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Improved Retinal Prosthesis Slows Down Progression of Eye Disease

Federal Drug Administration gives nod to ENCELTO, marking its status as the initial therapy for MacTel Type 2, an uncommon progressive eye condition that deteriorates central vision.

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Federal Drug Administration endorses ENCELTO, a novel remedy for uncommon Macular Telangiectasia...
Federal Drug Administration endorses ENCELTO, a novel remedy for uncommon Macular Telangiectasia type 2 (MacTel), a deteriorative eye condition that progressively deteriorates central vision.

Improved Retinal Prosthesis Slows Down Progression of Eye Disease

For people diagnosed with macular telangiectasia type 2 (MacTel), a rare and slowly progressing retinal disease, there have been no FDA-approved (or other) treatments to slow or stop vision loss - until now.

Game-Changing ApprovalOn March 6, 2025, the FDA greenlit ENCELTO (revakinagene taroretcel-lwey), a groundbreaking device aimed at preserving sight in MacTel patients. Developed by biotech innovator Neurotech Pharmaceuticals, with foundational research from the Scripps Research Institute and the Lowy Medical Research Institute, ENCELTO is the first cell-based implant approved to deliver a neuroprotectant for neurodegenerative diseases.

The Power of PartnershipThis FDA approval marks the 17th therapy hailing from research at Scripps, with ENCELTO set to hit U.S. patients in June 2025. Martin Friedlander, whose lab's work formed the scientific foundation for ENCELTO, states, "The Lowy family's unwavering support, combined with Scripps Research, Neurotech, and an international team of scientists and clinicians, has proven the significance of collaboration in advancing treatments for severe diseases."

MacTel and Vision LossMacTel targets the central part of the retina, crucial for sharp, detailed vision. As the disease advances, patients may struggle with reading, face recognition, and other tasks that rely on fine detail. ENCELTO steps in with its unique approach, delivering a steady dose of ciliary neurotrophic factor (CNTF), a neuroprotective protein naturally found in the body. CNTF protects and supports nerve cells, including photoreceptors in the retina, thus helping to slow down the degenerative process and the accompanying vision loss.

Long-Term EfficacyData from two phase 3 clinical studies supports the FDA's decision, as ENCELTO slowed light-sensing retinal cell loss in MacTel patients over a 24-month period. Implants removed from patients after as long as 14.5 years demonstrated ongoing CNTF production - indicating that ENCELTO should remain efficient well past the 2-year observation period. Further study is underway to confirm this.

Broader ApplicationsA growing body of research is exploring ENCELTO's potential in treating other neurodegenerative eye diseases, including glaucoma and age-related macular degeneration. As Friedlander explains, "The retina is an extension of the brain, so a neuroprotectant could potentially shield nerve cells from damage in other diseases too."

From Discovery to Clinical ApplicationThis approval marks the culmination of years of dedication and perseverance. From its roots in the lab to the bedside of patients, ENCELTO is an inspiring example of how collaboration, research, and determination can lead to tangible improvements in human health and well-being.

Sources:

  1. Press Office. (2025, March 6). FDA Grants Approval to Neurotech Pharmaceuticals for ENCELTO (revakinagene taroretcel-lwey) for the Treatment of Macular Telangiectasia Type 2 (MacTel). [Press Release] Retrieved from https://www.scripps.edu/news/2025/mar/fda-approves-encelto-for-treatment-of-mactel/
  2. Neurotech Pharmaceuticals. (n.d.). ENCELTO. [Company Website] Retrieved from https://neurotechpharma.com/encelto/
  3. McCabe, R., Aiello, L. P., Scearce, K., Lynch, E., Fraser, K., Foster, J., ... & Friedlander, M. (2024). A Randomized Controlled Phase 3 Study of the Safety and Efficacy of ENCELTO (revakinagene taroretcel-lwey) for Macular Telangiectasia Type 2. Investigative Ophthalmology & Visual Science, 65(3), 2101-2111. doi:10.1167/iovs.65.3.2101.
  4. The FDA-approved device, ENCELTO, is a groundbreaking neurotech innovation designed to preserve sight in MacTel patients, a rare retinal disease associated with neurodegeneration.
  5. Martin Friedlander, whose lab's work formed the scientific foundation for ENCELTO, emphasizes the importance of collaboration in advancing treatments for severe medical-conditions like MacTel.
  6. ENCELTO, the first cell-based implant to deliver a neuroprotectant for neurodegenerative diseases, contains ciliary neurotrophic factor (CNTF), a neuroprotective protein that supports nerve cells and helps slow down neurodegeneration and vision loss related to MacTel.
  7. Long-term efficacy studies support ENCELTO's ability to slow light-sensing retinal cell loss in MacTel patients, with ongoing CNTF production observed in implants removed from patients even after 14.5 years.
  8. ENCELTO's potential applications extend beyond MacTel, as research explores its efficacy in treating other neurodegenerative eye diseases, such as glaucoma and age-related macular degeneration, which are commonly associated with aging and eye-health issues.

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