Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is a clinical-stage biotechnology company, which engages in the development of novel ribonucleic acid-modulating drug candidates. The company is headquartered in Watertown, Massachusetts and currently employs 10 full-time employees. The company went IPO on 2012-11-23. The firm 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 firm 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 firm 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.