#ECFS2018 – Preclinical Data Shows Potential of ELX-02 to Reverse Effects of CFTR Nonsense Mutations

Preclinical studies show that Eloxx Pharmaceuticals’ lead therapy candidate ELX-02 can reverse the effects of CFTR gene nonsense mutations in mice and cystic fibrosis (CF) patient-derived organoids. These findings further support the clinical development of ELX-02 as a treatment for CF patients with CFTR nonsense mutations, which represent…