Medical Director

Roger H. Karlin, M.D.

Scientific Advisory Board

Roscoe Brady, M.D.
Department of Neurosciences - National Institute of Health (Bethesda, MD)

Ruben Matalon, M.D.
Department of Neurosciences - University of Texas at Galveston (Galveston, TX)

Andrew Freese, M.D.
Department of Neurosurgery - University of Minnesota (Minneapolis, MN)

Christopher Janson, M.D.
Department of Neurosurgery - University of Minnesota (Minneapolis, MN)

Scott W.J. McPhee, Ph.D.
Research Scientist in Department of Surgery in the Division of Neurosurgery - University of Medicine & Dentistry of New Jersey (Camden, NJ)

Jeremy S. Francis, Ph.D.
Research Scientist in Department of Surgery in the Division of Neurosurgery - University of Medicine & Dentistry of New Jersey (Camden, NJ)


Dr. Paola Leone, Ph.D. - Professor of Cell Biology and Director of the Cell & Gene Therapy Center at the Rowan University School of Osteopathic Medicine

Following post-doctoral studies in Montreal, Dr. Leone was an Associate Research Scientist at Yale University from 1996-1998. From 1998-2001, she was Associate Director of the CNS Gene Therapy Center at Jefferson Medical College and an Assistant Professor in the Department of Neurosurgery. Dr. Leone has been responsible for the development and characterization of viral (AAV, adenovirus, retrovirus) and non-viral vectors for the treatment of Canavan Disease and other disorders. At Yale and Thomas Jefferson Universities, she was the IND/FDA sponsor of two separate 'Gene Therapy for Canavan Disease' studies.

Dr. Leone is currently Principal Investigator on the Canavan gene therapy protocol using adeno-associated viral vectors. Her laboratory is conducting in-vivo studies of both viral vectors and stem cells and their use in neural transplantation for therapeutic applications on a variety of neurodegenerative disorders, brain and spinal injuries and stroke. She also leads investigations on pharmacological, genetic and stem cell therapies on animal models of Canavan Disease, Amyotrophic Lateral Sclerosis, Parkinson's Disease, Tay Sach's Disease and other neurological disorders.

Evan Snyder, M.D. & Ph.D. Harvard Medical School, Professor of Pediatrics, Pediatric Neurology, Neonatology; Director, Stem Cells and Regeneration Program, The Burnham Institute

Dr. Snyder is the world-renowned leading researcher and co-founder of the stem cell field. His work focuses on understanding the mechanisms underlying Canavan disease and other childhood neurodegenerative diseases. Dr. Snyder's work investigates Canavan at a molecular and cellular basis - particularly as programmed into the central nervous system at the stem cell level. Dr. Snyder's work focuses on studying implanted stem cells in Canavan animal models with the goal of treating afflicted Canavan children. 

Richard Jude Samulski, Ph.D. - Director and Professor of Pharmacology at the Gene Therapy Center at the University of North Carolina School (Chapel Hill) of Medicine

Dr. Samulski received his Ph.D. in Medical Microbiology and Immunology from the University of Florida.  Dr. Samulski’s graduate work demonstrated the first use of AAV as a viral vector and culminated in the first US patent involving non-AAV genes inserted into AAV. 

After completing post-doctoral training at Princeton, Dr. Samulski was hired to provide his expertise in AAV biology as a member of the scientific advisory board of Avigen, a new AAV research company.  Later, in 1993, Dr. Samulski co-founded an AAV-based gene therapy company called Merlin.  This research group was headed by Dr. Xiao and was the first group to demonstrate AAV-mediated long-term gene transduction in muscle. This finding precipitated a merger of Merlin with Somatix, Inc., which then merged in 1995 with Cell Genesys.  

In total, Dr. Samulski has worked with AAV for 25 years, and for the past 8 years, has been director of the University of North Carolina Gene Therapy Center.  Dr. Samulski has over 20 patents filed or issued in the area of AAV vectors. 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 frequently serves as a gene therapy consultant to the FDA.  Through the UNC gene therapy center, Dr. Samulski produced FDA-approved AAV clinical vector used to treat children with the neurological disorder of Canavan’s disease (now a total of 7 children – ages 2 - 6). He has received the first lifetime achievement award at the American Society of Gene Therapy (ASGT).