Krystal gets UK approval for redosable gene therapy

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorization to Krystal Biotech’s gene therapy, Vyjuvek, for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene, from birth.

Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based topical gene therapy, is the first genetic medicine approved in the UK for the treatment of DEB and is designed to address the root cause of the disease by delivering functional copies of the human COL7A1 gene to provide wound healing and sustained functional type VII collagen protein expression with redosing. The approval in the UK also includes flexible administration options similar to those granted in the U.S., EU and Japan, allowing for dosing at home or in a health care setting.

Vyjuvek was approved in the U.S. in May 2023 for the treatment of wounds in patients 6 months of age and older with the serious, rare skin disease, DEB, with mutation(s) in the COL7A1 gene. The FDA approved a label update in September 2025, expanding the eligible patient population to include DEB patients from birth and providing patients and their caregivers the ability to apply Vyjuvek themselves.

DEB is an inherited disorder that makes the skin extremely fragile, causing painful blisters, wounds, and scarring from minor friction because the layers of the skin do not anchor together properly.