Macular Telangiectasia type 2, or MacTel, is a rare eye disease that causes severe central vision loss. In December 2025, a 63-year-old Florida man became the first person in Florida, and one of the first in the United States, to receive a new implant that uses gene therapy to slow the disease’s symptoms.
Thomas Albini, M.D., and his team at the Bascom Palmer Eye Institute at the Miller School of Medicine performed the surgery to place the microscopic implant, called revakinagene taroretcel-lwey and sold commercially by Neurotech Pharmaceuticals as Encelto. It’s the first FDA-approved treatment designed specifically to treat MacTel.
This milestone was enabled by decades of research to develop treatments for retinal diseases — and much of this effort happened at Bascom Palmer.
MacTel affects about one in 10,000 people. It damages the retina in both eyes and typically leaves patients with about 20/60 vision, which causes significant problems with activities like driving or reading, explained Dr. Albini, a professor of clinical ophthalmology. The implant is FDA-approved for use at any stage of the disease.
The patient received the implant in only one eye, so doctors can ensure his body tolerates it before considering surgery for his other eye. Although it will not improve the patient’s vision, it should slow his vision loss to about half its previous rate.
Foundational Research at Bascom Palmer
MacTel was first identified in the 1980s by J. Donald Gass, M.D., a Bascom Palmer faculty member who described dozens of retinal diseases during his career.
“When this disease was first identified, an exceptional clinician was able to define it and study it in a small number of patients,” Dr. Albini said. “Even though it’s a fairly rare condition, he was able to recognize that this was a distinct entity.”
Then, beginning in the 1990s, Rong Wen, M.D., Ph.D., professor of ophthalmology at Bascom Palmer, studied the role of a protein called ciliary neurotrophic factor, or CNTF, in inherited retinal diseases in animals. He found that the protein promoted the survival of important retinal cells.
Scientists at Bascom Palmer and other institutions began studying ways to use the protein to treat retinal diseases in people.
Dr. Albini has been involved in clinical trials involving CNTF for more than 15 years, studying its application in diseases including macular degeneration, retinitis pigmentosa, ischemic optic neuropathy and glaucoma. But the results were best for patients with MacTel, he said.
“We are hopeful that we will find other diseases that this works for.”
A Genetically Engineered Solution
The key to bringing a CNTF-based treatment to patients was genetic engineering.
“Part of the problem with the [CNTF] molecule was that it disappears from the eye very rapidly,” Dr. Albini explained. “If you’re going to deliver it as an injection, you would have to do an injection every single day into somebody’s eye, which isn’t practical.”
Instead, the implant uses lab-cultured human retinal pigment epithelial cells that are engineered to produce CNTF. The tiny implant contains about 300,000 of these cells, which maintain a consistent, high concentration of the protein in a patient’s eye over a long period.
The surgery is done under a microscope, and the implant is sutured into a 3-millimeter incision behind the lens of a patient’s eye. It can be removed if the patient has issues with it, and the cells in the implant receive nutrients from the eye’s natural fluids.
The implant releases CNTF into the eye’s vitreous cavity, where it floats to the retina and the tissues beneath it. Its most beneficial effect is on Müller cells — it prevents them from degenerating and helps keep them alive. Because Müller cells sit next to the photoreceptors deep inside the retina, they, in turn, help preserve the photoreceptors. Both of these types of retinal cells are damaged during progression of MacTel.
Dr. Albini said he expects the implant to last for the rest of the patient’s life. One of the patients who received an implant in clinical trials had it for 14 years before deciding to have it removed, and it was still producing a steady state of CNTF.
Similar treatments could help patients with other retinal diseases. Dr. Albini’s team was involved in a recent study examining CNTF applications in glaucoma, and preliminary data showed some benefit in slowing disease progression. The next step will be a large clinical trial in glaucoma patients to confirm the findings. But unlike for MacTel, there are many other treatments for glaucoma already on the market, Dr. Albini noted.
“We are hopeful that we will find other diseases that this works for,” he said. “It’s certainly great for the minority of patients who have this orphan disease, and it’s likely to contribute to something else in the future.”
“This surgery shows how the pathway that goes from the clinical, to the laboratory and back to the clinic isn’t straight most of the time.”
An Unpredictable Path to Patients
Dr. Albini said the story of this implant demonstrates the many levels of research and clinical work that go into bringing a new treatment to patients.
Developing a new product takes time, he said — in this case, decades between identifying the potential benefits of CNTF and helping the first MacTel patients with the new implant. Treating MacTel wasn’t even one of the early goals for the implant — the first clinical trials tested it for inherited retinal diseases.
“It shows how sometimes the benefit of science is unpredictable,” Dr. Albini said.
Translating foundational research to the market also requires the investment and dedication of many people. When researchers and clinicians work together closely at places like Bascom Palmer, they can share their findings — especially the unexpected ones, Dr. Albini noted.
“This surgery shows how the pathway that goes from the clinical, to the laboratory and back to the clinic isn’t straight most of the time,” he said. “There are some unanticipated changes of direction, and it’s lucky that you have all these people working close to each other so that these different findings can be communicated.”
