Retinitis Pigmentosa Treatment in 2026: Approved, Under Review and in Trials

Last updated: November 5, 2026

One gene therapy is approved for retinitis pigmentosa (RP): LUXTURNA, and only for people with variants in both copies of the RPE65 gene¹. The FDA is reviewing a second, MOGENRY, for adults with RP and severe vision loss whatever gene is involved, and the company expects a decision in the first half of 2027^2,3. At least a dozen other treatments are in clinical trials, including two gene therapies for X-linked RP that have reported results from late-stage trials^5,8.

Each status below was checked on November 5, 2026 against company announcements, ClinicalTrials.gov and FDA records, and this page is updated after FDA decisions and major trial results. Results described as company-reported have not been published in a peer-reviewed journal. For symptoms, causes and diagnosis, see our retinitis pigmentosa guide.

Where each RP treatment stands

On this page

• Where each treatment stands
• Approved gene therapy
• Optogenetics and photoswitches
• Gene and RNA therapy trials
• Oral antioxidants: NAC and NPI-001
• Neuroprotection
• Cell therapy and stem cells
• What you can do now
• Where integrative care fits
• Frequently asked questions
• Sources

Approved gene therapy: LUXTURNA

LUXTURNA (voretigene neparvovec-rzyl) is the only gene therapy the FDA has approved for RP. Its label covers patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy who have viable retinal cells, as judged by the treating doctor, and it is not recommended for babies under 12 months^1,29. It is injected under the retina during eye surgery, one eye at a time¹. Because RP can be caused by many different genes, most people with RP are not eligible, so a genetic test is the first step³⁰.

Optogenetics and photoswitches

When most light-sensing cells have been lost, these treatments try to make surviving cells deeper in the retina respond to light. They are designed to work whatever gene caused the RP and are aimed at people with advanced vision loss. None restores normal sight.

MOGENRY (MCO-010): under FDA review

Nanoscope announced on September 9, 2026 that the FDA had accepted its application to approve MOGENRY for adults with RP and severe vision loss. It is a one-time injection into the eye². Japan’s regulator accepted an application on October 1, 2026 under priority review³. As of November 5, the company had not announced an FDA decision date; it expects possible approvals in both countries in the first half of 2027^3,4.

In the RESTORE trial, 27 adults whose treated eye saw worse than 20/1600 received one of two doses or a sham injection³¹. The company reports that the trial met its main goal at 52 weeks² and that improvement from baseline was still present about four years after treatment³². The most common side effects were inflammation inside the eye and raised eye pressure, mild to moderate and treated with eye drops³³. These results are company-reported.

RTx-015

Ray Therapeutics’ RTx-015 is an optogenetic gene therapy given by injection into the eye. Its phase 2/3 PERCEPT trial, registered in October 2026, is recruiting about 60 adults with advanced RP from any gene and compares two doses with a sham treatment¹³. The company reports vision improvements in an earlier small study but has not released detailed results³⁴. The FDA and the European Medicines Agency have given it designations meant to speed development^35,34.

GS030

GenSight’s GS030 combines an optogenetic gene therapy with goggles that project light onto the retina. In its first 10 patients, published in the New England Journal of Medicine on October 8, 2026, light sensitivity increased in 7. There was one severe side effect, a blockage of the retina’s main artery that cleared within minutes²². GS030 is not approved anywhere, and no next trial has been announced²¹.

KIO-301

KIO-301 is a photoswitch: a drug injected into the eye that makes surviving nerve cells in the retina respond to light. In a six-person first study, published in Nature Medicine in April 2026, it caused no serious side effects, and some participants showed changes in light perception and functional vision³⁶. A placebo-controlled phase 2 trial, ABACUS-2, is recruiting adults with very low vision, and the company expects results in the third quarter of 2027^19,20.

Gene and RNA therapy trials

Gene-specific treatments deliver a working copy of a gene, or change how a faulty gene is read, so they only help people with that gene. Your genetic test result tells you which of these trials you might qualify for.

