Amgen Inc.

Ptc Therapeutics, Inc.

Other

Tularik Limited

Holger Beckmann

Neil Gregory Almstead

Ellen Welch

Richard Gerald Wilde

Bei Shan

Patrick A Baeuerle

James Jan Takasugi

Jin-Long Chen

Steven Marc Rubenstein

Stuart W Peltz

Kevin Czaplinski

Kevin Lustig

Marc Learned

Novel acetylamino benzoic acid compounds, methods of using and pharmaceutical compositions comprising an acetylamino 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.

Use of Acetylamino benzoic acid compounds for nonsense suppression and the treatment of disease

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.

Use of nucleoside compounds for nonsense suppression and the treatment of genetic diseases

Acetylamino benzoic acid compounds and their use for nonsense suppression and the treatment of disease

Methods for identifying modulators of nuclear hormone receptor function comprise the steps of (a) forming a mixture comprising a nuclear hormone receptor, a peptide sensor and a candidate agent, but not a natural coactivator protein of the receptor, wherein the sensor provides direct, in vitro binding to the receptor under assay conditions; (b) measuring an agent-biased binding of the sensor to the receptor; and (c) comparing the agent-biased binding with a corresponding unbiased binding of the sensor to the receptor. In particular embodiments, the sensor comprises an amphipathic alpha helix nuclear hormone interacting domain comprising a recited nuclear hormone transcriptional coactivator motif sequence, the sensor is present at sub-micromolar concentration, the binding reaction occurs in solution, the sensor comprises a fluorescent label and the measuring step comprises detecting fluorescence polarization of the label. Reagents include labeled sensor peptides and reaction mixtures consisting essentially of nuclear hormone receptor, a peptide and a candidate agent.

Nuclear hormone receptor drug screens

The present application provides methods of assaying for compounds that inhibit premature translation termination and nonsense mediated RNA decay in cells.

Methods of assaying for compounds that inhibit premature translation termination and nonsense-mediated RNA decay

Nuclear hormone receptor fluorescence polarization assay

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Freiburg, Germany

Holger Beckmann