Christopher P. Austin, M.D., is the director of the National Center for Advancing Translational Sciences (NCATS) at the U.S. National Institutes of Health (NIH). NCATS’ mission is to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions. Before becoming NCATS director in September 2012, he was the director of the NCATS Division of Preclinical Innovation, which focuses on translating basic science discoveries into new treatments, particularly for rare and neglected diseases, and developing new technologies and paradigms to improve the efficiency of therapeutic and diagnostic development. In this role, Dr. Austin founded and directed numerous initiatives including the NIH Chemical Genomics Center (NCGC), the Therapeutics for Rare and Neglected Diseases (TRND) program and the Toxicology in the 21st Century (Tox21) program. From 2016 to 2018, he was the elected chair of the International Rare Disease Research Consortium (IRDiRC). Before joining NIH in 2002, Dr. Austin directed research programs on genomics-based target discovery, pharmacogenomics and neuropsychiatric drug development, with a particular focus on schizophrenia, at Merck. He received his A.B. in biology from Princeton University and his M.D. from Harvard Medical School and completed clinical training in internal medicine and neurology at Massachusetts General Hospital and a research fellowship in genetics at Harvard.

Krystof S. Bankiewicz, M.D., Ph.D., is a professor of neurosurgery at The Ohio State University. Previous positions include 20 years as Kinetics Foundation Chair in Translational Research and Professor in Residence of Neurological Surgery and Neurology at the University of California at San Francisco, where he currently holds the position of professor emeritus, and vice chair for research and chief of the Molecular Therapeutics Section at the National Institutes of Health (NIH). He cofounded MedGenesis Therapeutix, Inc., Voyager Therapeutics, Inc. and Brain Neurotherapy Bio, Inc. and has served on various scientific advisory boards. He is a recognized expert in neuro-restorative medicine, including delivery of therapeutics to the brain and gene therapy with successful translation of multiple drugs and gene therapies to the clinic. Dr. Bankiewicz has both industry and academic experience, is an inventor on numerous patents and has published more than 230 peer-reviewed research articles. Throughout his career, he has maintained a strong focus on the development of translational approaches to drug, gene and cell replacement therapies and has displayed the ability to synthesize distinct technologies into powerful new approaches to the treatment of serious diseases, including brain cancer, Parkinson’s disease, Huntington’s disease, Alzheimer’s disease, pediatric neurotransmitter deficiency and lysosomal storage disorders. Dr. Bankiewicz pioneered two ongoing gene therapy clinical trials in Parkinson’s disease and one in pediatric neurotransmitter deficiency. He also invented several devices currently used clinically to administer gene therapy to the brain. He received his M.D. from Jagiellonian University in Kraków, Poland, his Ph.D. and D.Sc. from the Institute of Neurology and Psychiatry in Warsaw, Poland and the title of Professor from the President of the Republic of Poland. He is a member of Polish Academy of Science. He was a recipient of a Fogarty Fellowship and trained in surgical neurology under Dr. Irvin Kopin and Dr. Edward Oldfield at the National Institutes of Neurological Disorders and Stroke at NIH.

Ying Kai Chan, Ph.D., is a research associate in the Department of Genetics at Harvard Medical School and a research scientist at the Wyss Institute for Biologically Inspired Engineering, both at Harvard University. His research interests are at the interface of viruses and the human immune system, with a focus on molecular engineering of desired viral and immune functions. He is currently exploring innovative technologies to modulate innate immune and inflammatory responses to AAV gene therapy. Previously Dr. Chan developed live attenuated vaccine candidates for infectious diseases such as dengue and Zika viruses. He received his Ph.D. in microbiology and immunobiology and a certificate in human biology and translational medicine from Harvard Medical School. Prior to that, he received degrees from Stanford University and Washington University in St. Louis.