X-linked RP: laru-zova and bota-vec

Beacon’s pivotal VISTA trial of laru-zova enrolled 85 males aged 12 to 48 with RPGR-related X-linked RP^37,5. After 12 months, 31% on the high dose and 24% on the low dose gained 15 or more letters on a dim-light eye chart, compared with none of the untreated group⁵. Beacon plans to begin a rolling FDA application by the end of 2026 and had not announced a submission as of November 5⁶. These results are company-reported.

MeiraGTx’s bota-vec targets the same gene. Its phase 3 LUMEOS trial did not meet its main goal, a maze-walking test. The company reports that several other measures improved; for example, 45% of treated patients gained more than 10 letters on a dim-light eye chart⁸. It says it still plans to apply for approval in the United States, Europe and Japan^8,9.

OCU400: a gene therapy for many forms of RP

Ocugen’s OCU400 delivers a “modifier” gene that helps regulate many other genes in the light-sensing cells, so it is being tested in RP caused by the RHO gene and by other genes¹⁰. The phase 3 liMeliGhT trial has enrolled 140 people aged 3 and older. Ocugen expects results in the first quarter of 2027 and plans an FDA application in the second quarter of 2027^11,12. A regulator in The Bahamas has given provisional approval for an OCU400 access program there, which is not an FDA approval¹², and the U.S. expanded access listing is no longer available³⁸.

Ultevursen: RNA therapy for USH2A

Ultevursen is an antisense RNA therapy injected into the eye every six months. It is for people with RP caused by variants in exon 13 of the USH2A gene, with or without Usher syndrome^17,18. Its phase 2b LUNA trial finished enrolling in July 2026, exceeding its target of 81 people, and main results are estimated for mid-2028^17,18.

Oral antioxidants: NAC and NPI-001

NAC Attack, a phase 3 trial led by Johns Hopkins with the National Eye Institute, is testing oral N-acetylcysteine (NAC) in 485 adults aged 18 to 65 with RP from any gene, each treated for 45 months. Main results are expected after May 2029¹⁴. NAC is sold over the counter, but the trial’s lead investigator advises against taking it outside the trial³⁹.

NPI-001 is a related antioxidant tablet being tested in up to 80 adults with RP caused by Usher syndrome. The first patient in its phase 3 trial was treated in October 2026, at sites in the United States and Australia, and results are not expected before 2029^16,15. An earlier 49-person trial did not find a significant difference in its main measure, retinal sensitivity; the company reported slower loss of light-sensing cells on retinal scans^40,41.

Neuroprotection

Neuroprotective treatments aim to keep the remaining cone cells alive rather than replace a gene. SparingVision’s SPVN06, a one-time gene therapy that delivers two protective proteins, finished dosing in a 33-person phase 1/2 trial in February 2026. The company reports a favorable safety profile so far and expects first data in 2027^24,23.

Cell therapy and stem cells

jCyte’s jCell injects retinal progenitor cells into the eye. An earlier 84-person phase 2b trial did not show a significant improvement in vision at 12 months⁴². A 31-person phase 2 trial has finished enrolling, and the company expects it to be completed in early 2027^25,26.

At Cedars-Sinai, neural progenitor cells placed under the retina survived for at least a year in 13 people with RP, and vision stayed stable, according to conference data presented in July 2026²⁸. This is an early safety trial²⁷.

A warning about clinics selling stem cell treatments

In 2017, doctors reported three women with macular degeneration who were left with severe vision loss after a clinic in Florida injected cells processed from their own fat into both eyes^43,44. Be cautious if a clinic asks you to pay for an experimental treatment, offers to treat both eyes on the same day, or points to a ClinicalTrials.gov listing as proof that a treatment works. A listing only means a study was registered⁴⁴.

The Foundation Fighting Blindness keeps a longer list of earlier-stage RP research⁴⁵.

