Maria Escolar

Dr. Maria Escolar Chief Medical Officer at Forge Biologics and Adjunct Professor of Pediatrics and Neurodevelopmental Disabilities Pediatrician at the University of Pittsburgh. She began her research on rare neurodegenerative disorders in 2000, when she established the Program for the Study of Neurodevelopment in Rare Disorders (NDRD), a clinical research program at The University of North Carolina (UNC) at Chapel Hill, which was a multidisciplinary program that aimed to improve the lives of children and families living with rare neurodegenerative disorders while learning about the natural history of the disorders. From 2010 to 2011, Dr. Escolar also served as Associate Director for Translational Research at UNC Gene Therapy Center under the direction of Dr. Jude Samulski and in 2011, she was recruited by the University of Pittsburgh to develop a larger Program for Rare Diseases, since inception of the NDRD program she evaluated patients from 48 states in the US and 39 countries.

Dr. Escolar is best known for her work focused on rare, genetic, neurodegenerative diseases: such as Krabbe, MLD, & ALD leukodystrophies, Mucopolysaccharidosis disorders (MPS I, II, and III), Gaucher, GM1, GM2, Nieman Pick C, Mucolipidosis, Neuronal ceroid lipofuscinosis, Alpha-mannosidosis, neurodegeneration with brain iron accumulation (PKAN), vanishing white matter disease and many others. Dr. Escolar has published over 80 original research papers in peer-reviewed journals, including two original publications in the New England Journal of Medicine. She is an expert in natural history studies, evaluation of treatment outcomes and identifiers of disease progression that can predict long-term outcomes. She uses standardized neurobehavioral, neuroradiological, and neurodevelopmental and biological markers to determine the extent of disease progression in patients suffering from rare diseases. Dr. Escolar has strong interests in developing novel quantitative neuroimaging techniques that allow for longitudinally tracking in the brain throughout the disease process. She was funded by the NIH to develop neuroimaging tools for newborn screening and generating atlases of normal brain development that allows for comparison between normal and aberrant neurological development and further contributes to the scientific community’s understanding of neurodegeneration in the developing brain. Lastly, Dr. Escolar is a leader in clinical trial design with a specific expertise in gene therapy trials. She has served as site PI for multiple gene therapy trials, including trials for MLD, Hunter’s Syndrome, Sanfilippo Syndrome, Gaucher and GM2. Dr. Escolar has also developed a novel therapy for Krabbe disease, a treatment using adeno-associated virus gene therapy after hematopoietic stem cell transplantation. Forge Biologics Inc. has licensed the therapy and advanced it to a Phase I/II clinical trial.