贝林戈吉欧佩尔帕韦克 (Vyjuvek)

概览

Beremagene geperpavec is gene therapy used to treat wounds associated with dystrophic epidermolysis bullosa.

作用机制

Collagen VII (C7, COL7), encoded by the COL7A1 gene, is an anchoring fibril component that holds the epidermis and dermis together to maintain skin integrity. COL7 is synthesized by epidermal keratinocytes and dermal fibroblasts. Dystrophic epidermolysis bullosa (DEB) is an inherited disorder caused by COL7A1 gene mutations, leading to reduced or deficient levels of biologically active and functional COL7. Deficient levels of COL7 result in no functional anchoring fibrils and impaired dermal-epidermal adhesion. DEB is associated with blistering, wounding, and scarring of the skin and other organs starting at birth. Beremagene geperpavec is a herpes simplex virus type 1 (HSV-1)-based vector genetically modified to express COL7. Upon topical application to wounds, beremagene geperpavec enters both keratinocytes and fibroblasts and the vector genome is deposited in the nucleus. Once in the nucleus, transcription of the encoded human COL7A1 is initiated to produce and secrete COL7 by the cell in its mature form. COL7 molecules arrange themselves into long, thin bundles that form anchoring fibrils.

适应症

Beremagene geperpavec is indicated for the treatment of wounds in patients six months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

• Wound