Our Approach to Precision Medicine for Heart Failure
We have developed a translational discovery platform using patient-specific cardiac disease models. Our platform has three critical components:
Our Patient BioBank
Through our exclusive relationship with the Stanford Cardiovascular Institute (CVI), we have built an extensive BioBank of samples from patients with a variety of inherited heart disorders such as hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM) and channelopathies. The BioBank continues to grow as new patients and family members are recruited to the Stanford CVI.
Patient-derived Disease Models
We use stem cell technology to generate cardiac disease models from our BioBank. Using a few key transcription factors, adult somatic cells, such as skin and blood, are collected under informed consent and reprogrammed into induced pluripotent stem (iPS) cells. Patient-specific cardiomyocytes are differentiated from the pluripotent stem cells. The patient-derived cardiomyocytes exhibit a variety of disease phenotypes, including hypertrophy, arrhythmic beating, aberrant calcium handling properties and contractility defects.
Precision Medicine Drug Discovery
Disease models are screened using a combination of molecular and phenotypic assay to identify novel drug candidates that rescue the gene mutation-specific phenotypes. Drugs with mutation specific responses may be assessed in a broader set of disease models to investigate potential use in other patient sub-populations. Our platform also enables the simultaneous assessment of acute cardiotoxicity (e.g. QT prolongation, E-C uncoupling).
What is different about our Technology Platform?
Our translational discovery platform has the potential to collapse the discovery process, saving years of time, millions of dollars and will enable next generation precision medicine.