Research & Development

Pipeline driven by patient needs and engagement

Our research addresses different aspects of EB pathogenesis. Our gene therapy program (EB01) is focused on the correction of causal mutations. Program EB02 is a phenotypic screen focused on identification of small molecules able to target EB associated SCC. EB03 program focuses on bolstering cancer treatment for RDEB patients with clinically diagnosed SCC lesions. EB04 program addresses inhibition of immune system to treat SCC in EB patients. 

Our programs are modality agnostic, consisting of small molecules, gene therapy and biotherapeutics. We are developing the programs internally but are also open for partnering.