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Celladon Corporation Announces Pre-clinical Study Demonstrating Inhalable MYDICAR Reverses Pulmonary Arterial Hypertension (PAH)
Date:7/30/2013

ism, when clots in the legs travel to the lungs and cause blockages. When the lung is damaged from these conditions, the tissue starts to quickly produce new and enlarged cells, which narrows pulmonary arteries. This increases the pressure inside them. The high pressure in these arteries resists the heart's effort to pump through them and the blood flow between the heart and lungs is reduced. The right side of the heart then must overcome the resistance and work harder to push the blood through the pulmonary arteries into the lungs. Over time, the right ventricle becomes thickened and enlarged and heart failure develops.

The MYDICAR gene therapy that Dr. Hajjar developed uses a modified adeno-associated viral-vector (AAV-vector). It works by introducing a healthy SERCA2a gene into cells, but this gene does not incorporate into a patient's chromosome, according to the study's lead author, Lahouaria Hadri, PhD, an Instructor of Medicine in Cardiology at Icahn School of Medicine at Mount Sinai. "The clinical trials in congestive heart failure have shown already that the gene therapy is very safe," says Dr. Hadri.

The clinical application of MYDICAR for patients with PAH will differ from those with heart failure. When treating heart failure patients, MYDICAR is infused through the coronary arteries of heart failure patients using catheters, while in PAH patients, MYDICAR will need to be administered through inhalation.

This study was supported by National Institutes of Health grants (K01HL103176, K08111207, R01 HL078691, HL057263, HL071763, HL080498, HL083156, and R01 HL105301).

About MYDICAR in Heart Failure
MYDICAR is a genetically targeted enzyme replacement therapy intended to restore levels of SERCA2a, a regulator of calcium cycling in the heart and cardiac contractility. SERCA2a levels decline in all forms of late-stage HF resulting in deficient heart function. With MYDICAR, the SERCA2a gene is delivered using recombinant adeno-a
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