Gene therapies in development for monogenic, complex retinal diseases

WAIKOLOA, Hawaii — Gene therapy for retinal disease is in various stages of development, with more than a dozen retinal clinical trials underway, Elias Reichel, MD, told colleagues at Retina 2019.
For the most part, these trials involve gene replacement using adeno-associated virus (AAV) to deliver the therapy, which thus far has shown persistent expression in humans and no significant safety concerns.
“[Leber’s congenital amaurosis] is the first condition that has been successfully treated with gene therapy,” Reichel said. Luxturna (voretigene neparvovec-rzl, Spark

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