Retinitis pigmentosa is a group of inherited eye diseases that damage the light-sensing cells in the retina. It affects roughly 1 in 4,000 people worldwide. Until recently, no treatments existed; now one gene therapy is FDA-approved for a specific genetic form, and dozens of trials are testing gene therapies, stem cells, and devices that bypass damaged cells.
What's actually going on in research
Trials are testing gene therapies for specific mutations, stem cell transplants to replace dying photoreceptors, retinal implants that send visual signals directly to the brain, and drugs that slow cell death. Researchers are also studying CRISPR gene editing to correct mutations in the eye, and optogenetic therapies that make surviving retinal cells light-sensitive again.
Gene therapies
Multiple gene therapies are in trials, each targeting specific mutations that cause retinitis pigmentosa. Luxturna, approved in 2017 for RPE65 mutations, proved gene therapy can work in the eye and opened the path for others.
Optogenetics
These therapies insert light-sensing proteins into surviving retinal cells, turning them into substitute photoreceptors. Early trials show some people regain basic visual function like detecting movement and shapes.
CRISPR editing
Trials are testing whether CRISPR can directly edit mutations in the eye to stop vision loss. The eye's small size and immune privilege make it an ideal testing ground for gene editing.
What to know before you search
Eligibility usually depends on genetic mutation type, amount of remaining vision, disease stage, and sometimes age at onset.
What types of trials are currently open
- Gene therapy trials — Testing treatments that deliver working copies of specific mutated genes into retinal cells. Each trial usually targets one genetic form of retinitis pigmentosa.
- Stem cell trials — Transplanting lab-grown retinal cells to replace damaged photoreceptors. Trials test whether new cells survive, integrate, and restore vision.
- Optogenetic trials — Adding light-sensing proteins to surviving retinal cells so they can detect light even after photoreceptors die.
- Device trials — Testing retinal implants and vision prosthetics that bypass damaged cells and send visual signals directly to the brain or remaining retinal tissue.
- Drug trials — Testing medications that slow photoreceptor death, reduce inflammation, or protect remaining vision.
Recently added Retinitis Pigmentosa trials
Study to Evaluate the Efficacy of a Single Intravitreal Injection of RTx-015 in Participants With Retinitis Pigmentosa (PERCEPT)
The purpose of this clinical trial is to evaluate the effectiveness and safety of a single injection into the eye of an investigational gene therapy in participants with retinitis pigmentosa (RP).
Track your eye health with researchers over one year
ECSTASI is a Natural History Study, which will be monitoring how standard of care (SoC) affect advanced rod-cone dystrophy (RCD), a type of eye disease also known as retinitis pigmentosa. No experimental, investigational or any other drug or treatment will be tested during the study. This study collects information in 3 parts: Part 1: retrospective health information from medical records (up to past 10 years), Part 2: prospective health information from in-person visits (up to 1 year), and Part 3: prospective health information from medical records (up to 1 year). Participants will decide if they wish to participate in all or only to some of these parts.
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