Manuela Corti, P.T., Ph.D., is an assistant professor in the Child Health Research Institute and Powell Gene Therapy Center at the University of Florida. She is a clinical scientist engaged in translational research focusing on understanding the contribution of neurological impairment in neuromuscular disorders by combining expertise in clinical assessment with novel therapies that rely on correcting the fundamental genetic defect. Her specific research is dedicated to developing AAV gene therapy programs for neuromuscular diseases and immunomodulation strategies to prevent immune responses against the AAV capsid and the transgene and to allow for AAV administration in pre-existing immunity. Dr. Corti’s research interests also include outcome measures and clinical trial readiness for neuromuscular diseases such as Pompe disease, Friedreich’s ataxia, Canavan disease and Duchenne muscular dystrophy. She received her Ph.D in rehabilitation science from the University of Florida.

Ronald G. Crystal, M.D., is a professor and chairman of the Department of Genetic Medicine at the Weill Medical College of Cornell University, where he also is the Bruce Webster Professor of Internal Medicine, director of the Belfer Gene Therapy Core Facility and attending physician at the New York-Presbyterian Hospital/Weill Cornell Medical Center. Previously he served as chief of the Pulmonary Branch of the National Heart, Lung, and Blood Institute. In the 1970s and 1980s, his research focused on the pathogenesis and therapy of lung disorders. His laboratory’s work formed the basis of the current understanding of the pathogenesis of lung fibrosis and the hereditary form of emphysema associated with alpha 1-antitrypsin deficiency, a disease for which he developed the FDA-approved therapy now used to treat thousands of patients worldwide. In the late 1980s, Dr. Crystal shifted his focus to gene therapy, a field in which he is a pioneer. He was the first to use a recombinant virus as a vehicle for in vivo gene therapy and has carried out human trials of gene therapy for cystic fibrosis, cardiac ischemia, cancer and central nervous system disorders. Dr. Crystal has received numerous professional honors and serves on a number of advisory boards to government and industry. He has published more than 900 scientific articles, and his work has been cited more than 50,000 times in the scientific literature. He has edited several textbooks, is responsible for numerous biomedical patents and is a founder of several biotechnology companies focused on developing gene therapy therapeutics. He received degrees in physics from Tufts University and the University of Pennsylvania and his M.D. from the University of Pennsylvania. He completed his postgraduate training in internal medicine at the Massachusetts General Hospital and in pulmonary medicine at the University of California, San Francisco.

Olivier Danos, Ph.D., is senior vice president and chief scientific officer at REGENXBIO. He is a pioneer in the field of gene therapy and has dedicated his career to advancing the use of this technology to develop life-saving therapies for patients. Previously he was a senior vice president in charge of cell and gene therapy at Biogen. Over the past 20 years, he has played leadership roles in cell and gene therapy as director of the Gene Therapy Consortium of the University College of London, at the Necker Hospital – Enfants Malades in Paris, as chief scientific officer of Genethon and as senior director of research at Somatix Therapy Corporation. Dr. Danos has held senior research positions in France at the Centre National de la Recherche Scientifique and at the Institut Pasteur. He is the former president and a founding member of the European Society of Gene and Cell Therapy. He received his master’s degree in genetics and molecular biology from the University of Paris Orsay and his Ph.D. from the Pasteur Institute and University of Paris Diderot.


Steven Gray, Ph.D., is an associate professor in the Department of Pediatrics at the University of Texas Southwestern (UTSW) Medical Center. He also is the director of the UTSW Viral Vector Facility and maintains affiliations with the Department of Molecular Biology, the Department of Neurology and Neurotherapeutics, the Eugene McDermott Center for Human Growth and Development and the Hamon Center for Regenerative Science and Medicine at UTSW. Dr. Gray’s core expertise is in AAV gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the nervous system. As AAV-based platform gene transfer technologies 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 preclinical studies for Rett syndrome; giant axonal neuropathy (GAN); Tay-Sachs, Sandhoff, Krabbe, AGU, Charcot-Marie-Tooth, Batten, and Austin diseases and have expanded into human clinical studies to test a gene therapy approach for GAN. He received his B.S. with honors from Auburn University and his Ph.D. in molecular biology from Vanderbilt University. He performed a postdoctoral fellowship focusing on gene therapy in the laboratory of Jude Samulski at the University of North Carolina, Chapel Hill.


