Could luxturna be right for you?

luxturna is for individuals with an inherited retinal disease caused by mutations in both copies of the RPE65 gene and who have enough remaining cells in the retina

A gene is the basic unit of hereditary information that is passed on to a child from their parents. Typically, every person has 2 copies of a gene, one from each parent. In the case of RPE65 gene mutations, both of the genes passed onto the child were mutated.

There are many inherited retinal diseases

For example, Leber congenital amaurosis and retinitis pigmentosa are two inherited retinal diseases. These are clinical diagnoses that are not based on mutations in specific genes, such as mutations in both copies of the RPE65 gene. To get a genetic diagnosis, you will need a genetic test.

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)

Our Simple Screener can help you find out if you are a possible candidate for an RPE65 genetic test, which can only be ordered by your healthcare professional.

TAKE THE SIMPLE SCREENER

RPE65 is 1 out of more than 260 genes that may be responsible for an inherited retinal disease. Confirming a gene mutation with a genetic test is the first step to learning if LUXTURNA may be right for you

How does luxturna work?

luxturna is a one-time gene therapy for each eye

Gene therapy is a method for treating a genetic disease. One approach to gene therapy is delivering a new or functional gene into a cell.

luxturna works to restore the visual cycle

The visual cycle is a process that allows you to see. LUXTURNA provides a working RPE65 gene to act in place of a mutated RPE65 gene. This working gene has the potential to make the visual cycle work properly again.

With the gene delivered by LUXTURNA, cells in the retina of the eye can produce the RPE65 protein

How is luxturna administered?

LUXTURNA is given by a healthcare professional as a surgical injection beneath the retina of each eye

One eye is treated at a time. After the first eye is treated, the second eye will be treated at least 6 days later.

Administration is performed at specific Ocular Gene Therapy Treatment Centers

Each treatment center is staffed with:

  • Healthcare professionals, including retinal specialists
  • Nurses
  • Genetic counselors

The staff at the treatment centers has experience treating patients with inherited retinal diseases and has been specially trained on administration of LUXTURNA.

How was the effect of LUXTURNA measured?

You may be used to tests such as:

  • Visual Acuity
  • Visual Field
  • Light Sensitivity

A new test was created to measure functional vision, which is how vision loss affects the ability to perform daily life activities. This test is called the Multi-Luminance Mobility Test.

Click to expand and learn more about how this study was designed

The clinical trial included 31 participants, 4-44 years old. Participants were eligible for the clinical trial if they:

  • Were 3 years of age or older
  • Had a genetic diagnosis of mutations in both copies of the RPE65 gene
  • Had enough remaining cells in the retina
  • Had visual acuity of 20/60 or worse in both eyes and/or visual field less than 20 degrees
  • Were able to perform the Multi-Luminance Mobility Test within the light level range evaluated, but unable to pass at the darkest light level

Two groups of participants were treated with LUXTURNA, each at a different time. The first group was treated before the second group in order to compare results between treated and untreated participants. The second group was then treated 1 year after the first group.

  • LUXTURNA was administered to the participants' first eye, and 6-18 days later, LUXTURNA was administered to their second eye
  • Participants followed a prescribed course of medication to reduce risks related to an immune response
  • Each participant was evaluated 1 year after treatment to measure the effect of LUXTURNA

LUXTURNA has 9 years of safety experience and has been studied in a range of patients between 4 and 44 years old

What were the results?

LUXTURNA improved functional vision. This increased participants' ability to perform activities of daily living

Two groups of participants were treated with LUXTURNA, each at a different time. The first group was treated before the second group in order to compare results between treated and untreated participants. The second group was then treated 1 year after the first group.

One year after treatment, participants in the first group were able to navigate through the Multi-Luminance Mobility Test course using both eyes at 2 light levels darker than before treatment.

55% of participants in both groups showed an improvement of at least 2 light levels darker after treatment

A majority of participants were able to complete the course at the darkest light level after treatment

65% of participants in the first group and 89% of participants in the second group were able to navigate through the Multi-Luminance Mobility Test course at the darkest light level, which was equal to a moonless summer night.

First Group

%

Second Group

%

See the Multi-Luminance Mobility Test in action

Watch how LUXTURNA improved functional vision in the clinical trial

Before treatment

at the darkest light level (fail)

1 year after treatment

at the darkest light level (pass)

The camera used automatically adjusts the level and temperature of light that it captures. Because of this feature, there may be slight variations in shade when filming at low light levels. Both videos were filmed in low-light environments.

What are the most serious and most common side effects with 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, 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).

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

  • Redness of the eye
  • Cataract (clouding of the lens inside 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

You should discuss any side effects with your healthcare professional.

Photographs are not of actual patients.

Rather than clinging to me when walking in dark areas, he now runs ahead on his own.

—Mother of Chris, Age 7

For illustrative purposes based on actual patient experience. Results will vary.