Health Canada approves Novartis' vision loss gene therapy

15 October 2020

Health Canada has approved Luxturna (voretigene neparvovec), a one-time gene therapy for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

The Novartis (NOVN: VX) drug is designed to provide functioning copies of the RPE65 gene to act in place of mutated RPE65 genes. These functioning genes produce the RPE65 protein to help improve vision and prevent progression towards total blindness.

Elise Héon, an ophthalmologist in the Department of Ophthalmology and Vision Sciences and the Director of the Eye Genetics Program at The Hospital for Sick Children in Toronto, said: “The effects of RPE65-mediated inherited retinal diseases can be life-changing. Previously, there was no treatment available and the progression towards complete blindness was inevitable.

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