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Vyjuvek

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health2 years ago•1 min saved

FDA approves groundbreaking gene therapy for rare skin condition.

The FDA has approved Vyjuvek, a redosable gene therapy made by Krystal Biotech, for patients with dystrophic epidermolysis bullosa, a genetic disease that causes painful blisters and persistent wounds. The topical treatment delivers a healthy copy of the gene that encodes the protein type VII collagen to the targeted skin cells, enabling healing to occur. Patients with the condition live covered in bandages to try to stop new blisters from forming and to dress their wounds.

via STAT|

#fda #gene-therapy #health

healthcare2 years ago•1 min saved

FDA approves groundbreaking rub-on gene therapy for rare skin disorder.

The US FDA has approved Krystal Biotech's Vyjuvek, a topical gene therapy for patients with dystrophic epidermolysis bullosa, a rare genetic skin disorder. The therapy is expected to be available in the US in Q3 2023 and is Krystal's first to be approved in the country. The FDA approval is supported by data from early-to-mid-stage and late-stage studies, which showed Vyjuvek completely healed wounds in about 65% of the participants.

via Reuters|

#dystrophic-epidermolysis-bullosa #fda #gene-therapy

health2 years ago•3 min saved

FDA Approves Topical Gene Therapy for Treatment of Skin Condition Causing Persistent Wounds

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.

via FDA.gov|

#col7a1 #dystrophic-epidermolysis-bullosa #fda