JPWO2020005877A5 - - Google Patents

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JPWO2020005877A5
JPWO2020005877A5 JP2020572894A JP2020572894A JPWO2020005877A5 JP WO2020005877 A5 JPWO2020005877 A5 JP WO2020005877A5 JP 2020572894 A JP2020572894 A JP 2020572894A JP 2020572894 A JP2020572894 A JP 2020572894A JP WO2020005877 A5 JPWO2020005877 A5 JP WO2020005877A5
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methyl
thiazolo
benzothiazole
fluoro
indazole
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JP2021528466A (ja
JP7515415B2 (ja
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JP2020572894A 2018-06-27 2019-06-25 ハンチントン病を処置するためのヘテロアリール化合物 Active JP7515415B2 (ja)

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JP2024072881A JP2024105367A (ja) 2018-06-27 2024-04-26 ハンチントン病を処置するためのヘテロアリール化合物

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US201862690540P 2018-06-27 2018-06-27
US62/690,540 2018-06-27
PCT/US2019/038895 WO2020005877A1 (en) 2018-06-27 2019-06-25 Heteroaryl compounds for treating huntington's disease

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JP2021528466A JP2021528466A (ja) 2021-10-21
JPWO2020005877A5 true JPWO2020005877A5 (enExample) 2022-06-21
JP7515415B2 JP7515415B2 (ja) 2024-07-12

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JP2020572894A Active JP7515415B2 (ja) 2018-06-27 2019-06-25 ハンチントン病を処置するためのヘテロアリール化合物
JP2024072881A Abandoned JP2024105367A (ja) 2018-06-27 2024-04-26 ハンチントン病を処置するためのヘテロアリール化合物

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US (1) US12139499B2 (enExample)
EP (1) EP3814345B8 (enExample)
JP (2) JP7515415B2 (enExample)
KR (1) KR20210038845A (enExample)
CN (1) CN112805280B (enExample)
AU (2) AU2019294482B2 (enExample)
BR (1) BR112020026534A2 (enExample)
CA (1) CA3104516A1 (enExample)
EA (1) EA202092899A1 (enExample)
IL (1) IL279714A (enExample)
MX (1) MX2020014315A (enExample)
SG (1) SG11202012869SA (enExample)
WO (1) WO2020005877A1 (enExample)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108697709A (zh) 2015-12-10 2018-10-23 Ptc医疗公司 用于治疗亨廷顿病的方法
AU2018282154B2 (en) 2017-06-05 2022-04-07 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
WO2019005993A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
CN111182898B (zh) 2017-06-28 2024-04-16 Ptc医疗公司 用于治疗亨廷顿氏病的方法
MX2020001425A (es) 2017-08-04 2020-08-06 Skyhawk Therapeutics Inc Metodos y composiciones para modular el empalme.
BR112020019373A2 (pt) 2018-03-27 2020-12-29 Ptc Therapeutics, Inc. Compostos para o tratamento da doença de hutington
EP3814360B8 (en) 2018-06-27 2024-11-06 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
IL279688B2 (en) 2018-06-27 2025-01-01 Ptc Therapeutics Inc Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease
WO2020005877A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
CN114007613A (zh) 2019-02-05 2022-02-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
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
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
JP2023500251A (ja) 2019-11-01 2023-01-05 ノバルティス アーゲー ハンチントン病の進行を遅らせる処置のためのスプライシングモジュレーターの使用
US20230322754A1 (en) * 2020-04-28 2023-10-12 Kymera Therapeutics, Inc. Irak inhibitors and uses thereof
AU2021272972A1 (en) 2020-05-13 2022-12-08 Chdi Foundation, Inc. HTT modulators for treating Huntington's disease
IL299543A (en) * 2020-07-02 2023-02-01 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
JP2023532331A (ja) * 2020-07-02 2023-07-27 リミックス セラピューティクス インコーポレイテッド 核酸をスプライシングするため及び増殖性疾患を処置するための修飾因子としての5-[5-(ピペリジン-4-イル)チエノ[3,2-c]ピラゾール-2-イル]インダゾール誘導体及び関連する化合物並びにスプライシングを調節するための化合物及び方法
