FDA Approves Topical Gene Therapy for Treatment of Skin Condition Causing Persistent Wounds
TL;DR Summary
The FDA has approved Vyjuvek, a topical gene therapy 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. DEB is a rare genetic skin disorder that causes painful and debilitating blisters and wounds. Vyjuvek is a genetically modified herpes-simplex virus used to deliver normal copies of the COL7A1 gene to the wounds. The safety and effectiveness of Vyjuvek was established in a randomized, double-blinded, placebo-controlled study involving a total of 31 subjects with DEB.
- FDA Approves First Topical Gene Therapy for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa FDA.gov
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