Real‐world effects of beremagene geperpavec in epidermolysis bullosa

BACKGROUND: Dystrophic epidermolysis bullosa (DEB) is a severe inherited skin fragility disorder caused by mutations in COL7A1, leading to wounds, inflammation and impaired quality of life. Beremagene geperpavec (B-VEC) is the first approved topical gene therapy delivering functional COL7A1 via a modified HSV-1 vector, enabling local collagen VII expression and improved wound healing. OBJECTIVES: To evaluate the real-world, multidimensional long-term outcomes of B-VEC treatment in patients with DEB, including wound healing, systemic inflammation, (auto)-antibody profiles and patient-reported outcomes. METHODS: In this multicentre observational study, 38 patients with DEB received B-VEC treatment between January 2024 and June 2026. The primary endpoint was the change in wound surface area from baseline during B-VEC treatment. Secondary endpoints included changes in systemic inflammatory and haematological parameters [C-reactive protein (CRP), interleukin-6 (IL-6), serum amyloid A (SAA), leucocytes and haemoglobin (Hb)], (auto-)antibody profiles [anti-C7, BP180, BP230 and herpes simplex virus (HSV)-1/2 IgG], body mass index (BMI) and patient-reported outcomes, including pain, pruritus, treatment satisfaction and wound dressing time. RESULTS: B-VEC treatment was associated with a significant reduction in wound surface area across healing wounds. Patients having persistent wounds exhibited lower BMI and higher leucocyte counts and received systemic biologic therapies less frequently. Anti-BP180, BP230 and HSV-1/2 titres remained stable, while anti-C7 levels increased during treatment. IIF was negative in most patients and did not differ between groups. Systemic inflammation showed a mixed pattern, with reduced leucocyte counts and SAA levels, and slightly increased CRP and IL-6 under treatment. Hb and BMI increased during treatment. Most patients reported high treatment satisfaction and reduction in pain, pruritus and wound dressing time. CONCLUSIONS: B-VEC therapy provides clinically meaningful improvements in wound healing, patient-reported outcomes and selected systemic parameters in DEB. Treatment response is influenced by host factors such as BMI, systemic inflammation and concomitant therapies. Baseline (auto)-antibody profiles do not appear to predict treatment efficacy. Overall, B-VEC represents a significant advancement in the multidimensional management of DEB in real-world clinical practice.