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Retina Genetics
Retinitis pigmentosa · Treatment guide

RP treatments that work for any gene.

More than 80 genes can cause retinitis pigmentosa, and most do not have their own gene therapy yet. This guide covers treatments that are being tested no matter which gene you have.

See all RP trials

Which treatments might fit me?

Every study has its own rules. These are general patterns, not a substitute for talking with your retina specialist.

Get a genetic test.

Even for treatments that work for any gene, most studies want a confirmed diagnosis, and your gene may make you eligible for a gene-specific study too. See our genetic testing guide.

Still have useful vision.

Treatments that protect the cells you still have aim to slow vision loss. They work best before too many cells are lost.

Very low vision.

Treatments that teach surviving cells to sense light and some cell therapies are for people who have already lost most of their sight.

At a glance.

Tap a name to read more. Recruiting status is updated every day from ClinicalTrials.gov.

TreatmentTypeHow it is givenStageRecruiting?
N-acetylcysteine (NAC)Protects cellsPillPhase 3Not now
OCU400Protects cellsEye surgeryPhase 3Not now
MCO-010Restores light sensingEye injection (in clinic)Under FDA reviewNot now
GS030Restores light sensingEye injection (in clinic)Phase 1/2Not now
RTx-015Restores light sensingEye injection (in clinic)Phase 1Not now
BS01Restores light sensingEye injection (in clinic)Phase 1/2Recruiting now
KIO-301Restores light sensingEye injection (in clinic)Phase 2Recruiting now
jCellCell therapyEye injection (in clinic)Phase 2Not now
OpCT-001Cell therapyEye surgeryPhase 1/2Recruiting now
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Treatments that try to protect the cells you still have.

These aim to slow vision loss. They are meant for people who still have useful vision, and they do not depend on which gene caused the RP.

N-acetylcysteine (NAC)

Johns Hopkins University, funded by the National Eye Institute

  • Phase 3

An antioxidant that is already sold as a supplement. The idea is that after rods die, cones are slowly damaged by oxidative stress, and NAC may reduce that damage.

How it is given
Pills taken by mouth, twice a day
Results so far
A small earlier study suggested a possible benefit, but it had no comparison group. NAC Attack is designed to find out whether it really helps.
Who could join
NAC Attack enrolled adults 18 to 65 with RP. Please do not start NAC on your own: ask your retina specialist first, because the dose used in the study is high.
Latest news
The NAC Attack study has finished enrolling about 480 people. Each person takes NAC or a placebo for about 4 years, so final results are not expected for several years.

Studies

Sources: NAC Attack study protocol (medRxiv, 2025)

OCU400 NR2E3 modifier gene therapy

Ocugen

  • Phase 3

A gene therapy that adds a healthy copy of NR2E3, a gene that acts like a master switch for many other genes in the retina. The goal is to help the retina stay healthier no matter which gene caused the RP.

How it is given
One-time eye surgery to place the treatment under the retina
Results so far
Earlier small studies were company-reported. The phase 3 results will be the first large controlled test.
Who could join
liMeliGhT included people with RP from RHO gene changes and from other genes. It is no longer enrolling.
Latest news
Enrollment in the liMeliGhT study (140 people) finished in March 2026. Ocugen expects results in early 2027 and plans to apply for FDA approval after that.

Studies

Sources: Ocugen: liMeliGhT enrollment complete

2 of 3

Treatments that teach surviving cells to sense light.

Most of these are called optogenetics. In advanced RP the rods and cones are gone, but other retinal cells (bipolar cells and ganglion cells) often survive. These treatments give those cells a light-sensing protein so they can take over some of the job. They are mostly for people with very low vision.

MCO-010 sonpiretigene isteparvovec, MOGENRY

Nanoscope Therapeutics

  • Under FDA review

Adds a light-sensing protein to bipolar cells. It is designed to work in normal room light, without special goggles.

How it is given
One injection into the eye, done in the clinic (no surgery)
Results so far
In the RESTORE study (27 adults with advanced RP), people given MCO-010 improved more on the eye chart than people given a sham (pretend) injection. The company reports the gains lasted about 3 years in follow-up.
Who could join
RESTORE enrolled adults with severe vision loss from RP. It has ended. Ask your retina specialist about access if the treatment is approved.
Latest news
The FDA accepted Nanoscope's application in 2026. The company says a decision is expected in the first half of 2027. If approved, it would be the first optogenetic treatment.

Studies

Sources: Retinal Physician: FDA accepts MCO-010 application; Ophthalmology Times: ARVO 2026 long-term data

GS030

GenSight Biologics

  • Phase 1/2

Adds a light-sensing protein (ChrimsonR) to ganglion cells. The goggles turn the scene into bright amber light that the treated cells can respond to.

