Every study has its own rules. These are general patterns, not a substitute for talking with your retina specialist.
Get a genetic test.
Almost every study asks for a genetic test report showing changes in the ABCA4 gene. If you have not had one, ask your doctor. See our genetic testing guide.
Earlier disease.
If your vision is still fairly good, studies of medicines and some gene therapies aim to slow the damage and protect the vision you have.
Pills or eye injections that try to slow the damage. They do not replace the ABCA4 gene, so they may help people with many different ABCA4 changes. Most are being tested in people whose vision is still fairly good, because the goal is to protect the retina that is left.
Tinlarebant LBS-008
Belite Bio
Under FDA review
Lowers a blood protein (RBP4) that carries vitamin A to the eye. Less vitamin A in the retina means fewer toxic waste products build up.
How it is given
A pill taken once a day
Results so far
In the phase 3 DRAGON study (104 people aged 12 to 20), the damaged areas of the retina grew about 36% more slowly than with placebo over 2 years. Reading vision stayed about the same in both groups, which is expected over 2 years. In earlier studies, some people noticed a yellow tint to their vision or slower adjustment to the dark; these effects were mostly mild.
Who could join
DRAGON enrolled ages 12 to 20 with Stargardt and at least one ABCA4 change.
Latest news
The FDA accepted the application with priority review in August 2026. A decision is expected by February 12, 2027.
A specially modified form of vitamin A. It is built to clump together into toxic waste much more slowly than normal vitamin A does.
How it is given
A capsule taken once a day
Results so far
In the TEASE-1 study (50 people), damaged areas grew more slowly than in the untreated group. It was well tolerated, with no reports of night blindness. Results were published in JAMA Ophthalmology in 2026.
Who could join
NORTHSTAR: ages 8 to 45, typical Stargardt disease, and a genetic test report.
Latest news
The NORTHSTAR phase 3 study began dosing in June 2026 and plans to enroll about 230 people.
CITY-RBP4(City Therapeutics). An injection under the skin that lowers RBP4, the same target as tinlarebant. First study in healthy volunteers and people with Stargardt, ages 12 to 45. NCT07836452Recruiting now
Disulfiram(University of Rochester). An older medicine (Antabuse) being tested for whether it can improve vision in several retinal degenerations. Small early study, adults 18 and older. NCT06319872Recruiting now
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Gene therapies for ABCA4.
These treatments try to fix the root cause by giving the retina a working ABCA4 gene, or by repairing the faulty one. You need a genetic test showing changes in ABCA4. The ABCA4 gene is too large for the usual delivery virus, so most of these use two viruses that each carry half of the gene. All are given once, by eye surgery that places the treatment under the retina.
SB-007
SpliceBio
Phase 1/2
Recruiting now
Two viruses each deliver half of the ABCA4 gene. Inside the cell, the two halves of the protein join to make the full, working ABCA4 protein.
How it is given
One-time eye surgery (injection under the retina)
Results so far
Results have not been reported yet.
Who could join
Ages 12 to 65 with changes in both copies of ABCA4.
Latest news
The second part of the study, which compares treated people with an untreated group, started in January 2026.
Two viruses each carry part of the ABCA4 gene. The cell joins the two messages together to make full-length ABCA4.
How it is given
One-time eye surgery (injection under the retina)
Results so far
Early results from the company (2026): improvements in vision in several patients, and no serious side effects so far. These are early, small and not yet peer-reviewed.
Who could join
Ages 6 and older with changes in both copies of ABCA4 and poor vision in the eye to be treated.
JWK006(West China Hospital). An ABCA4 gene therapy given under the retina. Small study in China, ages 10 to 18. NCT06300476Ongoing, not recruiting
HG005(HuidaGene Therapeutics). A gene treatment given under the retina. Small early study in China, ages 6 to 17. NCT07063251Recruiting now
ZVS106e(Zhongshan Ophthalmic Center). A two-part ABCA4 gene therapy. Small early study in China, ages 8 and older. NCT07241169Recruiting now
Base editing(Beam Therapeutics and others). Tools that correct a single letter of DNA inside the eye. Still in laboratory testing; not yet tested in people.
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Treatments that do not depend on your gene.
