UK Approves First Gene Therapy Gel for Rare Skin Disorder
The UK has approved the first gene therapy gel for treating dystrophic epidermolysis bullosa (DEB), a rare skin disorder. The gel, known as beremagene geperpavec, is applied weekly to wounds and promotes healing by delivering the necessary gene instructions. This approval marks a significant advancement in treatment options for patients with this condition.
- ▪Beremagene geperpavec is the first gene therapy approved for DEB at any age.
- ▪In clinical trials, 67% of wounds treated with the gel were completely healed after six months compared to 22% for placebo.
- ▪The treatment uses a modified herpes simplex virus to deliver genetic material without permanently altering DNA.
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The Medicines and Healthcare products Regulatory Agency (MHRA) has approved beremagene geperpavec (Vyjuvek, Krystal Biotech), a topical gene therapy for wounds in patients with dystrophic epidermolysis bullosa (DEB). It is the first such treatment approved for the condition at any age.Beremagene geperpavec is a gel, applied once a week directly to wounds, and can be used from birth onwards. It stimulates production of a missing protein which helps bind the inner and outer layers of the skin together, promoting wound healing and reducing the risk of further skin damage.
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Excerpt limited to ~120 words for fair-use compliance. The full article is at Medscape.
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