FDA approved first topical gene therapy for treatment of wounds in patients with dystrophic epidermolysis bullosa

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On May 19, 2023, the U.S. Food and Drug Administration (FDA) approved Krystal Biotech’s Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

DEB is a genetic disorder that affects the connective tissue in the skin and nails and results from mutation(s) in the COL7A1 gene. This gene encodes type VII collagen (COL7), which is an essential protein that helps strengthen and stabilize the outer and middle layers of the skin.

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Source: U.S. Food and Drug Administration
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