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HomeminewsRetinal Disease Gene Therapy

Retinal Disease Gene Therapy

Novartis has announced a licensing agreement with Spark Therapeutics covering development, registration and commercialisation rights to voretigene neparvovec in markets outside the US.

Voretigene neparvovec, (registered as Luxturna in the USA) received FDA approval on 19 December 2017 as a onetime gene therapy to restore functional vision in children and adult patients with biallelic mutations of the RPE65 (retinal pigment epithelial 65 kDa protein) gene.1 The condition causes Leber’s congenital amaurosis type 2 and other forms of severe retinal degenerative disease. Only a few thousand people worldwide are affected by the condition.

“No otherwise healthy child should have to go blind due to this devastating disease. Gene therapy is a promising new avenue to potentially address this unmet need,” said Shreeram Aradhye, Global Head of Medical Affairs and Chief Medical Officer, Novartis Pharmaceuticals.

The RPE65 protein is produced in the retinal pigment epithelium and supports and nourishes the retina. Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 isomerohydrolase activity, blocking the visual cycle and resulting in progressive vision loss and ultimately, blindness.1

No otherwise healthy child should have to go blind due to this devastating disease. Gene therapy is a promising new avenue to potentially address this unmet need

When approved in December 2017, Luxturna became the first directly administered gene therapy to be approved in the United States to target a disease caused by mutations in a specific gene.

References 1. US Food and Drug Administration. FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss. December 19, 2017. Last accessed: January 22, 2018.

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