FCX-013 Gene Therapy for Localized Scleroderma
FCX-013 Gene Therapy for Localized Scleroderma

FCX-013

At a Glance 

  • Chronic autoimmune 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
  • Approximately 90,000 patients in the U.S. have moderate to severe localized scleroderma1
  • Current treatments include systemic or topical corticosteroids which target inflammation, UVA light therapy and physical therapy; there are few treatment options to address excessive collagen accumulation in the skin and connective tissue
  • First gene therapy to offer the potential to treat the excessive collagen deposition in the skin and soft tissue at the site of localized scleroderma lesions

About Localized Scleroderma

Localized scleroderma is a chronic autoimmune skin disorder that manifests as excess production of extracellular matrix, specifically collagen, resulting in thickening of the skin and connective tissue. Moderate to severe forms of localized scleroderma can result in significant morbidity, including pain, restricted motion, disfigurement and developmental issues. The localized areas of skin thickening may extend to underlying tissue and muscle. In children this can impair growth and development. Lesions appearing across joints can be painful, impair motion and may be permanent.

Localized scleroderma encompasses several subtypes – totaling slightly less than 200,000 people in the U.S.2 – which are classified based on the depth and pattern of the lesion(s). The moderate to severe forms of the disorder include any subtype that affects function or produces symptoms of discomfort, tightness and pain. The U.S. population of patients who are considered to have moderate to severe localized scleroderma is estimated to be approximately 90,000.

Current treatments for localized scleroderma include systemic or topical corticosteroids, UVA light therapy and physical therapy. There are few treatment options to address excessive collagen accumulation in the skin and connective tissue.

LS clinical photo



About FCX-013

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 Precigen’s proprietary RheoSwitch Therapeutic System® (RTS®), a biologic switch activated by an orally administered compound (Veledimex) 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 (Veledimex) to facilitate protein expression. Once the fibrosis is resolved, the patient will stop taking the oral compound which will control further MMP-1 production.

The U. S. Food and Drug Administration (FDA) has granted allowance of Fibrocell’s Investigational New Drug Application for FCX-013 to begin clinical trials for the treatment of moderate to severe localized scleroderma.

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.

How Does FCX-013 Gene Therapy Work?

Dermal fibroblasts are collected from the patient, cultured and genetically modified to regulate collagen accumulation and then injected at the site of the localized disease.

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  1. Leitenberger, et. al. Distinct autoimmune syndromes in morphea: a case study of 245 adult and pediatric cases. Arch Dermatol. 2009 May; 145(5):545-550.
  2. The Scleroderma Foundation. What is Scleroderma? www.sclerodermra.org; accessed 10/09/2014.

 

Photo: © 2015 American College of Rheumatology.  Used with permission.