FCX-013 + veledimex

FCX-013 + veledimex is an investigational, two-component combination gene therapy for the treatment of moderate to severe localized scleroderma (morphea)[5][7][1]. FCX-013 consists of autologous dermal fibroblasts genetically modified ex vivo to express matrix metalloproteinase-1 (MMP-1), an enzyme that breaks down excess collagen. After local intradermal injection of FCX-013 into fibrotic skin lesions, patients receive oral veledimex, a small molecule activator ligand, which facilitates the regulated expression of MMP-1 by the modified fibroblasts through an inducible gene switch system (ecdysone receptor-based system). Veledimex is stopped when desired antifibrotic effect is achieved, as it controls transgene expression, providing a safety switch for MMP-1 production[1][3][5][7].

Other names

FCX-013FCX013FCX 013veledimex