Scientific Advisory Board
Renée JG Arnold, PharmD, RPh
President & CEO, Arnold Consultancy & Technology LLC
Renée JG Arnold completed her undergraduate training at the University of Maryland and received her Doctor of Pharmacy degree from the University of Southern California in Los Angeles. She also completed a one-year post-doctoral residency at University Hospital in San Diego/University of California at San Francisco School of Pharmacy. Dr. Arnold was most recently Principal, IMS Health; President and Co-Founder of Pharmacon International, Inc. Center for Health Outcomes Excellence.
Dr. Arnold is currently President & CEO, Arnold Consultancy & Technology LLC, headquartered in New York City, and Practice Lead, HEOR, Quorum Consulting, Inc., based in San Francisco, CA, where she oversees outcomes research and develops affiliated software for pharmaceutical and federal government programs. Her special interest in evidence-based health derives from her research that deals with use of technology to collect and/or model real-world data for use in rational decision-making by healthcare practitioners and policy makers. Dr. Arnold’s academic titles include Adjunct Associate Professor, Master of Public Health program, Department of Preventive Medicine at the Mount Sinai School of Medicine, where she has developed and teaches the pharmacoeconomics coursework. She is also Full Adjunct Professor at Long Island University College of Pharmacy and Health Sciences. Dr. Arnold is a founding member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and is an author/co-author of numerous articles and book chapters in the areas of pharmacology, pharmacoeconomics and cost containment strategies.
Dr. Robert Burgess, Ph.D
Faculty of The Jackson Laboratory in Bar Harbor, Maine
Dr. Robert Burgess has a long-standing interest in basic Neurobiology research. Dr. Burgess received his B.S. in Biochemistry and Physiology from Michigan State University in 1990, and his Ph.D. in Neuroscience from Stanford University in 1996. Following postdoctoral training at Washington University, St. Louis, Dr. Burgess joined the faculty of The Jackson Laboratory in Bar Harbor, Maine in 2001. His research program uses genetic approaches in mice to examine neurodevelopmental and neuromuscular diseases, including mouse models of Charcot-Marie-Tooth disease and other inherited peripheral neuropathies. Areas of study include the use of mouse models to understand pathophysiology, developing and improving animal models for preclinical research, and exploring therapeutic strategies using mouse models of human disease. The Burgess lab is funded by the National Institutes of Health and has also received support from numerous disease foundations. Dr. Burgess has published over 50 peer-reviewed publications, including papers in high impact journals such as Nature and Neuron.
Sean Ekins, Ph.D
CSO, Hereditary Neuropathy Foundation
Sean is CSO at the Hereditary Neuropathy Foundation. He divides his time between clients at Collaborations In Chemistry which currently includes: Collaborative Drug Discovery, Rutgers University and other biotech and consumer product companies. In addition he is CSO and President at Phoenix Nest focused on rare disease drug discovery. He graduated from the University of Aberdeen; receiving his M.Sc., Ph.D. and D.Sc. in Clinical Pharmacology. He was a postdoctoral fellow at Lilly Research Laboratories. He has worked as a senior scientist at Pfizer, Lilly, Associate Director of Computational Drug Discovery at Concurrent Pharmaceuticals Inc. (now Vitae Pharmaceuticals Inc), and Vice President of Computational Biology at GeneGo (now Thomson Reuters). Sean is Adjunct Professor, Division of Chemical Biology and Medicinal Chemistry, UNC Eshelman School of Pharmacy, University of North Carolina at Chapel Hill; Adjunct Associate Professor, School of Pharmacy Department of Pharmaceutical Sciences, University of Maryland and Adjunct Professor in the Department of Pharmacology at Rutgers University– Robert Wood Johnson Medical School, Piscataway, NJ. Sean is on the scientific advisory board for several companies and editorial boards of the Journal of Pharmacological and Toxicological Methods, Drug Discovery Today, Mutation Research Reviews, and Pharmaceutical Research. He has authored or co-authored >220 peer reviewed papers and book chapters as well as edited four books for Wiley. Sean co-developed the mobile apps ODDT (Open Drug Discovery Teams) and TB Mobile and has been awarded multiple NIH grants
Dr. Joel Freundlich, Ph.D
Associate professor of Pharmacology & Physiology and Medicine at Rutgers University–New Jersey Medical School
Dr. Freundlich is an associate professor of Pharmacology & Physiology and Medicine at Rutgers University–New Jersey Medical School. He is a faculty member of the Center for Emerging and Reemerging Pathogens. Prior to his return to academic research in 2006, he spent eight years in the pharmaceutical industry as a medicinal chemist. His undergraduate and master’s degree training were in chemical engineering at Cornell University as a McMullen Dean’s Scholar. He received his doctorate in organic chemistry from the Massachusetts Institute of Technology under the tutelage of 2005 Nobel Prize in Chemistry awardee Richard Schrock. An overarching goal of his current research is the discovery of small molecules that modulate novel biological targets in Mycobacterium tuberculosis – the causative agent of tuberculosis. To accomplish their aims, his lab typically employs computational, chemical, and biological techniques. His laboratory is funded by the National Institutes of Health.
