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Celladon Corporation Announces Pre-clinical Study Demonstrating Inhalable MYDICAR Reverses Pulmonary Arterial Hypertension (PAH)
Date:7/30/2013

SAN DIEGO, July 30, 2013 /PRNewswire/ -- Celladon Corporation, a clinical-stage biotechnology company committed to applying its first-mover, leadership position in the field of SERCA enzymes to transform the lives of patients with important, life-threatening diseases, announced today that an international team of researchers led by investigators at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai demonstrate that the deadly condition known as pulmonary arterial hypertension (PAH), which afflicts up to 150,000 Americans each year, may be reversible by using an inhalable form of Celladon's gene therapy product candidate MYDICAR (AAV1/ SERCA2a).

In their new study, reported in the July 30 issue of the journal Circulation, scientists demonstrated that MYDICAR administered through a nebulizer-like inhalation device can completely reverse PAH in rat models of the disease. In the lab, researchers also showed in pulmonary artery PAH patient tissue samples reduced expression of the SERCA2a, an enzyme critical for proper pumping of calcium in calcium compartments within the cells. MYDICAR could therefore be sought as a promising therapeutic intervention in PAH.

Krisztina Zsebo Ph.D., President and CEO of Celladon Corp and a co-author of the study said "Gene therapy represents an entirely new therapeutic modality for the treatment of PAH. We are very excited about the potential of using MYDICAR for this purpose. There is a tremendous need for new treatment options in PAH and we look forward to conducting additional studies investigating the potential benefit of MYDICAR in this patient population."

"The MYDICAR gene therapy could be delivered very easily to patients through simple inhalation — just like the way nebulizers work to treat asthma," says study co-senior investigator Roger J. Hajjar, MD, Director of the Cardiovascular Research Center and the Arthur &
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