The Food and Drug Administration's approval of voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics, Philadelphia, PA) on December 19, 2017, for the treatment of patients with biallelic mutations in RPE65 was a landmark development in the field of inherited retinal diseases (IRD).

We have witnessed how this development has changed the attitude of many patients with these rare diseases, even if the treatment is not indicated for their disease.

A renewed sense of hope exists that, after decades of there being no effective treatments, gene therapy may help us turn the corner and usher in a new era of treatments for IRDs.