Summary: The FDA has approved ENCELTO, the first treatment for macular telangiectasia type 2 (MacTel), a rare progressive retinal disease that impairs central vision. ENCELTO is a surgically implanted device that delivers a continuous dose of the neuroprotective protein ciliary neurotrophic factor (CNTF) to preserve retinal cells and slow disease progression.
Developed by Neurotech Pharmaceuticals from foundational research at Scripps Research and the Lowy Medical Research Institute, ENCELTO represents a major advance in retinal neuroprotection. Clinical trials demonstrate durable effects on photoreceptor preservation, and the approval is notable as the first cell-based implant to deliver a neuroprotectant for a neurodegenerative condition.
Key Facts:
- First-of-its-kind therapy: ENCELTO is the first FDA-approved treatment for MacTel and the first implantable device authorized to deliver a neuroprotective agent for a neurodegenerative disease.
- Demonstrated long-term activity: In studies, devices removed after as long as 14.5 years continued to produce CNTF at levels comparable to those at implantation, supporting sustained biologic activity beyond the 24-month trial period.
- Potential broader applications: Researchers are evaluating ENCELTO’s platform for other retinal neurodegenerative diseases such as glaucoma and age-related macular degeneration (AMD), exploring delivery of CNTF and other therapeutic molecules.
Source: Scripps Research Institute
Background: Before ENCELTO’s approval, people diagnosed with macular telangiectasia type 2 (MacTel) had no FDA-approved therapies to slow or halt vision loss. MacTel is a rare, slowly progressive disease that primarily affects the central macula, the retinal region responsible for high-acuity tasks such as reading and face recognition.
On March 6, 2025, the FDA approved ENCELTO (revakinagene taroretcel-lwey), a surgically implanted capsule designed to deliver CNTF directly to the back of the eye. The device is expected to become available to U.S. patients in June 2025.
The ENCELTO implant traces its origins to collaborative research led by physician-scientist Martin Friedlander at Scripps Research and supported by the Lowy Medical Research Institute. This approval is the 17th therapy tied to discoveries from Scripps Research and reflects years of basic science, translational work, and clinical development carried out with Neurotech Pharmaceuticals and an international consortium of investigators and clinicians.
MacTel was initially interpreted as a vascular disorder because early imaging showed abnormal capillaries near the fovea. Subsequent research led by Friedlander and colleagues demonstrated that the core problem is neurodegeneration: progressive loss of photoreceptors and supportive glial cells. Those findings motivated strategies focused on preserving retinal neurons rather than only treating vascular abnormalities.
ENCELTO uses a tiny, collagen-based capsule that contains genetically modified retinal pigment epithelial (RPE) cells engineered to secrete CNTF. Once implanted in the subretinal space, the capsule releases a controlled, continuous dose of CNTF while protecting the encapsulated cells from immune attack. CNTF is a naturally occurring neurotrophic protein that supports neuronal survival and function, acting as a neuroprotectant for photoreceptors and other retinal neurons.
The FDA decision was based on two Phase 3 clinical trials in which the implanted device slowed the loss of light-sensing retinal cells over 24 months. Beyond those controlled trials, explanted devices recovered years later retained CNTF production, suggesting a long-term therapeutic window. Ongoing Phase 4 and extended follow-up studies are assessing durability, safety, and real-world outcomes over longer periods.
Martin Friedlander, now president of the Lowy Medical Research Institute, emphasized the collaborative nature of the effort: the Lowy family’s commitment, academic research at Scripps, and industry development with Neurotech combined to produce a targeted, sustained therapy for a disease that previously had no approved options. Friedlander also notes that translating a lab discovery into a clinically available therapy is among the most rewarding outcomes for a clinician-scientist.
Beyond MacTel, investigators view the ENCELTO platform as a promising method to deliver neuroprotective molecules to the retina and potentially to address neurodegeneration in other contexts. Because the retina is an extension of the central nervous system, success in retinal neuroprotection opens avenues to explore similar strategies for other neurodegenerative conditions affecting vision or neural tissue.
As ENCELTO becomes available to patients, clinicians and researchers will continue monitoring long-term effectiveness and safety while investigating expanded uses and alternative payloads delivered by the implantable capsule. The approval marks an important milestone in retinal therapeutics and in the broader field of neuroprotective strategies for degenerative disease.
About this neurotech and visual neuroscience research news
Author: Press Office
Source: Scripps Research Institute
Contact: Press Office, Scripps Research Institute
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