Juliette Hordeaux, D.V.M., Ph.D is research director of lysosomal storage disease in the Gene Therapy Program at the University of Pennsylvania, where she organizes and oversees all aspect of lysosomal storage disease translational research, from proof of concept studies to Investigational New Drug–enabling studies. She works with an internal team of scientists and technicians and external industry partners. She has developed several gene therapy strategies for neuropathic lysosomal storage diseases. Dr. Hordeaux’s goals are to collect quality and focused preclinical data that will support regulatory submissions. She received her D.V.M. from the Veterinary School of Nantes, France, and her Ph.D. in biology, medicine and health from the University of Nantes. She has been European board certified in veterinary pathology since 2011.


Paola Leone, Ph.D., is a professor of cell biology and neuroscience at the Rowan University School of Osteopathic Medicine, where she directs research activities at the Cell and Gene Therapy Center. She is an internationally recognized leader in the field of gene therapy for neurodegenerative disease and directed the first clinical gene therapy application for neurological disease in the brain. She has an ongoing and longstanding translational research program centered on identifying pathophysiologic mechanisms in neurodegenerative disease for targeting by gene therapy-based technology platforms. Dr. Leone headed the first clinical gene therapy trial for a brain disease using both liposome-based technology and adeno-associated viral (AAV) vectors for application in the human pediatric leukodystrophy Canavan disease. She has been actively documenting the effects of gene therapy in Canavan disease patients for more than two decades and is currently pursuing novel gene therapy strategies with therapeutic potential in a wide range of model systems of neurodegenerative disease, including Canavan disease, Alzheimer’s disease, amyotrophic lateral sclerosis and traumatic brain injury. She is a distinguished member of numerous scientific advisory boards and has been honored with several awards, including a UNESCO Award and the MD Advantage Outstanding Medical Research Scientist Award. She received her Ph.D. in neuroscience from the University of Padua, Italy, and performed her postdoctoral training at Concordia University, Canada, and Yale University.

Brian Long, Ph.D., is an associate director in translational sciences and immunogenicity assessment at BioMarin, where he provides immunologic expertise to drug programs across developmental stages and develops immunogenicity and safety strategies for novel biologic therapeutics. He has more than 20 years of experience in immunology and infectious disease, autoimmunity, cell signaling, cancer biology and drug development. Previously Dr. Long was a research scientist in the Division of Experimental Medicine at the University of California, San Francisco (UCSF), where he worked on the development and standardization of humanized mouse models for the evaluation of HIV immunology, therapeutics and drug discovery. He received his Ph.D. in microbiology and immunology from the University of North Carolina, Chapel Hill and his postdoctoral training at the Gladstone Institutes and UCSF, where he investigated the role of innate immunity in HIV disease pathogenesis.

Caroline Sevin, M.D., Ph.D., is the head of the French reference center for childhood leukodystrophies at Bicêtre Hospital (Le Kremlin Bicêtre, France). She is involved in the diagnosis and treatment of children with childhood cerebral adrenoleukodystrophy (CCALD), metachromatic leukodystrophy (MLD) and other leukodystrophies. She is principal investigator (PI) or co-PI of several clinical trials in CCALD and MLD, including gene therapy for MLD. As a member of the leukodystrophy group in the European Reference Network on Rare Neurological Diseases (ERNRND), Dr. Sevin is involved in the production of European guidelines for MLD and X-linked adrenoleukodystrophy (X-ALD) as well as implementation of newborn screening in France. She is affiliated with the NeuroGenCell team at the Institute for Brain and Spinal Cord (ICM) in La Pitié Salpêtrière Hospital, Paris, France, under the direction of Dr. Nathalie Cartier. NeuroGenCell is dedicated to the development of gene therapy strategies for neurodegenerative diseases and has pioneered hematopoietic stem cell (HSC) gene therapy for X-ALD and brain gene therapy for MLD.

U.S. Department of Health and Human Services National Center for Advancing Translational Sciences