905 Long term use of topical beremagene geperpavec (B-VEC) in two patients with dystrophic epidermolysis bullosa

Dystrophic epidermolysis bullosa (DEB) is a rare genetic disorder due to mutations in COL7A1, the gene encoding type VII collagen (C7), and is characterized by skin fragility, painful wounds, and various cutaneous and systemic comorbidities. Currently there are no corrective, approved treatments for DEB. Beremagene geperpavec (B-VEC) is an investigational engineered HSV-1-based vector, which restores C7 expression to C7 deficient DEB skin and has shown efficacy in a 26-week phase 3 study. Here, we present clinical findings from two DEB patients who received long-term treatment with multiple doses of B-VEC: one recessive patient (RDEB) who received B-VEC for three years and one dominant (DDEB) patient who received B-VEC for over two years. At baseline, patients showed generalized erosions and C7 deficiency. During the course of B-VEC treatments, durable healing of wounds of varying sizes and anatomical locations, as well as improvement of cutaneous and systemic inflammation were seen. Weekly B-VEC was well-tolerated by both patients without any significant related adverse events. In these two case reports, continued wound healing and evidence of systemic improvement were observed with long-term administration of B-VEC without significant related adverse events.