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71. 发明申请

WO2004009610A3 USE OF NUCLEOSIDE COMPOUNDS FOR NONSENSE SUPPRESSION AND THE TREATMENT OF GENETIC DISEASES 审中-公开
标题翻译：核苷类化合物用于非感染抑制和治疗遗传性疾病的用途
公开(公告)号：WO2004009610A3
公开(公告)日：2005-10-06
申请号：PCT/US0323185
申请日：2003-07-23
申请人： PTC THERAPEUTICS INC , TULARIK INC , WILDE RICHARD G , ALMSTEAD NEIL G , WELCH ELLEN M , BECKMANN HOLGER
发明人： WILDE RICHARD G , ALMSTEAD NEIL G , WELCH ELLEN M , BECKMANN HOLGER
IPC分类号： C07H19/00 , C07H17/02 , A61K31/7064 , A61P7/00 , A61P25/00 , A61P37/00
CPC分类号： A61K31/506 , A61K31/7072 , C07H19/00
摘要： The invention encompasses nucleoside compounds, compositions comprising the compounds and methods for treating or preventing diseases associated with nonsense mutations of mRNA by administering these compounds or compositions.
摘要翻译：本发明包括核苷化合物，包含该化合物的组合物和通过施用这些化合物或组合物来治疗或预防与无义突变mRNA相关的疾病的方法。

72. 发明申请

WO2004087069A3 METHODS OF IDENTIFYING COMPOUNDS THAT TARGET tRNA SPLICING ENDONUCLEASE AND USES OF SAID COMPOUNDS AS ANTI-PROLIFERATIVE AGENTS 审中-公开
标题翻译：鉴定目标tRNA分离内切酶的化合物的方法和作为抗增殖剂的合成化合物的用途
公开(公告)号：WO2004087069A3
公开(公告)日：2005-08-25
申请号：PCT/US2004009572
申请日：2004-03-26
申请人： PTC THERAPEUTICS INC , TROTTA CHRISTOPHER R
发明人： TROTTA CHRISTOPHER R
IPC分类号： A01N61/00 , A61K20060101 , C12Q1/00 , C12Q1/68 , G01N33/566 , G01N33/573 , G01N33/574
CPC分类号： C12N9/22 , C12Q1/6897 , C12Q2539/105
摘要： The present invention relates to a method for screening and identifying compounds that modulate the activity tRNA splicing endonuclease. In particular, the invention provides assays for the identification of compounds that inhibit animalia tRNA splicing endonuclease. The methods of the present invention provide a simple, sensitive assay for high-throughput screening of libraries of compounds to identify pharmaceutical leads useful for treating and/or preventing cancer.
摘要翻译：本发明涉及筛选和鉴定调节活性tRNA剪接核酸内切酶的化合物的方法。特别地，本发明提供了用于鉴定抑制动物TRNA剪接内切核酸酶的化合物的测定。本发明的方法提供了用于鉴定可用于治疗和/或预防癌症的药物引用的化合物文库的高通量筛选的简单，灵敏的测定。

