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RDEB — Taking Steps to Better Understand and Treat This Devastating Connective Tissue Disorder

RDEB patients have a defect in the COL7A1 gene, leaving them unable to produce functional type VII collagen that helps anchor the dermal and epidermal layers of the skin. Therefore, the ideal RDEB treatment will target the underlying cause of the disease and correct the genetic defect in keratinocytes (skin cells) to restore type VII collagen function. Abeona Therapeutics is currently collaborating with Stanford University on such a therapy that could potentially be the first treatment option for RDEB patients and target different sized wounds including more serious large wounds. Progress in clinical research by industry and academia can help advance our understanding of how RDEB manifests in patients and how to best target the underlying genetic cause of this devastating disease.

Jean Tang, MD, PhD, Professor of Dermatology at Stanford, will present a talk titled Large Wounds: An Update on Natural History and EB-101 at the EB2020 Congress on Tuesday, January 21, 2020 about what her team along with Abeona have learned about the natural history of RDEB and how their findings are helping advance a targeted, potentially disease-modifying therapy for this patient population with a significant unmet medical need.