I am inspired by the many ways in which scientific discovery can positively transform our lives. My own curiosity has drawn me to the interface of the genetics, stem cell biology and neuroscience fields, ultimately leading to a career in academic science, biotechnology entrepreneurship and the pharmaceutical industry. As a Professor at Harvard University and a Principal Investigator at the Harvard Stem Cell Institute, my research group helped establish that pluripotent stem cells are a valuable source of many differentiated cell types, which can in turn be used to study how human genetic variation shapes cellular phenotypes contributing to disease. Because most cell-types from the human brain cannot be viably retrieved, even at autopsy, we naturally focused our early efforts on studying how genetic factors predispose us to nervous system disorders. Our studies of ALS have been particularly fruitful and demonstrated that alterations in RNA metabolism found in stem cell-derived motor neurons are predictive of those unfolding in patients. These fundamental findings served as the bases for further academic and entrepreneurial collaborations that have generated several therapeutic candidates for ALS, two of which are in active clinical studies. To further my goals of growing the scientific careers of colleagues and using new technologies to bring therapeutic solutions to unmet medical needs of patients with rare genetic disease, I joined BioMarin to lead Research and Early Development and now serve as Chief Scientific Officier