Development Status
Preclinical studies combining Targeted Genetics’ expertise in the development and manufacture of AAV-based therapies with Celladon’s portfolio of genes and cardiovascular expertise have been conducted. Preclinical data suggest that gene therapy targeting calcium cycling within the heart may improve myocardial contractility and reverse the progression of congestive heart failure. A study was conducted in a sheep model designed to have a reduction in heart function similar to that found in patients with Class 3 heart failure. Compared with control animals, six treated sheep showed significant improvement in heart function and contractility. At one month after treatment, average heart function in treated sheep improved, while average function in control animals declined. Given positive results from these studies and from other preclinical studies, this program moved into human clinical studies in the May 2007.
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