Castle Creek begins gene therapy clinical trial for scleroderma

By The Science Advisory Board staff writers

October 15, 2020 -- Castle Creek Biosciences has initiated a phase I/II clinical trial evaluating FCX-013, the company's investigational gene therapy for the treatment of moderate to severe localized scleroderma.

FCX-013 is an autologous fibroblast genetically modified using lentivirus. It is encoded for matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down collagen. A biological switch is incorporated into the therapy to control protein expression at the site of the localized scleroderma lesion. FCX-013 is administered via intradermal injection.

The open-label, single-cohort trial will evaluate the safety of the candidate and will enroll up to 10 patients. It will also assess fibrosis at targeted sclerotic lesions at various time points through 26 weeks postadministration of FCX-013 at the location of the fibrotic lesions where the genetically modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation.

FCX-013 has been granted orphan drug, rare pediatric disease, and fast track designations by the U.S. Food and Drug Administration.

Localized scleroderma is a chronic autoimmune skin disorder that leads to the excess production of collagen and causes thickening of the skin and connective tissue. In the U.S., 500,000 individuals experience moderate to severe localized scleroderma.

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