公开(公告)号：JP2011251981A

公开(公告)日：2011-12-15

申请号：JP2011156613

申请日：2011-07-15

Applicant: Ptc Therapeutics Inc ,  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

Inventor： KARP GARY MITCHELL ,  HWANG SEONGWOO ,  CHEN GUANGMING ,  ALMSTEAD NEIL GREGORY ,  MOON YOUNG-CHOON

IPC: C07D271/06 ,  A61K31/4245 ,  A61K31/4439 ,  A61K31/454 ,  A61K31/4545 ,  A61K31/5377 ,  A61P1/16 ,  A61P3/04 ,  A61P3/06 ,  A61P3/10 ,  A61P5/14 ,  A61P7/00 ,  A61P7/04 ,  A61P9/00 ,  A61P9/10 ,  A61P11/00 ,  A61P13/12 ,  A61P19/02 ,  A61P21/04 ,  A61P25/00 ,  A61P25/16 ,  A61P25/28 ,  A61P27/02 ,  A61P29/00 ,  A61P35/00 ,  A61P35/02 ,  A61P37/02 ,  A61P37/06 ,  A61P43/00 ,  C07D271/08 ,  C07D271/10 ,  C07D271/12 ,  C07D413/04 ,  C07D413/10

CPC classification number: A61K31/4245 ,  A61K9/0053 ,  A61K9/12 ,  A61K9/48 ,  A61K31/4439 ,  A61K31/454 ,  A61K31/4545 ,  A61K31/5377 ,  A61K31/655 ,  A61K45/06 ,  C07D271/06 ,  C07D271/12 ,  C07D285/14 ,  C07D413/02 ,  C07D413/04 ,  C07D413/10

Abstract: PROBLEM TO BE SOLVED: To provide a pharmaceutical composition and method, for treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay; or to provide a method for ameliorating one or more symptoms associated therewith.SOLUTION: This invention relates to: novel 1,2,4-oxadiazole benzoic acid compounds such as illustrated below; a method of using a 1,2,4-oxadiazole benzoic acid derivative; and a pharmaceutical composition comprising the 1,2,4-oxadiazole benzoic acid derivative.

Abstract translation: 待解决的问题：提供用于治疗或预防通过调节过早翻译终止或无义介导的mRNA衰变而改善的疾病的药物组合物和方法; 或提供改善与之相关的一种或多种症状的方法。 解决方案：本发明涉及：如下所示的新的1,2,4-恶二唑苯甲酸化合物; 使用1,2,4-恶二唑苯甲酸衍生物的方法; 以及包含1,2,4-恶二唑苯甲酸衍生物的药物组合物。 版权所有（C）2012，JPO＆INPIT

公开(公告)号：FI3814357T3

公开(公告)日：2024-07-26

申请号：FI19736566

申请日：2019-06-25

Applicant: PTC THERAPEUTICS INC ,  PTC THERAPEUTICS INC

Inventor： SYDORENKO NADIYA ,  ALAM MD RAUFUL ,  ARNOLD MICHAEL A ,  BABU SURESH ,  BHATTACHARYYA ANURADHA ,  CHEN GUANGMING ,  GERASYUTO ALEKSEY I ,  KARP GARY MITCHELL ,  KASSICK ANDREW J ,  MAZZOTTI ANTHONY R ,  MOON YOUNG-CHOON ,  NARASIMHAN JANA ,  PATEL JIGAR ,  TURPOFF ANTHONY ,  WOLL MATTHEW G ,  YAN WUMING ,  ZHANG NANJING

IPC: C07D487/04 ,  A61K31/4985 ,  A61K31/5025 ,  A61P25/00 ,  A61P25/14 ,  A61P25/28 ,  C07D491/04 ,  C07D495/04 ,  C07D519/00

Abstract: The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.In particular, the present description relates to substituted bicyclic heterocyclic and heteroaryl compounds compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

公开(公告)号：WO2004091502A2

公开(公告)日：2004-10-28

申请号：PCT/US2004011106

申请日：2004-04-09

Applicant: PTC THERAPEUTICS INC ,  KARP GARY MITCHELL ,  HWANG SEONGWOO ,  CHEN GUANGMING ,  ALMSTEAD NEIL GREGORY

Inventor： KARP GARY MITCHELL ,  HWANG SEONGWOO ,  CHEN GUANGMING ,  ALMSTEAD NEIL GREGORY

IPC: A61K31/4245 ,  C07D271/06 ,  C07D271/12 ,  C07D413/04 ,  C07D413/10 ,  A61K

CPC classification number: A61K31/4245 ,  A61K9/0053 ,  A61K9/12 ,  A61K9/48 ,  A61K31/4439 ,  A61K31/454 ,  A61K31/4545 ,  A61K31/5377 ,  A61K31/655 ,  A61K45/06 ,  C07D271/06 ,  C07D271/12 ,  C07D285/14 ,  C07D413/02 ,  C07D413/04 ,  C07D413/10

Abstract: Novel 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising an 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.

