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autologous COL7A1 gene-corrected skin equivalent graft
- Development stage
- Phase 1
- Lead developer
- Abrepa Pharmaceuticals
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Administration
- Topical
01
Overview
Autologous COL7A1 gene-corrected skin equivalent graft is an ex vivo gene therapy developed by the Institut National de la Santé et de la Recherche Médicale (INSERM) and Genethon for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a severe skin fragility disorder caused by mutations in the COL7A1 gene, which leads to a deficiency in type VII collagen (C7), the primary component of anchoring fibrils at the dermal-epidermal junction. The therapy involves harvesting keratinocytes and fibroblasts from the patient, transducing them with a lentiviral vector (pCCL-COL7A1-WPRE) carrying a functional COL7A1 cDNA, and expanding these cells to create a bilayered skin equivalent graft. This graft is then surgically applied to chronic wounds to restore C7 expression and anchoring fibril formation, thereby improving skin integrity and wound healing. It is currently being evaluated in Phase 1/2 clinical trials.
- Other names
- Autologous COL7A1-corrected skin graftsLentiviral-mediated COL7A1-corrected skin graftsautologous COL7A1 gene-corrected skin equivalent graft
02
Targets
COL7A1 (Collagen type VII alpha 1 chain)
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