S100A1 AAV gene therapy

S100A1 AAV gene therapy is an investigational gene therapy designed to treat heart failure and cardiomyopathy by restoring levels of the S100A1 protein. S100A1 is a calcium-binding protein that acts as a positive inotropic regulator of myocardial contractility; its levels are typically depleted in failing cardiomyocytes. By delivering the human S100A1 gene via adeno-associated virus (AAV) vectors—including AAV9, AAV6, and AAV5 serotypes—the therapy aims to normalize cardiomyocyte calcium cycling, improve sarcoplasmic reticulum calcium handling, and enhance mitochondrial energy homeostasis. Developed by researchers at Thomas Jefferson University and the University of Heidelberg, and associated with the biotech company AaviGen, the therapy has demonstrated the ability to rescue post-ischemic heart failure and reverse left ventricular dysfunction in large animal models. It is also being investigated for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy.

Other names

S100A1 gene therapyS-100A1 gene therapyS 100A1 gene therapy