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LUXTURNA is a prescription gene therapy product used for the treatment of patients with inherited retinal disease due to mutations in both copies of the RPE65 gene, which can only be confirmed through genetic testing. You must also have enough remaining cells in your retina (the thin layer of tissue in the back of your eyes) as determined by your healthcare professional.

What are the most serious and most common side effects of LUXTURNA?

Please see Important Safety Information for LUXTURNA.

How does LUXTURNA gene therapy work?

Gene therapy is a method for treating a genetic disease that delivers a new or functional gene into a cell.

RPE65 gene mutations lead to reduced or absent levels of the RPE65 protein. This blocks the visual cycle that converts light into electrical signals and causes vision loss.

LUXTURNA provides a functional copy of the RPE65 gene to act in place of a mutated RPE65 gene. The functioning gene has the potential to make the visual cycle work properly again.

What are the first steps toward treatment?

Mutations in both copies of the RPE65 gene, the only mutations for which LUXTURNA is indicated, must be confirmed with a genetic test ordered by your healthcare professional. In order to receive treatment, it must also be confirmed that you have enough remaining cells in your retina.

To find out more information about genetic testing or what the next steps are after a confirmed diagnosis, click below.


Where does treatment take place?

LUXTURNA is only administered at Ocular Gene Therapy Treatment Centers. Once your healthcare professional determines that treatment with LUXTURNA is right for you, Spark Therapeutics Generation Patient ServicesSM is available to connect you with a treatment center.

Interested in more information?

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