Duke Eye Center pioneers cell-based gene therapy for rare retinal disease

Craig T. Albanese, CEO - Duke University Hospital
Craig T. Albanese, CEO - Duke University Hospital
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A team at Duke Eye Center has become the first in a U.S. academic medical center to implant a new cell-based gene therapy for macular telangiectasia type 2 (MacTel), a rare retinal disorder that leads to progressive central vision loss. The procedure was performed on Yvette Crawley, 71, of Apex, North Carolina, who had experienced worsening vision since her early 60s and was diagnosed with MacTel in 2021.

MacTel causes the gradual degeneration of photoreceptors—light-sensitive cells at the back of the eye—making everyday tasks such as reading and driving increasingly difficult. Until recently, there were no approved treatments to slow the disease’s progression. That changed when the Food and Drug Administration approved revakinagene taroretcel-lwey, an encapsulated cell-based gene therapy, 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.”

The implanted device is about the size of a grain of rice and contains over 200,000 living cells inside a permeable membrane that gradually releases protein into the eye wall. Vajzovic explained that while existing degeneration cannot be reversed, “with this therapy, we can help decrease further degeneration of the photoreceptors and help patients retain the vision they do have.”

Crawley described her diagnosis as devastating. “This disease is relentless,” she said. “I was devastated when I got my diagnosis. You just think – ‘Oh, I live by myself. What’s this life going to be?’” She learned about the experimental treatment from Vajzovic during its clinical trial phase and remained hopeful it would one day become available.

“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,” Crawley said.

The Duke Reading Center played a key role in clinical trials for FDA approval of the therapy. The center specializes in analyzing retinal images and worked with Sina Farsiu, Ph.D., professor in ophthalmology and biomedical engineering.

“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 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.”

Vajzovic added: “This is an amazing example of Duke’s mission in action — translating groundbreaking research into real treatments. 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.”

Duke University Hospital is located in Durham, North Carolina, where it has served patients since its founding in 1925. It operates a pediatric ward and serves as a training hospital under President Craig T. Albanese. In 2022, it admitted more than 41,000 patients for treatment according to its annual report (https://corporate.dukehealth.org/news/duke-university-hospital-annual-report-2022).



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