Topical Gene Therapy for Dystrophic EB Shows Promise

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Topical Gene Therapy for Dystrophic EB Shows Promise
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Investigational topical treatment for dystrophic epidermolysis bullosa showed durable and statistically significant improvement. DermTwitter

INDIANAPOLIS – An investigational topical treatment for dystrophic epidermolysis bullosa known as beremagene geperpavec showed durable and statistically significant improvement in complete wound healing at 3 and 6 months compared with placebo, according to results from a small phase 3 study., encoding for type VII collagen and leading to skin fragility and wounds. No approved therapies are currently available. In the study, treatment was generally well tolerated.

The primary endpoint was complete wound healing at 6 months. Additional endpoints included complete wound healing at 3 months and change in pain associated with wound dressing changes. Of the total wounds that closed at 3 months, 67% of wounds treated with B-VEC were also closed at 6 months, compared with 33% of those treated with placebo . In other findings, a trend toward decreased pain was observed in wounds treated with B-VEC vs. those treated with placebo.

To date, all treatments for DEB"have been extremely labor intensive, including skin grafting and hospitalizations. It's a topical application that can be done in the office and potentially applied at home in the future. It's also durable. Not only are the [treated] areas closing, but they are staying closed.

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