Rare Skin Disorder Therapy Designated Orphan Drug
Fibrocell Science announced that the FDA has granted Orphan Drug designation to its genetically-modified autologous human fibroblasts for the treatment of dystrophic epidermolysis bullosa (DEB), a broad category of rare and severe genetic skin diseases that includes recessive DEB.
The genetically-modified fibroblasts combine Fibrocell's autologous fibroblast technology with Intrexon Corporation's UltraVector synthetic biology platform to target the source of this genetic disease that causes severe blistering and areas of missing skin.
Fibrocell Science plans on filing an Investigational New Drug application (IND) in the first half of 2015 and initiating a Phase 1 trial as well.
For more information visit FibrocellScience.com.