Genzyme Corporation

Anadys Pharmaceuticals, Inc.

Scriptgen Pharmaceuticals, Inc.

University of Massachusetts

Anadys Pharmaceutical, Inc.

James W Lillie

Jaime E Arenas

Andrew A Pakula

Lance Gavin Laing

Sharon T Cload

Bradford Hirth

John P Leonard

Elyse Bourque

Elina Makino

Hanlan Liu

Mary A Cromwell

Bing Wang

Paul Mason

Fazeela Morshed

Lingyun Li

Thomas O'Shea

Renato Sklerj

Michael R Green

Giovanni Perini

The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.

Glucosylceramide synthase inhibitors

The present invention provides methods for screening for bioactive compounds, in particular those that bind to RNA sequences involved in the pathogenesis of disease or in regulation of a physiological function. The methods involve assessing the stability and/or the conformation of an RNA target in the presence and absence of test ligands, and identifying as a ligand any test ligand that causes a measurable change in target RNA stability and/or conformation. In a preferred embodiment, the effect of a ligand on target RNA stability and/or conformation is assessed by measuring the fluorescence polarization of a fluorescently labeled probe.

Screen employing fluorescence anisotropy to identify compounds with affinity for nucleic acids

The present invention provides methods for high-throughput screening for bioactive compounds, in particular those that bind to RNA sequences involved in the pathogenesis of disease or in regulation of a physiological function. The methods involve measuring the conformation of an RNA target in the presence and absence of test ligands, and identifying as a ligand any test ligand that causes a measurable change in target RNA conformation.

Screen for compounds with affinity for nucleic acids

Disclosed is a method for screening for inhibitors of Hepatitis B Virus pX activity. The method involves contacting a test compound with (I) the pX protein of HBV, (ii) a transcription factor comprising the bZIP domain, or fragments that comprise a minimal a bZIP domain, and (iii) an oligoduplex comprising a target DNA sequence of the transcription factor to form a test mixture. After incubating the test mixture under appropriate conditions and for a sufficient time to allow pX-mediated dimerization and DNA binding of the transcription factor to occur, the level of DNA binding of the transcription factor in each test mixture is determined. A test compound is considered to be any compound that causes a decrease in the level of DNA binding in the test mixture relative to the level of DNA binding in control mixtures.

Methods for screening for inhibitors of the transcription-enhancing

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Natick, MA, United States of America

James W Lillie