ELX-02 Restores CFTR Activity in Organoids With G542X Mutations, Study Finds

ELX-02 Restores CFTR Activity in Organoids With G542X Mutations, Study Finds

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ELX-02 Restores CFTR Activity in Organoids With G542X Mutations, Study Finds

ELX-02 restored the levels and function of CFTR — the defective protein in CF — in “mini-organs,” or organoids, derived from patients carrying the most common CF-causing nonsense mutation, a study shows. “ELX-02 increased CFTR activity in a dose-dependent fashion,” with these functional increases being “similar to those obtained with tezacaftor/ivacaftor [Symdeko]” in organoids carrying the G542X mutation, Matthew Goddeeris, PhD, the vice president of research at Eloxx Pharmaceuticals, the investigational therapy’s developer, said in a…

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