Medical Advisory Board

Robert E. Sobol, M.D. has pioneered the development of novel biological therapies.  He was one of the first physicians to treat patients with monoclonal antibodies and gene therapy. Dr. Sobol is an internationally recognized expert in the fields of molecular cancer therapies and biomarkers. Dr. Sobol led the research teams responsible for the first experimental gene therapies for the treatment of brain tumors and colon carcinoma.

Dr. Sobol has served in the American Society of Gene and Cell Therapy as the Chair of both the Cancer Gene and Cell Therapy and the Bio Industry Liaison Committees. He is a member of several medical and scientific societies and was appointed to the Clinical Program Review Committee of the National Cancer Institute of the National Institutes of Health. He was the founding Editor-in-Chief of Cancer Gene Therapy, a Nature Publishing Group journal.

Dr. Sobol received a B.A. in Philosophy from Boston University and a M.D. from The Chicago Medical School. He subsequently trained at the University of Southern California Medical Center and at the University of California, San Diego and received Board Certifications in Internal Medicine and Medical Oncology.

Dr. Gerald S. Lipshutz, MD, MS, received his medical degree from the University of California, Los Angeles (UCLA) School of Medicine and completed his postgraduate training at the University of California, San Francisco School of Medicine. He is currently a Professor in the Departments of Surgery and Molecular and Medical Pharmacology at UCLA. Dr. Lipshutz is also a member of the UCLA Intellectual and Developmental Disabilities Research Center and the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, where he holds the Goldwyn Chair.

Dr. Lipshutz’s research focuses on developing innovative gene and cell therapies for single-gene metabolic disorders affecting the liver. He has been an invited participant in numerous National Institutes of Health (NIH) conferences and has served as a grant reviewer for both the Wellcome Trust in the United Kingdom and the NIH. He recently completed four years as a standing member of the NIH Genetic Disorders and Developmental Disabilities (GDD) Study Section.

An accomplished researcher and educator, Dr. Lipshutz has authored more than 80 peer-reviewed scientific publications. He also serves on the editorial boards of Molecular Therapy, Molecular Therapy: Methods & Clinical Development, and Gene Therapy. In addition, he is an active member of several professional organizations focused on surgery, transplantation, gene therapy, and metabolic disorders, including the American Society of Gene & Cell Therapy (ASGCT) and the Society for Inherited Metabolic Disorders (SIMD).

As Principal Investigator of the UCLA Lipshutz Hepatic Regenerative Medicine Laboratory, Dr. Lipshutz leads research into regenerative medicine approaches for the treatment of urea cycle disorders, including arginase deficiency and carbamoyl phosphate synthetase deficiency, as well as creatine deficiency disorders. His work aims to develop therapies that could one day replace liver transplantation for patients with single-enzyme metabolic diseases. He is also dedicated to advancing the understanding of the neurological effects of arginase deficiency and hyperargininemia.

Dr. Lipshutz currently serves as Principal Investigator on multiple NIH-funded, California Institute for Regenerative Medicine (CIRM)-funded, and industry-sponsored studies focused on the development of next-generation gene therapies for rare metabolic disorders.

Stephen Cederbaum is a Distinguished Professor Emeritus at UCLA Departments of Psychiatry and Biobehavioral Sciences, Pediatrics, and Human Genetics. Stephen has worked in the field of inborn errors of metabolism and newborn screening, particularly disorders of the urea cycle for more than 50 years. He has studied these disorders in patients and in animal models, using various forms of gene and mRNA therapy. He is considered a world authority on arginase deficiency which he has studied his entire career, in the clinic and in the laboratory. He helped found the parent support group that has become the ARD1-D Foundation and has been a continuous supporter to these families. More recently he has emphasized the pre-symptomatic DNA screening of individuals at risk for late onset autosomal dominant disorders. He lives in Santa Monica, California with his wife and they have two grown children.

Dr. Andreas Schulze is a metabolic pediatrician and professor in the Departments of Paediatrics and Biochemistry at University of Toronto. He is the Medical Director of the Newborn Screening Program at The Hospital for Sick Children (SickKids) Toronto and Senior Associate Scientist at the SickKids Research Institute.

Schulze receive a medical diploma and a doctorate in medicine from the Faculty of Medicine at Leipzig University in 1987. After completing graduate training and PhD in Physiological Biochemistry under the supervision of Dr. Hans-Joachim Boehme and Dr. Eberhard Hoffmann (1987-1992), Schulze pursued postgraduate clinical training in Pediatrics at the University Children’s Hospital in Heidelberg under Dr. Hans-Juergen Bremer and Dr. Georg F. Hoffmann (1992-1999). Schulze defended a Professorial Thesis (Habilitation) and received the Venia Legendi from the Ruprecht-Karls University Heidelberg in 2004. He is board certified in Physiological Biochemistry (1993) and in Pediatrics (1999).

Since 2007, Schulze works as clinician scientist at The Hospital for Sick Children in Toronto. As a clinician he takes care of children with inborn errors of metabolism and oversees the SickKids Newborn Screening Program. As a scientist, he established a research group and a research laboratory at the SickKids Research Institute. Dr. Schulze’s research is centered around creatine deficiency syndromes and regulation of creatine homeostasis. His research encompasses the metabolism of arginine, ornithine, and guanidino compounds, and includes small molecule drug discovery.