Epidermolysis Bullosa (EB) is a group of rare and genetic skin disorders in which the skin is so fragile, even minor trauma or friction can have devastating results, causing severe pain, blistering, scarring, infections, chronic wounds and immobility. Patients can experience agonizing, traumatic and time-consuming daily dressing changes. Severe forms of EB cause disfigurement, disability and early death. Recessive Dystrophic (RDEB) is a form of dystrophic EB. RDEB is a monogenic disease resulting in chronic skin blistering and wounding. Marked by devastating, progressive, painful blistering, RDEB is diagnosed at infancy and accompanied by a high mortality rate – 76% do not live beyond their 30s. The cost of bandaging alone can exceed $10,000 per month. The cause of RDEB is a mutation in the COL7A1 gene that encodes for COL7. There is no cure or approved treatment for EB
Also formulated with the Invisicare® technology, QRX004 is a topical lotion initially under development as a potential treatment for Recessive Dystrophic Epidermolysis Bullosa (RDEB).
QRX004 contains two active ingredients. The primary ingredient induces a read-through of nonsense mutations and leads to creation of robust and sustained type VII collagen. The result is improved wound closure, reduced blistering and stronger skin.