Potential Scleroderma Gene Therapy Moving into Preclinical Dosing Study, Fibrocell Reports

Patrícia Silva, PhD avatar

by Patrícia Silva, PhD |

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scleroderma gene therapy development

Fibrocell Science, Inc., recently highlighted the latest advancements in its pipeline of products focused on autologous cell and gene therapy treatments for several conditions, including scleroderma.

The company’s autologous fibroblast technology uses a patented manufacturing process that involves collecting small skin biopsies from patients, separating the tissue into its component cells, and then expanding the fibroblast cells in culture. In this way, each patient is treated with cells cultivated from his or her own dermal tissue (i.e., autologous cells).

“Recently, Fibrocell has made significant progress across our pipeline of personalized biologics, positioning the company to achieve value-creating milestones in 2016,” said David Pernock, company chairman and chief executive officer, in a press release. “We are encouraged by this progress that brings us closer to delivering potentially transformative therapies for patients suffering from debilitating diseases of the skin and connective tissue.”

Fibrocell has finished a proof-of-concept trial on FCX-013 for the treatment of linear scleroderma. The trial examined the efficacy of FCX-013 in reducing dermal thickness in fibrotic tissue. FCX-013 is an autologous dermal fibroblast transduced with a gene to regulate collagen accumulation at the site of the localized disease.

FCX-013 was tested in a bleomycin-induced scleroderma mice model, and results revealed that it was able to reduce the fibrotic tissue dermal thickness to levels identical to non-bleomycin treated skin. Moreover, the compound was also found to reduce the thickness of the subdermal muscle layer.

FCX-013 will now advance into preclinical dose-ranging trials for optimization. The company expects to submit an Investigational New Drug (IND) application for FCX-013 to the U.S. Food and Drug Administration (FDA) in 2017.

Fibrocell also recently completed patient dosing in a Phase 2, double-blind, randomized, and placebo-controlled clinical trial assessing the safety and efficacy of azficel-T for the treatment of patients with vocal fold scarring resulting in chronic or severe dysphonia. The company is also conducting a toxicology study, previously requested by the FDA and related to an IND application for FCX-007, its gene therapy product for patients with recessive dystrophic epidermolysis bullosa.

FCX-007 and FCX-013 are being developed in alliance with Intrexon Corporation.