Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa

• Published in: American journal of clinical dermatology Vol. 26; no. 4; pp. 623 - 635
• Main Authors: Marinkovich, M. Peter, Paller, Amy S., Guide, Shireen V., Gonzalez, Mercedes E., Lucky, Anne W., Bağcı, Işın Sinem, Agostini, Brittani, Fitzgerald, Kolleen, Chen, Shijie, Chen, Hubert, Conner, Meghan M., Krishnan, Suma M.
• Format: Journal Article
• Language: English
• Published: New Zealand Springer Nature B.V 01.07.2025; Springer International Publishing
• Subjects: Adolescent; Adult; Child; Clinical outcomes; Collagen Type VII - genetics; Epidermolysis Bullosa Dystrophica - genetics; Epidermolysis Bullosa Dystrophica - therapy; FDA approval; Female; Gene therapy; Genetic Therapy - adverse effects; Genetic Therapy - methods; Genetic Vectors - administration & dosage; Genetic Vectors - adverse effects; Herpes viruses; Herpesvirus 1, Human - genetics; Humans; Hypothesis testing; Male; Middle Aged; Original; Patient Reported Outcome Measures; Patient satisfaction; Patient Satisfaction - statistics & numerical data; Quality of Life; Questionnaires; Skin; Skin - pathology; Time Factors; Treatment Outcome; Wound healing; Wound Healing - genetics; Young Adult; United States > US

Patients with dystrophic epidermolysis bullosa have pathogenic variants in COL7A1, leading to skin fragility. Beremagene geperpavec-svdt (B-VEC) is a modified, herpes simplex virus type 1-based gene therapy vector that topically delivers COL7A1 to dystrophic epidermolysis bullosa wounds. In a phase...