This same SERCA2a dysfunction also occurs in heart failure. This new study utilizes Celladon's product candidate MYDICAR which is currently under investigation in an international 200 patient Phase IIb trial in the United States and Europe. In 2012, Celladon and the Mt. Sinai School of Medicine entered into a license agreement granting Celladon certain exclusive worldwide rights to develop and commercialize SERCA2a gene therapy products for PAH.
"What we have shown is that gene therapy restores function of this crucial SERCA2a enzyme in diseased lungs," says Dr. Hajjar. "We are delighted with these new findings because it suggests that MYDICAR which is already showing great benefit in congestive heart failure patients may be able to help PAH patients who currently have no good treatment options — and are in critical need of a life sustaining therapy."
When SERCA2a is down-regulated, calcium stays longer in the cells than it should, and it induces pathways that lead to overgrowth of new and enlarged cells. According to researchers, the delivery of the SERCA2a gene through the MYDICAR product in turn produces SERCA2a enzymes, which helps both heart and lung cells restore their proper use of calcium.
"We are now on a path toward PAH patient clinical trials in the near future," says Dr. Hajjar, a co-founder of Celladon who developed the gene therapy approach. Studies in large animal models are now underway. SERCA2a gene therapy has already been approved by the National Institutes of Health for human study.
A Simple MYDICAR Inhalation Corrects Deadly Dysfunction
PAH most commonly results from heart failure in the left side of the heart or from a pulmonary embol
|SOURCE Celladon Corporation|
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