon-and contraction-dependent functions of titin in sarcomere assembly
WO2012049665A1 (en) A modified human u1snrna molecule, a gene encoding for the modified human u1snrna molecule, an expression vector including the gene, and the use thereof in gene therapy
Tanwar et al. MITF is a novel transcriptional regulator of the calcium sensor STIM1: Significance in physiological melanogenesis
Bassett et al. Studies of the murine homolog of the multiple endocrine neoplasia type 1 (MEN1) gene, men1
Chang et al. Structure and expression pattern of teleost caspase recruitment domain (CARD) containing proteins that are potentially involved in NF-κB signalling
ES2991921T3 (es) Terapias basadas en la telomerasa transcriptasa inversa
Dormiani et al. Long-term and efficient expression of human β-globin gene in a hematopoietic cell line using a new site-specific integrating non-viral system
Zenke et al. Development of siRNA expression vector utilizing rock bream β-actin promoter: a potential therapeutic tool against viral infection in fish
Boonanuntanasarn et al. Characterization and organization of the U6 snRNA gene in zebrafish and usage of their promoters to express short hairpin RNA
WO2016104906A1 (ko) 형질전환동물 제작을 위한 형질주입 시스템
JP7854207B2 (ja) 筋強直性ジストロフィー1型治療薬
Skoblov et al. Human RFP2 gene promoter: unique structure and unusual strength
JPWO2005063301A1 (ja) Emt誘導剤
EP3898976A1 (en) Synthetic microrna mimics