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Celladon Corporation Announces Presentation of First-in-Human Gene Transfer to Treat Heart Failure at the 12th Annual Meeting of the American Society of Gene Therapy
Date:5/26/2009

Phase 1/2 Calcium Upregulation by Percutaneous Administration (CUPID) Data Highlight Safety Profile and Biological Effects of SERCA2a Enzyme Replacement

SAN DIEGO, May 26 /PRNewswire/ -- Celladon Corporation today announced that results from its genetically targeted enzyme replacement therapy for advanced heart failure, including Phase 1/2 data of MYDICAR(R) (AAV1/SERCA2a), will be presented by Krisztina M. Zsebo, PhD, President and CEO, Celladon Corporation and Howard Dittrich, MD, Cardiologist at University of California, San Diego School of Medicine, in a Scientific Symposium on Thursday, May 28 at the 2009 American Society of Gene Therapy Annual Meeting, San Diego Convention Center.

"A key underpinning of heart failure is the depletion of SERCA2a enzyme in heart muscle cells, which results in abnormal intracellular calcium cycling and poor contractility," said Zsebo. "Our research shows that recombinant adeno-associated viral vectors (AAV) are ideal for restoring SERCA2a enzyme production by means of cardiac muscle gene transfer. The path of investigation for MYDICAR(R) (AAV1/SERCA2a), from rodent studies to the ongoing Phase II clinical trial, has yielded valuable insights for the entire gene therapy field, dispelling many myths and revealing strategies to enhance vector uptake in humans with numerous applications."

While conventional drug therapies have improved survival of patients with congestive heart failure, the disease progresses relentlessly, resulting in more than 300,000 deaths each year, 6.5 million annual hospital days and an estimated $35 billion in direct and indirect healthcare expenditures. Recent advances in understanding the molecular basis of myocardial dysfunction, together with the evolution of safe and efficient gene transfer technology, have placed heart failure within reach of gene-based therapy. A Phase 1/2 clinical trial of dose escalation of MYDICAR administered dire
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