ZKN-0013

ZKN-0013 (also known as ZKN-013) is an investigational oral small molecule nonsense mutation readthrough agent, categorized as a Ribosome Modulating Agent (RMA). Originally discovered by Eloxx Pharmaceuticals and licensed to Almirall S.A., it was developed using the TURBO-ZM™ chemistry platform. The drug's mechanism involves targeting the human ribosome to facilitate the readthrough of premature stop codons caused by nonsense mutations, thereby enabling host cells to produce full-length, functional proteins. ZKN-0013 is primarily being developed for rare orphan dermatological diseases, most notably recessive dystrophic epidermolysis bullosa (RDEB), where it has demonstrated the ability to induce the production of functional COL7A1 protein in patient cells. It is also under investigation for familial adenomatous polyposis (FAP) associated with nonsense mutations and is currently in Phase 1 clinical development.

Other names

ZKN-013ZKN013ZKN 013ZKN-0013ZKN0013ZKN 0013