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Zoom link => https://us02web.zoom.us/j/86114044204
Motor neuron disease: a neurodegenerative disorder poised for successful therapy development
DBE MBBS MD FRCP FMedSci FAAN FANA FAAAS
Professor of Neurology,
Honorary Consultant Neurologist, Sheffield Teaching Hospitals NHS Foundation Trust,
Director of the Sheffield Institute for Translational Neuroscience (SITraN),
Director NIHR Sheffield Biomedical Research Centre,
Director of the cross-faculty Neuroscience Research Institute University of Sheffield
Zoom link => https://us02web.zoom.us/j/85270412441
Repeat Expansions Instigate Supply Chain Issues in Nucleus and Cytoplasm
More information on Eric Wang laboratory
Eric T. Wang – Principal Investigator
Eric received his BA in Biochemistry at Harvard College and PhD from the Harvard-MIT Division of Health Sciences & Technology. Following receipt of an NIH Early Independence Award, he started his lab as a Koch Institute Fellow, and subsequently moved to the University of Florida as an Assistant Professor. His interests lie at the intersection of genomics and cell biology, with a focus on better understanding disease pathogenesis underlying repeat expansion diseases. A family connection led him to focus on myotonic dystrophy, a multi-systemic disease affecting muscle, heart, and brain. He has worked with the Myotonic Dystrophy Foundation, the Muscular Dystrophy Association, and Promise to Kate to raise awareness and funding for research. He co-founded Strength, Science and Stories of Inspiration to support the research of young investigators. He also interacts extensively with drug companies to develop meaningful treatment options for people living with severe neurological diseases.
Zoom link => https://us02web.zoom.us/j/86864982792
Zoom link => https://us02web.zoom.us/j/85792428574
Intermediate filaments: from mechanisms in giant axonal neuropathy to therapeutic perspectives for IF-pathies
Pascale Bomont, PhD
ERC group leader, INSERM Research Director
NeuroMyoGene Institute, Lyon, France
Studying a rare neurodegenerative disease called giant axonal neuropathy (GAN), Pr Bomont will present the discovery of the gigaxonin-E3 ligase as a pivotal regulator of Intermediate Filament homeostasis, autophagy activation and motor neuron specification. Using the complementarity of various biological systems in human, mouse and zebrafish, the laboratory investigates the roles of these key cellular pathways in neurodegeneration, and is currently developing therapeutic approaches for GAN, that can be beneficial to other IF-pathies.
Pascale Bomont is a Research director at Inserm, University of Lyon1
She received her PhD in Human Genetics in 2002 (IGBMC, Strasbourg, France). Performing linkage and bioinformatics analysis on the Human Genome, she identified the genetic locus and mutated genes for several neuropathies. Focusing on Gigaxonin, a novel E3-ubiquitin ligase adaptor she found mutated in Giant Axonal Neuropathy (GAN), she conducted a postdoctoral training in Cell Biology (LICR, San Diego, USA) to investigate the broad cytoskeleton (Intermediate Filament) alteration in the pathology. Moving back to France, she was recruited at INSERM in 2007 (as assistant Professor), and was awarded by the ATIP-Avenir prize in 2011 to run a multidisciplinary research program on GAN, at Montpellier University (Associate Professor in 2012). She developed tools and biological systems in patients, mouse and zebrafish to generate a differential diagnosis test between GAN and CMTs, and to unravel the key roles of the Gigaxonin-E3 ligase in controlling cytoskeleton architecture, autophagy machinery and neuromuscular integrity.
She is now full Professor at University Lyon1, and runs an ERC-program at the NeuroMyogene institute on neurofilament biology.
Zoom link => https://us02web.zoom.us/j/87417667754
Generation of novel therapeutics for neuromuscular diseases through collaboration with disease and target biology academic experts and argenx antibody expertise
Carol Walton and René Bigirimana
Carol Walton, Director of Business Development at argenx and René Bigirimana, Scenior scientist at argenx
Argenx strives to combine its technical antibody expertise with external expertise on biological targets through collaborations. Within our Immunology Innovation Program (IIP), we aim to generate novel therapeutic approaches for high unmet need peripheral neuromuscular indication by collaborating with external scientific and academic experts to gain novel insights in disease and target biology. argenx will provide an Introduction to its Immunology Innovation Program for collaboration with some of our illustrative collaboration on C2 antibody ARGX 117 for MMN and Musk agonist antibody ARGX-119.
Zoom link => https://us02web.zoom.us/j/81380105352
Directed Evolution of Novel Adeno-Associated Viruses for Clinical Gene Therapy
Hubbard Howe Jr. Distinguished Professor
Chemical and Biomolecular Engineering, Bioengineering, Molecular and Cell Biology, and the Helen Wills Neuroscience Institute
University of California at Berkeley
David Schaffer is the Hubbard Howe Professor of Chemical and Biomolecular Engineering, Bioengineering, and Molecular and Cell Biology at the University of California, Berkeley, where he also serves as the Director of the California Institute for Quantitative Biosciences (QB3-Berkeley). He received a B.S. from Stanford University in 1993 and a Ph.D. from MIT in 1998, both in chemical engineering. He then conducted a postdoctoral fellowship at the Salk Institute for Biological Studies before joining Berkeley in 1999. There, he applies engineering principles to optimize gene and stem cell therapies, work that includes developing the concept of applying directed evolution to engineer targeted and efficient viral gene therapy vectors as well as new technologies to investigate and control stem cell fate decisions. He has published >230 papers, has advised >90 graduate students and postdoctoral fellows, is an inventor on >50 patents, and developed technologies that are being used in 9 human clinical trials. In addition, he has co-founded six companies, including a clinical stage, public company (4D Molecular Therapeutics) and a company acquired by Pfizer. Finally, has received recognitions including the National Academy of Inventors, Andreas Acrivos Professional Progress Award, the American Institute of Chemical Engineers Pharmaceutical and Bioengineering Award, the American Chemical Society Marvin Johnson Award, and the Biomedical Engineering Society Rita Shaffer Young Investigator Award.