This panel will begin with a summary by J.F. Hipp of the process of drug discovery and development, including the critical need for biomarkers in early stage drug development. Next, two presentations will focus on rare genetic disorders in autism. J. Serrats will show how recent genetic findings provide a platform for neurobiologically-driven drug discovery and O. Khwaja will summarize recent advances in the use of genomic medicine in these rare disorders. Finally, G.J. Pandina will discuss the critical need for robust outcome measures in ASD in order to show efficacy of interventions, as well as a study of one such measure. These four talks will provide an inclusive picture of early through late stage drug development, including biomarkers and outcomes measures, and summarize how to leverage molecular genetic discoveries in autism for the development of novel therapeutics.