Dr. Steven J. Gray, Ph.D
Research Assistant Professor Dept. of Ophthalmology, U. of N. Carolina at Chapel Hill
Dr. Steven J. Gray is Research Assistant Professor Dept. of Ophthalmology, U. of N. Carolina at Chapel Hill. He was previously a Post Doc at UNC and before that had undertaken a PhD in Molecular Biology, at Vanderbilt University. His core expertise is in AAV gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the central and peripheral nervous system. His major focus is in AAV vector development to develop vector tailored to serve specific clinical and research applications involving the nervous system. These include the development of novel AAV capsids to treat epilepsy and Rett syndrome, as well as capsids amenable to widespread CNS gene transfer after intra-CSF administration. These also include development of vectors targeted to specific cell types such as oligodendrocytes, astrocytes, and retinal ganglion cells. As reagents have been developed to achieve global, efficient, and in some cases cell-type specific CNS gene delivery, his research focus has also included preclinical studies to apply these reagents toward the development of treatments for neurological diseases. Currently these include Rett Syndrome, Giant Axonal Neuropathy, Tay-Sachs, Krabbe, AGU, and Batten Disease. He has published over 27 peer reviewed papers and has one US patent. His research is funded by NIH and other research foundations.
Joseph J. Higgins, MD, FAAN
Medical Director, Neurology for Quest Diagnostics and the Laboratory Director at Athena Diagnostics. He is responsible for neurological diagnostic testing.
Prior to joining Quest Diagnostics in 2012, Dr. Higgins was a Professor at Weill Cornell Medical College where he directed the Autism Research Program and was the principal investigator on several private foundation and NIH research grants focused on clinical neurogenetics. For over 10 years, he held senior leadership positions in the federal and state governments at the National Institute of Neurological Disorders and Stroke and the New York State Department of Health. He is a fellow of the American Academy of Neurology with board certifications in Pediatrics by the American Board of Pediatrics and Neurology with Special Qualification in Child Neurology by the American Board of Psychiatry and Neurology. He has authored more than 90 peer‐reviewed publications, six book chapters, and eight reviews articles in the field of neurogenetics. He is the CLIA Laboratory Director at the Athena Diagnostics lab and is a Voluntary Clinical Professor of Pediatric Neurology at Weill Cornell Medical College.
Dr. Higgins earned his bachelor’s degree in biology from Marist College and finished his medical degree at New York University.
Brett Langley, Ph.D
Director of Neural Epigenetics at the Burke Medical Research Institute, Assistant Professor of Neurology and Neuroscience at Weill Medical College of Cornell University
Brett Langley completed his undergraduate (1995) and Masters degree with First Class Honors (1999) from the University of Waikato, and his Ph.D. degree in Molecular Medicine (2002) from the University of Auckland School of Medicine in New Zealand. Following his Ph.D. studies, Brett did postdoctoral research training Dr. Rajiv R. Ratan at the Beth Israel Deaconess Medical Center and Harvard Medical School. He then joined the Faculty at the Burke Medical Research Institute and was appointed to the position of Instructor of Neurology and Neuroscience at Weill Medical College of Cornell University.
His area of research focuses on understanding the molecular events that determine a neuron’s or axon’s fate following injury or during disease, and ultimately, whether they can be modulated therapeutically to promote better outcomes in patients.
Nadia Litterman, Ph.D
PhD is Collaborations Director at Collaborative Drug Discovery (CDD)
Nadia Litterman, PhD is Collaborations Director at Collaborative Drug Discovery (CDD), where she aims to identify and develop collaborations for drug discovery using CDD’s innovative informatics technologies. She has a long standing interesting in finding therapeutics for neuronal disorders and rare diseases, with more than 10 years of broad research experience in the areas of chemistry, cell biology, and neuroscience. During postdoctoral research in the laboratory of Dr. Lee Rubin at Harvard University, she utilized stem cell models of the rare, genetic, early onset motor neuron disorder Spinal Muscular Atrophy (SMA) to search for novel therapeutic strategies. She identified an opportunity for drug repurposing of an anti-cancer compound for SMA and found that this approach may be broadened for a second disease of motor neurons, Amyotrophic Lateral Sclerosis. As a National Science Foundation graduate fellow in the Pathology Department at Harvard Medical School, she led an investigation to explore biochemical processes that control protein trafficking in neurons. Her undergraduate research in the Chemistry department at Princeton University focused on free radical toxicity as it relates to protein misfolding and neurodegeneration.
