A new hope for vision arrives when progress feels out of reach. An innovative implant, tested in rigorous trials, now offers a credible way to slow central vision loss and protect fragile retinal cells. As the first therapy to earn approval for a Rare Eye Disease, it opens a path that patients and clinicians have long awaited.
Foundations of a breakthrough
Macular telangiectasia type 2, known as MacTel, slowly erodes central vision, so daily tasks become harder while peripheral sight persists. Clinicians had only monitoring and support, so patients faced a steady decline without any approved therapy. That status quo ends with a device designed to protect retinal cells rather than replace them.
Researchers led a coordinated global effort so the evidence would be strong and clear. Two phase 3 trials followed people for 24 months, and sites spanned 47 locations. The studies enrolled 228 participants and measured both structure and function, which matters because sight depends on anatomy and performance together.
The U.S. regulator granted approval after reviewing these results and weighing clinical need. The decision arrived after landmark trials and set a first for neuroprotective care in the retina. Patients now gain access to a treatment that aims to slow damage, so remaining vision can last longer with real-world benefit.
How the implant works in a Rare Eye Disease
ENCELTO sits in a small collagen-based capsule placed at the back of the eye. Inside, genetically engineered retinal pigment epithelial cells release ciliary neurotrophic factor, or CNTF. The capsule shields the cells from immune attack, while it lets the protective protein flow out in a steady, local dose.
This approach supports the retina while disease continues its course, so care becomes proactive rather than reactive. Light-sensing cells receive help before they fail, and therapy stays focused where it is needed. The design favors sustained action, because the device delivers protein continuously instead of relying on frequent visits.
Clinicians also value consistency, since dosing remains stable between checkups. Patients avoid the burden of repeated injections, while doctors can track function and structure over time. As a platform, the device can carry other factors, so future therapies may follow the same route and expand options within retinal care.
What the trials measured and what changed
Trial teams assessed the ellipsoid zone, a precise marker of photoreceptor health, because structural integrity predicts future vision. One trial showed a 54.8% reduction in the rate of ellipsoid zone loss versus a sham surgery. The second showed a 30.6% reduction, smaller yet statistically significant, which still reflects real protection.
Function also mattered, so investigators ran microperimetry to test retinal light response, and they recorded reading speed. Microperimetry pointed to a slower decline, which aligned with stronger structural preservation in one study. Reading speed and sensitivity results were mixed across trials, so clinicians will interpret them with context while they follow patients.
Pooling data from both trials improved the strength of several functional findings, because designs matched and endpoints aligned. The pattern across outcomes supports a meaningful impact where it counts most: photoreceptor survival. Because a Rare Eye Disease often progresses slowly, long follow-up and composite views help confirm benefit in daily life.
Safety, timing, and who may benefit in a Rare Eye Disease
Participants tolerated the implant well, and adverse effects were minimal in the observed period. Localized delivery limits systemic exposure, which helps safety while still bathing the retina in protective CNTF. Because the capsule remains in place, dosing does not drift between visits, so clinics can plan stable follow-up schedules.
Early intervention appears helpful because healthier tissue responds better to protection, so fewer cells slip past the point of no return. Even so, the implant showed value across baseline vision levels and disease stages. People further along still gained slowing, which supports use while clinicians refine timing for maximum preservation.
Teams now track patients beyond 24 months to see whether preservation holds, improves, or levels off. As data accrue, doctors will learn which profiles respond best, and why some eyes gain more. These insights will guide counseling, so expectations stay realistic while care plans use the strongest evidence available.
What comes next, and why this matters
ENCELTO’s approval in March 2025 followed phase 3 data produced by Scripps Research, the National Eye Institute, and collaborators, with Neurotech Pharmaceuticals as sponsor. Investigators published the analysis in NEJM Evidence on July 22, 2025, so peers can review methods, endpoints, and statistics. Transparent reporting supports adoption and informed decision-making.
Because the device proves that steady, targeted protein delivery can preserve neurons, the field gains a new playbook. Developers can adapt the platform to other neurovascular retinal conditions where cells die slowly. As real-world evidence grows, payers, clinics, and patients will better understand durability, logistics, and cost-benefit in typical practice.
Clinicians already see how structure and function interact, so they plan tests that matter for life quality. Microperimetry tracks sensitivity near fixation, while imaging monitors the threatened photoreceptor layer. The Rare Eye Disease community now has a treatment that addresses biology directly, so hope aligns with measurable change, not wishful thinking.
Broader implications and ongoing questions
Neuroprotection changes the mindset because care shifts from rescuing lost cells to keeping vulnerable cells alive. This strategy complements future gene or cell therapies, since preservation buys time and maintains function. Combinations may one day protect, repair, and replace in sequence, so outcomes improve beyond any single tool.
Researchers will study subgroup responses, so clinics can match patients to the most effective plan. Imaging signatures, baseline sensitivity, and disease tempo may explain who benefits most. Because the platform can carry different proteins, scientists can test new payloads, while the capsule and delivery method stay familiar to surgeons.
Health systems also weigh access, since a one-time procedure could reduce long-term visit load. Operating room resources, training, and follow-up pathways will shape real-world scale. As results mature, stakeholders can refine guidelines so timing, monitoring, and outcome measures stay aligned, and patients maintain vision longer with less uncertainty about care. Using a Rare Eye Disease as the proving ground made the case for change.
A forward path for patients and the teams who guide them
Patients and clinicians now share a clear, evidence-based option that slows central vision loss and protects retinal cells. The journey continues as follow-up extends beyond two years, and as new payloads reach the same capsule. Because the first step worked, the path ahead looks wider for those living with a Rare Eye Disease.