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FDA approves Krystal gene therapy for rare wound disorder

May 22, 2023

Dystrophic epidermolysis bullosa is one of four main types of the disorder. Depending on the inheritance pattern, the condition can be severe and disabling, causing blisters anywhere on the skin from minor trauma and friction.

The form Vyjuvek is cleared to treat is caused by mutations in a gene responsible for encoding collagen, a necessary protein that helps strengthen and stabilize the outer layers of skin.

Vyjuvek is designed to deliver functional copies of the gene, called COL7A1, directly into the wound via a modified herpes simplex virus type 1. The functional gene is meant to produce a type of collagen, thereby promoting wound healing.

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