Scientific Advisory Board

Kevin Campbell, PhD

Kevin P. Campbell, PhD, is an investigator with the Howard Hughes Medical Institute, the Roy J. Carver Biomedical Research Chair in Molecular Physiology and Biophysics, Executive Officer of the Department of Molecular Physiology and Biophysics, and Director of the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center at the Carver College of Medicine, University of Iowa. Dr. Campbell is internationally recognized for his discovery of dystroglycan, and for elucidation of its function as an extracellular matrix receptor. His work established that dystroglycan is involved in a variety of physiological and developmental processes, including maintenance of skeletal muscle function as well as formation and function of the central nervous system. He showed that complex post-translational processing of dystroglycan, including extensive glycosylation, is required for its ability to function as an extracellular matrix receptor and that abnormal glycosylation results in a variety of congenital and limb-girdle muscular dystrophies with or without associated defects in brain development and function.

Dr. Campbell is an elected member of the Institute of Medicine, the National Academy of Sciences, and the American Academy of Arts and Sciences. In 2009, Dr. Campbell received the March of Dimes Prize in Developmental Biology for his pioneering research on cell mechanisms involved in muscular dystrophy. Dr. Campbell will receive the Society for Glycobiology President’s Innovator Award in the fall of 2017.

Jesper Gromada, PhD Chief Scientific Officer and Co-Chair, SAB

Jesper Gromada joined Exonics Therapeutics as Chief Scientific Officer in 2018. He brings to the company more than 20 years of experience working in various roles at Novo Nordisk, Eli Lilly, Novartis and most recently at Regeneron Pharmaceuticals, where he served as Vice President. A major focus of Jesper’s work has been the development of therapeutics for patients with metabolic and skeletal muscle diseases. He has published more than 140 peer-reviewed manuscripts, including in high impact journals such as the New England Journal of Medicine, Cell, Science and Cell Metabolism, and has given numerous invited lectures nationally and internationally. Jesper holds a Ph.D. from University of Copenhagen, Denmark.

 

 

Beth McNally, MD, PhD

Elizabeth M. McNally, MD, PhD, is the Ward Professor of Genetic Medicine at Northwestern University’s Feinberg School of Medicine where she directs the Center for Genetic Medicine. As a cardiologist and geneticist, Dr. McNally’s work spans preclinical models to patients and families with genetic risks for inherited cardiovascular disorders, including those with neuromuscular disease. She serves on advisory boards for the Muscular Dystrophy Association and Parent Project Muscular Dystrophy. A previous American Heart Association Established Investigator award winner, Dr. McNally is a member of the Association of American Physicians and past president of the American Society for Clinical Investigation.

Jerry Mendell, MD

Jerry R. Mendell, MD, is the Curran-Peters Chair of Pediatric Research at Nationwide Children’s Hospital (formerly Ohio State University and Children’s Hospital) in Columbus, Ohio. Dr. Mendell holds joint appointments in Pediatrics, Neurology, Pathology, and Physiology and Cell Biology at The Ohio State University. An expert in Duchenne, his work at the Mendell lab focuses on correcting the gene defects in the muscular dystrophies, resulting in seminal studies including myoblast transfer in Duchenne, the first Duchenne gene therapy clinical trial, and the first gene therapy trial showing efficacy in muscular dystrophy. Dr. Mendell has published more than 340 articles with a focus on neuromuscular disease, and authored books on both muscle and nerve disease. He received his Medical Doctorate from the University of Texas Southwestern Medical School and completed his neurology residency at Columbia University’s New York Neurological Institute and a post-doctoral fellowship in the Medical Neurology Branch of the National Institutes of Health.

