Mydicar

Mydicar (AAV1/SERCA2a) is an investigational gene therapy developed by Celladon Corporation for the treatment of chronic heart failure. It utilizes an adeno-associated virus type 1 (AAV1) vector to deliver the SERCA2a gene (sarcoplasmic reticulum calcium ATPase 2a) directly to the heart muscle via a one-time intracoronary infusion. The SERCA2a enzyme is responsible for transporting calcium ions from the cytoplasm into the sarcoplasmic reticulum, a process essential for cardiac muscle relaxation and subsequent contraction. In failing hearts, SERCA2a levels are typically reduced, leading to impaired calcium cycling and decreased pumping efficiency. Mydicar aims to restore these enzyme levels to improve myocardial function. Despite showing promise in early-stage trials (CUPID), the therapy failed to meet its primary endpoints in the Phase 2b CUPID-2 trial in 2015, leading to the discontinuation of its clinical development. Imperial College London sponsored a specific Phase 2 trial to evaluate the safety and feasibility of this gene transfer in advanced heart failure patients with implanted left ventricular assist devices (LVADs).

Brand names

Mydicar

Other names

AAV1/SERCA2a gene therapy