Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company. The Company is engaged in the science of ribosome modulation, leveraging both its TURBO- ZM chemistry technology platform and its library of aminoglycosides to develop oral small-molecule ribosome modulating agents (RMAs) and eukaryotic ribosome selective glycosides (ERSGs) for the treatment of rare and ultra-rare genetic diseases. Its lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. The Company is also advancing ZKN-013 as an oral agent for the treatment of recessive dystrophic (RDEB)/ junctional epidermolysis bullosa (JEB) and familial adenomatous polyposis (FAP) patients with nonsense mutations. The Company also has preclinical programs focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases, as well as rare ocular genetic disorders.
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