Eloxx Pharmaceuticals reports positive data from Phase 1 studies of its ELX-02 drug for cystic fibrosis, CysticFibrosisNews.com reports.
ELX-02 is a eukaryotic ribosomal selective glycoside that binds to ribosomes, which are cellular units that serve as factories to produce proteins inside cells. The therapy is designed to increase a ribosome’s ability to skip over nonsense mutations and enable the production of sufficient amounts of full-length protein.
The therapy was well-tolerated in single- and multiple-ascending dose studies in those clinical trials, and raised levels of the crucial CFTR protein in patient-derived organoids, data showed. These results were presented at the 42nd European Cystic Fibrosis Society Conference that took place June 5–8 in Liverpool.