Historically, patients with rare inherited retinal diseases (IRDs) were told there was no treatment to stop them from losing their vision. But this is changing. In 2017, the world’s first ocular gene therapy treatment, Luxturna (voretigene neparvovec-rzyl), was approved by the US Food and Drug Administration for IRDs caused by mutations in the RPE65 gene, causing Leber congenital amaurosis. Gene therapy treatments for at least 20 other IRDs are being developed1 followed closely by stem cell therapies, RNA therapies and optogenetic treatments.
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