Gustavo D. Aguirre, VMD, PhD, PhDhc
University of Pennsylvania School of Veterinary Medicine

Gustavo D. Aguirre, VMD, PhD, PhDhc

Gustavo D. Aguirre is Professor of Medical Genetics and Ophthalmology at the School of Veterinary Medicine, University of Pennsylvania, and works with dog models of inherited eye and retinal degeneration. His lab focuses on model identification, disease gene discovery, establishing disease metrics and defining molecular pathways linking the gene and mutation to the disease. Since 2000, his work has focused on developing gene-based and other therapies for translational applications.

His research has led to the proof of concept studies that AAV-mediated RPE65 gene therapy reversed a congenital blinding disorder of children, Leber congenital amaurosis. This then led to several clinical trials and eventual commercialization of the therapy. Two of his other gene therapy studies in the dog model are in Phase 3 clinical trials, and two others are in the IND enabling pathway.

Dr. Aguirre earned his undergraduate, veterinary, and doctoral degrees at the University of Pennsylvania, where he also completed a residency in ophthalmology before serving as a post-doctoral fellow at the Wilmer Ophthalmological Institute of the Johns Hopkins University School of Medicine. He joined the faculty at Penn in 1973, where he rose to hold joint professorial appointments in the Veterinary and Medical Schools. From 1992 to 2004, he was the Caspary Professor of Ophthalmology at the James A. Baker Institute of Cornell University, before returning to Penn in 2004. His work has been supported by the NIH, FFB and other organizations.

Dr. Aguirre has received numerous awards, among which are an honorary Doctor of Philosophy degree from the Faculty of Mathematics and Natural Sciences, University of Göteborg, Sweden, The Foundation Fighting Blindness Trustee Award, Scientist of the Year, Heart SightMiami/Foundation Fighting Blindness Award, O.N.C.E. International Prize for R&D in Biomedicine and New Technologies for the Blind, the Alcon Research Institute Award, the Louis Braille Special Recognition Award from Associated Services for the Blind and Visually Impaired, and he was a co-recipient of the 2004 Paul Kayser International Award in Retina Research. In 2017, he received the Proctor Medal from the Association for Research in Vision and Ophthalmology for outstanding research in basic or clinical sciences as applied to ophthalmology, and in 2020, he was co-recipient of the Sanford and Sue Greenberg Prize to End Blindness.

He is a Fellow of the Association for Research in Vision and Ophthalmology, the College of Physicians of Philadelphia, the American Association for the Advancement of Science, and he has been a member of The National Academy of Medicine since 2012.