Clinical case reports off-label use of gene therapy for extensive wounds in recessive dystrophic epidermolysis bullosa, suggesting higher doses may be beneficial.
Dystrophic epidermolysis bullosa (DEB) is a rare, inherited blistering skin disease with limited treatment options. Beremagene geperpavec (B‐VEC) is a herpes simplex virus type 1‐based topical gene therapy that is approved by the Food and Drug Administration (FDA) for the treatment of DEB at a fixed weekly dose. While B‐VEC has shown significant promise in treating DEB, few studies have examined off‐label use of B‐VEC at higher dose volumes. We describe the successful off‐label use of a temporarily increased B‐VEC dose in a patient with recessive DEB (RDEB) with extensive wounds following an episode of acute urticaria, highlighting the potential utility of higher dose B‐VEC in managing patients with extensive wound burden.
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Yamada et al. (2025) studied this question.
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