Fibrocell and Intrexon: Highlights of Pre-Clinical Data for Potential First-in-Class Treatment for Recessive Dystrophic Epidermolysis Bullosa

Fibrocell Science, Inc., and Intrexon Corporation are highlighting a poster entitled “Pre-Clinical Development of a Genetically-Modified Human Dermal Fibroblast (FCX-007) for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)” being presented at the American Society of Human Genetics Annual Meeting from October 6-10, 2015 in Baltimore, MD. The poster contains additional details of the previously reported positive in vivo pre-clinical data for FCX-007, Fibrocell’s orphan gene-therapy product candidate for the treatment of RDEB.

RDEB is caused by a mutation of the COL7A1 gene—the gene which encodes for type VII collagen (COL7), a protein that forms anchoring fibrils to hold together the layers of skin. Without these fibrils, skin layers separate causing severe blistering, open wounds and scarring in response to any kind of friction, including normal daily activities like rubbing or scratching.

In this in vivo pre-clinical study, FCX-007 was evaluated for toxicology and biological proof-of-concept in RDEB and normal human skin xenografts implanted onto severe combined immunodeficiency (SCID) mice. Toxicology results for FCX-007 showed at two- and six-weeks post-administration:

• No test article-related findings;
• No tumors in the skin grafts or other organs;
• No statistical changes in blood chemistry; and
• No apparent systemic distribution of the vector.

In addition, data supporting the biological proof-of-concept indicated FCX-007 cells in a human skin xenograft model expressed COL7 that localized to the basement membrane zone (BMZ) where anchoring fibrils are formed.

The poster will be available in the “Publications” section of Fibrocell’s website at: http://www.fibrocellscience.com/technology/publications/.