Drs. Jean Bennett and Albert Maguire with their dogs, who received the experimental gene therapy (photo credit: Peggy Peterson).
By Alexandra Brodin
Scheie Vision Annual Report 2020
The Scheie Eye Institute congratulates Jean Bennett, MD, PhD, the F.M. Kirby Professor of Ophthalmology, and Albert Maguire, MD, Professor of Ophthalmology, on receiving the Sanford and Susan Greenberg Outstanding Achievement Prize from the End Blindness 2020 campaign.
End Blindness 2020 supports the development of curative solutions to blinding diseases. The Sanford and Susan Greenberg Prize recognizes individuals who have had the greatest impact on advancing research for vision restoration.
Drs. Bennett and Maguire were selected for their pioneering work to develop the first-ever FDA-approved gene therapy for an inherited disease. This gene therapy targets a form of Leber’s congenital amaurosis (LCA) caused by a mutation in the RPE65 gene. Patients who undergo this gene therapy receive a one-time subretinal injection of a viral vector containing a normal copy of the RPE65 gene. This treatment, known as Luxturna, restored vision in children and adults with RPE65-related LCA and received historic FDA approval in 2017.
The research that led to Luxturna will pave the way for the development of gene therapies for other blinding diseases. “Sanford and Susan Greenberg are extraordinary people who have turned Sanford’s terrible misfortune in losing his sight into an inspiration for others,” said Dr. Bennett. “Albert and I intend to use this award to continue to make the Greenberg’s dreams of sight restoration and prevention of blindness come true.”
Gustavo Aguirre, VMD, PhD, Professor of Medical Genetics and Ophthalmology at the University of Pennsylvania School of Veterinary Medicine, and William Hauswirth, PhD, Professor of Ophthalmology at the University of Florida, were also recipients of this award. The $1 million prize will be distributed evenly between the four awardees.
Dr. Bennett intends to donate part of her funds to Katherine Uyhazi, MD, PhD, for her start-up package with the University of Pennsylvania. Dr. Uyhazi has recently accepted a full-time faculty appointment in the Department of Ophthalmology and is working to develop stem cell-based therapies for retinal degenerative diseases. “Dr. Uyhazi’s efforts will provide the perfect complement to our gene therapy studies, as they could ultimately lead to tissue transplantation approaches to restore vision,” explained Dr. Bennett.
In 2018, Drs. Bennett, Maguire and collaborators also received the António Champalimaud Vision Award for their advances in retinal gene therapy, and Dr. Bennett was a co-recipient of the Sanford Lorraine Cross Award.