Abstract translation: 公开了新的1,2,4-恶二唑苯甲酸化合物，使用方法和包含1,2,4-恶二唑苯甲酸衍生物的药物组合物。 所述方法包括治疗或预防通过调节过早翻译终止或无义介导的mRNA衰变而改善的疾病或改善与其相关的一种或多种症状的方法。

公开(公告)号：WO2007084435A2

公开(公告)日：2007-07-26

申请号：PCT/US2007000996

申请日：2007-01-16

Applicant: PTC THERAPEUTICS INC ,  KARP GARY MITCHELL ,  HWANG PETER SEONGWOO ,  TAKASUGI JAMES JAN ,  REN HONGYU ,  WILDE RICHARD GERALD ,  TURPOFF ANTHONY ALLAN ,  AREFOLOV ALEXANDER ,  CHEN GUANGMING ,  CAMPBELL JEFFREY ALLEN

Inventor： KARP GARY MITCHELL ,  HWANG PETER SEONGWOO ,  TAKASUGI JAMES JAN ,  REN HONGYU ,  WILDE RICHARD GERALD ,  TURPOFF ANTHONY ALLAN ,  AREFOLOV ALEXANDER ,  CHEN GUANGMING ,  CAMPBELL JEFFREY ALLEN

IPC: C07D209/30 ,  C07D209/42 ,  C07D401/04 ,  C07D401/14

CPC classification number: C07D209/30 ,  C07D209/42 ,  C07D401/04 ,  C07D401/14 ,  C07D403/12 ,  C07D403/14 ,  C07D409/12 ,  C07D413/12 ,  C07D417/10 ,  C07D417/12 ,  C07D417/14

Abstract: The present invention provides compounds, pharmaceutical compositions, and methods of using such compounds or compositions for treating infection by a virus, or for affecting viral IRES activity.

Abstract translation: 本发明提供化合物，药物组合物和使用这些化合物或组合物治疗病毒感染或影响病毒IRES活性的方法。

公开(公告)号：WO2016025933A3

公开(公告)日：2016-04-07

申请号：PCT/US2015045433

申请日：2015-08-15

Applicant: PTC THERAPEUTICS INC

Inventor： WANG JIASHI ,  GERASYUTO ALEKSEY IGOREVICH ,  ARNOLD MICHAEL A ,  KARP GARY MITCHELL ,  QI HONGYAN ,  WOLL MATTHEW G ,  ZHANG NANJING ,  BRANSTROM ARTHUR A ,  NARASIMHAN JANA ,  DUMBLE MELISSA L ,  HEDRICK JEAN ,  WEETALL MARIA L

IPC: A61K31/4353

CPC classification number: A61K45/06 ,  C07D471/04 ,  C07D491/147

Abstract: The present description relates to substituted polycyclic compounds and forms and pharmaceutical compositions thereof and methods of using such compounds, forms or compositions thereof for treating or ameliorating Neisseria gonorrhoeae and Neisseria meningiditis. The present description relates to a compound of Formula (I), Formula (II), Formula (III) or Formula (IV): wherein the dashed line "----- " represents an optional double bond and R1, R2, R5, X and Z are as defined herein, and forms and compositions thereof, and also relates to uses of a compound of Formula (I), Formula (II), Formula (III) or Formula (IV) or a form thereof and methods for treating or ameliorating Neisseria gonorrhoeae (N. gonorrhoeae) or Neisseria meningitidis (N. meningitidis) in a subject in need thereof, comprising, administering an effective amount of the compound to the subject.

