LAD765

LAD765 (formerly known as ZKN-0013 or ZKN-013) is an investigational oral small molecule nonsense mutation readthrough agent. Developed using Eloxx Pharmaceuticals' proprietary TURBO-ZM platform and licensed to Almirall S.A. in March 2024, the drug is designed to enable host cells to bypass premature stop codons caused by nonsense mutations. By promoting the production of full-length, functional proteins, LAD765 addresses the underlying genetic cause of several rare diseases. It is currently in Phase 1 clinical development, with studies evaluating its safety, tolerability, and pharmacokinetics in healthy volunteers. The primary therapeutic targets for LAD765 include rare orphan dermatological conditions such as recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB), as well as familial adenomatous polyposis (FAP).

Other names

LAD765LAD-765LAD 765ZKN-0013ZKN0013ZKN 0013ZKN-013ZKN013ZKN 013