Tim Miller, PhD
Timothy J. Miller, PhD is President and Chief Executive Officer and a Director of Abeona Therapeutics Inc. He has 18 years of scientific research, product development and clinical operations expertise, with a focus on transitioning novel biotherapeutics through pre-clinical phases and into Phase 1 and 2 human clinical trials. Dr. Miller was President & CEO of Red5 Pharmaceuticals from 2013 until 2015 and was CEO-in-Residence at BioEnterprise Inc. in 2015. He was Senior Director of Product Development at SironRX Therapeutics from 2010 to 2013. Between 1996 and 2010 Dr. Miller held various positions at several biotech companies focusing on gene therapy and regenerative medicine. Dr. Miller earned his PhD in Pharmacology with a focus on Gene therapy/Cystic Fibrosis from Case Western University. He also holds a BS in Biology and MS in Molecular Biology from John Carroll University (Cleveland, OH).
He has raised over $100M for therapies in cystic fibrosis, cardiovascular disease, wound healing, scar prevention, and rare diseases to advance these therapies into clinical trials. As a serial entrepreneur, he has managed all aspects of research and development, manufacturing of biologics, and clinical program start-up in both public and private companies, with direct experience engaging Food and Drug Administration (FDA) and NIH advisory agencies on multiple Investigational New Drug (IND) submissions. During his career, he has contributed to multiple patent applications, managed intellectual property, and published research in several internationally recognized journals. He has a passion for supporting patient advocacy.
George Zavoico, PhD
JonesTrading Institutional Services
George B. Zavoico, PhD, is a Senior Equity Analyst, Healthcare, at JonesTrading Institutional Services, a leading equity trading firm with a focus on block trading and a growing capital markets business. He has over 11 years of experience as a life sciences analyst writing research on publicly traded equities. His principal focus is on biotechnology, biopharmaceutical, specialty pharmaceutical, and molecular diagnostics companies. He received The Financial Times / Starmine Award two years in a row for being among the top-ranked earnings estimators in the biotechnology sector. In 2009, Zavoico was hired as the first equity analyst at MLV & Co., a New York-based boutique investment bank and institutional broker-dealer at the time, where he helped establish its Healthcare research team. He returned to MLV in mid-2014 after serving for a brief period as a Senior Equity Analyst at H.C. Wainwright & Co. in early 2014, and then joined JonesTrading in early 2015.
Previously, Zavoico was an equity research analyst in the healthcare sector at Westport Capital Markets and Cantor Fitzgerald. Prior to working as an analyst, Zavoico established his own consulting company serving the biotech and pharmaceutical industries, providing competitive intelligence and marketing research, due diligence services and guidance in regulatory affairs. Zavoico began his career as a senior research scientist at Bristol-Myers Squibb Co., moving on to management positions at Alexion Pharmaceuticals Inc. and T Cell Sciences Inc. (now Celldex Therapeutics Inc.). Zavoico has a bachelor's degree in biology from St. Lawrence University and a PhD in physiology from the University of Virginia. He held post-doctoral fellowships at the University of Connecticut School of Medicine and Harvard Medical School / Brigham & Women's Hospital. He has published more than 30 papers in peer-reviewed journals and has coauthored four book chapters.
Sonya Dougal, PhD
The New York Academy of Sciences
Caitlin McOmish, PhD
The New York Academy of Sciences
Barry Byrne, MD, PhD
University of Florida Powell Gene Therapy Center
Dr. Barry J. Byrne is a clinician scientist interested in a variety of rare diseases, with specific attention to developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness, cardiac dysfunction and respiratory dysfunction. His research team has made significant contributions to the understanding and treatment of Pompe disease, a type of muscular dystrophy resulting from abnormal glycogen accumulation in the muscle. His current research has focused on developing new therapies using the missing cellular protein or the corrective gene to restore muscle function in Pompe and other inherited myopathies.
