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# Topical Gene Therapy for Wounds Approved
- URL: https://www.fdaweb.com/topical-gene-therapy-for-wounds-approved/
- Published: 2023-05-19T12:00:00.000Z
- Updated: 2026-09-14T18:33:10.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5154498

FDA has approved a Krystal Biotech BLA for Vyjuvek, a herpes-simplex virus Type 1 vector-based gene therapy for treating wounds in patients six months of age and older with dystrophic epidermolysis bullosa (DEB). The topical gene therapy is designed to treat DEB at the molecular level by providing the patient’s skin cells with two copies of the COL7A1 gene to make functional COL7 protein, which addresses the underlying disease-causing mechanism, according to the company.

“When COL7A1 is deficient, skin layers can separate, causing painful and debilitating blisters and wounds,” an [FDA release](https://www.fda.gov/news-events/press-announcements/fda-approves-first-topical-gene-therapy-treatment-wounds-patients-dystrophic-epidermolysis-bullosa?ref=fdaweb.com) says. “DEB usually presents itself at birth and is divided into two major types depending on the inheritance pattern: recessive dystrophic epidermolysis bullosa (RDEB) and dominant dystrophic epidermolysis bullosa (DDEB).”

Approval was based on data from a randomized, double-blinded, placebo-controlled study involving 31 subjects with DEB, FDA says. “Efficacy was established by improved wound healing, defined as the difference in the proportion of confirmed complete (100%) wound closure between the Vyjuvek-treated and the placebo-treated wounds at 24 weeks,” it says adding that 65% of the Vyjuvek-treated wounds completely closed while only 26% of the placebo-treated wounds did.