Sometimes called gene-agnostic or mutation-independent treatments. They do not fix ABCA4, so they work the same way no matter which gene change you have. Some try to protect the retina you still have. Others help surviving cells take over the job of the light-sensing cells that were lost; those are mostly for people with advanced vision loss.
OCU410ST
Ocugen
Phase 2/3
Does not replace ABCA4. It delivers a different gene, RORA, that helps control waste build-up and inflammation in the retina. Because of this, it can be used no matter which ABCA4 changes you have.
How it is given
One-time eye surgery (injection under the retina)
Results so far
In the earlier phase 1 study, treated eyes showed slower growth of damaged areas than untreated eyes, as reported by the company. No serious side effects related to the treatment have been reported.
Who could join
Ages 5 and older with a genetic diagnosis of Stargardt.
Latest news
All 63 participants have been treated. Ocugen plans to share main results and apply to the FDA in 2027.
Optogenetic therapy. It adds a light-sensing protein to other retinal cells (bipolar cells) that usually survive, so they can respond to light after the rods and cones are gone.
How it is given
One injection into the eye, done in the clinic (no surgery)
Results so far
In the STARLIGHT study (6 people with advanced vision loss), vision improved by an average of about 5 letters on the eye chart after 48 weeks, and by more when using a magnifier. No serious side effects were reported. This was a small study with no comparison group.
Who could join
STARLIGHT enrolled ages 16 and older with very low vision (about 20/400 to 20/1600) in the treated eye.
Latest news
For Stargardt, Nanoscope has agreed on a phase 3 study design with the FDA. For retinitis pigmentosa, the FDA accepted its application in 2026, with a decision expected in the first half of 2027.
Not a drug or a gene. The glasses send an image to the chip as invisible light, and the chip turns it into signals the remaining retina can pass to the brain.
How it is given
Eye surgery to place a tiny wireless chip under the retina, used with special camera glasses
Results so far
Results in Stargardt have not been reported yet. The same implant has been tested in people with advanced dry age-related macular degeneration.
Who could join
Adults 18 and older with an inherited retinal disease, central vision of 20/320 or worse, and a large enough area of damage at the center of the retina.
Lab-grown cells, made from stem cells, are placed under the retina to replace cells that have been lost. These studies are at an early stage and mainly test safety. They are usually for people with significant vision loss.
OpCT-001
BlueRock Therapeutics (Bayer)
Phase 1/2a
Recruiting now
Light-sensing cell precursors (photoreceptor precursors) grown from induced pluripotent stem cells, which are made from adult cells rather than embryos.
How it is given
Eye surgery (cells placed under the retina)
Results so far
Results have not been reported yet.
Who could join
Adults 18 and older with a genetic diagnosis of an inherited disease of the light-sensing cells, such as retinitis pigmentosa, Usher syndrome or cone-rod dystrophy, and low vision. Ask the study team whether Stargardt qualifies.
Not every treatment works. Knowing what has already been tried helps you make sense of the news.
Earlier treatments
Avacincaptad pegol (Zimura)(Astellas, formerly Iveric Bio). Eye injections that block part of the immune system (complement C5). In the phase 2b STAR study, damage grew at the same rate as with a sham (pretend) injection. It is approved for geographic atrophy in age-related macular degeneration, not for Stargardt. NCT03364153Completed
Emixustat(Kubota Vision). A daily pill that slows the vitamin A cycle. In the phase 3 SeaSTAR study, it did not slow the growth of damaged areas compared with placebo. NCT03772665Completed
SAR422459 (StarGen)(Sanofi). An early ABCA4 gene therapy using a lentivirus. The study was stopped; long-term safety follow-up of treated patients continues. NCT01367444Stopped early
MA09-hRPE(Astellas). Support cells (RPE) grown from embryonic stem cells. These first studies focused on safety. Astellas is now testing a newer cell product, ASP2020. NCT01469832Completed
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. Tsang lab patient handout 'Stargardt Tables' (July 2026 update), re-checked against ClinicalTrials.gov, company announcements and published papers.
Questions to ask your doctor.
Do my genetic test results confirm Stargardt disease caused by ABCA4?
Is my vision and the amount of damage in my retina a fit for any of these studies?
Which study sites are closest to me, and who should I contact?
What would joining a study involve: surgery, visits, travel, and how long?
Should I avoid vitamin A supplements, and should I wear sunglasses outdoors?
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.