dabocemagene autoficel

Dabocemagene autoficel is an ex vivo, autologous cell-based gene therapy developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disorder caused by mutations in the COL7A1 gene. The product consists of a patient’s own dermal fibroblasts that are genetically modified using a lentiviral vector to express functional type VII collagen (COL7). These corrected cells are injected into chronic or non-healing wounds, where they produce COL7 protein to restore anchoring fibrils at the dermal–epidermal junction and promote wound healing. Dabocemagene autoficel has received orphan drug, rare pediatric disease, fast track, and regenerative medicine advanced therapy designations from the FDA. It is currently in phase 3 clinical trials for RDEB[1][2][3][4][5][6][7][8].

Brand names

D-Fi

Other names

Genetically modified human dermal fibroblast-collagen VIIGenetically-modified autologous human dermal fibroblastsGM-HDF-COL7GM-HDF-COL-7GM-HDF-COL 7Fibroblast epidermolysis bullosa cell therapy - Fibrocell/IntrexonFibroblast epidermolysis bullosa dystrophica cell therapy - Fibrocell/IntrexonFibroblast epidermolysis bullosa dystrophica gene therapy - Fibrocell/IntrexonFibroblast epidermolysis bullosa gene therapy -Fibrocell/IntrexonFibroblast-RDEB-cell-therapy-Fibrocell/IntrexonFibroblast-RDEB-gene therapy-Fibrocell/Intrexon