The Salk Institute for Biological Studies

Other

The Scripps Research Institute

Irm LLC

Hamilton

Enrique Saez

Ronald Mark Evans

John Wityak

Xiaolin Li

Valentina Molteni

Juliet Nabakka

Beth Anaclerio

Jeffery W Kelly

Fang Liang

David Archer Ellis

Christina Cooley

Lars Plate

R Luke Wiseman

Julia M D Grandjean

Aparajita Madhavan

Bernard Kok

David No

Disclosed herein are compounds, their pharmaceutical salts, and pharmaceutical compositions that selectively activate the inositol-requiring enzyme 1 (IRE1)/X-box binding protein 1 (XBP1s) signaling pathway of the unfolded protein response (UPR), but that do not target the IRE1 kinase domain. The compounds are useful in treating diseases or conditions characterized by imbalances in proteostasis within the endoplasmic reticulum (ER) or secretory pathway, including those not associated with ER stress or activation of UPR.

IRE1 activating compounds for use in therapy

The invention provides compounds, pharmaceutical compositions comprising such compounds and methods of using such compounds to treat or prevent diseases or disorders associated with the activity of liver X receptors (LXRs).

Compounds and compositions as LXR modulators

In accordance with the present invention, there are provided various methods for modulating the expression of an exogenous gene in an isolated cell and in a mammalian subject employing modified ecdysone receptors. Also provided are modified ecdysone receptors, as well as homomeric and heterodimeric receptors containing same, nucleic acids encoding invention modified ecdysone receptors, modified hormone response elements, gene transfer vectors, recombinant cells, and transgenic animals containing nucleic acids encoding invention modified ecdysone receptor.

Methods for modulating expression of exogenous genes in mammalian systems

In accordance with the present invention, there are provided various methods for modulating the expression of an exogenous gene in a mammalian subject employing modified ecdysone receptors. Also provided are modified ecdysone receptors, as well as homomeric and heterodimeric receptors containing same, nucleic acids encoding invention modified ecdysone receptors, modified ecdysone response elements, gene transfer vectors, recombinant cells, and transgenic animals containing nucleic acids encoding invention modified ecdysone receptor.

Method for modulating expression of exogenous genes in mammalian systems, and products related thereto

In accordance with the present invention, there are provided various methods for modulating the expression of an exogenous gene in a mammalian subject employing modified ecdysone receptors. Also provided are modified ecdysone receptors, as well as homomeric and heterodimeric receptors containing same, nucleic acids encoding invention modified ecdysone receptors, modified hormone response elements, gene transfer vectors, recombinant cells, and transgenic animals containing nucleic acids encoding invention modified ecdysone receptor.

Formulations useful for modulating expression of exogenous genes in mammalian systems, and products related thereto

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San Diego, CA, United States of America

Enrique Saez