Fibrocell and Intrexon Announce IND Filing of FCX-007 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Fibrocell Science, Inc.,

, an autologous cell and gene therapy company focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet needs, and Intrexon Corporation

, a leader in synthetic biology, today announced the submission of an Investigational New Drug (IND) Application with the U.S. Food and Drug Administration for FCX-007, Fibrocell's lead orphan gene-therapy drug candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a congenital, progressive, devastatingly painful and debilitating genetic disorder that often leads to death. It is caused by a mutation of the COL7A1 gene, the gene which encodes for type VII collagen (COL7), a protein that forms anchoring fibrils. Anchoring fibrils hold together the layers of skin, and without them, 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. FCX-007 is a genetically-modified autologous fibroblast that encodes COL7. In addition to our recent announcement regarding the preliminary toxicology results using FCX-007 cells in a human skin graft model which demonstrated no findings of toxicology in RDEB human skin xenograft severe combined immunodeficiency (SCID) mice, Fibrocell and Intrexon announced additional positive proof-of-concept data from in vivo pre-clinical studies that showed: the presence of COL7 in the dermal-epidermal junction of the RDEB cultured grafts in RDEB human skin xenograft SCID mice; and no apparent systemic distribution of the vector in normal human skin xenograft SCID mice. "Filing the IND is a significant milestone for RDEB patients, their families and their caregivers as we advance FCX-007 toward human clinical trials," said David Pernock, Chairman and Chief Executive Officer of Fibrocell. "FCX-007 is a personalized gene therapy that offers the potential to treat the underlying cause of RDEB and give hope to patients suffering from this devastating disease." "The combination of Intrexon's biological engineering and Fibrocell's autologous fibroblast platform has led to a promising genetically-modified cellular therapy to potentially address unmet needs of RDEB patients," added Suma Krishnan, Senior Vice President, Product Development and Head of Intrexon's Human Therapeutics Division. "Moving forward into the clinic is another achievement in the development of FCX-007, and we are pleased to be one step closer in realizing the potential of this powerful therapeutic candidate." Fibrocell expects to initiate a Phase I/II clinical trial by year-end to evaluate the safety, mechanism of action, and efficacy of FCX-007.