Fibrocell Science Rings NASDAQ Bell, Investigates New Drug


Its been a busy week for the people at Fibrocell, the Exton based autologous cell therapy company focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases.  On Tuesday senior management took a trip to New York City and rang NASDAQ’s opening bell (above).

Then on Wednesday the company announced they and Intrexon Corp. are investigating a new drug – GM-HDF-COL7 – which could potentially treat recessive dystrophic epidermolysis bullosa, the most severe form of the rare connective tissue disorder dpidermolysis bullosa.

The companies are filing an investigational application with the U.S. Food and Drug Administration and are beginning clinical material manufacturing this month.  Intrexon is based in Germantown, Md.

RDEB is an extremely debilitating genetic disorder that causes severe blistering and areas of missing skin when a mutation or error happens within the collagen VII gene. RDEB has the highest rate of morbidity and mortality of all the genetic blistering disorders and is often lethal before the age of 30. So far, there is no cure or approved treatment.

“Fibrocell continues to focus its autologous fibroblast product pipeline on genetically-based orphan skin diseases for which there is a significant unmet need for treatments, and where our approach with Intrexon for creating personalized biologics provides distinctive advantages,” said David Pernock, Fibrocell’s chairman and CEO.

For those interested in the technical aspects of synthetic biology and the new drug (also known as genetically-modified human dermal fibroblast, collagen VII) see this webcast of the company’s recent research and development day.

Fibrocell is also pursuing medical applications for azficel-T—the company’s proprietary autologous fibroblast technology—for restrictive burn and vocal cord scarring.

Intrexon Corp. calls its synthetic biology approach and integrated technologies Better DNA®.

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