M. Peter Marinkovich, MD, on Increased Accessibility With Topical B-VEC DEB Gene Therapy


The associate professor of dermatology at Stanford University discussed the potential impact of Vyjuvek’s approval in the derm field.

“The thing that's really exciting for me about this type of therapy is, there are a lot of cell-based therapies that require a lot of cell manufacturing, and then you have to have a very specialized facility to apply these therapies, especially these cell-based therapies. But with this therapy, it's very easy. Just put it on the skin. And you can ship it off the shelf to the clinical site. And you just need very basic clinical conditions. So, one of the things that's really attractive to me about this therapy is its potential for use in underserved communities, underdeveloped countries, because it's so easy to use.”

Krystal Biotech’s beremagene geperpavec (B-VEC) became the first approved gene therapy in the dermatology field under the name Vyjuvek in May 2023.It’s been over half a year since the FDA approved the therapy for the treatment of dystrophic epidermolysis bullosa (DEB), and CGTLive spoke with some experts involved in its development to look back at the approval, its potential effect in the dermatology field, and their future outlook in the field.

One of the experts CGTLive spoke with is M. Peter Marinkovich, MD, an associate professor of dermatology at Stanford University, and an investigator on the pivotal GEM trials that evaluated B-VEC. Marinkovich discussed the impact of B-VEC's approval on the dermatology field, stressing the milestone of the first-in-field approval and the first redosable, topical gene therapy approval. He also shared his excitement on the accessibility of B-VEC in underserved areas, since it is easy to distribute and apply.

FDA approves first topical gene therapy for treatment of wounds in patients with dystrophic epidermolysis bullosa. News release. FDA. May 19, 2023. Accessed May 19, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-topical-gene-therapy-treatment-wounds-patients-dystrophic-epidermolysis-bullosa
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