In patients with recessive dystrophic epidermolysis bullosa (RDEB), the epidermis can 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 internal skin lining the mouth and throat are affected, disrupting patients’ ability to eat. Many die prematurely from aggressive squamous cell carcinoma, a common complication of DEB. These patients have no treatment options beyond daily wound care, pain management and protective bandaging. The epidermolysis bullosa patient community has done extraordinary work to spread awareness of the unmet medical need that patients face.
Phoenix Tissue Repair is working to create a first-ever systemic treatment for RDEB by replacing the collagen proteins (Col7) at the root of the disease. In November 2018, we initiated a Phase 1/2 clinical trial of PTR-01, a recombinant version of the human protein Collagen VII (rCol7) in adult patients with recessive dystrophic epidermolysis bullosa (RDEB). In this trial, we are evaluating safety and tolerability of PTR-01, as well as clinical proof of concept in RDEB patient population. For more information about this trial, visit the posting on ClinicalTrials.gov, or contact us at email@example.com