73. 发明申请

WO2005003316A2 RNA PROCESSING PROTEIN COMPLEXES AND USES THEREOF 审中-公开
标题翻译： RNA加工蛋白复合物及其用途
公开(公告)号：WO2005003316A2
公开(公告)日：2005-01-13
申请号：PCT/US2004/021334
申请日：2004-07-02
申请人： PTC THERAPEUTICS, INC. , TROTTA, Christopher, R. , PAUSHKIN, Sergey
发明人： TROTTA, Christopher, R. , PAUSHKIN, Sergey
IPC分类号： C12N
CPC分类号： C12Q1/6897 , C07K14/47 , C12N9/22
摘要： The invention provides human protein complexes with endonuclease activity. In particular, the invention provides human protein complexes with tRNA splicing endonuclease activity and/or 3' end pre-mRNA endonuclease activity. The invention also provides a splice variant of human Sen2, namely human Sen2deltaEx8, and human protein complexes comprising human Sen2deltaEx8. The human Sen2deltaEx8 complexes have pre-tRNA cleavage activity and/or 3' end pre-mRNA endonuclease activity. The invention also provides human protein complexes with pre-ribosomal RNA cleavage activity. The invention also provides antibodies that immunospecifically bind to a complex described herein or a component thereof, and methods of diagnosing, preventing, treating, managing or ameliorating a disorder utilizing such antibodies. The present invention also provides methods utilizing the complexes described herein, inter alia , in screening, diagnosis, and therapy. The invention further provides methods of preparing and purifying the complexes. The present invention further provides methods of identifying a compound that modulates the expression of a component of a complex described herein, the formation of a complex described herein or the activity of a complex described herein, and methods of preventing, treating, managing or ameliorating a disorder, such as a proliferative disorder, or a symptom thereof utilizing a compound identified in accordance with the methods.
摘要翻译：本发明提供具有内切核酸酶活性的人蛋白复合物。特别地，本发明提供具有tRNA剪接核酸内切酶活性和/或3'末端pre-mRNA内切酶活性的人蛋白复合物。本发明还提供人Sen2的剪接变体，即人Sen2deltaEx8，以及包含人Sen2deltaEx8的人蛋白复合物。人Sen2deltaEx8复合物具有pre-tRNA切割活性和/或3'末端pre-mRNA内切核酸酶活性。本发明还提供具有前核糖体RNA切割活性的人蛋白复合物。本发明还提供免疫特异性结合本文所述的复合物或其组分的抗体，以及利用此类抗体诊断，预防，治疗，控制或改善病症的方法。本发明还提供了利用本文所述的复合物，特别是在筛选，诊断和治疗中的方法。本发明还提供了制备和纯化复合物的方法。本发明还提供了鉴定调节本文所述复合物组分的表达，本文所述的复合物的形成或本文所述复合物的活性的化合物的方法，以及预防，治疗，治疗或改善障碍，例如增殖性疾病，或其症状，利用根据该方法鉴定的化合物。

74. 发明申请

WO2004091502A3 1,2,4-OXADIAZOLE BENZOIC ACID COMPOUNDS 审中-公开
公开(公告)号：WO2004091502A3
公开(公告)日：2004-10-28
申请号：PCT/US2004/011106
申请日：2004-04-09
申请人： PTC THERAPEUTICS, INC. , KARP, Gary, Mitchell , HWANG, Seongwoo , CHEN, Guangming , ALMSTEAD, Neil, Gregory
发明人： KARP, Gary, Mitchell , HWANG, Seongwoo , CHEN, Guangming , ALMSTEAD, Neil, Gregory
IPC分类号： C07D271/06
摘要： 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.

75. 发明申请

WO2004087884A3 TARGETING ENZYMES OF THE tRNA SPLICING PATHWAY FOR IDENTIFICATION OF ANTI-FUNGAL AND/OR ANTI-PROLIFERATIVE MOLECULES 审中-公开
公开(公告)号：WO2004087884A3
公开(公告)日：2004-10-14
申请号：PCT/US2004/009590
申请日：2004-03-26
申请人： PTC THERAPEUTICS, INC , TROTTA, Christopher, R.
发明人： TROTTA, Christopher, R.
IPC分类号： A01N61/00
摘要： The present invention relates to a method for screening and identifying compounds that modulate the activity of one or more components in the tRNA splicing pathway. In particular the invention relates to a method for screening and identifying compounds that modulate the activity tRNA splicing endonuclease and/or tRNA splicing ligase. The invention provides assays for the identification of compounds that inhibit animalia tRNA splicing endonuclease and/or animalia tRNA splicing ligase. The invention also provides assays for the identification of compounds that inhibit fungal tRNA splicing endonuclease and/or fungal tRNA splicing ligase. The methods of the present invention provide a simple, sensitive assay for high-throughput screening of libraries of compounds to identify pharmaceutical leads useful for treating and/or preventing cancer and/or fungal infections.

76. 发明申请

WO2023028534A1 INHIBITORS OF NLRP3 审中-公开
公开(公告)号：WO2023028534A1
公开(公告)日：2023-03-02
申请号：PCT/US2022/075421
申请日：2022-08-24
申请人： PTC THERAPEUTICS, INC.
发明人： ZHANG, Xiaoyan , ALAM, Rauful , BARRAZA, Scott, J. , BEJCEK, Lauren , GILBERT, Bradley, B. , GONG, Hua , HANDOKO, Handoko , HOSSEYNI, Seyedmorteza , HUARTE, Eduardo , JEON, Woohyung , LI, Jing , LIU, Yao , NIEDERER, Kyle , PARKER, Erica, N. , PILLAI, Meenu , RASTELLI, Ettore , SYDORENKO, Nadiya , TURPOFF, Anthony , WOLL, Matthew, G. , ZHANG, Nanjing , ZHANG, Yan , ZHENG, Tianyi
IPC分类号： C07D401/12 , C07D401/14 , C07D403/12 , C07D405/14 , C07D417/14 , C07D471/04 , C07D487/04 , C07D495/04 , C07D498/04 , C07D519/00 , A61P25/28 , A61K31/502 , A61K31/5025
摘要： The present invention relates to novel compounds of Formulae I-XI: wherein each A, A', Q, Q', W, Rw, Y, and Z, and -- are as defined herein, which inhibit NOD-like receptor protein 3 (NLRP3) inflammasome activity. The invention further relates to the processes for their preparation, pharmaceutical compositions and medicaments containing them, and their use in the treatment of diseases and disorders mediated by NLRP3.

