There is no question that retinal gene therapy has definitely arrived. Not only is it alive and well today, but looking at the current state of research, it will hopefully have a very bright future.
These were some of the messages contained in the 2019 Charles L Schepens MD Lecture Award given by Jean Bennett, MD, PhD, and Albert M. Maguire, MD, at the American Academy of Ophthalmology 2019 annual meeting in San Francisco.
In an informative and entertaining talk titled “Retinal Gene Therapy: From Theory to Practice,” they shared their experiences developing voretigene neparvovec-rzyl (Luxturna, Spark Therapeutics), the first gene therapy approved in the United States to treat an inherited disease.
“When we began this research, there was no such thing as gene therapy. There were no clinical trials, no companies, not even laboratory techniques to do retinal gene transfer. Our original Leber’s congenital amaurosis (LCA) team numbered just six people,” said Dr. Maguire, professor of Ophthalmology, University of Pennsylvania, Philadelphia. “Now gene therapy is a multibillion dollar business involving countless investigators, dozens of startups, and several large pharmaceutical companies, all employing thousands of people.”
Dr. Bennett provided a clinical status update.
“There are now more than 1,150 patients enrolled in retina gene therapy clinical trials that are being conducted at more than 30 sites, and at least 13 Centers of Excellence are administering Luxturna,” she said. “Importantly, there are many more gene therapy clinical trials being planned, and we hope that Luxturna is just the first of many successful gene therapies.”