How it is given
One injection into the eye, plus special light-projecting goggles
Results so far
In 10 people who were legally blind, 7 became more sensitive to light, and some could find or count objects while wearing the goggles. No serious treatment-related side effects were reported. The study was designed to test safety, not to prove benefit.
Who could join
PIONEER is no longer enrolling.
Latest news
Results from the PIONEER study were published in the New England Journal of Medicine in October 2026.

Studies

Sources: Retinal Physician: GS030 results; STAT News

RTx-015

Ray Therapeutics

  • Phase 1

Adds a light-sensing protein to ganglion cells, designed to work in everyday light levels.

How it is given
One injection into the eye, done in the clinic (no surgery)
Results so far
No peer-reviewed results yet.
Who could join
The ENVISION study is for adults with advanced RP or choroideremia.
Latest news
The FDA gave RTx-015 a special designation for promising regenerative treatments (RMAT) in April 2026, which allows closer work with the FDA.

Studies

Sources: Ophthalmology Times: RMAT designation

BS01

Bionic Sight

  • Phase 1/2
  • Recruiting now

Adds a light-sensing protein to ganglion cells. A camera device converts what you see into signals the treated cells can read.

How it is given
One injection into the eye, plus a wearable device
Results so far
Early company-reported results: people who were nearly blind could detect light and movement, and most in the higher-dose groups read more letters on the eye chart. These results have not yet been published in full.
Who could join
Adults with RP whose better-seeing eye is 20/160 or worse, or whose side vision is narrowed to 30 degrees or less.
Latest news
The FDA gave BS01 an RMAT designation in February 2025. The company says it is preparing a phase 3 study.

Studies

Sources: CGTLive: BS01 results

KIO-301

Kiora Pharmaceuticals, with Théa

  • Phase 2
  • Recruiting now

Not a gene therapy. A small-molecule 'photoswitch' that makes ganglion cells respond to light. Because it is a medicine, not a gene, its effect may wear off and it may need repeat injections.

How it is given
Injection into the eye, done in the clinic (no surgery), repeated over time
Results so far
In the first study (6 people with late-stage RP), the injections were safe in the short term and some people did better on vision tests. The study had no comparison group (Nature Medicine, 2026).
Who could join
ABACUS-2 is for adults with late-stage RP (no light perception or very low vision in both eyes). RP from most genes qualifies, and Usher syndrome type 2 is allowed.
Latest news
Kiora expects results from the ABACUS-2 study in the third quarter of 2027.

Studies

Sources: Kiora: ABACUS-1 published in Nature Medicine; Kiora Q2 2026 update

3 of 3

Cell therapies.

These add new cells to the eye. Some release substances that support the retina you have. Others aim to replace the lost light-sensing cells.

jCell famzeretcel

jCyte

  • Phase 2

Retinal progenitor cells from donated tissue. They are thought to release substances that help the remaining rods and cones survive and work better.

How it is given
One injection into the eye, done in the clinic (no surgery)
Results so far
In an earlier phase 2b study, more people given the higher dose gained 10 or more letters on the eye chart at 12 months than with a sham injection (about 39% vs 19%). The new study is testing this again.
Who could join
The current study is no longer enrolling.
Latest news
A new phase 2 study has finished enrolling. Its main results are expected after December 2026.

Studies

Sources: jCyte phase 2b results

OpCT-001

BlueRock Therapeutics (Bayer)

  • Phase 1/2
  • Recruiting now

New light-sensing cells (photoreceptors) grown from stem cells, placed under the retina to replace the ones that were lost.

How it is given
Eye surgery to place cells under the retina
Results so far
No results yet.
Who could join
Adults with a genetically confirmed disease of the light-sensing cells, including RP, and moderate to very low vision. People who have had gene therapy, cell therapy or a retinal implant cannot join.
Latest news
The FDA has given it orphan drug designation, which supports development of treatments for rare diseases.

Studies

Sources: Bayer: orphan drug designation

Studies that have ended.

Not every treatment works. Knowing what has already been tried helps you make sense of the news.

Earlier treatments

  • Argus II retinal implant (Second Sight Medical Products). An electronic implant with a camera on glasses. It was approved in the U.S. in 2013 and gave some people with advanced RP the ability to see light and large shapes, but the company stopped making it in 2020.

A word of caution. Be careful with clinics that charge money for 'stem cell' treatments of the eye. Real clinical trials do not charge you to take part. Some people have lost their sight after unproven stem cell injections. Talk to your retina specialist before agreeing to any treatment.

Last checked by our team: October 26, 2026. Drafted from the handouts we give in clinic, re-checked against ClinicalTrials.gov, company announcements and published papers.

Questions to ask your doctor.

  • Which gene causes my RP, and is there a study for my specific gene?
  • Is my vision a better fit for treatments that protect cells, or for ones that restore light sensing?
  • If MCO-010 is approved, would I be a candidate?
  • Should I take any supplements, and are any of them risky for me?
  • Have I seen a low vision specialist? See our list of low vision services.

This guide is for general education and does not recommend any treatment or study. Listing a study does not mean we run, recruit for or endorse it.