Jeffery Molkentin, PhD

Jeffery D. Molkentin, PhD. Is a Professor in the Department of pediatrics, University of Cincinnati and Cincinnati Children’s Hospital. He received his B.S. from Marquette University in Milwaukee Wisconsin (USA) in 1989. He then received his PhD from the Medical College of Wisconsin in 1994, after which he performed postdoctoral training with Dr. Eric Olson in Texas (USA) from 1994-1997, followed by his first faculty appointment in 1997 at the Cincinnati Children’s Hospital Medical Center of the University of Cincinnati (USA). Dr. Molkentin has published over 390 original articles during this time, he has a Scopus H-index of 103. Dr. Molkentin was a Pew Scholar early in his career and is now a full investigator of the Howard Hughes Medical Institute in the USA since 2008. He has also won a number of awards such as the Louis N and Arnold M Katz award to young investigators and more recently the Basic Research Prize, both of the American Heart Association. Dr. Molkentin also won the Lucian Award from McGill University. He has also served on NIH study section and was an organizer of various national and international scientific meetings. Finally, Dr Molkentin has placed approximately 25 of his past trainees into academics as laboratory principle investigators. Dr. Molkentin’s research program continues to focus on the identification of candidate genes and signaling pathways involved in cardiac hypertrophy, contractility, cell death, heart failure, fibrosis, regeneration and muscular dystrophy, as well as mitochondria-dependent necrosis.

Eric Olson, PhD Founder and Co-Chair, SAB

Eric Olson, PhD, is Professor and Chair of the Department of Molecular Biology at the University of Texas Southwestern Medical Center in Dallas, where he also holds the Robert A. Welch Distinguished Chair in Science, the Annie and Willie Nelson Professorship in Stem Cell Research, and the Pogue Distinguished Chair in Research on Cardiac Birth Defects. Dr. Olson has dedicated his career to deciphering the mechanisms that control development and disease of the heart, cardiovascular system and skeletal muscle tissue. Dr. Olson has co-founded multiple biotechnology companies to translate basic discoveries into new therapeutics for muscle disease, and is a member of the National Academy of Sciences, the National Academy of Medicine and the American Academy of Arts and Sciences.

Volker Straub, MD

Volker Straub, MD, is the Harold Macmillan Professor of Medicine and Professor for Neuromuscular Genetics at the Newcastle University Institute of Genetic Medicine’s Newcastle Muscle Center, and director of Newcastle University’s John Walton Muscular Dystrophy Research Centre, in Newcastle upon Tyne, United Kingdom. One of Dr. Straub’s interests is translational research. He was a founder of the European Union-funded network of excellence, TREAT-NMD (www.treat-nmd.eu), the now global TREAT-NMD Alliance, which aims to accelerate the development and delivery of treatments for patients with neuromuscular diseases. Dr. Straub has a long-standing interest in the pathogenesis of genetic muscle diseases, with research using zebrafish and mouse models, applying muscle imaging, next generation sequencing and other –omics technologies. Dr. Straub was trained as a pediatric neurologist at the University of Düsseldorf and the University of Essen in Germany. In 2003, Dr. Straub joined Newcastle University in the United Kingdom, where he also serves as deputy director of the Institute of Genetic Medicine.

Luk Vandenberghe, PhD

Luk H. Vandenberghe, PhD, is an Associate Professor at Harvard Medical School and Associate Member of the Broad Institute of Harvard and MIT in Boston, MA, USA. He directs the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear Infirmary in Boston, USA, a part of the Ocular Genomics Institute, a bench to bedside research program to study, diagnose, and develop treatments for diseases of the eye.

He received a degree in cellular and genetic engineering from the University of Leuven, Belgium. His previous work led to the discovery of novel AAV serotypes such as AAV9, novel insights into AAV structure-function, and vector immunobiology. His laboratory aims to gain a deeper understanding of mechanisms of gene transfer, to develop technologies to overcome hurdles to gene therapy clinical applications, and to translate specific gene therapy programs in vision, hearing, and other fields. Recent studies leverage structural and evolutionary information on AAV as a starting point for the rational design of synthetic viral vector systems, a first generation of which is referred to as AncAAVs which are now progressing to the clinic for a number of indications. Dr. Vandenberghe previously co-founded GenSight Biologics and Akouos. He also is a founder, board member, and advisor to Odylia Therapeutics, a non-profit catalyzing translation for gene therapies within the challenging field of ultra-rare disorders. Dr. Vandenberghe has over 50 peer reviewed publication and more than a dozen licensed patents, mostly related to gene therapy methods, technologies, and applications.