The FDA approval of revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals) marks a watershed moment in the management of macular telangiectasia type 2 (MacTel), a neurodegenerative retinal disease.^1,2 For decades, clinicians could offer patients little more than monitoring and supportive care while the condition silently progressed.^1,2 Now, with a device capable of delivering continuous neurotrophic support directly to the retina, the treatment landscape is poised for change, according to ophthalmologists.

In this Q&A with the Eye Care Network, Martin Friedlander, MD, PhD, and Rishi P. Singh, MD, FASRS, discuss how this new technology may alter disease trajectories, reshape clinical workflows, heighten awareness and diagnosis of MacTel, and open the door to broader neuroprotective strategies across retinal medicine.

Note: Transcript edited for clarity and length.

How might this device change the treatment landscape for patients with MacTel?

Friedlander: There’s nothing to treat patients with MacTel at this point. It's a 100% game-changer that we can delay… we've seen patients, or a couple of patients, [who] had the device for a number of years, and it's really interesting, after 1 or 2 years, the loss of the ellipsoid zone. It just flattens out. Not every patient, but in many of the patients we see an absolute halting of progression of disease, which is very exciting. If you can preserve useful normal vision for a decade or two, that's very meaningful for these patients.

Singh: The approval of this device represents a major breakthrough for patients with MacTel, a condition for which we previously had no FDA-approved treatment options. By delivering continuous neurotrophic support to the retina, this device offers a novel approach that could slow disease progression and help preserve vision in affected individuals. This shift from passive monitoring to an active treatment strategy is a game-changer for both patients and clinicians.

Is there a learning curve associated with implantation of the device?

Friedlander: It's definitely surgeon-dependent. The few devices we've had to explant typically have to do with extrusion of the device and can sometimes be traced back to less-than-optimal surgical implantation. My understanding is, I believe Neurotech is going to start out with a limited number of clinics which have experience with the device.

Singh: As with any novel surgical implantation, there is a learning curve for surgeons, particularly in terms of placement technique and patient selection. However, the procedure builds on existing vitreoretinal surgical approaches, making it a feasible addition to a retina specialist’s surgical repertoire. With appropriate training, adoption should be relatively smooth for experienced surgeons.

What does approval mean for increasing awareness and diagnosis of MacTel among ophthalmologists and optometrists?

Friedlander: I think we're already seeing that. The work we do here is done at the Lowy Medical Research Institute. LMRI is a private, nonprofit, privately funded research institute, which was endowed by the Lowy family, who approached us almost 20 years ago when they had one of their sons diagnosed with macular telangiectasia. At the time we knew virtually nothing about the disease other than we thought it was a retinal vascular disease. We have over 4000 patients enrolled in our MacTel Registry. This is a disease with a prevalence of 0.04% so it's not a common disease, an orphan disease, but we have many, many patients who are involved with our group.

RELATED: Angiogenesis 2025: Encapsulated cell therapy for MacTel shows long-term efficacy in phase 3 trials

There's a huge effort ongoing in terms of informing the public and physicians. The number of publications in this area, the number of talks related to this, especially now, having something which we can actually put into patients to slow progression of disease has been quite impactful in terms of educating the physician-public as well as patient-public.

Singh: The availability of an FDA-approved treatment is likely to drive greater awareness of MacTel among eye care professionals. Historically, MacTel has been underdiagnosed due to its subtle clinical presentation, but the introduction of an intervention will encourage earlier detection and referral patterns among ophthalmologists and optometrists. This, in turn, could lead to improved patient outcomes as we diagnose and manage the disease at earlier stages.

Could the device pave the way for similar therapies and other neurodegenerative retinal diseases?

Friedlander: I think we view the Neurotech device as a platform technology which will be useful for delivering not only neurotrophics, but perhaps other molecules, like angiostatics or other neurotrophics other than CNTF [ciliary neurotrophic factor] to treat other neurovascular glial degenerative diseases like glaucoma, age-related macular degeneration, retinitis pigmentosa. NeuroTech has already had several studies in the area of atrophic AMD and retinitis pigmentosa and glaucoma, none of which have yielded statistically significant outcomes, but have very promising, encouraging, preliminary data where we're definitely following up with that.

Singh: This approval is an exciting step forward, not only for MacTel but for the broader field of retinal neuroprotection. By demonstrating the potential of sustained-drug delivery to support retinal neurons, this device could pave the way for future treatments targeting other neurodegenerative retinal conditions, such as geographic atrophy in age-related macular degeneration or inherited retinal diseases. It underscores the growing recognition of neuroprotection as a viable therapeutic strategy in ophthalmology.

Martin Friedlander, MD, PhD
E: [email protected]
Friedlander is professor, Department of Molecular Medicine, Scripps Research Institute; chief, Retina Services, Division of Ophthalmology, Scripps Clinic; and president, The Lowy Medical Research Institute in La Jolla, California.

Rishi P. Singh, MD, FASRS
E: [email protected]
Singh is vice president/chief medical officer of Martin North and South Hospitals; professor of ophthalmology, Cleveland Clinic Lerner College of Medicine; Cleveland Clinic Martin Health in Stuart, Florida. He is also a member of the editorial advisory board for Modern Retina.

REFERENCE

1. Neurotech’s ENCELTO (revakinagene taroretcel-lwey) approved by the FDA for the treatment of macular telangiectasia type 2 (MacTel). News release. Neurotech Pharmaceuticals. Published March 6, 2025. Accessed March 6, 2025. https://www.neurotechpharmaceuticals.com/neurotechs-enceltotm-revakinagene-taroretcel-lwey-approved-by-the-fda-for-the-treatment-of-macular-telangiectasia-type-2-mactel/

2. Friedlander M, Stevenson S. Angiogenesis 2025: Encapsulated cell therapy for MacTel shows long-term efficacy in phase 3 trials. Ophthalmology Times. February 12, 2025. Accessed March 6, 2025. https://www.ophthalmologytimes.com/view/angiogenesis-2025-encapsulated-cell-therapy-for-mactel-shows-long-term-efficacy-in-phase-3-trials