WO2022194802A1 (en) * 2021-03-17 2022-09-22 F. Hoffmann-La Roche Ag New thiadiazolopyrimidone derivatives
EP4308572B1 (en) * 2021-03-17 2025-04-23 F. Hoffmann-La Roche AG New thienopyrimidinone derivatives
JP2024514421A (ja) * 2021-03-17 2024-04-02 エフ. ホフマン-ラ ロシュ アーゲー 新規チアゾロピリミジノン誘導体
TW202304446A (zh) 2021-03-29 2023-02-01 瑞士商諾華公司 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途
AU2022394333A1 (en) 2021-11-17 2024-06-27 Chdi Foundation, Inc. HTT modulators for treating Huntington’s disease
CN116262758B (zh) * 2021-12-15 2025-02-28 上海博悦生物科技有限公司 7-甲基噻唑并[5,4-d]嘧啶类化合物、制备方法及其用途
JP2025525683A (ja) * 2022-05-20 2025-08-06 バイオジェン・エムエイ・インコーポレイテッド ハンチントン病を治療するための複素環式化合物
KR20240152239A (ko) * 2023-04-11 2024-10-21 보로노이 주식회사 피라졸로피리딘 유도체 화합물 및 이의 용도
WO2024218207A1 (en) * 2023-04-20 2024-10-24 F. Hoffmann-La Roche Ag Thieno[3,2-b]pyridine derivatives
WO2025111408A1 (en) * 2023-11-21 2025-05-30 Biogen Ma Inc. Compounds for treating spinal muscular atrophy and huntington's disease

Family Cites Families (207)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BE637271A (enExample) 1963-04-04 1900-01-01
US3558618A (en) 1968-04-01 1971-01-26 Dow Chemical Co Novel 4h-pyrazino(1,2-a)pyrimidine-4-ones
GB1383409A (en) 1972-09-09 1974-02-12 Pfizer Ltd Derivatives of 2-amino- and 4-amino-quinazoline and pharmaceutical compositions containing them
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
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
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
FR2567518B1 (fr) 1984-07-11 1987-11-13 Sanofi Sa Nouveaux composes a noyau heterocyclique azote, leur preparation et les medicaments qui en contiennent
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
WO1993023398A1 (en) 1992-05-13 1993-11-25 E.I. Du Pont De Nemours And Company Substituted pyrido[1,2-a]pyrimidinone derivatives as fungicides
PT698092E (pt) 1993-05-11 2007-10-29 Univ North Carolina Oligonucleótidos complementares de uma cadeia codificadora que combatem splicing aberrante e métodos para a sua utilização
CA2222322A1 (en) 1995-06-06 1996-12-12 Abbott Laboratories Quinolizinone type compounds
US5916916A (en) 1996-10-10 1999-06-29 Eli Lilly And Company 1-aryloxy-2-arylnaphthyl compounds, intermediates, compositions, and methods
US5869500A (en) 1996-12-13 1999-02-09 Hoffmann-La Roche Inc. Pyridone compounds useful in treating Alzheimer's disease
CA2269561C (en) 1998-04-22 2007-06-05 Dainippon Ink And Chemicals, Inc. Naphthalene derivative and liquid crystal composition comprising the same
WO2000017197A1 (en) 1998-09-21 2000-03-30 Biochem Pharma Inc. Quinolizinones as integrin inhibitors
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
ATE312820T1 (de) 1999-10-28 2005-12-15 Trine Pharmaceuticals Inc Pumpeninhibitoren zur freisetzung von medikamenten
DE60128655T2 (de) 2000-01-24 2008-02-07 Astrazeneca Ab Durch einen morpholinrest substituierte therapeutische verbindungen
US20050054836A1 (en) 2000-11-09 2005-03-10 Cold Spring Harbor Laboratory Chimeric molecules to modulate gene expression
US6774134B2 (en) 2000-12-20 2004-08-10 Bristol-Myers Squibb Company Heterocyclic substituted 2-methyl-benzimidazole antiviral agents
HUP0400639A3 (en) 2000-12-21 2010-03-29 Vertex Pharma Pyrazole compounds useful as protein kinase inhibitors and pharmaceutical compositions containing them
CA2445697A1 (en) 2001-04-26 2002-11-07 Daiichi Pharmaceutical Co., Ltd. Drug efflux pump inhibitor
MXPA04005510A (es) 2001-12-07 2006-02-24 Vertex Pharma Compuestos basados en pirimidina utiles como inhibidores de gsk-3.
