On May 9, 2017 Fibrocell Science, Inc. (NASDAQ: FCSC), a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, and Intrexon Corporation (NYSE: XON), a leader in synthetic biology, reported that two abstracts will be presented in oral sessions at the 20th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) from May 10-13, 2017 in Washington, D.C (Press release, Intrexon, MAY 9, 2017, View Source [SID1234518983]). Schedule your 30 min Free 1stOncology Demo! The schedule for each presentation is as follows:
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Session: Pharmacology, Toxicology and Assay Development
Presentation: Pre-Clinical Development of a Genetically-Modified Human Dermal Fibroblast (FCX-007) for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Presenter: Anna Malyala, PhD, Director, New Product Development, Fibrocell Science
Abstract ID: 301
Presentation on Thursday, May 11, 2017, 4:45 – 5:00 p.m. ET
Room: Maryland ABC Room
Session: Somatic Stem Cell Therapies
Presentation: Development of an Autologous Gene-Modified Cell Therapy for the Treatment of Linear Scleroderma
Presenter: Darby Thomas, PhD, Director, Rare Diseases, Human Therapeutics Division, Intrexon Corporation
Abstract ID: 740
Presentation on Saturday, May 13, 2017, 11:00 – 11:15 a.m. ET
Location: Delaware AB Room
About FCX-007
FCX-007 is Fibrocell’s clinical-stage, gene therapy product 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 the gene for COL7 and is being developed in collaboration with Intrexon Corporation. By genetically modifying autologous fibroblasts ex vivo to produce COL7, culturing them and then treating 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 while avoiding systemic distribution. The FDA has granted Orphan Designation to FCX-007 for the treatment of Dystrophic Epidermolysis Bullosa, which includes RDEB. In addition, FCX-007 has been granted Rare Pediatric Disease Designation and Fast Track Designation by the FDA for treatment of RDEB.
About FCX-013
Fibrocell is in pre-clinical development of FCX-013, its gene therapy candidate for the treatment of linear scleroderma, a form of localized scleroderma. FCX-013 incorporates Intrexon’s proprietary RheoSwitch Therapeutic System, a biologic switch activated by an orally administered compound to control future protein expression once the initial fibrosis has been resolved. FCX-013 is designed to be injected under the skin at the location of the fibrosis where the genetically-modified fibroblast cells will produce a protein to break down excess collagen accumulation. The patient takes an oral compound to facilitate protein expression. Once the fibrosis is resolved, the patient will stop taking the oral compound which will stop further production of the subject protein by FCX-013. Fibrocell has received Orphan Drug Designation from the FDA for FCX-013 for the treatment of localized scleroderma.