What are retinal dystrophies?

Hereditary retinal dystrophies are a group of various genetic, chronic and disabling disorders of visual function, generally due to dysfunction of the photoreceptors and / or the retinal pigment epithelium (RPE) and / or the choriocapillaris. Hereditary retinal dystrophies can manifest alone or as part of a syndrome. They usually progress over the years and produce a partial reduction or a more global loss of vision.

The RPE65 gene is responsible for the production of an enzyme necessary for the normal functioning of retinal cells (all-trans-retinol isomerase).

What is Luxturna?

Luxturna (voretigene neparvovec) is the first gene therapy to treat a hereditary disease of the retina, indicated for children and adults with vision loss due to mutations in both copies of the RPE65 gene, as long as their retina has a sufficient number of cells viable.

Luxturna is composed of vital vectors that possess normal copies of the damaged gene (RPE65). Its administration is by intravitreal injection (inside the eyeball). Once Luxturna has been injected, the virus carries the normal gene to the cells of the retina and enables them to produce the missing enzyme, which improves the functionality of the cells of the retina and slows the progression of the disease.

Is there clinical experience with Luxturna?

Luxturna has been successfully applied to patients with retinal dystrophies due to RPE65 gene mutations. Treatment with Luxturna significantly improved the patients’ vision, which resulted in a significant improvement in their mobility abilities.

Are there any risks associated with Luxturna?

All treatments have potential adverse effects and Luxturna is no exception. Luxturna should not be administered to patients who:

• Are allergic to voretigén neparvovec or any of the other ingredients of this medicine.
• Have an eye infection at the time of injection.
• Presents, at the time of injection, an ocular inflammatory condition. inflammation in the eye.

The most common side effects associated with the use of Luxturna are conjunctival hyperaemia (redness of the conjunctiva), cataracts (clouding of the lens) and increased pressure inside the eye.

Luxturna must be administered by ophthalmologists who have training in its use and sufficient experience in the administration of intravitreal treatments.