Dr. Byrne is the Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida. After obtaining a BS degree in Chemistry from Denison University, he pursued his medical education, as well as a PhD in Microbiology and Immunology, at the University of Illinois. He completed his pediatric residency, cardiology fellowship training and post-doctoral training in Biological Chemistry at Johns Hopkins University. Joining the University of Florida in 1997, he has served in a variety of clinical, research and educational roles, and is now the Earl and Christy Powell University Chair in Genetics.
Maria Escolar, MD, MS
University of Pittsburgh
After receiving her medical degree in Colombia in 1986, Dr. Maria Luisa Escolar came to the United States to complete a residency in Pediatrics and a fellowship in Child Development and Behavioral Pediatrics at New York Hospital-Cornell Medical Center. Within one year of encountering her first patient with Krabbe disease in 2000, Dr. Escolar established the Program for the Study of Neurodevelopment in Rare Disorders and relocated her program to Children's Hospital of Pittsburgh of UPMC in 2011. This Program is the only one of its kind in the United States and focuses on understanding the behavioral and neurological abnormalities of inherited brain disorders caused by genetic mutations, such as the mucopolysaccharidoses (MPS) and leukodystrophies. Dr. Escolar's research focuses on developing better methods to understand the impact of brain abnormalities on neurobehavioral function. She has developed new methods to understand function in neurodegeneration and a new neuroimaging tool able to quantitate changes in myelination as early as birth. She has published more than 60 papers in medical journals, including two articles published in the New England Journal of Medicine. Dr. Escolar is board certified in Pediatrics and Neurodevelopmental Disabilities.
Kevin Flanigan, MD
Nationwide Children's Hospital
Dr. Flanigan trained in Neurology and Neuromuscular Disease at Johns Hopkins University, and pursued a post-doctoral fellowship in Human Molecular Biology and Genetics at the University of Utah. After 14 years on the faculty in Utah, he moved to Ohio State University as Professor of Pediatrics and Neurology in 2009. He is the Robert F. & Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research, Director, of Nationwide Children's Center for Gene Therapy and co-Director of the NCH Muscular Dystrophy Association Clinic. He has been a member of the Executive Board of the World Muscle Society since 2001, and is a member of the Executive Committee of TREAT-NMD, the international alliance directed toward establishing the infrastructure to ensure that promising new therapies reach patients as quickly as possible. His laboratory work is directed toward the molecular characterization and therapy of neuromuscular diseases, and the identification of genetic modifiers of disease. He is also conducted multiple clinical trials in Duchenne muscular dystrophy, including trials of gene modifying therapies such as nonsense suppression and exon skipping.
Steven J. Gray, PhD
University of North Carolina at Chapel Hill
Dr. Steven Gray earned his PhD in molecular biology from Vanderbilt University in 2006, after receiving a BS degree with honors from Auburn University. He performed a postdoctoral fellowship focusing on gene therapy in the laboratory of Jude Samulski at UNC. He is currently an assistant professor at UNC in the Department of Ophthalmology. He is also a member of the Gene Therapy Center and the Carolina Institute for Developmental Disabilities (CIDD).
Dr. Gray's core expertise is in AAV gene therapy vector engineering, followed by optimizing approaches to deliver a gene to the nervous system or eye. His major focus is in AAV vector development to develop vectors tailored to serve specific clinical and research applications involving the nervous system or eye. These include the development of novel AAV capsids amenable to widespread CNS gene transfer or specialized ocular gene transfer. 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, Krabbe, AGU, and Batten Disease, and have expanded into human clinical studies to test a gene therapy approach for GAN.
Dr. Gray has published over 50 peer-reviewed papers in journals such as New England Journal of Medicine, Molecular Therapy, Nature Biotechnology, Gene Therapy, and The Proceedings of the National Academy of Sciences. He also has 3 pending patents. His research is funded by the National Institute for Neurological Disorders and Stroke, as well as numerous large and small research foundations. Dr. Gray was recently recognized with the 2016 Healthcare Hero award by the Triangle Business Journal, and his work on GAN was featured in a story by the CBS National Evening News in 2015.
