Joe Dougherty, PhD grew up in Saint Louis, Missouri. He majored in Psychology with a minor in Chemistry at Truman State University in Kirksville, Missouri before joining the Geschwind lab at UCLA for his PhD studies in Neuroscience. The Geschwind lab was a key leader in applying high-throughput approaches for measuring the expression of all the genes to questions of brain development and function.  There, Joe studied the role of specific genes and gene expression in the development of the cell types of the mammalian brain, focusing on how neural stem cells become neurons.  He later trained as a postdoctoral researcher with Nat Heintz at Rockefeller University where he learned mouse genetics and transgenesis and applied new tools such as the TRAP technology to understand how gene expression differs across cell types the brain, especially those thought to be important in psychiatric diseases. ​ Following his postdoctoral work, Dr. Dougherty took a position of Assistant Professor at Washington University in St. Louis where, in 2010, he started his own lab jointly affiliated with the Departments of Genetics and Psychiatry.  There, he built a creative and successful team focused on understanding how human genetic risk factors for conditions like autism and IDD alter the development and function of the brain, using mice as a tool (>96% of human genes have a clear parallel in mouse, often with a shared function in brain development).  As many genes that cause disease in humans are regulators of gene expression, over the last 15 years, he and his lab have focused on a dual mission of understanding 1) how regulation and dysregulation of gene expression leads to changes in brain, and 2) training the next generation of neuroscientists.  To date, nearly 100 students have trained in his lab as undergraduates, graduate students, and postdocs, collectively contributing to over 100 publications describing new discoveries in brain and behavior. Now a full Professor and Co-Director of the Washington University Intellectual and Developmental Disability Research Center, Dr. Dougherty has pivoted his lab away from pure basic science discoveries toward areas that can provide insight into the conditions of patients. Notably, around 2020 MYT1L had recently been discovered as a key regulator of gene expression that, when altered, could cause challenges such as autism and developmental delay.  A family followed by a local clinician had recently discovered that their child had MYT1L mutation, and Dr. Dougherty (along with the Maloney and Kroll labs) embarked on a partnership with this family and clinician to develop new basic science tools to understand the impact of MYT1L mutation on brain development.  He developed a mouse model of this patient’s mutation, enabling the team to define what genes are regulated by MYT1L, how the mutation changes their expression, and how this impacts brain development and behavior.  This built the foundation for a research program spanning from basic science of MYT1L gene regulation to the testing of pharmacological and genetic interventions for restoring MYT1L function. Dr. Dougherty’s team is excited to continue these investigations to drive a deeper understanding of this critical gene, with the ultimate goal of new treatment options for patients and their families.

Joe Katakowski, PhD is responsible for leading the development strategy and internal R&D efforts for projects within the RTW Foundation portfolio. Prior to joining RTW Foundation, Joe was a staff scientist at Regeneron where he led a team focused on preclinical development of gene therapies and AAV vector engineering. Before Regeneron, Joe was a principal scientist at Pfizer where he developed and led multiple immuno-oncology programs from early-stage discovery up to IND application, working with a diverse array of modalities including lipid/polymeric nanoparticles, PROTACs, ADCs, antibodies, and small molecules. Prior to Pfizer, Joe was a senior scientist at Innovimmune, a biotech startup. During his PhD, Joe developed unique delivery technologies for nucleic acid-based drugs, seeking to modulate immune responses for autoimmune and oncology indications. His PhD work led to two separate first author publications in Molecular Therapy, as well as additional publications throughout his research career. ​ Joe has a BS in human biology from Michigan State University, an MS in cellular & molecular biology from Eastern Michigan University and a PhD in immunology & biomedical sciences from the Albert Einstein College of Medicine.

