FCX-013 Gene Therapy for Linear Scleroderma
FCX-013 Gene Therapy for Linear Scleroderma

FCX-013

At a Glance 

  • Chronic, localized autoimmune disease that causes debilitating linear scars and skin thickening that can lead to pain, reduced joint mobility and deformity
  • Initially targeting approximately 40,000 patients in the U.S. patients who have scleroderma over a major joint and exhibit severe joint pain1
  • Current treatments – systemic or topical corticosteroids, UVA light therapy and physical therapy – only address symptoms
  • First gene therapy to offer the potential to address the underlying cause of linear scleroderma by regulating collagen accumulation at the site of the localized disease

About Linear Scleroderma

Linear scleroderma, a form of localized scleroderma, is a chronic autoimmune skin disorder that manifests as excess production of extracellular matrix—specifically Type I collagen and Type III collagen—resulting in thickening of the skin and connective tissue. The localized areas of skin thickening may extend to underlying tissue and muscle in children which can impair growth and development. Lesions appearing across joints can be painful, impair motion and may be permanent.

Localized scleroderma is comprised of a number of forms or sub-types totaling slightly less than 200,000 people in the U.S.2 The U.S. population of patients who have linear scleroderma over a major joint and exhibit severe joint pain is estimated to be approximately 40,000.

Current treatments for linear scleroderma, including systemic or topical corticosteroids, UVA light therapy and physical therapy, only address the symptoms of the disorder.

LS clinical photo



About FCX-013

FCX-013 is an autologous fibroblast cell genetically modified to express a protein to breakdown excess collagen I and III at the site of the localized disease. FCX-013 incorporates Intrexon’s proprietary RheoSwitch Therapeutic System®, a biologic switch activated by an orally administered compound which allows us to control future protein expression once the initial fibrosis has been resolved. FCX-013 is currently in pre-clinical development for the treatment of linear scleroderma, a form of localized scleroderma.

The U. S. Food and Drug Administration has granted Orphan Drug Designation to FCX-013 for the treatment of localized scleroderma, which includes linear scleroderma. In addition, FCX-013 has been granted Rare Pediatric Disease Designation for the treatment of moderate to severe localized scleroderma, which includes the linear subtype. 

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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Explore Advocacy & Educational Resources


  1. The Scleroderma Foundation. “Localized Scleroderma” brochure, pages 4, 6-7: http://www.scleroderma.org/site/DocServer/Loaclized.pdf?docID=317; accessed 07/20/15.
  2. The Scleroderma Foundation. What is Scleroderma? www.sclerodermra.org; accessed 10/09/2014.

 

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