Focus of the Report: This report evaluates the use of voretigene neparvovec-rzyl (Luxturna) for the treatment of vision loss due to inherited retinal dystrophies (IRDs) from confirmed biallelic RPE65 mutations.
Technology Description: Voretigene neparvovec-rzyl is an adeno-associated, viral vector–based ocular gene therapy that delivers a functional copy of the RPE65 gene directly to the retinal pigment epithelial cells of the eye. The healthy RPE65 gene provides a sustained therapeutic benefit via continual expression of functional RPE65 protein, which converts light into electrical signals in the retina, restoring vision loss.
Controversy: Patients with RPE65-mediated IRD have few treatment options and are likely to develop visual impairment that severely impacts vision-dependent activities of daily living. Voretigene neparvovec-rzyl is a novel gene therapy with the goal of correcting the underlying genetic defect and restoring visual function. This therapy is associated with a notably high cost, which may present a barrier for treatment.
Is voretigene neparvovec-rzyl effective in treating patients with IRDs involving vision loss caused by RPE65 gene mutations?
Is voretigene neparvovec-rzyl safe?
Have specific patient selection criteria been identified for the use of voretigene neparvovec-rzyl?
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