Abstract translation: 本说明书涉及取代的多环化合物及其形式及其药物组合物，以及使用这些化合物，其形式或组合物治疗或改善淋病奈瑟氏球菌和脑膜炎奈瑟球菌的方法。 本发明涉及式（I），式（II），式（III）或式（IV）的化合物：其中虚线“-----”表示任选的双键，并且R 1，R 2，R 5 ，X和Z如本文所定义，以及其形式和组成，并且还涉及式（I），式（II），式（III）或式（IV）的化合物或其形式的用途及其用途 在有需要的受试者中治疗或改善淋病奈瑟氏球菌（淋病奈瑟氏球菌）或脑膜炎奈瑟氏球菌（脑膜炎奈瑟氏球菌），包括向受试者施用有效量的化合物。

公开(公告)号：WO2013119916A3

公开(公告)日：2013-10-24

申请号：PCT/US2013025292

申请日：2013-02-08

Applicant: PTC THERAPEUTICS INC ,  HOFFMANN LA ROCHE

Inventor： QI HONGYAN ,  CHOI SOONGYU ,  DAKKA AMAL ,  KARP GARY MITCHELL ,  NARASIMHAN JANA ,  NARYSHKIN NIKOLAI ,  TURPOFF ANTHONY A ,  WEETALL MARIA L ,  WELCH ELLEN ,  WOLL MATTHEW G ,  YANG TIANLE ,  ZHANG NANJING ,  ZHANG XIAOYAN ,  ZHAO XIN ,  GREEN LUKE ,  PINARD EMMANUEL ,  RATNI HASANE

IPC: A61K31/53 ,  A01N43/90 ,  A61K31/519 ,  C07D239/70

CPC classification number: C07D471/04 ,  A61K31/519 ,  C07D239/70 ,  C07D487/04 ,  C07D519/00

Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.

Abstract translation: 本文提供了化合物，其组合物，并用于治疗脊髓性肌肉萎缩。

公开(公告)号：WO2007084435A8

公开(公告)日：2009-11-12

申请号：PCT/US2007000996

申请日：2007-01-16

Applicant: PTC THERAPEUTICS INC ,  KARP GARY MITCHELL ,  HWANG PETER SEONGWOO ,  TAKASUGI JAMES JAN ,  REN HONGYU ,  WILDE RICHARD GERALD ,  TURPOFF ANTHONY ALLAN ,  AREFOLOV ALEXANDER ,  CHEN GUANGMING ,  CAMPBELL JEFFREY ALLEN ,  LI CHUNSHI ,  PAGET STEVEN ,  ZHANG NANJING ,  ZHANG XIAOYAN ,  ZHU JIN

Inventor： KARP GARY MITCHELL ,  HWANG PETER SEONGWOO ,  TAKASUGI JAMES JAN ,  REN HONGYU ,  WILDE RICHARD GERALD ,  TURPOFF ANTHONY ALLAN ,  AREFOLOV ALEXANDER ,  CHEN GUANGMING ,  CAMPBELL JEFFREY ALLEN ,  LI CHUNSHI ,  PAGET STEVEN ,  ZHANG NANJING ,  ZHANG XIAOYAN ,  ZHU JIN

IPC: C07D209/30 ,  A61K31/405 ,  A61P31/12 ,  C07D209/42 ,  C07D401/04 ,  C07D401/14 ,  C07D403/12 ,  C07D403/14 ,  C07D409/12 ,  C07D413/12 ,  C07D417/10 ,  C07D417/12 ,  C07D417/14

CPC classification number: C07D209/30 ,  C07D209/42 ,  C07D401/04 ,  C07D401/14 ,  C07D403/12 ,  C07D403/14 ,  C07D409/12 ,  C07D413/12 ,  C07D417/10 ,  C07D417/12 ,  C07D417/14

Abstract: The present invention provides compounds, pharmaceutical compositions, and methods of using such compounds or compositions for treating infection by a virus, or for affecting viral IRES activity.

Abstract translation: 本发明提供化合物，药物组合物和使用这些化合物或组合物治疗病毒感染或影响病毒IRES活性的方法。

公开(公告)号：WO2007084413B1

公开(公告)日：2008-01-17

申请号：PCT/US2007000923

申请日：2007-01-16

Applicant: PTC THERAPEUTICS INC ,  KARP GARY MITCHELL ,  AREFOLOV ALEXANDER ,  REN HONGYU ,  TURPOFF ANTHONY ALLAN ,  WILDE RICHARD GERALD ,  TAKASUGI JAMES JAN ,  HWANG PETER SEONGWOO ,  CHEN GUANGMING ,  CAMPBELL JEFFREY ALLEN

