Fibrocell fibroblasts get orphan drug status

The FDA designates Fibrocell's (FCSC) genetically-modified autologous human fibroblasts an orphan drug to treat dystophic epidermolysis bullosa (DEB) including recessive DEB (RDEB).

There are between 2,800 and 5,600 RDEB patients in the U.S. They usually do not live beyond 30 years of age. There is no cure for the disease. The only treatment is basic wound care.

Fibrocell combines its fibroblasts with Intrexon's (XON) UltraVector synthetic biology platform in a therapy that inserts the gene that encodes functional collagen VII into the patient's own fibroblasts. In RDEB sufferers, the fibroblasts lack the ability to produce functional collagen VII, the essential protein that forms anchoring fibrils responsible for attaching the layers of the skin together.

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