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
ATE548354T1 (de) 2002-07-24 2012-03-15 Ptc Therapeutics Inc Ureido-substituierte benzoesäureverbindungen und ihre verwendung für die nonsense-suppression und behandlung von erkrankungen
AU2003262833A1 (en) 2002-08-23 2004-03-11 The Board Of Trustees Of The Leland Stanford Junior University Fluorescent glycosides and methods for their use
PL375025A1 (en) 2002-09-30 2005-11-14 Neurosearch A/S Novel 1,4-diazabicycloalkane derivatives, their preparation and use
CA2765093A1 (en) 2002-11-12 2004-05-27 Abbott Laboratories Bicyclic-substituted amines as histamine-3 receptor ligands
US20070078144A1 (en) 2003-01-29 2007-04-05 Stockwell Brent R Agents for treating neurodegenerative diseases
US20040224952A1 (en) 2003-05-07 2004-11-11 Cowart Marlon D. Fused bicyclic-substituted amines as histamine-3 receptor ligands
AU2004249730A1 (en) 2003-06-20 2004-12-29 Novartis Vaccines And Diagnostics, Inc. Pyridino(1,2-A)pyrimidin-4-one compounds as anticancer agents
GB0315494D0 (en) 2003-07-02 2003-08-06 Biofocus Plc Compounds which bind to the active site of protein kinase enzymes
CN1829709A (zh) 2003-08-01 2006-09-06 健亚生物科技公司 对抗黄病毒的双环咪唑衍生物
US7160888B2 (en) 2003-08-22 2007-01-09 Warner Lambert Company Llc [1,8]naphthyridin-2-ones and related compounds for the treatment of schizophrenia
US20050074801A1 (en) 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
US7309699B2 (en) 2003-12-22 2007-12-18 Abbott Laboratories 3-Quinuclidinyl amino-substituted biaryl derivatives
US20050245531A1 (en) 2003-12-22 2005-11-03 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US7655657B2 (en) 2003-12-22 2010-02-02 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US20050137203A1 (en) 2003-12-22 2005-06-23 Jianguo Ji 3-quinuclidinyl amino-substituted biaryl derivatives
ES2360222T3 (es) 2003-12-24 2011-06-02 Biota Scientific Management Pty. Ltd. Agentes policíclicos para el tratamiento de infecciones respiratorias por virus sincicial.
SE0400184D0 (sv) 2004-01-30 2004-01-30 Fyrkloevern Scandinavia Ab New therapeutical use
TW200536830A (en) 2004-02-06 2005-11-16 Chugai Pharmaceutical Co Ltd 1-(2H)-isoquinolone derivative
ZA200609267B (en) 2004-04-08 2009-02-25 Targegen Inc Benzotriazine inhibitors of kinases
AU2005232672A1 (en) 2004-04-08 2005-10-27 Neurogen Corporation Substituted cinnolin-4-ylamines
DE602005002562T2 (de) 2004-05-04 2008-01-31 Warner-Lambert Company Llc Pyrrolylsubstituierte pyridoä2,3-düpyrimidin-7-one und derivate davon als therapeutische mittel
US7399767B2 (en) 2005-01-21 2008-07-15 Ortho-Mcneil Pharmaceutical, Inc. Heterocyclic benzo[c]chromene derivatives useful as modulators of the estrogen receptors
US7879992B2 (en) 2005-01-31 2011-02-01 Isis Pharmaceuticals, Inc. Modification of MyD88 splicing using modified oligonucleotides
GB0501999D0 (en) 2005-02-01 2005-03-09 Sentinel Oncology Ltd Pharmaceutical compounds
JP2006219453A (ja) 2005-02-14 2006-08-24 Tokyo Univ Of Pharmacy & Life Science キノリン環を母核とする金属識別型二波長性蛍光分子
US7563601B1 (en) 2005-06-01 2009-07-21 City Of Hope Artificial riboswitch for controlling pre-mRNA splicing
WO2006138418A2 (en) 2005-06-14 2006-12-28 President And Fellows Of Harvard College Improvement of cognitive performance with sirtuin activators
BRPI0613564A2 (pt) 2005-07-04 2011-01-18 Novo Nordisk As compostos, seus usos na preparação de composições farmacêuticas e composições farmacêuticas compreendendo os mesmos
