JPWO2020005877A5 - - Google Patents

Download PDF

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
methyl
thiazolo
benzothiazole
fluoro
indazole
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.)
Granted
Application number
JP2020572894A
Other languages
English (en)
Japanese (ja)
Other versions
JP7515415B2 (ja
JP2021528466A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/038895 external-priority patent/WO2020005877A1/en
Publication of JP2021528466A publication Critical patent/JP2021528466A/ja
Publication of JPWO2020005877A5 publication Critical patent/JPWO2020005877A5/ja
Priority to JP2024072881A priority Critical patent/JP2024105367A/ja
Application granted granted Critical
Publication of JP7515415B2 publication Critical patent/JP7515415B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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)

Priority Applications (1)

Application Number Priority Date Filing Date Title
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

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024072881A Division 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

Publications (3)

Publication Number Publication Date
JP2021528466A JP2021528466A (ja) 2021-10-21
JPWO2020005877A5 true JPWO2020005877A5 (OSRAM) 2022-06-21
JP7515415B2 JP7515415B2 (ja) 2024-07-12

Family

ID=67185796

Family Applications (2)

Application Number Title Priority Date Filing Date
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

Family Applications After (1)

Application Number Title Priority Date Filing Date
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

Country Status (13)

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

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX382671B (es) 2015-12-10 2025-03-13 Ptc Therapeutics Inc Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington
JP7376471B2 (ja) 2017-06-05 2023-11-08 ピーティーシー セラピューティクス, インコーポレイテッド ハンチントン病を処置するための化合物
MX2019015578A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
MY201938A (en) 2017-08-04 2024-03-25 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
JP7399870B2 (ja) 2018-03-27 2023-12-18 ピーティーシー セラピューティクス, インコーポレイテッド ハンチントン病を処置するための化合物
EA202092899A1 (ru) 2018-06-27 2021-05-14 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Гетероарильные соединения для лечения болезни гентингтона
LT3814357T (lt) 2018-06-27 2024-06-25 Ptc Therapeutics, Inc. Heterocikliniai ir heteroarilų junginiai, skirti hantingtono ligos gydymui
US11685746B2 (en) 2018-06-27 2023-06-27 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
CN114650822A (zh) 2019-11-01 2022-06-21 诺华股份有限公司 剪接调节子用于减慢亨廷顿氏病进展的治疗的用途
US20230322754A1 (en) * 2020-04-28 2023-10-12 Kymera Therapeutics, Inc. Irak inhibitors and uses thereof
MX2022013856A (es) 2020-05-13 2023-04-05 Chdi Foundation Inc Moduladores htt para tratar la enfermedad de huntington.
KR20230118067A (ko) 2020-07-02 2023-08-10 레믹스 테라퓨틱스 인크. 5-[5-(피페리딘-4-일)티에노[3,2-c]피라졸-2-일]인다졸 유도체, 및 핵산 스플라이싱의 조절제로서의 및 증식성 질환의 치료를 위한 관련 화합물
JP2023532332A (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 核酸のスプライシング及び増殖性疾患の処置のためのモジュレーターとしての2-(インダゾール-5-イル)-6-(ピペリジン-4-イル)-1,7-ナフチリジン誘導体及び関連する化合物
WO2022194800A1 (en) * 2021-03-17 2022-09-22 F. Hoffmann-La Roche Ag New thienopyrimidinone derivatives
CA3212341A1 (en) * 2021-03-17 2022-09-22 F. Hoffmann-La Roche Ag New thiazolopyrimidinone derivatives
JP2024510487A (ja) * 2021-03-17 2024-03-07 エフ. ホフマン-ラ ロシュ アーゲー 新規なチアジアゾロピリミドン誘導体
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
JP2024540477A (ja) 2021-11-17 2024-10-31 シーエイチディーアイ ファウンデーション,インコーポレーテッド ハンチントン病を治療するためのhttモジュレータとしてのn-(2h-インダゾール-5-イル)ピラジン-2-カルボキサミド誘導体及び類似化合物
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
EP4526292A1 (en) * 2022-05-20 2025-03-26 Biogen MA Inc. Heterocyclic compounds for treating huntington's disease
