Topical Gene Therapy Heals Dystrophic Epidermolysis Bullosa Wounds

People with untreatable dystrophic epidermolysis bullosa (DEB) may soon have access to an investigational gene therapy delivered in a topical gel that is currently under review by the US Food and Drug Administration (FDA).

In a phase 3 study of patients with DEB, "we found that repeated topical application of B-VEC [beremagene geperpavec], an HSV-1–based gene therapy, resulted in a greater likelihood of complete wound healing than the topical application of placebo at up to 6 months," the study authors write. The study was published in The New England Journal of Medicine on December 15. "Longer and larger trials are warranted to determine the durability of effect and risks of this approach," the authors note.