US7473784B2 (en) * 2005-08-01 2009-01-06 Bristol-Myers Squibb Company Benzothiazole and azabenzothiazole compounds useful as kinase inhibitors
US7576110B2 (en) 2005-09-22 2009-08-18 Abbott Laboratories Benzothiazole cyclobutyl amine derivatives
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
US20070179123A1 (en) 2005-11-08 2007-08-02 Chiang Peter K Methods and compositions for treating diseases associated with pathogenic proteins
KR101789603B1 (ko) 2005-11-10 2017-11-21 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 Tnf 수퍼패밀리 수용체에 대한 스플라이스 스위칭올리고머 및 염증성 질환 치료용 약제학적 조성물
EP1963323B1 (en) 2005-12-06 2010-07-14 Neurosearch A/S Novel diazabicyclic aryl derivatives and their medical use
AU2006329202A1 (en) 2005-12-21 2007-06-28 Painceptor Pharma Corporation Compositions and methods for modulating gated ion channels
EP2161038B1 (en) 2006-01-26 2013-12-25 Isis Pharmaceuticals, Inc. Compositions and their uses directed to Huntingtin
EP2388327A1 (en) 2006-01-27 2011-11-23 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
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
KR101280333B1 (ko) * 2006-03-28 2013-07-02 하이 포인트 파마슈티칼스, 엘엘씨 히스타민 h3 수용체 활성을 갖는 벤조티아졸
WO2007130383A2 (en) 2006-04-28 2007-11-15 Northwestern University Compositions and treatments using pyridazine compounds and secretases
US20080247964A1 (en) 2006-05-08 2008-10-09 Yuelian Xu Substituted azaspiro derivatives
TW200812588A (en) 2006-05-15 2008-03-16 Neurogen Corp CRF1 receptor ligands comprising heteroaryl fused bicycles
JP2009537599A (ja) 2006-05-23 2009-10-29 ノイロサーチ アクティーゼルスカブ 新規8,10−ジアザ−ビシクロ[4.3.1]デカン誘導体及びそれらの医学的使用
CA2658462C (en) 2006-07-20 2011-09-27 Amgen Inc. Substituted pyridone compounds and methods of use
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
EP2066653B1 (en) 2006-08-03 2012-09-12 Rottapharm S.p.A. 6-1h-imidazo-quinazoline and quinolines derivatives, new potent analgesics and anti-inflammatory agents
WO2008020302A2 (en) 2006-08-17 2008-02-21 Pfizer Products Inc. Heteroaromatic quinoline-based compounds as phosphodiesterase (pde) inhibitors
JP2010507619A (ja) 2006-10-25 2010-03-11 ノイロサーチ アクティーゼルスカブ オキサジアゾール及びチアジアゾール化合物並びにニコチン性アセチルコリン受容体調節物質としてのその使用
US8314119B2 (en) 2006-11-06 2012-11-20 Abbvie Inc. Azaadamantane derivatives and methods of use
WO2008077188A1 (en) 2006-12-22 2008-07-03 Avexa Limited Bicyclic pyrimidinones and uses thereof
US20080171792A1 (en) 2006-12-28 2008-07-17 Jobdevairakkam Christopher New Use of highly concentrated formulations of 4-phenylbutyrate for treatment of certain disorders
FR2914188B1 (fr) 2007-03-28 2012-06-22 Trophos Nouvelle composition a base d'oxime de cholest-4-en-3-one
EP2014656A3 (en) 2007-06-11 2011-08-24 High Point Pharmaceuticals, LLC New heteocyclic h3 antagonists
SI2193133T1 (sl) 2007-09-27 2015-12-31 Fundacion Centro Nacional De Investigaciones Oncologicas Carlos Iii Imidazolotiadiazoli za uporabo kot zaviralci protein-kinaze
CA2700841A1 (en) 2007-09-27 2009-04-02 The United States Of America, As Represented By The Secretary, Departmen T Of Health And Human Services Isoindoline compounds for the treatment of spinal muscular atrophy and other uses
US8153813B2 (en) 2007-12-20 2012-04-10 Abbott Laboratories Benzothiazole and benzooxazole derivatives and methods of use
WO2009114874A2 (en) * 2008-03-14 2009-09-17 Intellikine, Inc. Benzothiazole kinase inhibitors and methods of use
