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：WO2014209841A2

公开(公告)日：2014-12-31

申请号：PCT/US2014043577

申请日：2014-06-23

Applicant: HOFFMANN LA ROCHE ,  HOFFMANN LA ROCHE ,  PTC THERAPEUTICS INC

Inventor： DAKKA AMAL ,  GREEN LUKE ,  KARP GARY ,  NARASIMHAN JANA ,  NARYSHKIN NIKOLAI ,  PINARD EMMANUEL ,  QI HONGYAN ,  RATNI HASANE ,  RISHER NICOLE ,  WEETALL MARLA ,  WOLL MATTHEW

IPC: A61K31/4985

CPC classification number: C07D519/00 ,  C07D263/56 ,  C07D413/12 ,  C07D471/04 ,  C07D487/04 ,  C07D487/10

Abstract: The present invention provides compounds of formula (I) wherein A, B, X, Y, R1 and R2 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.

Abstract translation: 本发明提供其中A，B，X，Y，R 1和R 2如本文所述的式（I）化合物及其药学上可接受的盐。 此外，本发明涉及制备式（I）化合物，包含它们的药物组合物及其作为药物的用途。

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。

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：EP2812004A4

公开(公告)日：2015-11-11

申请号：EP13747100

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 MARLA L ,  WELCH ELLEN ,  WOLL MATTHEW G ,  YANG TIANLE ,  ZHANG NANJING ,  ZHANG XIAOYAN ,  ZHAO XIN ,  GREEN LUKE ,  PINARD EMMANUEL ,  RATNI HASANE

IPC: A61K31/53 ,  A61K31/4375 ,  A61K31/519 ,  A61P25/00 ,  C07D471/04 ,  C07D487/04 ,  C07D519/00

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：EP2750504A4

公开(公告)日：2015-03-11

申请号：EP12828218

申请日：2012-08-29

Applicant: PTC THERAPEUTICS INC

Inventor： BRANSTROM ARTHUR ,  JOSYULA VARA PRASAD VENKATA NAGENDRA ,  ARNOLD MICHAEL ANDREW ,  GERASYUTO ALEKSEY I ,  KARP GARY ,  WANG JIASHI ,  BAIRD JOHN DAVID ,  CHEN GUANGMING ,  GINZBURG OLYA ,  YANG TIANLE ,  SMITH SEAN WESLEY ,  PEDDI SRINIVASA ,  WOLL MATTHEW G ,  ZHANG XIAOYAN ,  ZHANG NANJING ,  QI HONGYAN ,  NARASIMHAN JANA

IPC: A01N43/40

CPC classification number: C07D471/04 ,  A61K31/437 ,  A61K31/4439 ,  A61K31/4709 ,  A61K31/5377 ,  A61K45/06 ,  C07D401/04 ,  C07D401/14 ,  C07D405/14 ,  C07D413/04

Abstract: The present description relates to compounds and forms and pharmaceutical compositions thereof and methods for use thereof to treat or ameliorate bacterial infections caused by wild-type and multi-drug resistant Gram-negative and Gram-positive pathogens.

公开(公告)号：EP3013345A4

公开(公告)日：2016-11-30

申请号：EP14816651

申请日：2014-06-23

Applicant: HOFFMANN LA ROCHE ,  PTC THERAPEUTICS INC

Inventor： DAKKA AMAL ,  GREEN LUKE ,  KARP GARY ,  NARASIMHAN JANA ,  NARYSHKIN NIKOLAI ,  PINARD EMMANUEL ,  QI HONGYAN ,  RATNI HASANE ,  RISHER NICOLE ,  WEETALL MARLA ,  WOLL MATTHEW

IPC: A61K31/497 ,  A61K31/4965 ,  C07D401/00

CPC classification number: C07D519/00 ,  C07D263/56 ,  C07D413/12 ,  C07D471/04 ,  C07D487/04 ,  C07D487/10

Abstract: The present invention provides compounds of formula (I) wherein A, B, X, Y, R1 and R2 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.

Abstract translation: 本发明提供其中A，B，X，Y，R 1和R 2如本文所述的式（I）化合物及其药学上可接受的盐。 此外，本发明涉及制备式（I）化合物，包含它们的药物组合物及其作为药物的用途。

公开(公告)号：EP2750505A4

公开(公告)日：2015-08-05

申请号：EP12828566

申请日：2012-08-29

Applicant: PTC THERAPEUTICS INC

Inventor： BRANSTROM ARTHUR ,  JOSYULA VARA PRASAD VENKATA NAGENDRA ,  ARNOLD MICHAEL ANDREW ,  GERASYUTO ALEKSEY I ,  KARP GARY ,  WANG JIASHI ,  BAIRD JOHN DAVID ,  PEDDI SRINIVASA ,  ARASU TAMIL ,  DU WU ,  GORSKE YI JIN KIM ,  TURPOFF ANTHONY ALLAN ,  WILDE RICHARD GERALD ,  GINZBURG OLYA ,  ZHANG NANJING ,  QI HONGYAN ,  ZHANG XIAOYAN ,  SMITH SEAN WESLEY ,  YANG TIANLE ,  NARASIMHAN JANA

IPC: A01N43/40 ,  A61K31/44

CPC classification number: C07D515/04 ,  A01N43/40 ,  A01N43/50 ,  A01N43/56 ,  A01N43/60 ,  A01N43/647 ,  A01N43/84 ,  A01N43/90 ,  A01N47/38 ,  A61K31/4412 ,  A61K31/4427 ,  A61K31/4439 ,  A61K31/496 ,  A61K31/5377 ,  A61K31/5383 ,  A61K45/06 ,  C07D213/80 ,  C07D401/10 ,  C07D487/08 ,  A61K2300/00

Abstract: The present description relates to compounds and forms and pharmaceutical compositions thereof and methods for use thereof to treat or ameliorate bacterial infections caused by wild-type and multi-drug resistant Gram-negative and Gram-positive pathogens.

公开(公告)号：CO2024017547A2

公开(公告)日：2025-03-27

申请号：CO2024017547

申请日：2024-12-20

Applicant: PTC THERAPEUTICS INC

Inventor： BHATTACHARYYA ANURADHA ,  CHEN GUANGMING ,  JIANG YAO ,  NARASIMHAN JANA ,  WOLL MATTHEW G ,  ZHANG NANJING ,  ZHANG XIAOYAN ,  ARNOLD MICHAEL A ,  ANNAND JAMES R ,  ARACHCHILAGE GAYAN MIRIHANA ,  CHIU JACKIE ,  DAHIYA GAURAV ,  KIZHAKKAYIL MANGADAN ARUN RAJ ,  MORRILL CHRISTIE ,  MOTIKA STEPHEN E

IPC: A61K31/4365 ,  A61K31/517 ,  A61K31/519 ,  C07D495/04

Abstract: Un aspecto de la presente descripción se refiere a compuestos útiles para mejorar el corte y empalme de pre ARNm en una célula. En particular, otro aspecto de la presente descripción se refiere a compuestos heteroarilo sustituidos, formas y composiciones farmacéuticas de los mismos y métodos de uso para tratar o mejorar la ataxia espinocerebelosa tipo 3 (SCA3), también conocida como enfermedad de Machado-Joseph (MJD).