The field is expanding beyond treating inherited skin diseases to include adapting gene-therapy platforms for conditions like ichthyosis, Darier disease, and Hailey–Hailey disease, with researchers exploring iPS/CRISPR-based tissue regeneration as a future direction.

B-VEC (beremagene geperpavec), developed by Krystal Biotech and sold as Vyjuvek, showed two-thirds of wounds healing versus under a quarter with placebo over six months, with some benefits lasting two years or more.

A growing landscape of options—from topical gels to grafts and injectable cell therapies—presents decision factors such as wound type, durability, and surgical needs, while price, access, and scalability remain major hurdles.

pz-cel (prademagene zamikeracel) by Abeona Therapeutics, approved by the FDA in spring 2025, targets COL7A1 for RDEB with graft-based cell therapy aimed at deep, non-healing wounds, and trials show wound healing and pain relief with some patients choosing repeat treatment.

The overarching takeaway is that these therapies shift focus from symptom management to molecular repair, offering meaningful improvements and extended lifespans, even as they are not cures and require further development and broader accessibility.

Castle Creek Biosciences is developing a graft-based approach that delivers gene-corrected cells and can be cryopreserved for repeated dosing, potentially providing prophylactic benefits.

Historical context traces back to Italian work, notably De Luca, starting in 2006 with gene-corrected skin grafts, highlighting ongoing funding and market viability challenges for these therapies.

Krystal Biotech is also pursuing eye-drop applications of B-VEC and exploring expanded uses beyond skin, with trials examining non-skin manifestations.

A family with Epidermolysis bullosa experienced dramatic healing after B-VEC, marking the era as the first topical gene-replacement therapy for a non-cancerous skin disorder, with positive outcomes reported among four Troop children.