Despite important therapeutic improvements in pharmacological and device therapies, the prognosis for patients with advanced cardiovascular disease is poor, even with optimal pharmacological and device management. Heart failure continues to be a major cause of morbidity and mortality in the US. It is the leading medical cause of hospitalization and is expected to result in an estimated direct and indirect cost to the healthcare system in 2009 of $37.2 billion.¹

Non-pharmacological therapies (such as heart transplantation and the use of implantable left ventricular assist devices) are considered only in the later stages of the disease, and access to such therapies is restricted to a fraction of patients who could potentially benefit from them. Specifically lacking are interventions that reverse the myocardial contractile deficiency that initially created the heart failure state.^2,3

In this context, alternative approaches such as cell and gene therapy have attracted increased attention. Gene therapies have historically been challenged by ethical concerns over potential manipulation of the human genome. However, in the last decade gene transfer agents composed of defined sets of proteins and nucleic acids have become available that are much more amenable to rigorous quality control and potency assessments than their cell therapy counterparts, which presents challenges for consistent manufacturability. In the US, oversight of the clinical development and ultimate approval of new drugs and biological agents comes under the auspices of the US Food and Drug Administration (FDA). The FDA is familiar with viralbased therapies from the review of vaccine products and has approved a number of agents based on naturally occurring viruses also composed of defined proteins and nucleic acids, as shown in Table 1.

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