Research Portfolio
Key
Gene therapy and 
Gene editing
Cell therapy
Drug therapy
Protein therapy
EB cancer therapy
Research
Ocular stem cell regeneration
Stem cell regeneration of the ocular surface in Recessive Dystrophic Epidermolysis Bullosa
RDEB SCC gene sequencing
Deep sequencing Recessive Dystrophic Epidermolysis Bullosa squamous cell carcinoma
Lenticol M
Mensenchymal stromal cells engineered to express Collagen VII for the treatment of Recessive Dystrophic Epidermolysis Bullosa
Lenticol F
Phase I study of lentiviral-mediated COL7A1 gene-modified autologous fibroblasts in adults with Recessive Dystrophic Epidermolysis Bullosa
ADSTEM
A phase I/II study to evaluate allogeneic mesenchymal stromal cells for the treatment of skin disease in adults with Recessive Dystrophic Epidermolysis Bullosa
Limbal stem cells
Limbal stem cells for treatment of corneal wounds in Epidermolysis Bullosa
TALEN-based gene editing
A TALEN-based approach to developing safer, more effective treatments for people with Epidermolysis Bullosa
EBSTEM
Clinical trial of mesenchymal stromal cells in Recessive Dystrophic Epidermolysis Bullosa