77. 发明申请

WO2022173823A1 METHODS FOR TREATING COVID-19 WITH SEPIAPTERIN 审中-公开
公开(公告)号：WO2022173823A1
公开(公告)日：2022-08-18
申请号：PCT/US2022/015810
申请日：2022-02-09
申请人： PTC THERAPEUTICS MP, INC. , VIRGINIA COMMONWEALTH UNIVERSITY
发明人： CLARK, Gene, Chatman , MEZZAROMA, Eleonora , RABENDER, Christopher , MIKKELSEN, Ross, B. , YAKOVLEV, Vasily , SMITH, Neil
IPC分类号： A61K31/519 , A61P11/00 , A61P31/12
摘要： The invention features methods of treating a SARS-CoV-2 infection in a subject, the method comprising administering to the subject an effective amount of sepiapterin or a pharmaceutically acceptable salt thereof.

78. 发明申请

WO2022169868A1 COMPOUNDS FOR TREATING SPINOCEREBELLAR ATAXIA TYPE 3 审中-公开
公开(公告)号：WO2022169868A1
公开(公告)日：2022-08-11
申请号：PCT/US2022/014934
申请日：2022-02-02
申请人： PTC THERAPEUTICS INC.
发明人： ARACHCHILAGE, Gayan Mirihana , ARNOLD, Michael A. , BARRAZA, Scott , BHATTACHARYYA, Anuradha , KARP, Gary Mitchell
IPC分类号： A61K31/519
摘要： The present description relates to methods of treating spinocerebellar ataxia type 3 (SCA3) using substituted thieno[3,2-d]pyrimidine compounds, forms, and pharmaceutical compositions thereof.

79. 发明申请

WO2021226478A1 DHODH INHIBITOR FOR THE TREATMENT OF COVID-19 审中-公开
公开(公告)号：WO2021226478A1
公开(公告)日：2021-11-11
申请号：PCT/US2021/031326
申请日：2021-05-07
申请人： PTC THERAPEUTICS, INC.
发明人： NARYSHKIN, Nikolai, A. , GRACI, Jason , BAIRD, John , COLACINO, Joseph , WEETALL, Marla, L. , CAO, Liangxian
IPC分类号： A61K31/437 , A61K31/4545 , A61K31/5377
摘要： One aspect described herein includes a method for treating coronavirus 2019 disease (COVID-19) caused by severe respiratory syndrome coronavirus 2 (SARS-CoV-2) by administering an effective amount of a DHODH inhibitor. Another aspect described herein includes a method of treating COVD-19 by administering 4-chlorophenyl (S)-6-chloro-1-(4-methoxyphenyl)-1,3,4,9-tetrahydro-2H-pyrido[3,4-b]indole-2-carboxylate (PTC299), or a pharmaceutically acceptable salt thereof.

80. 发明申请

WO2020185648A1 COMPOUND FORM HAVING ENHANCED BIOAVAILABILITY AND FORMULATIONS THEREOF 审中-公开
公开(公告)号：WO2020185648A1
公开(公告)日：2020-09-17
申请号：PCT/US2020/021648
申请日：2020-03-09
申请人： PTC THERAPEUTICS, INC.
发明人： UDDIN, Akm, Nasir , DALI, Mandar, Vasant , VAZE, Onkar, Shripad
IPC分类号： C07D487/04
摘要： The present description generally relates to amorphous forms of compounds having enhanced aqueous solubility and dissolution rates and amorphous solid dispersions and oral pharmaceutical formulations thereof, and to processes for preparing the same. The present description specifically relates to amorphous forms of 5-fluoro-2-(6-fluoro-2-methyl-1H-benzimidazol-1-yl)-N-[4-(trifluoromethyl)phenyl]pyrimidine-4,6-diamine and amorphous solid dispersions and oral pharmaceutical formulations thereof, and to processes for preparing the same.

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