pCCL-INVO-SP lentiviral vector

pCCL-INVO-SP is an investigational ex vivo gene therapy developed for the treatment of Netherton syndrome (NS), a rare and severe autosomal recessive skin disorder caused by mutations in the SPINK5 gene. The therapy utilizes a self-inactivating (SIN), HIV-1-based lentiviral vector to deliver a codon-optimized SPINK5 transgene, which encodes the serine protease inhibitor LEKTI (lymphoepithelial Kazal-type-related inhibitor). Expression of the transgene is driven by a human involucrin promoter (INVOp), ensuring compartment-specific expression in the differentiated layers of the epidermis. In this therapeutic strategy, autologous keratinocyte stem cells are harvested from the patient, transduced with the pCCL-INVO-SP vector, expanded into epithelial sheets, and subsequently grafted back onto the patient's skin to restore functional LEKTI expression and correct the serine protease dysregulation characteristic of NS.

Other names

pCCL-INVOp-SPINK5pCCL-INVOp-SPINK-5pCCL-INVOp-SPINK 5lentiviral SPINK5 gene-modified autologous keratinocyte epithelial sheets