02/17/26
In this article, Wills Eye Retina Chief Carl D. Regillo, MD and colleagues explore the shift to gene therapy for the treatment of macular disease. As the authors explain, "Gene therapy entered the ophthalmology space in 2017 with the approval of voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics) for Leber congenital amaurosis, a rare inherited retinal disease.2 This landmark approval opened the door for the extension of this concept to common, noninherited retinal diseases such as nAMD, diabetic retinopathy (DR), and diabetic macular edema (DME). Gene therapies for retinal conditions use viral vectors to transduce retinal cells with instructions to manufacture specific proteins." The advantage of gene therapy is that it promises to reduce or eliminate the onerous cycle of repeated intravitreal (IVT) injections.