In April 2025, prademagene zamikeracel (Zevaskyn) became the first FDA-approved cell-based genetic therapy, marking a significant advancement in the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a condition that previously had limited effective options for managing large chronic wounds.

A phase 3 trial published in June 2025 demonstrated that genetically corrected skin grafts significantly improved healing and pain relief in patients with RDEB, with 81% of treated wounds achieving at least 50% healing compared to just 16% in control wounds.

At the 24-week mark of the trial, mean pain scores decreased by an average of 3.07 for treated wounds, while controls only saw a reduction of 0.90, with 16% of treated wounds achieving complete healing.

The grafting process involved the extraction of keratinocytes from 8-mm punch biopsies, which were genetically modified and cultured before being surgically applied as up to six graft sheets per patient.

Despite the promising results, the procedure is complex and labor-intensive, requiring rapid transportation of biopsies to a specialized manufacturing facility, which raises significant cost considerations.

Long-term patient monitoring is essential due to the use of retroviruses in the treatment; however, previous studies have indicated no serious adverse effects or complications associated with the therapy.

To assist patients in overcoming insurance and logistical challenges related to accessing treatment, Abeona Therapeutics offers a support program.

Experts are optimistic that this innovative approach could be adapted for other genetic skin diseases, such as ichthyosis and Gorlin syndrome, and may enhance treatments for other forms of epidermolysis bullosa (EB).