Model BBP-589 - Recombinant Collagen 7 For Dystrophic Epidermolysis Bullosa (Rdeb)

BridgeBio is working to create a potential first-ever systemic treatment for dystrophic epidermolysis bullosa (DEB), a disease caused by loss of function of Collagen 7 (C7) protein. PTR-01 is a C7 protein replacement therapy designed to restore the missing collagen protein (C7) at the root of the disease. Mutations in the gene that encodes C7 enable the epidermis to separate from the dermis. Even minor friction or trauma can cause debilitating blistering, tearing and scarring of the skin, along with severe pain and itching. The disease can also affect the lining of the mouth, esophagus, eye, anus and vagina. BridgeBio completed a Phase 2 clinical trial evaluating safety and tolerability of PTR-01, as well as clinical proof of concept, and has initiated a Phase 2 extension study.