WO2024215070A1 (ko) * 2023-04-11 2024-10-17 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
NL297170A (OSRAM) 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
NZ266386A (en) 1993-05-11 1997-11-24 Univ North Carolina Use of antisense rna oligonucleotides in regulating gene expression
JPH11510478A (ja) 1995-06-06 1999-09-14 アボツト・ラボラトリーズ キノリジノン型化合物
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
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
US6214986B1 (en) 1998-10-07 2001-04-10 Isis Pharmaceuticals, Inc. Antisense modulation of bcl-x expression
AU7961100A (en) 1999-10-28 2001-05-08 Daiichi Pharmaceutical Co., Ltd. Drug discharge pump inhibitors
DE60128655T2 (de) 2000-01-24 2008-02-07 Astrazeneca Ab Durch einen morpholinrest substituierte therapeutische verbindungen
CA2437942C (en) 2000-11-09 2013-06-11 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
WO2002068415A1 (en) 2000-12-21 2002-09-06 Vertex Pharmaceuticals Incorporated Pyrazole compounds useful as protein kinase inhibitors
EP1389463A4 (en) 2001-04-26 2008-09-17 Daiichi Seiyaku Co MEDICAMENT FOR INHIBITING A DRUG REMOVAL PUMP
EP2198867A1 (en) 2001-12-07 2010-06-23 Vertex Pharmaceuticals, Inc. Pyrimidine-based compounds useful as GSK-3 inhibitors
GB0205281D0 (en) 2002-03-06 2002-04-17 Novartis Ag Organic compounds
CA2493458A1 (en) 2002-07-24 2004-01-29 Ptc Therapeutics, Inc. Ureido substituted benzoic acid compounds, their use for nonsense suppression and the treatment of diseases caused by such mutations
WO2004019002A2 (en) 2002-08-23 2004-03-04 The Board Of Trustees Of The Leland Stanford Junior University Fluorescent glycosides and methods for their use
US7220741B2 (en) 2002-09-30 2007-05-22 Neurosearch A/S 1,4-diazabicycloalkane derivatives, their preparation and use
WO2004043458A1 (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
MXPA05013142A (es) 2003-06-20 2006-03-17 Chiron Corp Compuestos de piridinio [1,2-a]pirimidin-4-ona como agentes anticancer.
GB0315494D0 (en) 2003-07-02 2003-08-06 Biofocus Plc Compounds which bind to the active site of protein kinase enzymes
ZA200600424B (en) 2003-08-01 2007-05-30 Genelabs Tech Inc Bicyclic imidazol derivatives against flaviviridae
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
US20050137203A1 (en) 2003-12-22 2005-06-23 Jianguo Ji 3-quinuclidinyl amino-substituted biaryl derivatives
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
US8598193B2 (en) 2003-12-24 2013-12-03 Biota Scientific Management Pty Ltd. Polycyclic agents for the treatment of respiratory syncytial virus infections
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
CN1964717A (zh) 2004-04-08 2007-05-16 神经能质公司 经取代∴啉-4-基胺
DE602005002562T2 (de) 2004-05-04 2008-01-31 Warner-Lambert Company Llc Pyrrolylsubstituierte pyridoä2,3-düpyrimidin-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
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
CA2614116A1 (en) 2005-07-04 2007-01-11 Novo Nordisk A/S Novel medicaments
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
US8258109B2 (en) 2005-10-20 2012-09-04 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of LMNA expression
WO2007056580A2 (en) 2005-11-08 2007-05-18 Pharmadyn, Inc. Methods and compositions for treating diseases associated with pathogenic proteins
EP2535413A3 (en) 2005-11-10 2016-04-13 Roche Innovation Center Copenhagen A/S Splice switching oligomers for the TNF superfamily receptors and their use in treatment of disease
US8173658B2 (en) 2005-12-06 2012-05-08 Neurosearch A/S Diazabicycylic aryl derivatives and their medical use
CN101360738A (zh) 2005-12-21 2009-02-04 佩因赛普托药物公司 调节门控离子通道的组合物和方法
WO2007089611A2 (en) 2006-01-26 2007-08-09 Isis Pharmaceuticals Inc. Compositions and their uses directed to huntingtin
EP1984499B1 (en) 2006-01-27 2015-05-27 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
