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Therapeutic Dosing of Synthetic Antimi Further Demonstrates the Potential of microRNA-208 Inhibition in Treatment of Chronic Heart Failure

Research by miRagen Therapeutics published in Circulation

BOULDER, Colo.--(BUSINESS WIRE)--Sep 6, 2011 - miRagen Therapeutics, Inc., a preclinical-stage biopharmaceutical company focused on improving patients' lives by developing innovative microRNA (miRNA)-based therapeutics for cardiovascular and muscle disease, announced that new preclinical data published in today's online edition of Circulation suggest that therapeutic inhibition of microRNA-208 (miR-208) may improve cardiac function and survival rates during heart failure.

In this study, chemically-synthesized inhibitors of miR-208 were shown to suppress pathological cardiac remodeling in a model of heart failure induced by chronic high blood pressure, while enhancing cardiac function and survival. While previous data have demonstrated the essential role of miR-208 in pathological cardiac remodeling [van Rooij, Science 2007], this is the first study to show that the inhibition of miR-208 can halt heart disease progression. These results also suggest that antimiR-208 treatment may be effective in the treatment of diastolic heart failure (DHF). DHF, together with systolic heart failure (SHF), are two clinical subsets of the syndrome of chronic heart failure that are most commonly encountered in clinical practice. Both SHF and DHF represent areas of significant unmet medical need.

“These findings greatly enhance our understanding of miR-208's important role in the pathogenesis and progression of heart failure,” said Eric N. Olson, Ph.D., Chief Scientific Advisor and Co-founder of miRagen Therapeutics, Inc. “In addition, these results provide further evidence of the potential of systemically-delivered oligonucleotide-based therapeutics in the setting of heart disease.”

“We are very pleased to share these exciting results, which further underscore the potential for miR-208 inhibition to provide meaningful improvement across a range of clinical measures of heart failure,” said William S. Marshall, Ph.D., President and Chief Executive Officer of miRagen Therapeutics, Inc. “Not only do these findings provide additional validation of miR-208 as a promising target for therapeutic intervention, but they also enhance our understanding of microRNA biology and help to advance our mission to develop innovative microRNA-based therapeutics to treat patients afflicted with cardiovascular disease.”

About microRNAs: MicroRNAs have emerged as an important class of small RNAs encoded in the genome. They act to control the expression of sets of genes and entire pathways and are thus thought of as master regulators of gene expression. Recent studies have demonstrated that microRNAs are associated with many disease processes. Because they are single molecular entities that dictate the expression of fundamental regulatory pathways, microRNAs represent potential drug targets for controlling many biologic and disease processes.

About miRagen Therapeutics: miRagen Therapeutics, Inc., was founded in 2007 to develop innovative microRNA-based therapeutics for cardiovascular and muscle disease. Only recently discovered, microRNAs are short, single-stranded RNA molecules encoded in the genome that regulate gene expression and play a vital role in influencing cardiovascular and muscle disease. Cardiovascular disease is the leading cause of death globally and represents an enormous burden on global healthcare systems. Principally funded through venture capital investments, miRagen combines world recognized leadership in cardiovascular medicine with unprecedented in-house expertise in microRNA biology and chemistry.

Posted: September 2011