Scientific Advisors

Patrick Aebischer, M.D.

President of Ecole polytechnique federale de Lausanne (EPFL)

Patrick Aebischer is a leading academic researcher and industry entrepreneur with extensive experience in the field of cell and gene transfer, particularly for the treatment of neurodegenerative diseases. Dr. Aebischer currently serves as president of the Ecole polytechnique fédérale de Lausanne (EPFL), a leading university that combines teaching, research and technology transfer, along with an extensive network of universities, and industry and political leaders, to advance technological innovation in physical science and engineering. Dr. Aebischer was an associate professor of Medical Sciences at Brown University in Providence, RI, and was elected chairman of the section of artificial organs, biomaterials and cellular technology of the Division of Biology and Medicine of Brown University before becoming professor and medical director of the Surgical Research Division and Gene Therapy Center at the Centre Hospitalier Universitaire Vaudois in Lausanne, Switzerland. Dr. Aebischer is the recipient of numerous honors including the Robert Bing Prize of the Swiss Academy of Medical Sciences and the Pfizer Foundation Prize for Clinical Neurosciences. He is a fellow of the American Institute for Medical and Biological Engineering and the Swiss Academy of Medicine Sciences, a founder of CytoTherapeutics, Inc. (1989), Modex Therapeutiques, Inc. (1996) and Amazentis S.A. (2007) and a board member of Nestlé Inc., Lonza Inc. and the World Economic Forum, as well as chairman of the Novartis Venture Fund.

Dr. Aebischer holds an M.D. and trained as a neuroscientist at the University of Geneva, Switzerland.

Mavis Agbandje-McKenna, Ph.D.

Professor, University of Florida; Director, Center for Structural Biology, University of Florida College of Medicine

Mavis Agbandje-McKenna is a leading structural biologist and pioneer in the biophysical characterization of adeno-associated viruses (AAVs). She is a professor at the University of Florida and director of the Center for Structural Biology in the College of Medicine. Dr. Agbandje-McKenna’s laboratory uses structure-based approaches (X-ray crystallography and cryo-electron microscopy), combined with biochemistry, biophysics, molecular biology and in vivo animal studies, for studies aimed at improving the efficacy of AAV vectors.

Dr. Agbandje-McKenna holds a Ph.D. in biophysics from the University of London.

Robert Brown, D.Phil, M.D.

Professor and Chair of Neurology, University of Massachusetts Medical Center and Medical School; Director, Day Neuromuscular Research Laboratory at the University of Massachusetts Medical School

Robert Brown is an internationally recognized researcher and physician, renowned for his groundbreaking basic and clinical research on the inherited and genetic basis of neurodegenerative and neuromuscular diseases, in particular ALS. In 1993, Dr. Brown led a team of researchers that discovered the first gene linked to the inherited form of ALS, called SOD1. Dr. Brown is currently professor and chair of neurology at the University of Massachusetts Medical Center and Medical School. Dr. Brown is also director of the Day Neuromuscular Research Laboratory, which he founded in 1984 to investigate neuromuscular diseases, including ALS.

Dr. Brown holds an M.D. from Harvard Medical School and a D.Phil from Oxford University. He trained in neurology at the Massachusetts General Hospital.

Massimo Pandolfo, M.D.

Professor of Neurology and Director of the Laboratory of Experimental Neurology, Université Libre de Bruxelles (ULB); Chief of Neurology, ULD-Erasme University Hospital, Brussels

Massimo Pandolfo is a leader in neurogenetics and translational neuroscience. He is Professor of Neurology and director of the Laboratory of Experimental Neurology at the Université Libre de Bruxelles (ULB) in Brussels, Belgium. He is also chief of neurology at the ULB-Erasme University Hospital in Brussels. He is a fellow of the American Academy of Neurology (AAN), member of the AAN Science Committee, chair of the AAN Translational Neuroscience and International Subcommittees, and member of the European Academy of Neurology Scientific Panels on neurogenetics and on translational neurology. In 1996, Dr. Pandolfo led the team that identified the Friedreich’s ataxia gene, and has since been at the forefront of basic, translational and clinical research on Friedreich’s ataxia. In addition to his seminal work on Friedreich’s ataxia, Dr. Pandolfo has contributed to the study of other inherited ataxias, of the genetics and pharmacogenetics of epilepsy, of several rare inherited neurological diseases (brain degeneration with iron accumulation, paroxysmal non-kinesogenic dyskinesia, aceruloplasminemia), and of the genetic predisposition to stroke. Dr. Pandolfo’s current research focuses on dissecting the pathogenesis of inherited ataxias and epilepsies and on the development of small molecule and gene therapy-based approaches for the treatment of Friedreich’s ataxia and other neurodegenerative diseases.

Dr. Pandolfo obtained his M.D. from the University of Milan, Italy, where he also completed his residency in neurology.