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Fibrocell (FCSC) and partner Intrexon (XON) must conduct toxicology-specific study of FCX-007 in non-grafted mice model of severe combined immunodeficiency

September 25, 2015 9:18am

An FDA slap on the wrist! The new toxicology study should be initiated in Q4/15. FCSC closed at $6.49 – SELL, XON still a BUY

The Bottom Line: The status of the previously filed IND application for FCX-007, FCSC's orphan gene-therapy drug candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) included feedback from the U.S. FDA on the IND – we need more toxicology!

FCSC expects to amend its IND in response to FDA feedback and include data from new toxicology study in Q1/16. Then, FCSC plans to start P1/2 clinical trial … now in Q2/16 – if accepted!

Recessive dystrophic epidermolysis bullosa (RDEB) is the most severe form of dystrophic epidermolysis bullosa (DEB), a congenital, progressive, devastatingly painful and debilitating genetic disorder that often leads to death. RDEB is caused by a mutation of the COL7A1 gene, the gene which encodes for type VII collagen, a protein that forms anchoring fibrils. Anchoring fibrils hold together the layers of skin, and without them, skin layers separate causing severe blistering, open wounds and scarring in response to any kind of friction, including normal daily activities like rubbing or scratching. Children who inherit the condition are often called "butterfly children" because their skin is as fragile as a butterfly's wings. We estimate there are approximately 1,100 -- 2,500 RDEB patients in the U.S. Currently, treatments for RDEB address only the sequelae, including daily bandaging, hydrogel dressings, antibiotics, feeding tubes and surgeries.

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