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RNAi Therapeutic Delivery Using EGEN's Nanoparticle System for PAH Treatment

Published on June 7, 2013 at 7:04 AM

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 anti-miR (an oligonucleotide that targets miRNA) will slow or reverse established PAH-related vascular remodeling and restore normal pulmonary function. EGEN's Staramine-mPEG nanoparticle delivery system overcomes delivery challenges by targeting the site of the miRNA-145 expression; namely the lung.

"Our approach may provide a potential breakthrough in the treatment of PAH because of the strong mechanistic linkages between up regulation of miRNA-145 that we are targeting and the vascular remodeling associated with the disease," said Dr. William Gerthoffer, Professor and Chair, Biochemistry & Molecular Biology, University of South Alabama College of Medicine. "Use of the Staramine-mPEG delivery system is a natural fit for this anti-miR approach where targeted delivery to the lung is a requirement."

"There is a clear unmet medical need for novel agents that can treat PAH," said Jason Fewell, Ph.D., VP of Preclinical Research and Development at EGEN, Inc. "We have been very encouraged by the positive efficacy results that have been achieved by Dr. Gerthoffer's group in their rat PAH model using our proprietary delivery technology and believe that targeting miRNAs has tremendous potential as a novel therapeutic for this devastating disease."

Source: http://www.egeninc.com

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