Given the prevalence and societal impact of autism spectrum disorder (ASD), there is an urgent need to develop innovative treatments that will improve core social deficits, for which there is currently no reliable pharmacological treatment, prevention or cure. Development of novel biological interventions will depend upon the successful translation of basic neuroscience research into safe and effective medicines. This article outlines steps to bring neuroscience research from ‘the bench’ to treatment at ‘bedside’, from phenotyping the disorder to animal models to patient treatment. Although these steps appear simplistic, this is a daunting challenge because of the inherent complexity of the human brain, our lack of understanding of disease neurobiology underlying ASD, and the incredible heterogeneity of the disorder. For ASD, perhaps more than any other neurological or psychiatric disorder, progress will depend on integrative multidisciplinary approaches between basic scientists from varying neuroscience disciplines and clinicians to make ‘bench to bedside’ treatment a reality.