CA2645581A1 (en) * 2006-03-28 2007-10-04 High Point Pharmaceuticals, Llc Benzothiazoles having histamine h3 receptor activity
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
WO2007133756A2 (en) 2006-05-15 2007-11-22 Neurogen Corporation 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
EP2079724B1 (en) 2006-07-20 2010-05-26 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
DK2066653T3 (da) 2006-08-03 2012-12-10 Rottapharm Spa 6-1H-Imidazoquinazolin- og -quinolinderivater, nye stærke analgetika og antiinflammatoriske midler
WO2008020302A2 (en) 2006-08-17 2008-02-21 Pfizer Products Inc. Heteroaromatic quinoline-based compounds as phosphodiesterase (pde) inhibitors
US20100029685A1 (en) 2006-10-25 2010-02-04 Neurosearch A/S Oxadiazole and thiadiazole compounds and their use as nicotinic acetylcholine receptor modulators
US8314119B2 (en) 2006-11-06 2012-11-20 Abbvie Inc. Azaadamantane derivatives and methods of use
EP2269992A1 (en) 2006-12-22 2011-01-05 Avexa Limited Bicyclic pyrimidinones for the treatment of viral infections
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
DK2193133T3 (en) 2007-09-27 2015-10-05 Fundacion Ct Nac Investigaciones Oncologicas Carlos Iii IMIDAZOLOTHIADIAZOLES USED AS PROTEINKINASE INHIBITORS
US20100267712A1 (en) 2007-09-27 2010-10-21 The United States of America, as represented by the Secretary, Department of Health and 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
CA2728376C (en) 2008-06-20 2015-05-12 Rottapharm S.P.A. 6-1h-imidazo-quinazoline and quinolines derivatives/mao inhibitors for treating depression
EP2138493A1 (en) 2008-06-26 2009-12-30 Sanofi-Aventis Substituted pyrimidone derivatives
AU2009266421B2 (en) 2008-07-02 2013-07-11 Avexa Limited Compounds having antiviral properties
US8932818B2 (en) 2008-08-13 2015-01-13 Ptc Therapeutics, Inc. Screening assays for compounds that modulate programmed ribosomal frameshifting
WO2010024903A1 (en) 2008-08-29 2010-03-04 Yangbo Feng BENZO[d]OXAZOLES AND BENZO[d]THIAZOLES AS KINASE INHIBITORS
JP2012505898A (ja) 2008-10-16 2012-03-08 シェーリング コーポレイション ピロリジン、ピペリジンおよびピペラジン誘導体ならびにそれらの使用方法
GB2465405A (en) 2008-11-10 2010-05-19 Univ Basel Triazine, pyrimidine and pyridine analogues and their use in therapy
US8283360B2 (en) * 2008-12-19 2012-10-09 Merck Sharp & Dohme Corp. Bicyclic heterocyclic derivatives and methods of use thereof
KR20110132564A (ko) 2009-02-11 2011-12-08 선오비온 파마슈티컬스 인코포레이티드 히스타민 h3 역 작용제 및 길항제, 및 이의 사용 방법
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
WO2010144909A1 (en) 2009-06-12 2010-12-16 Novartis Ag Fused heterocyclic compounds and their uses
BRPI1015043A2 (pt) 2009-06-19 2019-07-09 Abbott Lab derivados de diazahomoadamantano e métodos de uso dos mesmos
KR102279458B1 (ko) 2009-09-11 2021-07-21 아이오니스 파마수티컬즈, 인코포레이티드 헌팅틴 발현의 조절
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
WO2011062853A1 (en) 2009-11-20 2011-05-26 Merck Sharp & Dohme Corp. Quinolizidinone carboxamide m1 receptor positive allosteric modulators
JP2013517233A (ja) 2010-01-13 2013-05-16 インスティチュート・パスツール・コリア 抗感染ピリド(1,2−a)ピリミジン
EP3561060A1 (en) 2010-02-08 2019-10-30 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
US9006198B2 (en) 2010-02-08 2015-04-14 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP2536738A4 (en) 2010-02-08 2014-09-17 Isis Pharmaceuticals Inc METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISEASES OR SUFFERING IN CONNECTION WITH REPEAT EXTENSIONS
BR112012028445A2 (pt) 2010-05-06 2016-07-19 Bristol Myers Squibb Co compostos de heteroarila bicíclica como moduladores de gpr119
EP2575866A4 (en) 2010-05-24 2013-10-16 Presidio Pharmaceuticals Inc HCV NS5A INHIBITORS
CN103167883B (zh) 2010-07-19 2016-08-03 F·C·贝内特 肌强直性营养障碍蛋白激酶(dmpk)表达的调节
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
ES2772258T3 (es) 2011-02-07 2020-07-07 Biogen Ma Inc Agentes moduladores de S1P