What you can do now

• Get genetic testing. It shows whether LUXTURNA or a gene-specific trial could apply to you. In the United States, an eye care professional can order testing, with genetic counseling, at no cost through the Foundation Fighting Blindness⁴⁶.
• Look for trials that fit your gene and stage. Search ClinicalTrials.gov for “retinitis pigmentosa” plus your gene, ask your retina specialist, and check the Foundation Fighting Blindness website. Each trial has its own age and vision requirements. Tell any trial team about every supplement, herb and integrative treatment you use.
• Treat the complications. Cataract surgery can help some people with RP⁴⁷, and swelling at the center of the retina (cystoid macular edema), which affects about 1 in 4 people with RP, can sometimes be treated with an eye drop or an oral medicine⁴⁸.
• Start low vision rehabilitation early. Low vision aids and training programs can help⁴⁹. Our guide to low vision tools and rehabilitation explains what is available.
• Check supplements with your retina specialist. The evidence for vitamin A is uncertain and high doses carry real risks; our RP guide explains the details.
• Help others understand. Our RP vision simulator shows family, friends and teachers roughly how RP can change what you see.

Where integrative care fits

Netra Eye Institute offers integrative eye care, including acupuncture, nutrition and lifestyle guidance, Traditional Chinese Medicine and Ayurveda. For RP we offer it alongside care from a retina specialist, never instead of it. There is not yet reliable evidence that any of these approaches slows RP, and we do not promise that they will improve or preserve vision.

If you are in a clinical trial, or hoping to join one, the trial comes first. NAC Attack asks participants not to take supplements other than vitamin A¹⁴, and the NPI-001 trial excludes people taking NAC products¹⁵. We adjust or pause anything the trial team asks us to. Read more in holistic and natural treatment for retinitis pigmentosa.

Frequently asked questions

Is there a cure for retinitis pigmentosa?

No. LUXTURNA treats one genetic form, caused by variants in both copies of the RPE65 gene¹, and the FDA is reviewing MOGENRY for adults with RP and severe vision loss from any gene². Neither restores normal vision.

How soon will there be a cure for retinitis pigmentosa?

No one can give a date. The next milestones are FDA and Japanese decisions on MOGENRY, expected in the first half of 2027³, a planned FDA application for laru-zova in X-linked RP⁶, and OCU400 phase 3 results in early 2027¹¹. These treatments aim to slow vision loss or restore some function, not to cure RP.

What is the newest treatment for retinitis pigmentosa?

MOGENRY (MCO-010), a one-time eye injection for adults with RP and severe vision loss from any gene, is the newest RP treatment to reach FDA review². The newest late-stage trial is the phase 2/3 PERCEPT trial of RTx-015, registered in October 2026¹³.

Can gene therapy help everyone with RP?

Not yet. LUXTURNA works only for RPE65-related disease¹, and laru-zova and bota-vec target the RPGR gene in X-linked RP^5,8. Gene-agnostic treatments such as MOGENRY and OCU400 aim to work whatever the gene, but neither is approved yet^2,10.

How do I join a retinitis pigmentosa clinical trial?

Get genetic testing first, because gene therapy trials select people by gene; in the United States, an eye care professional can order it at no cost through the Foundation Fighting Blindness⁴⁶. Then search ClinicalTrials.gov, ask your retina specialist, and check each trial’s age and vision requirements.

