Gene Therapy to Treat Localized Scleroderma скачать в хорошем качестве

Gene Therapy to Treat Localized Scleroderma

At the recent BIO International Convention held in Phildelphia, PA, we talked with John Maslowski, President and Chief Executive Officer (CEO) of Fibrocell Science, about his company’s gene therapy pipeline, including a therapy to treat localized scleroderma. Localized scleroderma is an autoimmune skin disorder that results in excess production of collagen, that in turn, causes the thickening of the skin and connective tissue. Maslowski said “I think we are all familiar with systemic scleroderma, which has absolutely, fatal and deliberating effects on the body. There is scleroderma of the skin. And by skin, it actually can go deeper than that. It can affect down to the muscle layer and even to the fascia. It can be very painful and have high morbidity.” “These are not just mild cosmetic disorders,” noted Maslowski, but conditions that can dramatically affect growth and development, especially in children. Currently, there is no approved therapy for localized scleroderma. Maslowski noted that several medications, including various corticosteroids, have been investigated for this condition but to date, no therapy has been approved. FCX-013 is a gene therapy that produces excess metalloproteinase 1, a protein that can help break down collagen. The company has begun a clinical trial with the gene therapy. The first, phase 1 study is in adults and the company hopes to include children when they begin the phase 2 clinical trial. For more information, fibrocell.com.