Dr. Lucia Notterpek, Ph.D
William T. and Janice M. Neely Professor and Chair, Department of Neuroscience at the University of Florida, Gainesville
Dr. Lucia Notterpek, is the William T. and Janice M. Neely Professorand Chair, Department of Neuroscience at the University of Florida, Gainesville. She has a PhD in Neuroscience from UCLA and was a Postdoctoral Fellow in Neurobiology at Stanford University. The primary goal of her research is to contribute toward the development of effective therapies for demyelinating disorders of the nervous system, such as hereditary neuropathies and multiple sclerosis. To accomplish this aim, one must understand the subcellular mechanisms that underlie the process of myelination during development and the maintenance of the myelin sheath in the mature nervous system. Approaches used include small molecule therapies and in vitro cellular modeling of demyelinating disorders. The main model system she uses for her studies is Charcot-Marie-Tooth disease type 1A. Using optical imaging she is examining the response of myelinating cells to the accumulation of damaged and misfolded proteins and investigating approaches to prevent and reverse these events. Therapeutic approaches under investigations include small molecule enhancers of protein degradation and protein folding, as well as miRNA-mediated gene suppression. Targeting of therapeutic and imaging molecules to specific cells is being achieved by the use of lipid nanoparticles.
David Pleasure, MD
Professor of Neurology and Pediatrics, Director of Research, Shriner's Hospital for Children, Director, Institute for Pediatric Regenerative Medicine and Distinguished Professor, UC Davis
David obtained his MD from Columbia, and continued his training at the NINDS and the Marine Biological Laboratory, Woods Hole. He studies how inflammation of white matter contributes to certain autoimmune diseases like multiple sclerosis, cerebral palsy and several inherited childhood diseases such as adrenoleukodystrophy. He also focuses on periventricular leukomalacia (PVL) – a disorder of developing white matter in premature infants – that frequently results in cerebral palsy. Dr. Pleasure’s studies on immature oligodendroglia – cells that make myelin – contributed significantly to the understanding of both disorders. His research employs genetically modified mice, and is conducted in The Institute for Pediatric Regenerative Medicine (IPRM), a consortium of 14 faculty members and 27 pre/postdoctoral trainees that he founded in 2005. He has published over 230 peer reviewed papers.
Dr. Michael Sereda, M.D., Ph.D
Professor of Neurology and Group leader in the Department of Neurogenetics, Max Planck Institute (MPI) of Experimental Medicine, Göttingen, Germany
Dr. Michael Sereda M.D. is Professor of Neurology and Group leader in the Department of Neurogenetics, Max Planck Institute (MPI) of Experimental Medicine, Göttingen, Germany. He was a postdoctoral fellow in the group of Prof K.-A. Nave, PhD at MPI. He also completed a Residency in Neurology in the Dept. of Clinical Neurophysiology and Neurology, University of Göttingen and is currently Consultant Neurologist there. Hisdomain of research is Schwann cell and molecular biology as well as axon-glia interaction and disease mechanisms of Charcot-Marie-Tooth disease. He has generated a transgenic rat model of CMT1A which is helpful in the analysis of modifier genes, epigenetic factors, and in the evaluation of experimental treatment strategies. He has also recently identified biomarkers of disease severity in CMT1A patients and is currently validating markers in patients from across Europe which should help in performing clinical trials in the near future.
Dr. Scott Stromatt
Dr. Stromatt has a medical degree from the University of Chicago and a MBA from the University of Colorado. He is board certified in Internal Medicine and has over 26 years of biopharmaceutical experience, including three years as a biotechnology analyst for a Wall Street investment firm.
Dr. Stromatt has conducted over 55 Phase 1 to Phase 4 clinical trials in a wide variety of clinical indications, including oncology, rheumatology, pulmonology, gastroenterology and neurology. In the field of neurology, he ran clinical trials with a novel growth factor for Lou Gehrig’s disease or amyotrophic lateral sclerosis. He has worked on various regulatory filings including INDs, NDAs, BLAs and MAAs. Additionally, he has experience in medical affairs with extensive market and brand management support.
Dr. Stromatt is currently the Chief Medical Officer at Emergent BioSolutions, a biotechnology company focused on oncology, autoimmune disease and infectious diseases.
Dianna E. Willis, Ph.D
Head of the Laboratory for Axonal and RNA Biology, Director of the Center for Pain Research at the Burke Medical Research Institute and an Assistant Professor of Neuroscience at Weill Cornell Medical College
Dianna E. Willis, Ph.D., is the head of the Laboratory for Axonal and RNA Biology, Director of the Center for Pain Research at the Burke Medical Research Institute and an Assistant Professor of Neuroscience at Weill Cornell Medical College. Her research has focused on understanding how local protein synthesis within axons contributes to the proper function of neurons and to their ability to respond to injury. Her lab is also interested in how aberrant axonal translation may lead to maladaptive plasticity as is evident in neuropathic pain and neuropathy.