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R & D Product Overview Enzyme Replacement
R & D Product Overview Enzyme Replacement
| Enzyme Replacement Therapy |
A New Paradigm: Treating Heart Failure as an Enzyme DeficiencyThe target of Celladon’s drugs is central to the ability of the heart to pump blood, and is determined by the movement of cytosolic calcium ions (Ca2+) in and out of an organelle in the heart cells called the sarcoplasmic reticulum (SR). The key factor controlling this movement of Ca2+ into the SR is the ATPase SERCA2a, an enzyme that declines significantly in all forms of heart failure as the disease progresses. The search for a positive inotropic drug (increases contractility) that could relieve symptoms and improve the prognosis in patients with heart failure has been extensive. However, all current inotropic drugs used to increase contractility of the heart have a negative effect on long-term survival. By targeting SERCA2a, Celladon’s first-in-class drugs reduce the O2 cost of contractility and improve survival in preclinical studies.
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