Autism Assessment & Treatment | Icahn School of Medicine

SEAVER AUTISM CENTER FOR RESEARCH AND TREATMENT

Drug Discovery and Development

With our genetics-first approach, researchers at the Seaver Autism Center for Research and Treatment have identified numerous genes associated with autism spectrum disorder, providing unprecedented opportunities to develop targeted therapies. Our multidisciplinary team of experts employs a comprehensive, drug discovery pipeline that spans from initial screening in cellular models to rigorous testing in animal models, culminating in carefully designed clinical trials. This seamless integration of basic science and clinical research allows us to rapidly move promising compounds from the laboratory to potential patient therapies.

Our drug discovery efforts are particularly focused on addressing the needs of individuals with profound autism, a group that has historically been underserved in both research and treatment development. By leveraging our extensive genetic discoveries and our array of preclinical models, including patient-derived stem cells and genetically modified rodent models, we are able to identify and validate novel therapeutic targets with unprecedented precision. Our specialists in drug discovery and development have crafted comprehensive strategies for developing these novel therapeutics, with a particular emphasis on gene therapy approaches for genetic mutations associated with autism, as well as small-molecule screens in human organoids. As we look to the future, we are confident that our rigorous, collaborative approach will yield biologically based treatments for profound autism within the next decade, revolutionizing care for individuals and families affected by these conditions.