ELX-02 is a compound designed to restore CFTR function in people with CF who have nonsense mutations. Nonsense mutations (also known as "x" or "stop" mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. ELX-02 is intended allow lung cells to override these premature stop signals and make full-length, functional CFTR protein.
A phase 2 study is planned to test the safety and tolerability of ELX-02. The study is for people with CF who have at least one copy of the G542X CFTR mutation.
This program is sponsored by Eloxx Pharmaceuticals and is being conducted within the Therapeutics Development Network.
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