Alfonso Sabater, MD, PhD, on Bringing Gene Therapy to Ophthalmology


The associate professor of clinical ophthalmology at University of Miami discussed potential future areas of investigation for gene therapy in ocular diseases.

“Right now we're focused on EB, but potentially this therapy could be used, for example, patients with Fuchs dystrophy, a genetic condition that affects the inner layer of the cornea... We've just brainstormed a little bit about this, this could be a potential indication, but there might be other uses, other conditions in the eye that could benefit from gene therapy. There is, I would say, a significant interest in this field. And I'm sure that we will see this treatment for new indications in the near future.”

Beremagene geperpavec (B-VEC; Krystal Biotech), a topical and redosable gene therapy, was approved for the treatment of dystrophic epidermolysis bullosa (DEB) in patients 6 months or older in May 2023 under the name Vyjuvek. The 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 his thoughts on the recent approval and the future potential for the gene therapy beyond dermatologic indications. He shared his goal to investigate B-VEC in more patients with ocular DEB to further support the positive results seen so far in the sole patient treated. In addition to patients with ocular DEB, he touched on other potential indications, such as Fuchs dystrophy. Sabater emphasized his excitement for the future of gene therapy in ophthalmology.

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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