Eloxx Pharmaceuticals Inc

F:3970

Eloxx Pharmaceuticals, Inc. engages in the development of novel ribonucleic acid-modulating drug candidates. The company is headquartered in Watertown, Massachusetts and currently employs 28 full-time employees. The company went IPO on 2012-11-23. The firm is focused on developing ribonucleic acid (RNA)-modulating drug candidates, each designed to be a eukaryotic ribosomal selective glycoside (ERSG), formulated to treat rare and ultra-rare premature stop codon diseases. The firm's lead product candidate, ELX-02, is a small molecule designed to restore production of full-length functional proteins. ELX-02 is in clinical development for systemic administration for cystic fibrosis. The firm is also conducting investigational new drug (IND)-enabling preclinical studies of ERSG compounds for autosomal dominant polycystic kidney disease (ADPKD) and in rare inherited retinal disorders (IRDs) by intravitreal administration with an initial focus on Usher Syndrome. Its preclinical candidate pool consists of a library of ERSG drug candidates identified based on read-through potential and cytoplasmic ribosomal selectivity.