EP3467109A1 (en) 2011-02-08 2019-04-10 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
WO2013019938A1 (en) 2011-08-02 2013-02-07 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
US20140303235A1 (en) 2011-08-11 2014-10-09 Isis Pharmaceuticals, Inc. Linkage modified gapped oligomeric compounds and uses thereof
EP2560008B1 (en) 2011-08-18 2016-11-30 Korea Institute of Science and Technology Pharmaceutical compositions for preventing or treating degenerative brain disease and method of screening the same
WO2013033223A1 (en) 2011-08-29 2013-03-07 Isis 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
ES2606630T3 (es) 2011-11-28 2017-03-24 Novartis Ag Derivados de trifluorometil-oxadiazol novedosos y su uso en el tratamiento de enfermedad
USRE47689E1 (en) 2011-12-30 2019-11-05 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
CA2862084C (en) 2012-01-26 2021-05-11 Ptc Therapeutics, Inc. 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy
EA037123B1 (ru) 2012-02-10 2021-02-09 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения спинальной мышечной атрофии
WO2013130689A1 (en) 2012-03-01 2013-09-06 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
EP2828247B1 (en) 2012-03-23 2019-01-16 PTC Therapeutics, Inc. 4h-chromen-4-one derivatives for treating spinal muscular atrophy
EP2844643B1 (en) 2012-04-03 2016-11-30 Bristol-Myers Squibb Company Pyrimidinedione carboxamide inhibitors of endothelial lipase
US9296701B2 (en) 2012-04-24 2016-03-29 Vertex Pharmaceuticals Incorporated DNA-PK inhibitors
AU2013261130A1 (en) 2012-05-15 2014-10-23 Novartis Ag Benzamide derivatives for inhibiting the activity of ABL1, ABL2 and BCR-ABL1
US9212209B2 (en) 2012-07-13 2015-12-15 Indiana University Research And Technology Corporation Screening methods for 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
EP4144845B1 (en) 2012-10-12 2024-04-24 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
EP4086347A3 (en) 2012-10-12 2023-01-11 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
PL2911671T3 (pl) 2012-10-25 2018-11-30 Usher Iii Initiative, Inc. Pirazolopirydazyny oraz sposoby leczenia zwyrodnieniowych chorób siatkówki i utraty słuchu związanych z zespołem ushera
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
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
US10260069B2 (en) 2013-02-04 2019-04-16 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
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
BR112015028475A2 (pt) 2013-05-14 2017-07-25 Hoffmann La Roche novos aza-oxo-indois para o tratamento ou profilaxia de infecção virótica respiratória sincitial
KR102314290B1 (ko) 2013-06-25 2021-10-21 에프. 호프만-라 로슈 아게 척수성 근위축증을 치료하기 위한 화합물
CN105636953B (zh) 2013-07-31 2018-01-02 诺华股份有限公司 1,4‑二取代的哒嗪衍生物及其用于治疗与smn缺乏相关的病症的用途
JP6689197B2 (ja) 2013-08-19 2020-04-28 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト スクリーニング方法
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
WO2015095446A1 (en) 2013-12-19 2015-06-25 Ptc Therapeutics, Inc. Methods for modulating the amount of rna transcripts
EP4137572A1 (en) 2014-01-16 2023-02-22 Wave Life Sciences Ltd. Chiral design
EP3094629B1 (en) 2014-01-17 2018-08-22 Novartis AG 1-(triazin-3-yl/pyridazin-3-yl)-piper(-azine)idine derivatives and compositions thereof for inhibiting the activity of shp2
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
RU2683309C2 (ru) 2014-03-14 2019-03-28 Раквалиа Фарма Инк. Азаспиропроизводные в качестве антагонистов trpm8
US10005745B2 (en) 2014-03-19 2018-06-26 The Regents Of The University Of California Amyloid targeting agents and methods of using the same
RS59718B1 (sr) 2014-05-15 2020-01-31 Hoffmann La Roche Jedinjenja za lečenje spinalne mišićne atrofije
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
