MyoKardia is a South San Francisco-based clinical-stage biopharmaceutical company that aims to develop targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. The company's lead product candidate is mavacamten, an orally-administered small molecule in Phase III clinical trials designed to reduce left ventricular contractility and prevent or reverse HCM progression. The company also develops MYK-491, an orally-administered small molecule in Phase IIa clinical trials designed to restore normal cardiac muscle contractility in diseased DCM hearts. The company has also developed various preclinical programs such as MYK-224 & LUS-1 for HCM treatment and ACT-1 targeting genetic DCM. MyoKardia has a collaboration with 23andMe, Inc. to better research consumer genetics and rare cardiovascular diseases.