Characterisation of the wound microbiome and antimicrobial resistance profiles in clinical isolates from epidermolysis bullosa patients
7 March 2026Perception of Parents Regarding Oral Health-Related Quality of Life of Children With Epidermolysis Bullosa: A Cross-Sectional Study
1 April 2026Expert Opin Biol Ther. 2026 Mar 18:1-6. doi: 10.1080/14712598.2026.2639393. Epub ahead of print. PMID: 41764660.
An evaluation of prademagene zamikeracel for the treatment of recessive dystrophic epidermolysis bullosa
Authors: Lesiak A, Marinkovich MP
Affiliation:
- Laboratory of Autoinflammatory, Genetic and Rare Skin Disorders, Medical University of Lodz, Lodz, Poland.
- Department of Dermatology, Paediatric Dermatology and Oncology, Medical University of Lodz, Lodz, Poland.
- Department of Dermatology, Stanford University School of Medicine, Stanford, CA, USA.
Abstract:
ntroduction: Prademagene zamikeracel is an autologous, genetically corrected epidermal graft therapy designed to address the underlying molecular defect in recessive dystrophic epidermolysis bullosa (RDEB). The product is manufactured from a patient’s own keratinocytes, which are modified ex vivo to overexpress a normal copy of COL7A1, the gene coding for type VII collagen (C7), which is essential for anchoring fibril formation and stable dermal-epidermal cohesion. In individuals with biallelic COL7A1 variants, loss of C7 results in severe skin fragility, painful and widespread wounds, debilitating scarring, and a high risk of aggressive cutaneous squamous cell carcinoma.
Areas covered: This review outlines the key scientific foundations, translational advances, and clinical trial outcomes that supported regulatory approval of prademagene zamikeracel for use in adult and pediatric patients with RDEB.
Expert opinion: The development of prademagene zamikeracel represents a significant advance in regenerative therapy for RDEB, demonstrating that durable restoration of C7 expression and long-term wound improvement can be achieved through skin grafting.
Keywords: Prademagene zamikeracel; recessive dystrophic epidermolysis bullosa; type VII collagen.