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The Journal of Cardiac Failure Publishes Results From Celladon Corporation's First-in-Human Phase 1/2 Clinical Trial in Advanced Heart Failure
Date:4/8/2009

Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID Trial) Demonstrated Safety and Biological Effects of SERCA2a Enzyme Replacement Using Gene Transfer

SAN DIEGO, April 8 /PRNewswire/ -- Celladon Corporation today announced the publication of results from a Phase 1/2 trial of MYDICAR(R) (AAV1/SERCA2a), a genetically targeted enzyme replacement therapy for advanced heart failure, in the April 2009 issue of the Journal of Cardiac Failure. The CUPID clinical trial demonstrated acceptable safety and produced quantitative evidence of biological activity across a number of parameters important for assessing heart failure. MYDICAR is designed to restore levels of an enzyme, SERCA2a, known to play a key role in the progression of heart failure. Repairing this molecular defect in animal studies has been shown to reverse the disease and restore cardiac function.

"Despite important therapeutic advances in pharmacologic and device therapies, the prognosis of patients with chronic heart failure remains extremely poor. Moreover, heart transplantation and the use of implantable assist devices are considered only in the later stages of the disease, and access to such therapies is restricted to a fraction of patients in need," said Brian E. Jaski, M.D., Medical Director of Advanced Heart Failure, Sharp Memorial Hospital, San Diego Cardiac Center, San Diego, a principal investigator on the study, and a lead author of the publication. "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. Our objective 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."

The results of this study include data from the Phase 1 portion of this ongoing Phase 1/2 study for Cohorts 1 a
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SOURCE Celladon Corporation
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