Ptc Therapeutics, Inc.

Pct Therapeutics, Inc.

Peter Seongwoo Hwang

Young-Choon Moon

Neil Gregory Almstead

James Jan Takasugi

Gary Mitchell Karp

Jeffrey Allen Campbell

Guangming Chen

Richard Gerald Wilde

Hongyu Ren

Anthony A Turpoff

Alexander Arefolov

Ellen Welch

Xiaoyan Zhang

Nanjing Zhang

Steven David Paget

Chunshi Li

Jin Zhu

Hongyan Qi

Langdon Miller

Seemon H Pines

Zhengxian Gu

James J Takasugi

Don Corson

Jeff Campbell

William Lennox

Neil G Almstead

Soongyu Choi

Liangxian Cao

Marla L Weetall

Samit Hirawat

William Jospeh Lennox

Thomas W Davis

Christopher Robert Trotta

Donald Thomas Corson

Harry H Miao

Paul D Kennedy

Jason D Graci

Tamil Arasu

Arasu Tamil

Christine Espiritu

Concetta Freund

Takashi Komatsu

Ellen M Welch

Seemon Pines

Thomas Davis

Maria L Weetall

The present invention relates to crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid, pharmaceutical compositions and dosage forms comprising the crystalline forms, methods of making the crystalline forms and methods for their use for the treatment, prevention or management of diseases ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay.

Crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid for the treatment of disease

Provided herein are improved processes for the synthesis of substituted tetrahydro beta-carboline derivatives. In particular, provided herein are improved processes useful for the preparation of (S)-4-chlorophenyl 6-chloro-1-(4-methoxyphenyl)-3,4-dihydro-1H-pyrido[3,4-b]indole-2(9H)-carboxylate. Formula (I): 

Processes for the preparation of substituted tetrahydro beta-carbolines

Compounds, such as compounds of Formula (I), that selectively inhibit pathological production of human vascular endothelial growth factor (VEGF) and compositions comprising such Compounds are described. Compounds that inhibit viral replication or the production of viral RNA or DNA or viral protein and compositions comprising such Compounds are described. Also described are methods of reducing VEGF using such Compounds and methods for treating cancer and non-neoplastic conditions involving the administration of such Compounds. Further described are methods of inhibiting viral replication or the production of viral RNA or DNA or viral protein using such Compounds and methods for treating viral infections involving the administration of such Compounds. The Compounds may be administered as a single agent therapy or in combination with one or more additional therapies to a human in need of such treatments. 

Methods for treating cancer and non-neoplastic conditions

Crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4] oxadiazol-3-yl]-benzoic acid

The present invention relates to functional proteins encoded by nucleic acid sequences comprising a nonsense mutation. The present invention also relates to methods for the production of functional proteins encoded by nucleic acid sequences comprising a nonsense mutation and the use of such proteins for prevention, management and/or treatment of diseases associated with a nonsense mutation(s) in a gene.

Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewith

Crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid

Novel hydroxylated 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising a hydroxylated 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.

Hydroxylated 1,2,4-oxadiazole benzoic acid compounds, compositions thereof and their use in bioassays

Provided herein are processes for the preparation of compounds useful for the treatment, prevention or management of diseases associated with a nonsense mutation. More specifically, provided herein are processes for the synthesis of 1,2,4-oxadiazoles. In particular, provided herein are processes useful for the preparation of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid.

Processes for the preparation of 1,2,4-oxadiazole benzoic acids

Hydroxylated 1,2,4-oxadiazole benzoic acid compounds and compositions thereof

In accordance with the present invention, compounds that inhibit viral replication, preferably Hepatitis C Virus (HCV) replication, have been identified, and methods for their use provided. In one aspect of the invention, compounds useful in the treatment or prevention of a viral infection are provided. In another aspect of the invention, compounds useful in the treatment or prevention of HCV infection are provided.

Methods for treating hepatitis C

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.

Growing community of inventors

Edison, NJ, United States of America

Peter Seongwoo Hwang