Description
In collaboration with Celladon Corporation, Targeted Genetics is evaluating AAV-based delivery of SERCA2a and phospholamban (PLB) gene variants. The SERCA2a and phospholamban genes encode proteins that are key regulators of the heart’s ability to contract. Studies conducted in the laboratory of Dr. Roger J. Hajjar, Director of the Cardiovascular Laboratory of Integrative Physiology and Imaging at Massachusetts General Hospital and co-founder of Celladon, demonstrate that SERCA2a activity is decreased in heart tissue obtained from heart failure patients and that delivery of the SERCA2a gene can normalize contractility in animal models of heart failure. Research conducted in the laboratory of Dr. Kenneth R. Chien, Director of the Cardiovascular Research Center, Massachusetts General Hospital and Director of the Harvard Stem Cell Institute, Harvard Medical Center and co-founder of Celladon, indicates that inhibition of phospholamban activity using PLB gene variants leads to normalization of heart function in heart failure models.
The pathway through which these genes help to regulate the contractility of the heart has been considered a promising drug development target for some time, yet efforts to develop small molecule therapeutics targeting this pathway have been unsuccessful. We believe that gene delivery may provide a new approach to unlocking the therapeutic potential of this target.
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