WO2009126635A1 (en) 2008-04-09 2009-10-15 Abbott Laboratories 2-amino-benzothiazole derivates useful as inhibitors of rock kinases
WO2009131958A2 (en) 2008-04-21 2009-10-29 Institute For Oneworld Health Compounds, compositions and methods comprising triazine derivatives
WO2009151546A2 (en) 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
WO2009152868A1 (en) 2008-06-20 2009-12-23 Rottapharm S.P.A. 6-1h-imidazo-quinazoline and quinolines derivatives, new mao inhibitors and imidazoline receptor ligands
EP2138493A1 (en) 2008-06-26 2009-12-30 Sanofi-Aventis Substituted pyrimidone derivatives
BRPI0913934A2 (pt) 2008-07-02 2015-10-20 Avexa Ltd compostos tendo propriedades antivirais
WO2010019243A1 (en) 2008-08-13 2010-02-18 Ptc Therapeutics, Inc. Methods for treating viral infections
WO2010024903A1 (en) 2008-08-29 2010-03-04 Yangbo Feng BENZO[d]OXAZOLES AND BENZO[d]THIAZOLES AS KINASE INHIBITORS
EP2350067A2 (en) 2008-10-16 2011-08-03 Schering Corporation Pyrrolidine, piperidine and piperazine derivatives and methods of use thereof
GB2465405A (en) 2008-11-10 2010-05-19 Univ Basel Triazine, pyrimidine and pyridine analogues and their use in therapy
WO2010071819A1 (en) * 2008-12-19 2010-06-24 Schering Corporation Bicyclic heterocyclic derivatives and methods of use thereof
SG173639A1 (en) 2009-02-11 2011-09-29 Sunovion Pharmaceuticals Inc Histamine h3 inverse agonists and antagonists and methods of use thereof
FR2945289A1 (fr) 2009-05-11 2010-11-12 Sanofi Aventis Derives de 2-cycloamino-5-(pyridin-4-yl)imidazo°2,1-b! °1,3,4!thiadiazole, leur preparation et leur application en therapeutique
EP2440058A4 (en) 2009-06-12 2012-11-21 Dana Farber Cancer Inst Inc MELTED HETEROCYCLIC COMPOUNDS AND APPLICATIONS THEREOF
US8846661B2 (en) 2009-06-19 2014-09-30 Abbvie Inc. Diazahomoadamantane derivatives and methods of use thereof
RU2562861C2 (ru) 2009-09-11 2015-09-10 Айсис Фармасьютикалс, Инк. Модуляция экспрессии гентингтина
WO2011050245A1 (en) 2009-10-23 2011-04-28 Yangbo Feng Bicyclic heteroaryls as kinase inhibitors
WO2011057204A2 (en) 2009-11-06 2011-05-12 The Johns Hopkins University Lrrk2-mediated neuronal toxicity
US8754220B2 (en) 2009-11-20 2014-06-17 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide M1 receptor positive allosteric modulators
US20130012506A1 (en) 2010-01-13 2013-01-10 Zaesung No Anti-infective pyrido (1,2-a) pyrimidines
WO2011097641A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
CA2789038A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
JP6006120B2 (ja) 2010-02-08 2016-10-12 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. 対立遺伝子多様体の選択的低減
EP2566860B1 (en) * 2010-05-06 2014-09-10 Bristol-Myers Squibb Company Bicyclic heteroaryl compounds as gpr119 modulators
CA2800509A1 (en) 2010-05-24 2011-12-01 Presidio Pharmaceuticals, Inc. Inhibitors of hcv ns5a
KR20180105730A (ko) 2010-07-19 2018-09-28 아이오니스 파마수티컬즈, 인코포레이티드 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법
WO2012019106A2 (en) 2010-08-06 2012-02-09 Board Of Regents Of The University Of Nebraska Positive and negative modulators of nmda receptors
WO2012075393A2 (en) 2010-12-02 2012-06-07 President And Fellows Of Harvard College Activators of proteasomal degradation and uses thereof
CN102617548A (zh) 2011-01-31 2012-08-01 北京赛林泰医药技术有限公司 作为gpr受体激动剂的双环杂芳基化合物及其组合物和应用
WO2012104823A2 (en) 2011-02-04 2012-08-09 Novartis Ag Pyridopyrimidinone compounds in the treatment of neurodegenerative diseases
CN106278999B (zh) 2011-02-07 2019-10-25 比奥根Ma公司 S1p调节剂
