Intrexon Corporation

Rohm and Haas Company

Percipiad, Inc.

Precigen, Inc.

Robert Eugene Hormann

Colin M Tice

Orestes Chortyk

Glenn Richard Carlson

Thomas R Opie

Inna Shulman

David W Potter

Andrew Meyer

Sheela K Chellappan

Dat Phat Le

Dean Ervin Cress

Tarlochan Singh Dhadialla

Howard Smith

Thomas Meteyer

Bing Li

Enrique Luis Michelotti

Subba Reddy Palli

Rolf Hilfiker

Susan Margaret De Paul

Arthur John Kudla

Eva Rödel

Ronald Phillip Herzig, Jr

Mohan Philip

Silvia Lapenna

Laurence Neil Dinan

Ray A James

Zev Lidert

Edward M Sioma

Malla Padidam

Daryl Eugene Gilbert

Eva Rodel

Ronald Phillip Herzig

Susan M Depaul

The present invention provides boron-containing isoquinoline compounds as protein kinase-modulating compounds. These compounds are useful as neuroprotective and neuro-regenerative agents for the amelioration of glaucoma and other ocular neuropathies.

Boron-containing rho kinase inhibitors

The present disclosure provides boron-containing diacylhydrazines having Formula I: and the pharmaceutically acceptable salts and solvates thereof, wherein R, R, R, R, and Rare defined as set forth in the specification. The present disclosure also provides the use of boron-containing diacylhydrazines is ecdysone receptor-based inducible gene expression systems. Thus, the present disclosure is useful for applications such as gene therapy, treatment of disease, large scale production of proteins and antibodies, cell-based screening assays, functional genomics, proteomics, metabolomics, and regulation of traits in transgenic organisms, where control of gene expression levels is desirable.

Boron-containing diacylhydrazines

The present invention provides diacylhydrazine ligands and chiral diacylhydrazine ligands for use with ecdysone receptor-based inducible gene expression systems. Thus, the present invention is useful for applications such as gene therapy, large scale production of proteins and antibodies, cell-based screening assays, functional genomics, proteomics, metabolomics, and regulation of traits in transgenic organisms, where control of gene expression levels is desirable. An advantage of the present invention is that it provides a means to regulate gene expression and to tailor expression levels to suit the user's requirements.

Chiral diacylhydrazine ligands for modulating the expression of exogenous genes via an ecdysone receptor complex

The present disclosure provides boron-containing diacylhydrazines having Formula I: and the pharmaceutically acceptable salts and solvates thereof, wherein A, R, and Rare defined as set forth in the specification. The present disclosure also provides the use of boron-containing diacylhydrazines is ecdysone receptor-based inducible gene expression systems. Thus, the present disclosure is useful for applications such as gene therapy, treatment of disease, large scale production of proteins and antibodies, cell-based screening assays, functional genomics, proteomics, metabolomics, and regulation of traits in transgenic organisms, where control of gene expression levels is desirable.

Boron-containing diacylhydrazine compounds

The present disclosure provides crystalline polymorphic and amorphous forms of (R)-3,5-dimethyl-benzoic acid N-(1-tert-butyl-butyl)-N'-(2-ethyl-3-methoxy-benzoyl)-hydrazide (Compound 1) or (S)-3,5-dimethyl-benzoic acid N-(1-tert-butyl-butyl)-N&#x2032;-(2-ethyl-3-methoxy-benzoyl)-hydrazide (Compound 2). The present disclosure further provides compositions comprising crystalline polymorphic and amorphous forms of Compound 1 or Compound 2 and an excipient, methods of making crystalline polymorphic or amorphous forms of Compound 1 or Compound 2, and methods of using crystalline polymorphic or amorphous forms of Compound 1 or Compound 2 to regulate gene expression in a cell or in a subject.

Crystalline diacylhydrazine and the use thereof

This invention relates to a method to modulate exogenous gene expression in which an ecdysone receptor complex comprising: a DNA binding domain; a ligand binding domain; a transactivation domain; and a ligand is contacted with a DNA construct comprising: the exogenous gene and a response element; wherein the exogenous gene is under the control of the response element and binding of the DNA binding domain to the response element in the presence of the ligand results in activation or suppression of the gene. The ligands comprise a class of ketones.

Ketone ligands for modulating the expression of exogenous genes via an ecdysone receptor complex

The present invention relates to non-steroidal ligands for use in nuclear receptor-based inducible gene expression system, and a method to modulate exogenous gene expression in which an ecdysone receptor complex comprising: a DNA binding domain; a ligand binding domain; a transactivation domain; and a ligand is contacted with a DNA construct comprising; the exogenous gene and a response element; wherein the exogenous gene is under the control of the response element and binding of the DNA binding domain to the response element in the presence of the ligand results in activation or suppression of the gene.

Bioavailable diacylhydrazine ligands for modulating the expression of exogenous genes via an ecdysone receptor complex

The present invention relates to non-steroidal ligands for use in nuclear receptor-based inducible gene expression system, and a method to modulate exogenous gene expression in which an ecdysone receptor complex comprising: a DNA binding domain; a ligand binding domain; a transactivation domain; and a ligand is contacted with a DNA construct comprising: the exogenous gene and a response element; wherein the exogenous gene is under the control of the response element and binding of the DNA binding domain to the response element in the presence of the ligand results in activation or suppression of the gene.

Diacylhydrazine ligands for modulating the expression of exogenous genes in mammalian systems via an ecdysone receptor complex

The present invention relates to steroidal ligands for use in nuclear receptor-based inducible gene expression systems. The invention further relates to methods of modulating the expression of genes of interest with a system containing one or more nuclear receptor complexes and one or more steroidal ligands. Further aspects include ligand compositions including therapeutic compositions.

Steroidal ligands and their use in gene switch modulation

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Melrose Park, PA, United States of America

Robert Eugene Hormann