About Us

We are a clinical-stage biotechnology company applying our leadership position in the field of gene therapy, and calcium dysregulation to develop novel therapies for diseases with tremendous unmet medical needs. Our lead programs target Sarco/endoplasmic reticulum Ca2+ -ATPase, or SERCA, enzymes, which are a family a family of enzymes that play an integral part in the regulation of intra-cellular calcium in all human cells. Calcium dysregulation is implicated in a number of important and complex medical conditions and diseases, such as heart failure, which is a clinical syndrome characterized by poor heart function, resulting in inadequate blood flow to meet the body’s metabolic needs, as well as diabetes and neurodegenerative diseases. We are applying our leading expertise in the field of SERCA2 biology towards the development of gene therapies and small molecule compounds to correct SERCA2 deficiencies and the resulting calcium imbalances within diseased cells. MYDICAR, our most advanced product candidate, uses gene therapy to target SERCA2a, which is an enzyme that becomes deficient in patients with heart failure.