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EGEN, Inc. Advances a Systemically Delivered RNAi Therapeutic for the Treatment of Pulmonary Arterial Hypertension (PAH)
Date:6/5/2013

HUNTSVILLE, Ala., June 5, 2013 /PRNewswire/ -- EGEN, Inc., a clinical stage biopharmaceutical  company, announced today that  EGEN's proprietary lung targeting Staramine-mPEG nanoparticle delivery system has been used to administer an inhibitor of microRNA-145 (anti-miRNA-145) in a rat model of severe occlusive PAH.  Treatment was shown to significantly reverse the pulmonary hypertension associated with the advanced stages of the disease and restore cardiovascular function.  This program, in collaboration with Dr. William Gerthoffer at the University of South Alabama College of Medicine, is focused on evaluating new approaches that can be used to prevent vascular remodeling that occurs in patients with PAH which leads to the deleterious cardiovascular effects associated with the disease.  The results of this work were recently presented at the 2013 American Thoracic Society Annual Meeting held in Philadelphia, PA.  The abstract titles were:  "Targeted Pulmonary Delivery of a microRNA Inhibitor Reverses Severe Pulmonary Arterial Hypertension in Rats", J.M. Mclendon presenting author, and "Treatment with Anti-microRNA in an Experimental Model of Occlusive Pulmonary Arterial Hypertension Reverses Vascular Remodeling", S.R Joshi presenting author.

MicroRNAs (miRNAs) are endogenously produced non-coding RNAs that bind to partially complimentary target sites in messenger RNA leading to degradation of transcript or translation inhibition.  In this way, miRNAs can post-transcriptionally regulate gene expression in a cell.  Increasingly, it is being recognized that miRNA disregulation may be the root cause of many diseases.  In PAH, an increase in one particular miRNA (miRNA-145) has been linked to pulmonary vascular remodeling.  Thus, it has been hypothesized that inhibiting the miRNA with an
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