Through the study and development of technologies that overcome current translational hurdles, Dr. Vandenberghe is helping to position gene therapy as a broadly applicable clinical modality in ophthalmology. Previously, he developed a novel adeno-associated viral vector (AAV) platform, and in translational studies, he characterized those for safety and efficacy parameters in large animal models. His current research focuses on gene therapy for neurosensory disorders, especially retinal blindness. He is on the editorial board of Molecular Therapy and is an active member of the American Society of Cell and Gene Therapy, for which he chairs the educational committee. In addition, he co-founded GenSight Biologics, a biotech entity that pursues innovative ocular gene therapy strategies.