Gene Therapy Fast Tracked for Rare Congenital Skin Disorder

Recessive Dystrophic Epidermolysis Bullosa is a rare, congenital, debilitating genetic skin disorder
Recessive Dystrophic Epidermolysis Bullosa is a rare, congenital, debilitating genetic skin disorder

Fibrocell Science announced that the Food and Drug Administration (FDA) has granted Fast Track designation to FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB).

RDEB is a rare, congenital, debilitating genetic skin disorder that presents as severe painful blisters, open wounds, and scarring due to the deficiency of the protein type VII collagen (COL7). It is a progressive disease that often leads to death. There are currently no pharmacologic treatments for RDEB; treatment is limited to symptomatic therapies such as daily bandaging, hydrogel dressings, antibiotics, feeding tubes and surgeries. 

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Fibrocell, in collaboration with Intrexon Corporation, is currently investigating FCX-007 in a Phase 1/2 trial for the treatment of RDEB. It was previously granted Orphan Drug designation for the treatment of Dystrophic Epidermolysis Bullosa (DEB) and Rare Pediatric Disease designation for RDEB.

FCX-007, a genetically-modified autologous fibroblast that encodes the gene for COL7, is being developed as an injectable for direct administration to the affected areas, thus avoiding systemic distribution.

For more information visit Fibrocell.com.

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