In a historic move that underscores the growing potential of gene-based therapies, the U.S. Food and Drug Administration (FDA) has approved Zevaskyn (pz-cel), the first-ever cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB), a rare and debilitating genetic skin disorder.
Developed by Abeona Therapeutics, Zevaskyn represents a major leap forward in regenerative and personalized medicine. RDEB is caused by mutations in the COL7A1 gene, which is responsible for producing type VII collagen, a protein crucial for anchoring the layers of skin together. Without this protein, the skin becomes prone to blistering, tearing, and non-healing wounds.
Zevaskyn works by extracting a patient’s own skin cells, correcting the genetic defect in a laboratory setting using viral vectors and then culturing the modified cells into skin grafts. These grafts are transplanted back onto the patient, where they produce functional type VII collagen, helping to restore skin integrity.
Clinical trials reported not only accelerated wound healing but also long-term durability of treated areas and a substantial improvement in pain scores. The therapy is expected to be offered at select specialized treatment centers in the U.S. starting in Q3 2025, with priority for patients suffering from the most severe forms of the disease.
Experts believe this approval may pave the way for future gene therapies targeting other rare or previously untreatable genetic disorders.
Disclaimer: This article is intended for informational purposes only and should not be considered medical advice. Patients are advised to consult qualified healthcare professionals before making any decisions related to their medical care. The availability and suitability of Zevaskyn may vary depending on individual patient conditions, regional regulatory policies and treatment center capabilities.