EP2673361B1 (en) 2011-02-08 2016-04-13 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US8703763B2 (en) 2011-03-02 2014-04-22 Hoffmann-La Roche Inc. Bridged piperidine derivatives
US9447075B2 (en) 2011-08-02 2016-09-20 The Brigham And Women's Hospital, Inc. Pyridazine derivatives as EAAT2 activators
US8871756B2 (en) 2011-08-11 2014-10-28 Hoffmann-La Roche Inc. Compounds for the treatment and prophylaxis of Respiratory Syncytial Virus disease
WO2013022967A1 (en) 2011-08-11 2013-02-14 Isis Pharmaceuticals, Inc. Gapped oligomeric compounds comprising 5'-modified deoxyribonucleosides in the gap and uses thereof
US20130046093A1 (en) 2011-08-18 2013-02-21 Korea Institute Of Science And Technology Pharmaceutical compositions for preventing or treating degenerative brain disease and method of screening the same
US9976138B2 (en) 2011-08-29 2018-05-22 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
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
GB201119538D0 (en) 2011-11-10 2011-12-21 Viral Ltd Pharmaceutical compounds
JP5966014B2 (ja) 2011-11-28 2016-08-10 ノバルティス アーゲー 新規トリフルオロメチル−オキサジアゾール誘導体および疾患の処置におけるその使用
WO2013101974A1 (en) 2011-12-30 2013-07-04 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
BR112014018027B1 (pt) 2012-01-26 2020-10-27 Ptc Therapeutics, Inc composto, composição farmacêutica e usos dos mesmos e métodos para aumentar a inclusão de éxon 7 de ame2 em rnam que é transcrito a partir do gene ame2 e para aumentar a quantidade de proteína smn
EP2812004B1 (en) 2012-02-10 2018-06-27 PTC Therapeutics, Inc. Compounds for treating spinal muscular atrophy
BR112014021531B1 (pt) 2012-03-01 2022-10-04 Ptc Therapeutics, Inc. Composto, composição farmacêutica e usos dos mesmos
CN104470909B (zh) 2012-03-23 2018-04-24 Ptc医疗公司 用于治疗脊髓性肌萎缩的化合物
WO2013151877A1 (en) 2012-04-03 2013-10-10 Bristol-Myers Squibb Company Pyrimidinedione carboxamide inhibitors of endothelial lipase
EP2841428B1 (en) 2012-04-24 2018-08-22 Vertex Pharmaceuticals Incorporated Dna-pk inhibitors
MX2014013376A (es) 2012-05-15 2015-08-14 Novartis Ag Derivados de benzamida para inhibir la actividad de abl1, abl2, y bcr-abl1.
WO2014012050A2 (en) 2012-07-13 2014-01-16 Indiana University Research & Technology Corporation Compounds for treatment of spinal muscular atrophy
US8729263B2 (en) 2012-08-13 2014-05-20 Novartis Ag 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions
EP4086347A3 (en) 2012-10-12 2023-01-11 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2014059341A2 (en) 2012-10-12 2014-04-17 Isis Pharmaceuticals, Inc. Antisense compounds and uses thereof
US9227976B2 (en) 2012-10-25 2016-01-05 Usher Iii Initiative, Inc. Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with usher syndrome
AU2013334139B2 (en) 2012-10-25 2017-08-03 Usher Iii Initiative, Inc. Pyrazolopyridazines and methods for treating retinal-degenerative diseases and hearing loss associated with Usher Syndrome
JP6255711B2 (ja) 2012-11-01 2018-01-10 株式会社リコー エレクトロクロミック化合物、エレクトロクロミック組成物及び表示素子
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
ES2817050T3 (es) 2013-02-04 2021-04-06 Ionis Pharmaceuticals Inc Compuestos antisentido selectivos y usos de los mismos
CN105026398B (zh) 2013-03-05 2018-05-18 默克专利股份公司 用于治疗疾病诸如癌症的三唑并[4,5-d]嘧啶衍生物
MX2015015691A (es) 2013-05-14 2016-03-04 Hoffmann La Roche Aza-oxo-indoles novedosos para el tratamiento y profilaxis de la infeccion del virus sincital respiratorio.
