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GMEB-SASS
- Development stage
- Phase 2
- Lead developer
- CHU de Québec-Université Laval
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Topical
01
Overview
GMEB-SASS (Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute) is an investigational autologous, tissue-engineered skin substitute developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The therapy consists of a bilayered skin graft produced from the patient's own keratinocytes and fibroblasts, which are genetically modified ex vivo using a retroviral vector to express the COL7A1 gene. This gene encodes type VII collagen, the protein responsible for forming anchoring fibrils that secure the epidermis to the dermis. In RDEB, mutations in COL7A1 lead to skin fragility and severe blistering; GMEB-SASS aims to permanently restore skin integrity by replacing damaged areas with healthy, collagen-producing skin cells.
- Other names
- Genetically Modified Epidermolysis Bullosa Self-Assembled Skin SubstituteGMEB-SASS graft
02
Targets
COL7A1 (Collagen type VII alpha 1 chain)
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