Technology Overview
The iPierian approach begins by rigorously selecting a patient population to represent a disease, including patient-specific information such as age of onset, severity/progression, and family/sibling history. Skin biopsy-derived fibroblasts from these patients are reprogrammed into induced pluripotent stem (iPS) cells and then differentiated into functional disease-relevant cells – such as motor neurons, cortical neurons, or glia. After an iPS cell line has been differentiated into the cell types of interest, a disease phenotype is investigated, revealing a disease-relevant difference between the patient-derived cells and those from healthy controls. From this disease phenotype, cell-based assays are then developed for high-throughput screening, enabling the discovery of novel targets and disease-modifying therapeutics. This iPierian advantage puts the patients at the forefront of the drug discovery process, producing drug candidates that likely have a higher probability of success in the clinic.
