IND Filed for Gene Therapy to Treat Scleroderma

Fibrocell Science has submitted an Investigational New Drug Application (IND) with the FDA for a gene therapy candidate to treat moderate to severe localized scleroderma.

Localized scleroderma, a chronic, immune-mediated skin disorder that affects approximately 90,000 US patients, typically begins between the ages of 20 to 50. The disease manifests as excess collagen production that results in fibrosis of the skin and connective tissue. Moderate to severe forms of scleroderma can result in significant morbidity.

Fibrocell’s proposed therapy, FCX-013, is an autologous fibroblast that has been genetically modified and encoded for matrix metalloproteinase 1 (MMP-1), a protein that is responsible for breaking down collagen. FCX-013 is designed for subcutaneous injection at the location of the fibrotic lesions, where the genetically modified fibroblast cells will produce MMP-1 to break down accumulated collagen. At the same time, the patient will take an oral compound that will help facilitate MMP-1 expression. Once the fibrosis has resolved, the patient will cease taking the oral compound.

If eventually approved, FCX-013 will provide a new therapeutic option for patients with scleroderma. Currently available treatments are symptomatic and supportive in nature; systemic or topical corticosteroids may be used, and light therapy or physical therapy may be employed. Medications that help to control fibrosis include D-penicillamine and cholchicine, and angiotensin-converting enzyme inhibitors may be used to treat kidney disease associated with scleroderma. The localized administration of FCX-013 could help patients avoid the adverse effects that are associated with systemic therapy.

Fibrocell is also working on gene therapies to target other disease that affect the skin and connective tissue. The most advanced product candidate in the company’s pipeline is FCX-007, which is in phase 1 and 2 clinical development for the treatment of recessive dystrophic epidermolysis bullosa, a disorder characterized by widespread blistering of the skin that may lead to scarring. The company is also in the research phase for potential gene therapies to target arthritis and related conditions.