With advances in reprogramming and differentiation technologies, as well as with the recent availability of gene editing approaches, we are finally able to create more complex and phenotypically accurate cellular models based on pluripotent cell technology. This opens new and exciting opportunities for pluripotent stem cell utilization in early discovery, preclinical and translational research. CNS diseases and disorders are currently the main therapeutic area of application with some impressive success stories resulted in clinical trials. Cambridge Healthtech Institute’s Second Annual iPS Cells for Disease Modeling and Drug Discovery symposium is designed to bring together experts and bench scientists working with pluripotent cells and end users of their services, as well as researchers working on finding cures for specific diseases and disorders.