Dr. Isom is the Maurice H. Seevers Professor and Chair of the Department of Pharmacology, Professor of Molecular and Integrative Physiology, and Professor of Neurology at the University of Michigan Medical School. She has served as Director of the Program in Biomedical Sciences and Assistant Dean for Graduate Education in the University of Michigan Medical School. She received her PhD in Pharmacology at Vanderbilt University School of Medicine and then trained as a postdoctoral fellow in the laboratory of Dr. William A. Catterall at the University of Washington. Dr. Isom’s research program at the University of Michigan focuses on voltage-gated sodium channel function and the roles of sodium channel gene variants in developmental and epileptic encephalopathy (DEE), including Dravet syndrome. Her lab investigates SCN1A, SCN1B, and SCN8A DEE variants in mouse models and in human induced pluripotent stem cell (iPSC) neurons and cardiac myocytes. They developed the first large transgenic animal model of Dravet syndrome, a Scn1a haploinsufficient rabbit. Their body of work has provided preclinical evidence for neuro-cardiac mechanisms of Sudden Unexpected Death in Epilepsy. Dr. Isom collaborated with Stoke Therapeutics to develop the first antisense oligonucleotide precision therapeutic agent for Dravet syndrome, which is now in clinical trials. Dr. Isom is Co-PI of the NINDS-funded EpiMVP Center Without Walls. She serves as PI of the NIH funded, Pharmacological Sciences Training Program T32 grant, co-chairs the Dravet Syndrome Foundation Scientific Advisory Board, served on the Board of the American Epilepsy Society, co-chairs the American Epilepsy Society-NINDS Benchmarks committee, chaired the NIH ESTA study section, as well as served on editorial boards of scientific journals. She has received awards for research and mentoring, including a NINDS Javits R37 MERIT award and the University of Michigan Rackham Distinguished Graduate Mentoring Award. She is a Fellow of the American Association for the Advancement of Science, a Fellow of the American Society for Pharmacology and Experimental Therapeutics, and a Fellow of the American Epilepsy Society. Dr. Isom was elected to the National Academy of Medicine in 2021 and received the American Epilepsy Society Basic Science Research Award in 2022.

Moritz Mall, PhD grew up in Munich, Germany, and studied Biochemistry and Molecular Biology at LMU Munich and ETH Zurich. He completed his PhD at the European Molecular Biology Laboratory (EMBL) in Heidelberg, Germany, where he uncovered fundamental mechanisms of cell division. For his postdoctoral research, he was awarded a fellowship from the German Research Foundation (DFG) and joined the laboratory of Marius Wernig at Stanford University. There, he shifted his focus to studying cell fate determination, neuronal development, and disease through pioneering work in cellular reprogramming. Since 2012, Dr. Mall has been deeply engaged in uncovering the basic physiological role of the autism spectrum disorder (ASD)-associated transcription factor MYT1L. Over the past decade, his work has evolved from fundamental studies of MYT1L’s role in maintaining neuronal identity to generating the first human and mouse models of MYT1L deficiency. Through these efforts, his lab has revealed how MYT1L mutations contribute to neurodevelopmental delays, behavioral abnormalities, and altered gene regulation, offering critical insights into potential therapeutic strategies. His research has been recognized with major awards, including a European Research Council (ERC) Grant, and published in leading scientific journals. Returning to Germany in 2018, Dr. Mall established his independent research group at the German Cancer Research Center (DKFZ) in Heidelberg, becoming one of the founding group leaders of the Hector Institute for Translational Brain Research (HITBR). His team combines animal models, stem cells, and organoid technologies to explore cellular plasticity in development and disease, with a focus on cancer and MYT1L-associated neurodevelopmental disorders. A key aim of their research is to build precision medicine models for individual patients by using patient-derived stem cells to better understand disease mechanisms and to explore potential pharmacological treatment strategies. In addition to his scientific contributions, Dr. Mall has worked closely with families affected by MYT1L mutations. In 2024, together with Prof. Maja Hempel and their teams, he organized the first German-speaking MYT1L Family Day at Heidelberg University Hospital, bringing together more than 60 participants. Dr. Mall maintains active communication with numerous families worldwide, united by the shared mission of making a difference for patients and families affected by MYT1L. Dr. Mall and his collaborators envision building a blueprint for how neurodevelopmental disorders can be better understood — and ultimately treated — by bridging cutting-edge basic research with community-driven initiatives. His current work continues to explore both the fundamental and translational aspects of MYT1L function, with the goal of turning scientific discoveries into tangible hope for patients and families around the world.