Alfonso Sabater, MD, PhD, on Further Research With Gene Therapy in Eye Care


The associate professor of clinical ophthalmology at University of Miami discussed the first topical application of gene therapy to the eye.

“There's also a significant amount of research that has been done with new therapies, but these the first time that gene therapy was applied topically in the eye. That's definitely a step forward and it seems like it works, but it's only one patient. So, I like to be very careful when I say the treatment works, but it’s definitely worth it to continue doing research and exploring how we could use this platform to treat other conditions beyond EB.”

The FDA approved the Krystal Biotech’s gene therapy beremagene geperpavec (B-VEC) under the name Vyjuvek in May 2023 for the treatment of dystrophic epidermolysis bullosa (DEB) in patients 6 months or older. The topical and redosable gene therapy has also been used under a compassionate use approval in treating a single patient for ocular complications of DEB under the guidance of Alfonso Sabater, MD, PhD, associate professor, clinical ophthalmology, and medical director, ocular surface program, and director, Corneal Innovation Lab, University of Miami.

CGTLive spoke with Sabater to learn more about the current state of cell and gene therapy research in eye care. He shared his excitement to be a part of the first topical application of gene therapy to the eye, a major milestone in gene therapy research. He also shared his hope that gene therapy can be used to help treat other eye conditions in the future, although with the current challenges and expenses, he expects its use to be restricted only to the most severe conditions until manufacturing is improved.

Krystal Biotech receives FDA approval for the first-ever redosable gene therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the treatment of dystrophic epidermolysis bullosa. News release. Krystal Biotech. May 19, 2023. Accessed May 19, 2023.
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