Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, established in 2013. The company specializes in developing innovative ribonucleic acid modulating drug candidates aimed at treating rare and ultra-rare diseases caused by premature stop codon mutations. Its primary investigational drug candidate, ELX-02, is currently undergoing Phase 2 clinical trials for the treatment of patients with cystic fibrosis and nephropathic cystinosis who have nonsense mutations. Eloxx utilizes its proprietary ZM chemistry technology platform and a library of novel aminoglycosides to create Ribosome Modulating Agents (RMAs) and Eukaryotic Ribosome Selective Glycosides (ERSGs), which are small molecules designed to restore the production of functional proteins in patients affected by these genetic disorders.