Katherine A. High, MD
Dr. Kathy High, an accomplished hematologist with a longstanding interest in gene therapy for genetic disease, began her career studying the molecular basis of blood coagulation and the development of novel therapeutics for the treatment of bleeding disorders. Her pioneering bench-to-bedside studies of gene therapy for hemophilia led to a series of studies that characterized the human immune response to AAV vectors in a variety of target tissues. Kathy's work has evolved to encompass clinical translation of potential gene therapies for multiple inherited disorders. As the director of the Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia, Kathy assembled a multidisciplinary team of scientists and researchers working to discover new gene and cell therapies for genetic diseases and to facilitate rapid translation of preclinical discoveries into clinical application.
Kathy was a long-time member of the faculty at the University of Pennsylvania and of the medical staff at CHOP, where she was also an Investigator of the Howard Hughes Medical Institute. She served a five-year term on the FDA Advisory Committee on Cell, Tissue and Gene Therapies and is a past president of the American Society of Gene & Cell Therapy (ASGCT). She received her AB in chemistry from Harvard University, an MD from the University of North Carolina School of Medicine, a business certification from the University of North Carolina Business School Management Institute for Hospital Administrators and an MA from the University of Pennsylvania.
Brian Kaspar, PhD
Dr. Kaspar's research focuses on basic and translational studies related to neurological and neuromuscular disorders. The laboratory has strengths in animal models of neurodegenerative and neuromuscular disease, gene delivery, and stem cell biology. A main focus of the Kaspar laboratory is centered on the mechanism(s) of neurodegeneration in Amyotrophic Lateral Sclerosis (ALS) and Spinal Muscular Atrophy (SMA). Rodent models of this disease are employed to investigate various cell type involvements in disease onset and progression. Furthermore, Kaspar laboratory is actively developing novel methods to deliver genes and therapies more efficiently to the nervous system and testing novel targets to combat this debilitating, lethal disease. Additionally, the laboratory also investigates the biological control of embryonic and adult derived stem cells. Current studies with stem cells are evaluating cell cycle regulation along with developing methods for intricate control of differentiation to defined cellular phenotypes, such as complex motor neurons. Finally, the laboratory works on muscle enhancing strategies in order to combat musculoskeletal disorders.
R. Jude Samulski, PhD
Dr. Samulski received his PhD in Medical Microbiology and Immunology from the University of Florida. Dr. Samulski's graduate work (1978-82) involved cloning of AAV as a viral vector and culminated in the first US patent involving non-AAV genes inserted into AAV.
Dr Samulski has worked with adeno-associated virus (AAV) for 35 years, and was the Director of the University of North Carolina Gene Therapy Center for over 20 years. Dr. Samulski Research has focused on AAV-based gene therapy. Dr Samulski is a former member of the Recombinant DNA Advisory Committee (RAC), a committee tasked with assisting the FDA with approving or disapproving gene therapy clinical trials in the United States. He also has served as a gene therapy consultant to the FDA.
Jakub Tolar, MD, PhD
University of Minnesota
Dr. Tolar is originally from the Czech Republic, and received his medical education in Prague at the Charles University. In 1992, he came to the University of Minnesota, where he completed his PhD in Molecular, Cellular & Developmental Biology and Genetics. He is currently a distinguished McKnight Professor in the Department of Pediatrics, Blood and Marrow Transplantation, the Edmund Wallace Tulloch & Anna Marie Tulloch Chair in Stem Cell Biology, Genetics & Genomics, and the Director of the Stem Cell Institute. He is a member of the graduate faculty of the Microbiology, Immunology and Cancer Biology Program, the Molecular, Cellular, and Developmental Biology and Genetics Program, and the Stem Cell Biology Program.
Dr. Tolar's research focuses on finding new ways of treating children with lethal diseases—cancer, inborn errors of metabolism, and devastating genetic disorders—using stem cell transplantation. He is also looking for safer and more effective methods of repairing and using a patient's own cells in diseases such as epidermolysis bullosa, mucopolysaccharidosis type I (Hurler syndrome), Fanconi anemia, and dyskeratosis congenita.