MX363456B (es) 2013-06-25 2019-03-25 Hoffmann La Roche Compuestos para tratar atrofia muscular espinal.
CA2918805C (en) 2013-07-31 2023-02-28 Novartis Ag 1,4-disubstituted pyridazine derivatives and their use for treating smn-deficiency-related conditions
MX372669B (es) 2013-08-19 2020-04-23 Hoffmann La Roche Un compuesto que modifica el empalme del gen de foxm1 para usarse en la profilaxis o tratamiento de cáncer.
WO2015095449A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount rna transcripts
WO2015095446A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
US10195202B2 (en) 2013-12-19 2019-02-05 Ptc Therapeutics, Inc. Methods for modulating the amount of RNA transcripts
MX2016009290A (es) 2014-01-16 2017-02-28 Wave Life Sciences Ltd Diseño quiral.
CN105899493B (zh) 2014-01-17 2019-03-29 诺华股份有限公司 用于抑制shp2活性的1-(三嗪-3-基/哒嗪-3-基)-哌(-嗪)啶衍生物及其组合物
AR099134A1 (es) 2014-01-24 2016-06-29 Hoffmann La Roche Procedimiento para la preparación de n-[(3-aminooxetán-3-il)metil]-2-(1,1-dioxo-3,5-dihidro-1,4-benzotiazepín-4-il)-6-metil-quinazolín-4-amina
WO2015136947A1 (en) 2014-03-14 2015-09-17 Raqualia Pharma Inc. Azaspiro derivatives as trpm8 antagonists
JP2017512834A (ja) 2014-03-19 2017-05-25 アミディス・ダイアグノスティックス・インコーポレイテッド アミロイド標的剤及びその使用方法
HRP20230637T1 (hr) 2014-05-15 2023-09-29 F. Hoffmann - La Roche Ag Postupak za proizvodnju spojeva korisnih za liječenje spinalne mišićne atrofije
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CN106573811A (zh) 2014-06-17 2017-04-19 诺维信公司 从废水中去除生物磷
CA2952895C (en) 2014-06-25 2023-09-26 F. Hoffmann-La Roche Ag Imidazo[1,2-a]pyrazin-1yl-benzamide compounds for treating spinal muscular atrophy
US10472346B2 (en) 2014-10-31 2019-11-12 The General Hospital Corporation Potent gamma-secretase modulators
GB201419579D0 (en) 2014-11-03 2014-12-17 Iomet Pharma Ltd Pharmaceutical compound
WO2016087417A1 (de) 2014-12-02 2016-06-09 Bayer Cropscience Aktiengesellschaft Bicyclische verbindungen als schädlingsbekämpfungsmittel
ES2732023T3 (es) 2014-12-24 2019-11-20 Uniqure Ip Bv Supresión del gen de la huntingtina inducida por ARNi
ES2928714T3 (es) 2015-01-16 2022-11-22 Massachusetts Gen Hospital Compuestos para mejorar el empalme del ARNm
JP6884102B2 (ja) 2015-02-09 2021-06-09 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト がんの治療のための化合物
GB201502567D0 (en) 2015-02-16 2015-04-01 Sentinel Oncology Ltd Pharmaceutical compounds
WO2016144351A1 (en) * 2015-03-11 2016-09-15 E. I. Du Pont De Nemours And Company Heterocycle-substituted bicyclic azole pesticides
GB201506933D0 (en) 2015-04-23 2015-06-10 Sentinel Oncology Ltd Pharmaceutical compounds
EP3298017B1 (en) 2015-05-20 2019-08-14 H. Hoffnabb-La Roche Ag Compounds for treating spinal muscular atrophy
EP3310169B1 (en) 2015-05-30 2023-05-17 PTC Therapeutics, Inc. Methods for modulating rna splicing
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017023987A1 (en) 2015-08-03 2017-02-09 Samumed, Llc. 3-(1h-pyrrolo[3,2-c]pyridin-2-yl)-1h-pyrazolo[3,4-b]pyridines and therapeutic uses thereof
WO2017059251A1 (en) 2015-10-02 2017-04-06 Incyte Corporation Heterocyclic compounds useful as pim kinase inhibitors
KR102162062B1 (ko) 2015-11-12 2020-10-07 에프. 호프만-라 로슈 아게 척수성 근위축증의 치료용 조성물
CN108137601A (zh) 2015-11-12 2018-06-08 豪夫迈·罗氏有限公司 用于治疗肌萎缩性侧索硬化症的化合物
US10383867B2 (en) 2015-11-28 2019-08-20 Russell Dahl Quinoline derivatives and their use for treating endoplasmic reticulum stress-related diseases and disorders
CN108697709A (zh) * 2015-12-10 2018-10-23 Ptc医疗公司 用于治疗亨廷顿病的方法
