Eloxx Pharmaceuticals Email Format
Biotechnology ResearchMassachusetts, United States11-50 Employees
Eloxx Pharmaceuticals is a clinical-stage biotechnology company focused on developing RNA-modulating drug candidates to treat rare and ultra-rare diseases caused by premature stop codons. These mutations disrupt protein production from messenger RNA and have been identified in more than 1,800 rare conditions. The approach aims to enable the cytoplasmic ribosome to read through such stop codons, with the goal of restoring production of full-length proteins. Its lead candidate, ELX-02, is in the early stages of clinical development and is being explored for conditions including cystic fibrosis and cystinosis. The company is based in Watertown, Massachusetts, and conducts research activities in Rehovot, Israel.