EP3157875A2 (en) 2014-06-17 2017-04-26 Novozymes A/S Biological phosphorus removal from wastewater
BR112016026951B1 (pt) 2014-06-25 2022-09-20 F. Hoffmann-La Roche Ag Imidazo[1,2-a]pirazin-1-il-benzamida, seu uso, e composições farmacêuticas
JP6672296B2 (ja) 2014-10-31 2020-03-25 ザ ジェネラル ホスピタル コーポレイション 強力なγ−セクレターゼモジュレータ
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
SMT201900347T1 (it) 2014-12-24 2019-07-11 Uniqure Ip Bv Soppressione del gene huntingtina indotta da rnai
CA2973949C (en) 2015-01-16 2023-07-11 The General Hospital Corporation Compounds for improving mrna splicing
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
CN112679467B (zh) * 2015-03-11 2024-11-01 Fmc公司 杂环取代的二环唑杀有害生物剂
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
US10226448B2 (en) 2015-08-03 2019-03-12 Samumed, Llc 3-(1H-pyrrolo[3,2-C]pyridin-2-yl)-1H-pyrazolo[3,4-B]pyridines and therapeutic uses thereof
TW201718546A (zh) 2015-10-02 2017-06-01 英塞特公司 適用作pim激酶抑制劑之雜環化合物
AR106652A1 (es) 2015-11-12 2018-02-07 Hoffmann La Roche Compuestos para tratar la esclerosis lateral amiotrófica
BR112018009281B1 (pt) 2015-11-12 2023-10-31 F. Hoffmann-La Roche Ag Composições para tratar atrofia muscular espinhal e seu uso
US10383867B2 (en) 2015-11-28 2019-08-20 Russell Dahl Quinoline derivatives and their use for treating endoplasmic reticulum stress-related diseases and disorders
MX382671B (es) 2015-12-10 2025-03-13 Ptc Therapeutics Inc Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington
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
CA3224365A1 (en) * 2016-03-11 2017-09-14 Ac Immune Sa Bicyclic compounds for diagnosis and therapy of alpha-synuclein associated disorders
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
JP7044375B2 (ja) 2016-05-31 2022-03-30 ボード オブ リージェンツ,ザ ユニバーシティ オブ テキサス システム Ptpn11の複素環式阻害剤
WO2018013770A1 (en) * 2016-07-14 2018-01-18 Bristol-Myers Squibb Company Bicyclic heteroaryl substituted compounds
US20190264267A1 (en) 2016-07-25 2019-08-29 Wave Life Sciences Ltd. Phasing
TWI848901B (zh) 2016-10-24 2024-07-21 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
US11591336B2 (en) 2017-05-26 2023-02-28 D. E. Shaw Research, Llc Substituted pyrazolo[3,4-b]pyrazines as SHP2 phosphatase inhibitors
JP7376471B2 (ja) 2017-06-05 2023-11-08 ピーティーシー セラピューティクス, インコーポレイテッド ハンチントン病を処置するための化合物
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
MX2019015578A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
MY201938A (en) 2017-08-04 2024-03-25 Skyhawk Therapeutics Inc Methods and compositions for modulating splicing
WO2019165073A1 (en) 2018-02-21 2019-08-29 Relay Therapeutics, Inc. Shp2 phosphatase inhibitors and methods of use thereof
EP4506035A3 (en) 2018-03-21 2025-04-16 Relay Therapeutics, Inc. Shp2 phosphatase inhibitors and methods of use thereof
EP3768680A1 (en) 2018-03-21 2021-01-27 Relay Therapeutics, Inc. Pyrazolo[3,4-b]pyrazine shp2 phosphatase inhibitors and methods of use thereof
JP7399870B2 (ja) 2018-03-27 2023-12-18 ピーティーシー セラピューティクス, インコーポレイテッド ハンチントン病を処置するための化合物
LT3814357T (lt) 2018-06-27 2024-06-25 Ptc Therapeutics, Inc. Heterocikliniai ir heteroarilų junginiai, skirti hantingtono ligos gydymui
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
EA202092899A1 (ru) 2018-06-27 2021-05-14 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Гетероарильные соединения для лечения болезни гентингтона
KR20210151823A (ko) 2019-03-15 2021-12-14 스카이호크 테라퓨틱스, 인코포레이티드 비정상적인 스플라이싱을 보정하기 위한 조성물 및 방법
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
CN114650822A (zh) 2019-11-01 2022-06-21 诺华股份有限公司 剪接调节子用于减慢亨廷顿氏病进展的治疗的用途
JP2023522177A (ja) 2020-04-09 2023-05-29 ピーティーシー セラピューティクス, インコーポレイテッド ハンチントン病を処置するための化合物
IL302839A (en) 2020-11-12 2023-07-01 Ptc Therapeutics Inc Novel rna transcript
KR20240054264A (ko) 2021-07-30 2024-04-25 피티씨 테라퓨틱스, 인크. Heteroaryl compounds for treating huntington's disease

