Lonza is to carry out GMP manufacture of Celladon’s Phase IIb-stage targeted enzyme replacement therapy candidate Mydicar® (AAV1/SERCA2a) for clinical trials, and potentially for commercial supply. The viral vector-based treatment is designed to restore levels of the SERCA2a, a regulator of calcium cycling in the heart, and cardiac contractility. Lonza says it will manufacture Mydicar initially at the 1,000 liter scale and potentially up to 2,000 liters, at its recently completed and validated suite.
“This is an important milestone in the Mydicar® development program,” comments Krisztina Zsebo, Ph.D., Celladon president and CEO. “Lonza’s large-scale viral manufacturing expertise will greatly complement and support our future development and commercialization efforts for Mydicar.”
Levels of SERCA2a decline in all forms of late-stage heart failure, resulting in deficient heart function. Mydicar has been developed as a recombinant AAV-based vector carrying the SERCA2a-gene, which is administered in a single dose directly to the heart during routine outpatient cardiac catheterization procedure. The product is undergoing Phase II trials in patients with advanced heart failure. Earlier this year Celladon raised $53 million in a venture financing round to support continued clinical development of the product.