Phase III Gene Therapy Skin Grafts Accelerate Healing in Severe Epidermolysis Bullosa
Stanford Medicine researchers have reported success in a Phase III clinical trial using gene therapy–based skin grafts to treat recessive dystrophic epidermolysis bullosa (RDEB), a devastating genetic skin-blistering disease. The therapy, recently approved by the FDA, represents a major step forward in regenerative wound care for rare disorders.
Key Highlights:
- Improved Wound Healing: In a trial involving 11 RDEB patients, wounds treated with the engineered grafts healed significantly faster than untreated areas. Participants also experienced less pain and itching.
- FDA Approval: The treatment—marketed as ZEVASKYN™ (prademagene zamikeracel)—was approved by the FDA in April 2025, making it the first gene-corrected, cell-based therapy for RDEB.
- Patient Impact: Trial participants described the therapy as life-changing. One 20-year-old patient reported reduced pain and improved quality of life, allowing her to continue working during treatment.
- Durability & Safety: The grafts provided long-lasting healing with minimal side effects. No serious adverse events were reported, and follow-up showed continued skin integrity at the graft sites.
This milestone in wound care highlights the potential for autologous gene-modified grafts to transform treatment for rare and severe skin conditions, offering hope where conventional wound care often falls short.
Based on results published in June 2025 in The Lancet.
Keywords: gene therapy, skin grafts, epidermolysis bullosa, ZEVASKYN, RDEB