Part 1: AAV-mediated Gene Therapy for Developmental and Epileptic Encephalopathies

Introduction to AAV-mediated Gene Therapy presented by Dr. Michael W. Lawlor of the Medical College of Wisconsin. This recording is part one of a three-part educational webinar series intended for healthcare providers. This educational series is provided by the Dravet Syndrome Foundation in collaboration with Encoded Therapeutics.

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Several years ago, gene therapy still seemed a distant possibility for Dravet syndrome and other developmental and epileptic encephalopathies (DEEs). Over time, advances to our basic understanding of genetics and technology have allowed researchers and clinicians to develop new approaches to gene-based interventions. Now there are several of these approaches in various stages of preclinical research and clinical development for the potential treatment of Dravet syndrome.

This three-part educational webinar series provides background information on one such approach, AAV-mediated gene therapy. Throughout this series, Dr. Michael Lawlor and Dr. Sarah Weckhuysen take us through the basic concepts of gene therapy and AAV-mediated gene therapy (webinar 1), considerations for moving AAV-mediated gene therapy from preclinical research into clinical development programs (webinar 2), and the potential application of gene therapy to address the underlying genetic cause the of DEEs (webinar 3).

Presenter Biographies:

Michael W. Lawlor MD, PhD
Michael W. Lawlor, M.D., Ph.D., is a Professor of Pathology, Biomedical Engineering, Physiology, Cell Biology, Neurobiology, and Anatomy and the Associate Director of the Neuroscience Research Center at the Medical College of Wisconsin. He is a Board-Certified Anatomic Pathologist and Neuropathologist, and his research interests include pediatric muscle disease and gene therapy. Dr. Lawlor is an Editorial Board member of Muscle and Nerve and Journal of Neuropathology and Experimental Neurology. He is currently serving as an Independent Scientific Advisory Board member of Taysha Gene Therapies and a Scientific Advisory Board member for Solid Biosciences in support of their gene therapy programs. Dr. Lawlor earned his medical degree and doctorate from Loyola University School of Medicine and his residency, fellowship, and postdoctoral training was completed at Massachusetts General Hospital and Boston Children’s Hospital in association with Harvard Medical School.

Sarah Weckhuysen MD, PhD
Sarah Weckhuysen MD, PhD is an Assistant Professor at the University of Antwerp in Belgium with interest in both clinical epilepsy and genetic research. As a neurologist she worked in several tertiary epilepsy centers, including the Epilepsy Center Kempenhaeghe in the Netherlands, the Hôpital Pitié Salpêtrière in Paris, France, and currently the University Hospital of Antwerp in Belgium. She obtained a PhD on the topic of genetics of epileptic encephalopathies under the mentorship of Prof. P. De Jonghe, who discovered de novo SCN1A mutations as the genetic cause of Dravet syndrome back in 2001. Since 2018 Sarah is heading the epilepsy research team of the Applied & Translational Neurogenomics group at the VIB-Center for Molecular Neurology in Antwerp. The primary focus of her research group is the identification of novel genes and genetic mechanisms implicated in epilepsy, using state-of-the-art multi-omics approaches. The developmental and epileptic encephalopathies are of specific interest to her group, as she strongly believes that the study of the underlying genetic mechanisms can be the entry point for the development of more potent, targeted therapies. Her lab therefore uses patient derived induced pluripotent stem cells as a tool to translate the genetic work of her group into biological understanding and better treatments for people with epilepsy. Over the years, her work contributed to many novel epilepsy gene discoveries and disease characterizations. Dr. Weckhuysen serves as a member of the Dravet Syndrome Foundation Scientific Advisory Board.