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Study Published in Nature Biotechnology Demonstrates Ability of Moderna's Novel Therapeutic Paradigm to Repair and Regenerate Damaged Heart Tissue
Date:9/8/2013

Chien. "Over the past 20 years, many labs have attempted to repair damaged heart muscle in animal models by delivering VEGF-A and other factors to the heart using recombinant proteins, DNA plasmids, and engineered viruses—with little if any clinical success. The results we have seen using messenger RNA therapeutics™ mark the start of a new and exciting phase of drug development with potentially profound implications for patients."

In the study, the mice injected with VEGF-A-coded messenger RNA produced a "pulse-like" expression of a significant quantity of VEGF-A near the injection site. The production of VEGF-A in turn mobilized heart progenitor cells to produce functional, intact blood vessels in the heart and led to marked improvements in heart function and improved long-term survival of the mice for at least one year post-MI. Researchers attribute these effects to a reduction in the size of infarct region and an increase in capillary density in the affected region. In this way, the high-level, transient production of VEGF-A led to a sustained therapeutic improvement by altering the behavior of heart progenitor cells. 

Introduction of modRNA was not associated with an innate immune response, further supporting the safety and effectiveness of this novel approach.

"We are extremely proud that our messenger RNA therapeutics™ platform enabled Ken to realize his vision and clinical intuition of the last 20 years," said Stephane Bancel, president and founding CEO of Moderna. "Beyond the considerable implications for the treatment of heart injury, this publication provides an important validation of the potential of messenger RNA therapeutics™ across a wide range of diseases. The ability to stimulate protein production within cells will enable the treatment of many diseases that cannot be addressed today. What is also exciting is the fact that the Moderna team has considerably improved our
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SOURCE Moderna Therapeutics
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