465 GEM-3: phase 3 safety and immunogenicity results of beremagene geperpavec (B-VEC), an investigational, topical gene therapy for dystrophic epidermolysis bullosa (DEB)

Abstract

DEB is rare, severe, genetic disorder caused by mutations in the COL7A1 gene. B-VEC is an HSV-1-based topical, redosable gene therapy designed to restore functional COL7 protein in DEB patients. In a Phase 3, multi-center, intra-patient, randomized, double-blind, placebo-controlled study (NCT04491604), patients with confirmed COL7A1 mutations were enrolled. Each patient contributed 1 primary wound pair, randomized 1:1 to weekly treatment with B-VEC or placebo for 26 weeks. B-VEC dose ranged from 4e8-1.2e9 PFU/wound, determined by wound size at baseline.