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Study Published In Nature Biotechnology Demonstrates Ability Of Moderna's Novel Therapeutic Paradigm To Repair And Regenerate Damaged Heart Tissue

CAMBRIDGE, Mass., Sept. 8, 2013 /PRNewswire/ --  Moderna Therapeutics, the pioneering company developing messenger RNA therapeutics™, a revolutionary new treatment modality to enable the in vivo production of therapeutic proteins, announced today that two of its academic co-founders published a study in Nature Biotechnology demonstrating that Moderna's messenger RNA therapeutics™ platform induced in vivo production of proteins to stimulate blood vessel growth, repair damaged heart tissue, and improve outcomes in a mouse model of myocardial infarction (MI).

The research, led by Moderna's academic co-founder Kenneth Chien, M.D., Ph.D., with the help of academic co-founder Derrick Rossi, Ph.D., provides proof-of-concept that messenger RNA therapeutics™ provide an effective and robust platform for gene transfer that could be applicable across a number of diseases. The Nature Biotechnology paper is the first peer-reviewed scientific publication describing the ability of messenger RNA therapeutics™ to stimulate in vivo production of human proteins and to achieve a long-term therapeutic effect.

Researchers injected mice with a single dose of VEGF-A-coded messenger RNA in the heart shortly following myocardial infarction to trigger production of the VEGF-A protein. VEGF-A (vascular endothelial growth factor-A) is an important angiogenic factor protein that mediates the growth of blood vessels. Mice injected with messenger RNA went on to generate new cardiovascular cells and show improvements on measures of cardiac function and survival when compared with controls.

"Regeneration of heart tissue through the mobilization of heart progenitor cells holds tremendous potential for the treatment of myocardial infarction and other forms of heart injury but has remained an elusive goal for over a decade," said Dr. 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."

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