Voretigene Neparvovec
Luxturna is a gene therapy designed to treat patients with inherited retinal dystrophy caused by mutations in the RPE65 gene. This one-time treatment is administered via subretinal injection and works by delivering a functional copy of the RPE65 gene directly to retinal cells, allowing them to produce the normal protein needed for vision.
Luxturna is a gene therapy eye injection for treating inherited retinal disease due to RPE65 mutations.
Key facts at a glance
- RxNorm
- 1995543
- Generic
- Luxturna
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Luxturna is indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. This includes conditions such as Leber congenital amaurosis (LCA) and retinitis pigmentosa caused by RPE65 mutations, in patients who have sufficient remaining retinal cells.
Luxturna is an adeno-associated virus (AAV) vector-based gene therapy that delivers a functional copy of the human RPE65 gene to retinal cells through a subretinal injection. The viral vector allows the gene to enter cells, where it enables them to produce a functional RPE65 enzyme necessary for the visual cycle, thereby restoring vision function.
Luxturna is administered as a one-time gene therapy per eye by a surgeon experienced in ocular procedures. It is not a cure but may significantly improve functional vision. Potential risks include surgical complications, eye inflammation, and retinal detachment. Patients should discuss benefits and risks with their ophthalmologist and consider long-term follow-up care.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy