iPierian’s approach to developing novel therapeutics is enabled by the company’s proprietary capabilities in induced pluripotent stem cell (iPSC) technology. Skin biopsy-derived fibroblasts are collected from patients with neurodegenerative diseases, reprogrammed into iPSCs, and then differentiated into functional disease-relevant cells – such as cortical neurons, motor neurons, microglia, and astrocytes. After an iPSC 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 developed to enable the discovery and validation of novel targets and molecules.
Leveraging the company’s iPSC capabilities can provide insight into the earliest drivers of diseases such as Alzheimer’s, in contrast to conventional autopsy samples which typically only allow for study of end-stage pathophysiology. 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.