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Celladon Announces Presentation of Clinical Data From First-in-Human MYDICAR(R) Trial for Advanced Heart Failure at American Heart Association Scientific Sessions
Date:11/9/2008

Phase 1 Data Demonstrate Safety and Improvement in Heart Failure in a

Majority of Patients

NEW ORLEANS, Nov. 9 /PRNewswire/ -- Celladon Corporation announced today results from the first nine patients treated with MYDICAR(R), a genetically-targeted enzyme replacement therapy for advanced heart failure, showing the product was safe and demonstrating improvement across a number of key parameters. Phase 1 data from the "Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID Trial), a First-in-Human Phase 1/2 Clinical Trial" were presented at the American Heart Association Scientific Sessions 2008.

This first phase of the multi-center trial was designed to investigate safety and biological effects of restoring SERCA2a enzyme activity in heart muscle cells. The enzyme levels are decreased in late stages of heart failure, and extensive research shows loss of SERCA2a levels represents a common pathway resulting in a defect in the ability of the heart to contract properly. Replacing the enzyme may restore function and reverse heart failure.

"The CUPID trial is the first to attempt to rescue a failing heart by replacing an enzyme known to play a critical role in normal cardiac muscle cell activity," said Brian E. Jaski, M.D., Medical Director of Advanced Heart Failure, Sharp Memorial Hospital, San Diego Cardiac Center, San Diego, and a principal investigator on the study. "Our objective in this study is not only to improve the symptoms of heart failure, but restore physiologic function and reverse the severity of the disease in this chronic patient population."

Celladon scientists, led by company co-founder Roger J. Hajjar, M.D., Director of the Cardiovascular Research Center at Mount Sinai School of Medicine, New York, developed MYDICAR for restoring the SERCA2a calcium transporter in heart failure and validated the overall beneficial effects on cardiac function. MYDICAR
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SOURCE Celladon Corporation
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