Systematic review reports mixed visual gains, frequent atrophy after Luxturna gene therapy

A systematic review of 28 studies (376 individuals, 673 treated eyes) examined outcomes of voretigene neparvovec-rzyl for biallelic RPE65-associated inherited retinal disorders. The review found consistent improvements in light sensitivity and visual field, particularly in pediatric patients treated before advanced degeneration, while best-corrected visual acuity gains were variable and generally modest. Pooled analysis of three studies estimated that 75% of treated eyes developed new or progressive perifoveal atrophy, with chorioretinal atrophy and intraocular inflammation reported as the most frequent adverse events, though severe complications such as retinal tears and detachment were infrequent.

Most relevant for: Patients with biallelic RPE65-associated inherited retinal disorders being considered for or monitored after gene therapy, particularly pediatric cases treated before advanced degeneration.

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