Fibrocell Science, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted allowance of its Investigational New Drug (IND) Application for FCX-013 one of the company's gene therapy candidates to begin clinical trials for the treatment of moderate to severe localized scleroderma. Localized scleroderma is a chronic autoimmune skin disorder characterized by excessive collagen deposition resulting in thickening of the dermis and underlying tissue. Moderate to severe forms of localized scleroderma 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 (veledimex) to control protein expression. Fibrocell expects to initiate enrollment for an open label, single arm Phase 1/2 clinical trial in the third quarter of 2018. The primary objective of the trial is to evaluate the safety of FCX-013. Secondary analyses consist of several fibrosis assessments including histology, skin scores, ultrasound and additional measurements of targeted sclerotic lesions and control sites at various time points up to 16 weeks post-administration of FCX-013. Ten patients with any subtype of localized scleroderma are targeted for enrollment (approximately 5 patients per Phase). The Phase 1 portion will enroll adult patients, and dosing for the first three adult patients will be staggered prior to dosing the rest of the trial's population. Fibrocell intends to include pediatric patients in the Phase 2 portion of the trial after submission and approval of safety and activity data from the adult Phase 1 patients to the FDA and the Data Safety Monitoring Board for the trial.