Beremagene geperpavec-svdt (VYJUVEK™) is a topically applied, redosable, live, replication defective herpes simplex virus-1 (HSV-1) vector -based gene therapy that is being developed by Krystal Biotech to deliver functional human collagen type VII alpha 1 chain (COL7A1) genes in patients with both, dominant and recessive dystrophic epidermolysis bullosa. Beremagene geperpavec can transduce both keratinocytes and fibroblasts and restore functional COL7 protein. In May 2023, beremagene geperpavec received its first approval in the US for the treatment of wounds in patients ≥ 6 months of age with dystrophic epidermolysis bullosa with mutation(s) in the COL7A1 gene. A Marketing Authorization Application for beremagene geperpavec in Europe is planned for the second half of 2023. This article summarizes the milestones in the development of beremagene geperpavec leading to this first approval for dystrophic epidermolysis bullosa.
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Estimating Costs in Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa-Reply.
JAMA Dermatol
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Program on Regulation, Therapeutics, and Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Boston, Massachusetts.
Estimating Costs in Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa.
JAMA Dermatol
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Faculty of Medicine and Health, University of New South Wales, New South Wales, Australia.
Practical considerations relevant to treatment with the gene therapy beremagene geperpavec-svdt for dystrophic epidermolysis bullosa.
J Dermatolog Treat
December 2024
Department of Dermatology, Stanford University School of Medicine, Stanford, California, USA.
Background/purpose: Dystrophic epidermolysis bullosa (DEB), a rare genetic skin disease caused by loss-of-function mutations in , the gene encoding type VII collagen (COL7), is characterized by skin blistering, scarring, and extracutaneous manifestations that markedly reduce patient quality-of-life. Beremagene geperpavec-svdt ('B-VEC') is a gene therapy employing a non-integrating, replication-defective herpes simplex virus type 1 (HSV-1)-based vector encoding two copies of full-length human to restore COL7 protein after topical administration to DEB wounds. B-VEC was approved in the United States in 2023 as the first topical gene therapy and the first approved treatment for DEB.
View Article and Find Full Text PDFEmerging Gene Therapeutics for Epidermolysis Bullosa under Development.
Int J Mol Sci
February 2024
EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University, 5020 Salzburg, Austria.
The monogenetic disease epidermolysis bullosa (EB) is characterised by the formation of extended blisters and lesions on the patient's skin upon minimal mechanical stress. Causal for this severe condition are genetic mutations in genes, leading to the functional impairment, reduction, or absence of the encoded protein within the skin's basement membrane zone connecting the epidermis to the underlying dermis. The major burden of affected families justifies the development of long-lasting and curative therapies operating at the genomic level.
View Article and Find Full Text PDFOcular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa.
N Engl J Med
February 2024
From the Bascom Palmer Eye Institute, University of Miami Miller School of Medicine, Miami (A.T., I.S.-A., J.G., A.L.S.); and Krystal Biotech, Pittsburgh (H.C., B.A., K.C., T.P., S.K.).
Dystrophic epidermolysis bullosa is a rare genetic disease caused by damaging variants in , which encodes type VII collagen. Blistering and scarring of the ocular surface develop, potentially leading to blindness. Beremagene geperpavec (B-VEC) is a replication-deficient herpes simplex virus type 1-based gene therapy engineered to deliver functional human type VII collagen.
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