Its initial focus is on the treatment of heritable cardiomyopathies, a group of rare, genetically-driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction.

MyoKardia uses precision medicine techniques which involve discovering and developing therapies that integrate clinical and molecular information based on the biological basis of disease. This approach has led to the efficient discovery and development of transformative therapies in areas such as oncology, cystic fibrosis and hypercholesterolemia, but has yet to be broadly applied to cardiovascular diseases.

The company was launched in 2012 by Third Rock Ventures, and was founded by experts in cardiovascular disease, cardiac muscle biology and genetics.

It has used its precision medicine platform to generate an initial pipeline of therapeutic programs for the chronic treatment of the two most common forms of heritable cardiomyopathy—hypertrophic cardiomyopathy and dilated cardiomyopathy.