We are using the STAR-D platform to develop treatments for rare orphan dermatological indications caused by the absence of, or a mutation in, a single gene. In addition to our work in rare skin diseases, we are currently leveraging our platform to expand our pipeline to include other dermatological indications.
Our lead product candidate, KB103, is currently undergoing clinical investigation as a gene therapy to treat DEB. KB103 is a replication-deficient, non-integrating viral vector that has been engineered employing our STAR-D platform to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells. Other indications in the current pipeline include TGM1-deficient Autosomal Recessive Congenital Ichthyosis, and chronic wounds.