Ptc Therapeutics, Inc.

Pct Therapeutics, Inc.

Liangxian Cao

Thomas W Davis

Young-Choon Moon

Soongyu Choi

Hongyan Qi

Marla L Weetall

William Jospeh Lennox

Donald Thomas Corson

Christopher Robert Trotta

Nadarajan Tamilarasu

Seongwoo Hwang

Langdon Miller

Charles Romfo

Panayiota Trifillis

Samit Hirawat

Nadiya Sydorenko

Harry H Miao

Nikolai A Naryshkin

Richard Gerald Wilde

Steven David Paget

Hongyu Ren

Chang-Sun Lee

Ramil Y Baiazitov

Wu Du

Ronggang Liu

Matthew C Pellegrini

Don Corson

Jeff Campbell

William Lennox

Neil Gregory Almstead

Jeffrey Allen Campbell

Peter Seongwoo Hwang

Zhengxian Gu

Tamil Arasu

Jason D Graci

Daniel J Medina

Kenneth P Olive

Melissa L Dumble

Jaime A Eberle-Singh

Anuradha Roy

Anuradha Mehta

Matthew C Pelligrini

Marites A Rafanan

Charles M Romfo

William Joseph Lennox

Thomas Davis

Maria L Weetall

A method for treating pancreatic cancer in a subject in need thereof comprising, administering to the subject an effective amount of a tubulin polymerization inhibitor compound is described herein. More particularly, a method for treating pancreatic ductal adenocarcinoma in a subject in need thereof comprising, administering to the subject an effective amount of a substituted reverse pyrimidine tubulin polymerization inhibitor compound alone or in combination with other chemo-therapeutic agents is described herein.

Method for treating pancreatic cancer

A method of use for Cpd 1 as an inhibitor of dihydroorotate dehydrogenase (DHODH) function in treating or ameliorating a hematological cancer in a subject in need thereof comprising, administering an effective amount of Cpd 1 to the subject, having the structure: or a form or pharmaceutical composition thereof.

DHODH inhibitor for use in treating hematologic cancers

Amine substituted reverse pyrimidine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.

Substituted reverse pyrimidine Bmi-1 inhibitors

The present invention relates to methods for identifying compounds that modulate untranslated region-dependent expression of a target gene. The invention particularly relates to using untranslated regions of a target gene or fragments thereof linked to a reporter gene to identify compounds that modulate untranslated region-dependent expression of a target gene. The methods of the present invention provide a simple, sensitive assay for high-throughput screening of libraries of compounds to identify pharmaceutical leads.

Methods for identifying compounds that modulate untranslated region-dependent gene expression and methods of using same

The invention relates to the fields of screening assays, compounds, and methods for altering gene expression and protein levels. In particular, the invention includes assays to screen for agents capable of modulating gene expression in a UTR-dependent manner and agents capable of modulating gene expression.

Methods and agents for screening for compounds capable of modulating gene expression

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

In accordance with the present invention, tetracyclic caboline derivatives that inhibit the expression of VEGF post-transcriptionally have been identified, and methods for their use provided. In one aspect of the invention, these compounds useful in the inhibition of VEGF production, in the inhibition of angiogenesis, and/or in the treatment of cancer, diabetic retinopathy or exudative macular degeneration are provided. In another aspect of the invention, methods are provided for the inhibition of VEGF production, the inhibition of angiogenesis, and/or the treatment of cancer, diabetic retinopathy or exudative macular degeneration using the compounds of the invention.

Tetra-cyclic carboline derivatives useful in the inhibition of angiogenesis

Methods for treating prostate cancer involving the administration of a compound that selectively inhibits pathological production of human vascular endothelial growth factor (VEGF) are described. Methods for treating benign prostatic hyperplasia involving the administration of a compound that selectively inhibits pathological production of human VEGF are also described. The compound may be administered as a single-agent therapy or in combination with one or more additional therapies to a human in need of such treatment.

Methods for treating prostate conditions

Methods for treating Kaposi's sarcoma involving the administration of a compound that selectively inhibits pathological production of human VEGF are described. The compound can be administered as a single-agent therapy or in combination with one or more additional therapies to a human in need of such treatment.

Methods for treating Kaposi sarcoma

Compounds having Formula I: wherein the variables Ar, Ar, R, Rand Rare defined herein, pharmaceutical compositions, and methods of using such compounds or compositions for inhibiting Bmi-1 expression.

BMI-1 protein expression modulators

The methods described herein provide nucleic acid constructs and screening methods for identifying and validating compounds for use in the treatment of cancer, wherein the compounds down-regulate the post-transcriptional expression of Bmi-1.

Methods for screening for compounds for treating cancer

The invention relates to the fields of screening assays and compounds and methods for altering protein expression and levels of protein. In particular, the invention includes assays to screen for agents capable of modulating expression of VEGF and agents capable of modulating VEGF expression.

Methods and agents for screening for compounds capable of modulating VEGF expression

Growing community of inventors

Parlin, NJ, United States of America

Liangxian Cao