Fibrocell Science, Inc. a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, announced that the remaining two patients in the NC1+ cohort have been dosed in the Phase 1 portion of the Phase 1/2 clinical trial of FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), a devastating genetic skin disease with a high mortality rate. The cohort consists of three patients.
Dosing additional patients follows the Data Safety Monitoring Board’s recommendation to continue the Phase 1/2 clinical trial of FCX-007 following a planned review of safety data from the first patient treated. No product-related adverse events were reported.
Twelve-week post-treatment data for safety, mechanism of action and efficacy for multiple patients in the Phase 1 portion of the trial are expected in the third quarter of 2017.
Fibrocell is developing FCX-007 in collaboration with Intrexon Corporation (NYSE:XON), a leader in synthetic biology.
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. FCX-007 has been granted Orphan Drug, Rare Pediatric Disease and Fast Track Designations by the U.S. Food and Drug Administration (FDA).
