EXTON, Pa. & GERMANTOWN, Md.—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, which hold together the layers of skin. 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 the companies’ 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 CEO 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 1/2 clinical trial by year-end to evaluate the safety, mechanism of action, and efficacy of FCX-007.
FCX-007 is Fibrocell's novel gene-therapy drug candidate for the treatment of RDEB, a
congenital and progressive orphan skin disease caused by the deficiency of the protein type VII collagen (COL7). FCX-007 is a genetically modified autologous fibroblast that encodes COL7 and is being developed in collaboration with Intrexon. By genetically modifying autologous fibroblasts, ex vivo, to produce COL7, culturing them and then treating blisters and wounds locally via injection, FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas, avoiding systemic treatment.
RDEB is the most severe form of dystrophic epidermolysis bullosa (DEB), a congenital, progressive, devastatingly painful and debilitating genetic disorder that often leads to death. RDEB is caused by a mutation of the COL7A1 gene, the gene which encodes for type VII collagen, 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. Children who inherit the condition are often called "butterfly children" because their skin is as fragile as a butterfly's wings. There are approximately 1,100 - 2,500 RDEB patients in the U.S. Currently, there is no cure for RDEB and treatments address only the sequelae, including daily bandaging, hydrogel dressings, antibiotics, feeding tubes and surgeries.
Fibrocell is 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 medical needs. Fibrocell's most advanced drug candidate, azficel-T, uses its proprietary autologous fibroblast technology and is in a Phase 2 clinical trial for the treatment of chronic dysphonia resulting from vocal cord scarring. In collaboration with Intrexon, a leader in synthetic biology, Fibrocell is also developing gene therapies for orphan skin diseases using gene-modified autologous fibroblasts. The company has filed an IND for FCX-007, its lead orphan gene-therapy drug candidate, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is in pre-clinical development of FCX-013, its second gene-therapy drug candidate, for the treatment of linear scleroderma.
Intrexon Corporation creates biologically-based products to improve the quality of life and the health of the planet. The company's integrated technology suite provides its partners across diverse markets with industrial-scale design and development of complex biological systems delivering enhanced control, quality, function, and performance of living cells.
