XON’s partner Fibrocell (FCSC) has completed dosing of first cohort in Phase I/II trial of FCX-007 gene therapy for treatment of recessive dystrophic epidermolysis bullosa (RDEB).
RDEB is the most severe form of dystrophic epidermolysis bullosa (DEB), a congenital, progressive, 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 COL7, 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 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. FCX-007 has been granted Orphan Drug, Rare Pediatric Disease and Fast Track Designations by the FDA.
The FDA has also granted Rare Pediatric Disease Designation to FCX-013, Fibrocell’s second gene therapy candidate with XON for the treatment of moderate to severe localized scleroderma — a chronic autoimmune disease characterized by thickening of the skin and connective tissue.
AquaBounty Technologies, a majority-owned subsidiary of XON has announced that it has entered into an agreement with Bell Fish Company to buy its fish-farming facility in Indiana, for $14 million in cash.
This will provide the company with a land-based, contained aquaculture system to grow AquAdvantage Salmon near major demand centers.