Luxturna (voretigene neparvovec) is a gene therapy for inherited retinal dystrophy caused by mutations in both copies of the RPE65 gene — a condition that often presents as a form of Leber congenital amaurosis or early-onset retinitis pigmentosa. It works by delivering a working copy of the RPE65 gene directly into the cells of the retina, using a harmless modified virus as a carrier, so the eye can rebuild part of the chemical cycle that turns light into vision. It is a one-time treatment per eye, given by a delicate subretinal injection, and in the UK it is provided through an NHS-commissioned specialist pathway rather than as a self-pay operation. Our role is to provide expert assessment, arrange or interpret genetic testing, and guide suitable patients into the right specialist centre.
What is Luxturna and how does it work?
The retina contains light-sensing cells that rely on a continuous chemical cycle — the visual cycle — to keep working. The RPE65 gene makes an enzyme essential to that cycle. When both inherited copies of RPE65 are faulty, the cycle breaks down, the light-sensing cells gradually stop working and degenerate, and vision is lost — often starting with poor night vision and shrinking peripheral vision from early childhood.
Luxturna uses an adeno-associated virus (AAV) — a safe, non-replicating carrier — to deliver a healthy copy of the RPE65 gene into the retinal pigment epithelium cells. Once inside, the cells use the new gene to make the missing enzyme, restoring part of the visual cycle. The aim is to improve functional vision, particularly the ability to navigate in low light, and to slow the loss of remaining vision. It does not regrow cells that have already died, which is why timing and having enough viable retinal cells matters.
Who qualifies for Luxturna?
Luxturna is highly targeted. It is only suitable for a specific genetic diagnosis, confirmed by testing, and where there are enough surviving retinal cells for the therapy to act on.
Because inherited retinal disease is complex, assessment involves detailed retinal imaging, functional vision testing and genetic counselling. Other advanced retinal therapies under research or in use for different conditions — such as subretinal cell therapy for dry AMD and anti-VEGF injections for wet AMD — are not interchangeable with Luxturna, which is specific to RPE65 disease. Explore the full range on our treatments hub.
Has RPE65 inherited retinal disease been mentioned in your family? A specialist assessment with retinal imaging and genetic testing confirms whether Luxturna gene therapy could help.
Book a retinal-genetics assessmentHow Luxturna is given
Luxturna is delivered into the retina during a short operation called a vitrectomy with subretinal injection, performed by a vitreoretinal surgeon at a specialist centre, usually under general anaesthetic. Each eye is treated on a separate occasion, typically a number of days apart.
- A vitrectomy removes the gel inside the eye to give the surgeon access to the retina.
- A very fine cannula is used to make a tiny subretinal injection, lifting a small area of retina to place the gene-therapy fluid directly beneath it.
- The AAV carrier delivers the healthy RPE65 gene into the retinal pigment epithelium cells.
- The eye is closed and you recover from the anaesthetic. Short-course steroids are given around the treatment to reduce inflammation.
- The second eye is treated on a separate date once the first is settling.
Recovery & what to expect
Recovery is similar to other vitrectomy retinal surgery, with close monitoring because this is a specialised therapy. Visual changes — particularly improved navigation in dim light — may develop over the weeks and months that follow.
Day of treatment
Day-case or short-stay surgery under general anaesthetic. The eye is padded and a course of steroids is started to calm inflammation.
Days 1–7
Early review checks healing. Mild redness and a gritty feeling settle. Drops are used and strenuous activity is avoided.
Weeks 2–6
The second eye is treated. Some patients begin to notice easier movement in low light as the therapy takes effect.
Months 1–6
Functional vision is reassessed. Improvements in light sensitivity and navigation are measured with specialist testing.
Long term
Lifelong follow-up monitors the retina. Luxturna is a one-time treatment that aims to preserve and improve the vision you have.
Funding & cost in the UK
Luxturna is one of the most expensive medicines ever brought to market, with a list price of around £306,000 per eye (about £613,000 for both eyes). In the UK it is not sold as a private self-pay procedure. Instead, for patients with confirmed biallelic RPE65 inherited retinal dystrophy and sufficient viable retinal cells, it is funded by the NHS through specialised commissioning and delivered at a small number of designated centres, under a confidential pricing agreement with the manufacturer.
- NHS funding: available for eligible patients with a confirmed RPE65 diagnosis, following specialist multidisciplinary review.
- Our role: private assessment, retinal imaging, genetic testing and counselling, and support with referral into the correct specialist pathway.
- Genetic testing first: eligibility cannot be confirmed without genetic results showing faults in both copies of RPE65.
If you would like clarity on whether you or a family member might qualify, the first step is a specialist assessment. You can also start with a free online consultation.