Inventor： KARP GARY MITCHELL ,  AREFOLOV ALEXANDER ,  REN HONGYU ,  TURPOFF ANTHONY ALLAN ,  WILDE RICHARD GERALD ,  TAKASUGI JAMES JAN ,  HWANG PETER SEONGWOO ,  CHEN GUANGMING ,  CAMPBELL JEFFREY ALLEN

IPC: C07D209/30 ,  A61K31/405 ,  A61P31/12 ,  C07D209/42 ,  C07D401/04 ,  C07D401/14 ,  C07D403/12 ,  C07D403/14 ,  C07D409/12 ,  C07D413/12 ,  C07D417/10 ,  C07D417/12 ,  C07D417/14

CPC classification number: C07D209/30 ,  C07D209/42 ,  C07D401/04 ,  C07D401/14 ,  C07D403/12 ,  C07D403/14 ,  C07D409/12 ,  C07D413/12 ,  C07D417/10 ,  C07D417/12 ,  C07D417/14

Abstract: The present invention provides compounds, pharmaceutical compositions, and methods of using such compounds or compositions for treating infection by a virus, or for affecting viral IRES activity.

Abstract translation: 本发明提供化合物，药物组合物和使用这些化合物或组合物治疗病毒感染或影响病毒IRES活性的方法。

公开(公告)号：WO2007106317A2

公开(公告)日：2007-09-20

申请号：PCT/US2007004721

申请日：2007-02-23

Applicant: SCHERING CORP ,  PTC THERAPEUTICS INC ,  LAHSER FREDERICK C ,  KARP GARY MITCHELL

Inventor： LAHSER FREDERICK C ,  KARP GARY MITCHELL

CPC classification number: A61K31/401 ,  A61K31/506 ,  A61K45/06 ,  A61K2300/00

Abstract: Disclosed are medicaments, pharmaceutical compositions, pharmaceutical kits, and methods based on combinations of: (a) at least one HCV IRES inhibitor; and (b) at least one HCV protease inhibitor; and optionally (c) at least one other therapeutic agent; for concurrent or consecutive administration in treating or ameliorating one or more symptoms of HCV, or disorders associated with HCV in a subject in need thereof.

Abstract translation: 公开了药物，药物组合物，药物盒和基于以下组合的方法：（a）至少一种HCV IRES抑制剂; 和（b）至少一种HCV蛋白酶抑制剂; 和（c）至少一种其它治疗剂; 用于同时或连续给药以治疗或改善HCV的一种或多种症状或与有需要的受试者中与HCV相关的病症。

公开(公告)号：WO2016039936A3

公开(公告)日：2016-08-18

申请号：PCT/US2015045436

申请日：2015-08-15

Applicant: PTC THERAPEUTICS INC

Inventor： BRANSTROM ARTHUR A ,  NARASIMHAN JANA ,  DUMBLE MELISSA L ,  WEETALL MARLA L ,  KARP GARY MITCHELL ,  GERASYUTO ALEKSEY IGOREVICH

IPC: A61K31/44

CPC classification number: A61K31/497 ,  A61K31/4418 ,  A61K31/4427 ,  A61K31/4433 ,  A61K31/4439 ,  A61K31/444 ,  A61K31/4545 ,  A61K31/496 ,  A61K31/4995 ,  A61K31/506 ,  A61K31/5377 ,  A61K31/5383 ,  A61K45/06

Abstract: The present description relates to the use of monocyclic ring substituted 2-pyridinone compounds and forms thereof for treating or ameliorating Neisseria gonorrhoeae. The present description relates to use of a substituted 2-pyridinone compound for treating or ameliorating Neisseria gonorrhoeae (N. gonorrhoeae) in a subject in need thereof comprising, administering an effective amount of a compound of Formula (I) or a form thereof to the subject: wherein R1, R2 or R3 are as defined herein, and forms and compositions thereof.

Abstract translation: 本说明书涉及单环环取代的2-吡啶酮化合物及其形式用于治疗或改善淋病奈瑟氏球菌的用途。 本说明书涉及取代的2-吡啶酮化合物在有需要的受试者中用于治疗或改善淋病奈瑟氏球菌（淋病奈瑟氏球菌）的用途，包括：将有效量的式（I）化合物或其形式给予 主题：其中R1，R2或R3如本文所定义，以及其形式和组成。