Similar Documents

Publication Publication Date Title
JPWO2020005877A5 (OSRAM)
JPWO2019191229A5 (OSRAM)
JPWO2019191092A5 (OSRAM)
JPWO2020005873A5 (OSRAM)
JP7649257B2 (ja) ハンチントン病を処置するための化合物
JP7399869B2 (ja) ハンチントン病を処置するための化合物
JP2020522570A5 (OSRAM)
AU2019203444B2 (en) Bicyclic lactams and methods of use thereof
KR101812357B1 (ko) Cdk4/6의 억제제로서의 피롤로피리미딘 화합물
CN112805280A (zh) 用于治疗亨廷顿氏病的杂芳基化合物
JP2021503004A5 (OSRAM)
HRP20210177T1 (hr) Supstituirani spojevi pirazolo[1,5-a]piridina kao inhibitori ret kinaze
JP2015504057A5 (OSRAM)
JP2014506929A5 (OSRAM)
WO2020005882A1 (en) Heteroaryl compounds for treating huntington's disease
HRP20151232T1 (hr) Derivati 2,3-dihidro-1h-inden-1-il-2,7-diazaspiro[3.6]nonana i njihova upotreba kao antagonista ili inverznih agonista grelinskog receptora
JP2011500774A5 (OSRAM)
JP2015537010A5 (OSRAM)
RU2015122895A (ru) Пуриновые ингибиторы человеческой фосфатидилинозит 3-киназы дельта
CA2649521A1 (en) Novel piperazine compound, and use thereof as hcv polymerase inhibitor
IL297044A (en) Compounds for the treatment of Huntington's disease
JPWO2020231977A5 (OSRAM)
US12180226B2 (en) Spirotricycle RIPK1 inhibitors and methods of uses thereof
NZ771342B2 (en) Heteroaryl compounds for treating huntington's disease
NZ771414B2 (en) Heterocyclic and heteroaryl compounds for treating huntington's disease