Molecules to Medicine Episode 2: Efficacy and Target Engagement

Understanding the pathological mechanisms of disease and how a potential therapy impacts these processes increases the likelihood of its progression through drug development pipelines. In this episode, Dr. Natasa Giallourou, uses a study in autism spectrum disorder to demonstrate how metabolomics was integral to identifying a disease biomarker which led to the development of a candidate treatment. Metabolomics was further used to provide evidence of target engagement and efficacy.