Fibrocell Science, a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, announced the submission of an Investigational New Drug (IND) Application with the US Food and Drug Administration (FDA) for FCX-013, the company’s gene therapy candidate for the treatment of moderate to severe localized scleroderma.
Localized scleroderma is a chronic autoimmune skin disorder that manifests as excess production of collagen resulting in thickening, or fibrosis, of the skin and connective tissue. Moderate to severe forms of localized scleroderma, including the linear subtype, can result in significant morbidity, including pain, restricted motion, disfigurement and developmental issues. FCX-013 is an autologous fibroblast genetically modified to express matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down collagen. FCX-013 incorporates Intrexon Corporation’s proprietary RheoSwitch Therapeutic System, a biologic switch activated by an orally administered compound to control protein expression once the initial fibrosis has been resolved.
“We are pleased to announce the submission of the IND as it represents a significant milestone in advancing development of FCX-013 for the treatment of moderate to severe localized scleroderma,” said John Maslowski, president and chief executive officer of Fibrocell. “Adequate treatment options do not exist for this vulnerable patient population. We are committed to developing our novel gene therapy candidate to address this important unmet medical need of patients suffering from this chronic, painful and debilitating disorder.”
The FDA has granted Orphan Drug Designation to FCX-013 for the treatment of localized scleroderma. In addition, FCX-013 has been granted Rare Pediatric Disease Designation for the treatment of moderate to severe localized scleroderma.
Fibrocell is in pre-clinical development of FCX-013, its gene therapy candidate for the treatment of moderate to severe localized scleroderma. FCX-013 is an autologous fibroblast genetically modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down collagen. FCX-013 incorporates Intrexon’s proprietary RheoSwitch Therapeutic System, a biologic switch activated by an orally administered compound to control protein expression at the site of the localized scleroderma lesions. FCX-013 is designed to be injected under the skin at the location of the fibrotic lesions where the genetically-modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation. With the FCX-013 therapy, the patient will take an oral compound to facilitate protein expression. Once the fibrosis is resolved, the patient will stop taking the oral compound which will halt further MMP-1 production.
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