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Maria Escolar

Maria Escolar

Chief Medical Officer, University of Pittsburgh
Maria Escolar, MD, MS is a pioneer in clinical trials design and is currently leading the development of gene therapy for Krabbe disease as Chief Medical Officer at Forge Biologics. She transitioned from Tenured Professor to Adjunct Professor of Pediatrics at the University of Pittsburgh in 2023 after she was recruited full time to Forge. She has 25 years of experience as a translational researcher and practicing clinician with over 95 publications, garnering international recognition for her work in neurodevelopment of children with leukodystrophies, mucopolysaccharidosis and other genetic, neurodegenerative conditions.

Dr. Escolar, a Neurodevelopmental Pediatrician, began her research on rare neurodegenerative disorders in 2002, when she established a translational multidisciplinary research program, the Program for the Study of Neurodevelopment in Rare Disorders (NDRD) at The University of North Carolina (UNC) Chapel Hill. The program aims to improve the lives of children with rare neurodegenerative disorders, describe their natural history and develop therapies. Dr. Escolar has strong interests in developing novel quantitative neuroimaging techniques and was NIH funded to develop neuroimaging tools that detect aberrant brain development as early as birth. Dr. Escolar served as Associate Director for Translational Research at UNC Gene Therapy Center under the direction of Dr. Jude Samulski until 2011 when she was recruited to the University of Pittsburgh where the NDRD program expanded globally attracting patients from 48 states and 39 countries.

Dr. Escolar is an expert on outcome measures, natural history studies and clinical trial design.. She has served as site PI for multiple gene therapy trials with all routes of administration. Dr. Escolar developed a novel adeno-associated virus gene therapy after hematopoietic stem cell transplantation for Krabbe disease administered during myeloablation and/or transplant immune suppression. Forge Biologics Inc. licensed the therapy in 2020 and advanced it to a Phase I/II clinical trial.
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