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First Academic Medical Center Implants Cell-Based Therapy for Rare Eye Disease

Dr. Vajzovic implants encapsulated cell-based gene therapy for MacTel patient
Dr. Vajzovic implants encapsulated cell-based gene therapy for MacTel patient

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Matt Talhelm
Matt Talhelm
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DURHAM, N.C. – A team at the Duke Eye Center is the first team at a U.S. academic medical center to implant a new commercially available cell-based gene therapy for a rare, but debilitating, retinal disorder called macular telangiectasia type 2, or MacTel.

Patients with MacTel gradually lose their central vision over time due to the degeneration of light-sensitive cells in the back of the eye known as photoreceptors, making daily tasks like reading or driving difficult.

There was no treatment to slow the progression of this rare disease until the FDA approved the new encapsulated cell-based gene therapy – called revakinagene taroretcel-lwey – in March 2025.

“This implant offers real hope for patients facing vision loss because of MacTel,” said Lejla Vajzovic, M.D., professor in the Department of Ophthalmology at Duke University School of Medicine. “We are really allowing our patients to live fuller, more independent lives by preserving their vision.”

Vajzovic led the surgical team that implanted the cell-based gene therapy capsule at Duke Eye Center. In addition to being a first-of-its-kind procedure at an academic medical center, it’s also a first for North Carolina and the southeast region.

The implant, which is about the size of a grain of rice, contains more than 200,000 living cells within a permeable membrane that releases protein into the eye wall.

“Unfortunately, we cannot reverse the degeneration that’s already happened,” said Vajzovic, “but with this therapy, we can help decrease further degeneration of the photoreceptors and help patients retain the vision they do have.”

The implant was performed on Yvette Crawley, 71, from Apex, North Carolina. Crawley started noticing changes to her vision around the time she turned 60.

She was diagnosed with MacTel in 2021. With no treatment available at the time, Crawley expected to continue losing sight in her left eye.

“This disease is relentless,” said Crawley. “I was devastated when I got my diagnosis. You just think – ‘Oh, I live by myself. What's this life going to be?’”

Crawley came to the Duke Eye Center to see Vajzovic, who told her about the therapy when it was under clinical trial at the time. Crawley said she remained hopeful that those trials would one day lead to her receiving treatment for the disease that threatened to rob her of her vision.

“Because of the research that led to this treatment, it’s going to allow me to continue to make choices in my retirement life. I’m not going to be hindered or hampered by my potential loss of vision,” said Crawley.

The Duke Reading Center, which is a leading center specializing in the comprehensive analysis of retinal images, collaborated with Sina Farsiu, Ph.D., professor in the departments of Ophthalmology and Biomedical Engineering, to play a pivotal role in the clinical trials that led to FDA approval for this therapy.

“This significant milestone for patients is the result of years of rigorous research and collaboration,” said Glenn Jaffe, M.D., director of the Duke Reading Center and Robert Machemer M.D. Distinguished Professor in the Department of Ophthalmology.

“By developing and validating the methodology to assess a novel clinical trial endpoint, and by making the measurements to show a treatment effect, we’ve not only helped bring the first treatment for MacTel to patients but also opened new pathways for evaluating therapies in other retinal diseases,” Jaffe said.

“This is an amazing example of Duke’s mission in action — translating groundbreaking research into real treatments,” Vajzovic said. “We’ve helped bring this therapy from concept to clinic, and now we’re among the first centers in the country to offer it to patients.”

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