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Adley and Oaklyn, patients who received LUXTURNA
Adley and Oaklyn,
patients who received LUXTURNA


LUXTURNA is the first prescription gene therapy product to help improve functional vision in patients with an inherited retinal disease (IRD) due to a mutation in their RPE65 gene. It is a one-time gene therapy that has the potential to restore the visual cycle—the process that allows you to see.

Who LUXTURNA is for

LUXTURNA is used for the treatment of patients who have both of the following:

  • An inherited retinal disease (IRD) due to mutations in both copies of the RPE65 gene
  • Enough remaining cells in their retina as determined by a healthcare professional

RPE65 is a gene that is necessary to make a protein needed for vision

RPE65 gene mutations cause vision to deteriorate and may progress to complete blindness.

If you have mutations in both copies of the RPE65 gene, you may experience:

  • Night blindness (nyctalopia)
  • Loss of light sensitivity
  • Loss of sharpness or clarity of vision
  • Impaired dark adaptation
  • Repetitive uncontrolled movements of the eye (nystagmus)

IRDs and RPE65

A gene is the basic unit of hereditary information that is passed on to a child from their parents. Typically, every person has two copies of a gene, one from each parent. In the case of IRDs due to RPE65 gene mutations, both of the genes passed onto the child are mutated.

There are many IRDs

For example, Leber congenital amaurosis and retinitis pigmentosa are two types of IRDs. These are clinical diagnoses that are not based on mutations in specific genes, such as mutations in both copies of the RPE65 gene. RPE65 is one out of more than 270 genes that may be responsible for an IRD. Confirming your specific gene mutation or mutations with a genetic test is the first step to learning if LUXTURNA may be right for you.

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Gene therapy overview

Gene therapy has the potential to enable treatment of complex diseases. Several innovators behind gene therapy research are targeting IRDs, making gene therapy a potential turning point for patients and caregivers in the IRD community.


LUXTURNA (voretigene neparvovec-rzyl) 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.

Important Safety Information for LUXTURNA

The following serious side effects may occur during or after the administration of LUXTURNA:

  • Eye infections, including a serious infection inside of the eye called endophthalmitis, that may lead to blindness.
  • Permanent decline in visual acuity, or the sharpness of central vision.
  • Changes in the retina (the thin layer of tissue in the back of the eye) that can lead to vision loss including:
    • development of a hole, thinning, or loss of function of the retina, separation of the layers in the center of the retina, decreased thickness of the retina and the choroid (the layer of blood vessels that lines the back of the eye), or bleeding in the retina.
    • breaks in or wrinkling on the surface of the retina or detachment of the retina.
  • Increased pressure inside of the eye. You should follow-up with your healthcare professional as instructed to detect and treat any increased pressure in the eye as this may cause blindness.
  • Expansion of the air bubble formed in the eye after administration of LUXTURNA. You should avoid air travel, travel to high elevations, or scuba diving until your healthcare professional has told you that the air bubble formed in the eye following administration of LUXTURNA has disappeared. Engaging in these activities while the air bubble is present can cause permanent vision loss.
  • Formation or worsening of cataract (clouding of the lens inside of the eye).

Tell your healthcare professional right away if you have any of the following symptoms of these serious side effects:

  • Seeing floaters (specks that float about in your field of vision)
  • Pain in the eye
  • Any change in vision including decreased vision or blurred vision
  • Seeing flashes of light

The following are the most common side effects that may occur with LUXTURNA:

  • Redness of the eye
  • Cataract (clouding of the lens inside of the eye)
  • Increased pressure inside of the eye
  • Breaks in the retina
  • Dellen (thinning of the clear layer in the front of the eye)
  • Development of a hole in the center of the retina
  • Subretinal deposits (deposits under the retina)
  • Eye swelling, irritation or pain
  • Wrinkling on the surface of the center of the retina

Treatment with LUXTURNA is not recommended for patients younger than 12 months of age because the retina is still growing, which may affect how LUXTURNA works.

Because small quantities of LUXTURNA may be in your tears for a short period of time, for the first 7 days after administration of LUXTURNA, place any waste material from dressings, tears and nasal secretions in sealed bags prior to disposal.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit, or call 1-800-FDA-1088. You may also report side effects to Spark Therapeutics at 1-855-SPARKTX (1-855-772-7589).

This information does not take the place of talking to your healthcare professional about your medical condition or treatment. If you have questions about LUXTURNA after reading this information, ask your healthcare professional.

Please see the US Full Prescribing Information for LUXTURNA.