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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

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 directly to heart cells with minimally invasive cardiac catheterization is currently underway. This clinical study represents the first-in-human trial of enzyme replacement therapy in heart failure using AAV and has demonstrated acceptable safety and produced quantitative evidence of biological activity across a number of parameters important for assessing heart failure. Celladon is testing the hypotheses that restoring SERCA2a enzyme production will improve ventricular function in patients with advanced heart failure.

About Celladon

Celladon Corporation, based in La Jolla, California, was launched in October 2004 as a privately held biotechnology company founded with the goal of becoming the leader in developing molecular therapies for the treatment of heart failure. The company's products target the key enzyme deficiency in advanced heart failure, SERCA2a, which regulates calcium cycling and contractility in heart muscle cells. Celladon's first product candidate, MYDICAR, delivers the gene for the SERCA2a enzyme. MYDICAR is currently being tested in Phase 1 and 2 clinical trials. Celladon is also developing traditional small molecule activators of SERCA2a for the treatment of heart failure. To learn more about Celladon, visit Celladon's website at

SOURCE Celladon Corporation
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