Bringing Meaningful Therapies From One To

A deep understanding of human genetics is core to our approach. Defining a high impact drug target for a common disease with a heterogenous population has a very low probability of success and has long been a major challenge for drug discovery. By starting with a focus on rare diseases – where we’re looking at a defined population and know that one gene is leading to one disease – we aim to enhance our understanding of both the target and disease. Through this approach, we gain invaluable insights that can de-risk and guide therapeutic expansion to more prevalent diseases.