First participant dosed in novel AAV-gene therapy trial for DMD-associated cardiomyopathy

Medera, and its clinical division Sardocor, announced that the first participant was dosed with AAV1.SERCA2a in a Phase 1b clinical trial for cardiomyopathy secondary to Duchenne.

Cardiac complications are the leading cause of mortality in Duchenne, and this experimental gene therapy delivers the SERCA2a gene with the goal of correcting the calcium dysregulation in cardiac muscle that leads to progressive fibrosis and heart failure.  This experimental therapy is delivered locally to the heart via the intracoronary blood vessels.  The study plans to enroll up to 12 participants, and is currently recruiting.

Link to press release HERE

Link to clinical trial info: currently recruiting