JP6872550B2 (ja) 2015-12-10 2021-05-19 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft 架橋されたピペリジン誘導体
BR112018068066B1 (pt) * 2016-03-11 2023-11-28 Ac Immune Sa Compostos bicíclicos e seu uso, composição diagnóstica e farmacêutica, misturas, métodos de coleção de dados para diagnóstico, para determinar uma predisposição a, para monitorar transtorno residual e para prever a capacidade de resposta de um paciente sofrendo de transtorno ou anormalidade associado com agregados de alfa-sinucleína, métodos para determinar a quantidade de agregados de alfa-sinucleína e para preparar um composto, kit teste e kit para preparar uma preparação radio farmacêutica
US10526345B2 (en) * 2016-04-08 2020-01-07 Mankind Pharma Ltd. Compounds as GPR119 agonists
AR108325A1 (es) 2016-04-27 2018-08-08 Samumed Llc Isoquinolin-3-il carboxamidas y preparación y uso de las mismas
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017210134A1 (en) 2016-05-31 2017-12-07 Board Of Regents, University Of Texas System Heterocyclic inhibitors of ptpn11
US10730868B2 (en) * 2016-07-14 2020-08-04 Bristol-Myers Squibb Company Bicyclic heteroaryl substituted compounds
US20190264267A1 (en) 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
TW202500565A (zh) 2016-10-24 2025-01-01 美商傳達治療有限公司 Shp2磷酸酶抑制劑及其使用方法
US11857599B2 (en) 2017-04-03 2024-01-02 Acceleron Pharma Inc. Compositions and methods for treating spinal muscular atrophy
EP3630770B1 (en) 2017-05-26 2024-08-28 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine derivatives as shp2 phosphatase inhibitors
AU2018282154B2 (en) 2017-06-05 2022-04-07 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
CN111182898B (zh) 2017-06-28 2024-04-16 Ptc医疗公司 用于治疗亨廷顿氏病的方法
WO2019005993A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. METHODS OF TREATING HUNTINGTON'S DISEASE
MX2020001425A (es) 2017-08-04 2020-08-06 Skyhawk Therapeutics Inc Metodos y composiciones para modular el empalme.
US20200392161A1 (en) 2018-02-21 2020-12-17 Relay Therapeutics, Inc. Shp2 phosphatase inhibitors and methods of use thereof
WO2019183364A1 (en) 2018-03-21 2019-09-26 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine shp2 phosphatase inhibitors and methods of use thereof
CN112166110B (zh) 2018-03-21 2023-08-11 传达治疗有限公司 Shp2磷酸酶抑制剂及其使用方法
BR112020019373A2 (pt) 2018-03-27 2020-12-29 Ptc Therapeutics, Inc. Compostos para o tratamento da doença de hutington
IL279688B2 (en) 2018-06-27 2025-01-01 Ptc Therapeutics Inc Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease
WO2020005877A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
EP3814360B8 (en) 2018-06-27 2024-11-06 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
EP3937942A4 (en) 2019-03-15 2022-11-16 Skyhawk Therapeutics, Inc. COMPOSITIONS AND METHODS FOR CORRECTING ABERRANT SPLICING
AU2020277027A1 (en) 2019-05-13 2021-12-09 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
WO2021007378A1 (en) 2019-07-11 2021-01-14 Ptc Therapeutics, Inc. Compounds for use in treating huntington's disease
JP2023500251A (ja) 2019-11-01 2023-01-05 ノバルティス アーゲー ハンチントン病の進行を遅らせる処置のためのスプライシングモジュレーターの使用
KR20230009367A (ko) 2020-04-09 2023-01-17 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 화합물
US20220162610A1 (en) 2020-11-12 2022-05-26 Ptc Therapeutics, Inc. Novel rna transcript
EP4377317A1 (en) 2021-07-30 2024-06-05 PTC Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease

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