Sources

1. U.S. Food and Drug Administration. LUXTURNA prescribing information. Revised May 2022.
2. Nanoscope Therapeutics. FDA acceptance of the Biologics License Application for MOGENRY. 9 September 2026.
3. Nanoscope Therapeutics. PMDA acceptance and priority review of the New Drug Application for MOGENRY in Japan. 1 October 2026.
4. Nanoscope Therapeutics. Regulatory affairs appointment; MOGENRY under FDA and PMDA review. 5 November 2026.
5. Beacon Therapeutics. Positive topline data from the pivotal VISTA trial of laru-zova. 21 September 2026.
6. Beacon Therapeutics. Data from the pivotal VISTA trial presented at AAO 2026. 12 October 2026.
7. ClinicalTrials.gov. NCT04671433: LUMEOS trial of bota-vec.
8. MeiraGTx. Acquisition of bota-vec for X-linked retinitis pigmentosa. 16 April 2026.
9. MeiraGTx. Second quarter 2026 financial and operational results. 13 August 2026.
10. ClinicalTrials.gov. NCT06388200: liMeliGhT trial of OCU400.
11. Ocugen. Business update with third quarter 2026 financial results. 4 November 2026.
12. Ocugen. Provisional approval and priority designation in The Bahamas for an OCU400 access program. 25 September 2026.
13. ClinicalTrials.gov. NCT07901751: PERCEPT trial of RTx-015. First posted 28 October 2026.
14. ClinicalTrials.gov. NCT05537220: NAC Attack.
15. ClinicalTrials.gov. NCT07710196: SLO-RP Preserve trial of NPI-001.
16. Nacuity Pharmaceuticals. First patient dosed in phase 3 trial of NPI-001 for RP associated with Usher syndrome. 28 October 2026.
17. Foundation Fighting Blindness. Sepul Bio completes clinical trial enrollment for its USH2A retinitis pigmentosa treatment. 24 July 2026.
18. ClinicalTrials.gov. NCT06627179: LUNA trial of ultevursen.
19. ClinicalTrials.gov. NCT06628947: ABACUS-2 trial of KIO-301.
20. Kiora Pharmaceuticals. Second quarter 2026 results and corporate update. 7 August 2026.
21. ClinicalTrials.gov. NCT03326336: PIONEER trial of GS030.
22. Sahel JA, et al.. Optogenetic therapy for restoring aspects of visual function. N Engl J Med. 8 October 2026.
23. ClinicalTrials.gov. NCT05748873: PRODYGY trial of SPVN06.
24. SparingVision. PRODYGY trial patient dosing completed with SPVN06. 9 February 2026.
25. jCyte. JC02-88 study.
26. ClinicalTrials.gov. NCT06912633: JC02-88 trial of jCell.
27. ClinicalTrials.gov. NCT04284293: neural progenitor cells (CNS10-NPC) for RP.
28. International Society for Stem Cell Research. One-year survival of stem cell-derived neural progenitor cells in patients with retinitis pigmentosa. 9 July 2026.
29. U.S. Food and Drug Administration. Approved cellular and gene therapy products. Content current as of 30 October 2026.
30. MedlinePlus Genetics. Retinitis pigmentosa.
31. ClinicalTrials.gov. NCT04945772: RESTORE trial of MCO-010.
32. Ophthalmology Times. AAO 2026: MCO-010 sustains vision gains in RP through 4 years. 11 October 2026.
33. Ophthalmology Times. FDA accepts BLA for MOGENRY in retinitis pigmentosa. 9 September 2026.
34. Ray Therapeutics. EMA PRIME designation for RTx-015. 29 April 2026.
35. Ray Therapeutics. FDA Regenerative Medicine Advanced Therapy designation for RTx-015. 1 April 2026.
36. Casson RJ, et al.. Intravitreal photoswitch therapy in advanced retinitis pigmentosa: a phase 1 open-label trial. Nat Med. 14 April 2026.
37. ClinicalTrials.gov. NCT04850118: VISTA trial of laru-zova.
38. ClinicalTrials.gov. NCT06574997: OCU400 expanded access program. Updated 2 October 2026.
39. Foundation Fighting Blindness. Phase 3 clinical trial of NAC launched for RP patients. 12 April 2024.
40. ClinicalTrials.gov. NCT04355689: phase 1/2 trial of NPI-001.
41. Nacuity Pharmaceuticals. Data from the clinical trial of NPI-001 in RP associated with Usher syndrome. 11 September 2025.
42. ClinicalTrials.gov. NCT03073733: phase 2b trial of jCell, results. Results posted 21 January 2022.
43. Kuriyan AE, et al.. Vision loss after intravitreal injection of autologous "stem cells" for AMD. N Engl J Med. 2017.
44. Foundation Fighting Blindness. Unregulated stem cell therapy causes severe vision loss for three Florida women.
45. Foundation Fighting Blindness. Retinitis pigmentosa research advances. 15 September 2026.
46. Foundation Fighting Blindness. No-cost genetic testing.
47. Hamel C.. Retinitis pigmentosa. Orphanet J Rare Dis. 2006.
48. Huckfeldt RM, et al.. Management of cystoid macular edema in retinitis pigmentosa. Semin Ophthalmol. 2017.
49. National Eye Institute. Retinitis pigmentosa. Updated 6 August 2025.