Luxturna
Voretigene neparvovec-rzyl, Spark Therapeutics, for confirmed biallelic RPE65 mutation-associated retinal dystrophy.
FDASpecialty Rx · Gene and Cell Therapy
Ocular Gene and Cell Therapy Luxturna and Encelto.
Two FDA-approved therapies deliver genetic or cell-based treatment inside the eye, given by retina surgeons in specialized centers.
2017Luxturna approved, December 19
2025Encelto approved for MacTel type 2
52% vs 10%Luxturna patients improving 2 or more MLMT levels
1 implantper eye for Encelto
Two approaches
Replacing a gene, or delivering a protein
Comprehensive ophthalmologists and optometrists are often the first to suspect these conditions and to coordinate genetic testing and referral. Luxturna delivers a working copy of the RPE65 gene to retinal cells. Encelto is an encapsulated cell implant that releases ciliary neurotrophic factor inside the eye.
Encelto
Revakinagene taroretcel-lwey, Neurotech, for adults with idiopathic macular telangiectasia type 2.
LabelGenetic testing first
Luxturna requires a confirmed biallelic RPE65 mutation, so testing comes before referral for treatment.
FDASpecialist delivery
Luxturna is a subretinal injection after vitrectomy; Encelto is surgically implanted in the vitreous.
LabelDosing and safety
Both are one-time procedures per eye, with surgical follow-up.
- Confirm the diagnosis
- Refer to a treating center
- Co-manage follow-up
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Luxturna dose
1.5 x 10^11 vector genomes in 0.3 mL by subretinal injection; eyes treated at least 6 days apart; not for infants under 12 months.
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Luxturna reactions
Conjunctival hyperemia 22%, cataract 20% and increased intraocular pressure 15% were among the most common.
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Encelto implant
One intravitreal implant per eye. Delayed dark adaptation was reported in 23%.
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Encelto results
In Study 1, ellipsoid-zone loss was 0.075 mm² with Encelto versus 0.166 mm² with sham.
Care paths
Every path for inherited retinal disease and MacTel
Not every patient is a candidate for gene or cell therapy. Vision Rescue does not rank these paths; the treating doctor and the patient choose.
Monitoring
Regular exams and multimodal imaging to follow the condition.
Optometrists and ophthalmologistsGenetic testing
Testing and counseling to confirm the diagnosis and eligibility.
Eye care providers, retina specialists or genetic counselorsGene therapy
A one-time subretinal injection for confirmed biallelic RPE65 retinal dystrophy.
Retina surgeons at treating centersCell therapy implant
An intravitreal implant for adults with MacTel type 2.
Retina surgeons at treating centersLow vision support
Low vision care for daily tasks at any stage.
Optometrists, ophthalmologists and low vision servicesThe referring doctor's role
Recognize, test, refer
These are rare conditions, so a structured path matters.
Recognize
Night blindness and reduced fields in a child or young adult, or characteristic macular changes in middle age.
Test
Order or refer for genetic testing and multimodal imaging to confirm the diagnosis.
Refer
Coordinate with an inherited retinal disease or retina specialist, and support low vision needs.
Product details
Luxturna
| Generic name | Voretigene neparvovec-rzyl |
|---|---|
| Company | Spark Therapeutics |
| Approved | December 19, 2017 |
| Indication | Confirmed biallelic RPE65 mutation-associated retinal dystrophy |
| Primary outcome | MLMT change of 2 or more: 52% vs 10% |
Encelto
| Generic name | Revakinagene taroretcel-lwey |
|---|---|
| Company | Neurotech |
| Approved | 2025 |
| Indication | Adults with idiopathic macular telangiectasia type 2 |
| Common reaction | Delayed dark adaptation 23% |
Questions about ocular gene and cell therapy
Can optometrists or general ophthalmologists give these treatments?
No. Both are surgical procedures performed by retina specialists at treating centers.
Who qualifies for Luxturna?
Patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy and sufficient viable retinal cells, as determined by the treating physician.
What does Encelto treat?
Idiopathic macular telangiectasia type 2 in adults.
How is Luxturna given?
As a subretinal injection, with each eye treated on a separate day at least 6 days apart.
What are common side effects of Luxturna?
Conjunctival hyperemia, cataract and increased eye pressure were among the most common.
Where can patients get genetic testing?
Through their eye care provider, a retina specialist or a genetic counselor; programs vary by condition.
References
Be the first to recognize it
Know the signs of inherited retinal disease and MacTel, and the path to genetic testing and referral, in optometry and ophthalmology practices alike.
Stay current: Latest eye care industry news
Luxturna and Encelto are trademarks of their owners. Written for licensed eye care professionals. These therapies are administered only by qualified specialists. Review the full Prescribing Information.