On May 28, 2015 Fibrocell Science, Inc., (Nasdaq:FCSC), 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, and Intrexon Corporation (NYSE:XON), a leader in synthetic biology, together reported that a poster will be presented highlighting in vitro pre-clinical data for FCX-007, a gene-therapy drug candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), at the 2015 European Society of Human Genetics (ESHG) Annual Meeting in Glasgow, Scotland, United Kingdom from June 6-9, 2015 (Press release, Intrexon, MAY 28, 2015, View Source;p=irol-newsArticle&ID=2054246 [SID:1234506593]).
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John Maslowski, Vice President of Scientific Affairs at Fibrocell, will present the poster highlighting FCX-007, which is in development for RDEB, a congenital, orphan skin disease caused by the deficiency of the protein collagen VII (COL7). FCX-007 is a gene-modified autologous fibroblast that encodes for COL7, and is being developed in collaboration with Intrexon.
The details of the poster presentation session are as follows:
Session: PM04. Skeletal, connective tissue, ectodermal and skin disorders
Title: Development of a Genetically-Modified Human Dermal Fibroblast for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Poster Board #: PM04.60
Date: Monday, June 8, 2015
Time: 10:30 – 11:30 a.m. GMT
Location: Exhibition Hall at Scottish Exhibition & Conference Center
About FCX-007
FCX-007 is Fibrocell’s novel gene-therapy drug candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a congenital and progressive orphan skin disease caused by the deficiency of the protein type VII collagen (COL7). FCX-007 is a gene-modified autologous fibroblast that encodes for 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. The drug is currently in late stage pre-clinical development with an IND filing targeted for mid-2015.
About Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Recessive dystrophic epidermolysis bullosa (RDEB) is the most severe form of dystrophic epidermolysis bullosa (DEB), a congenital, progressive, devastatingly painful and debilitating genetic disorder that leads to death. 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. 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.
About Fibrocell Science, Inc.
Fibrocell Science, Inc. (Nasdaq:FCSC) 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 FDA-approved proprietary autologous fibroblast technology and is in a Phase II clinical trial for the treatment of chronic dysphonia resulting from vocal cord scarring or atrophy. In collaboration with Intrexon Corporation (NYSE:XON), a leader in synthetic biology, Fibrocell is also developing gene therapies for orphan skin diseases using gene-modified autologous fibroblasts. The Company’s lead orphan gene-therapy drug candidate, FCX-007, is in late stage pre-clinical development for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is also in pre-clinical development of FCX-013, its second gene-therapy drug candidate, for the treatment of linear scleroderma. For more information, visit www.fibrocellscience.com.