ELX-03

**ELX-03** is an investigational **small-molecule translational read-through agent** developed by **Eloxx Pharmaceuticals** for genetic diseases caused by **nonsense mutations**. It belongs to the company's eukaryotic ribosomal selective glycoside platform and is designed to promote ribosomal read-through of premature stop codons, thereby restoring production of full-length functional protein while showing reduced affinity for prokaryotic and mitochondrial ribosomes relative to older aminoglycosides. Publicly disclosed work has focused mainly on **inherited retinal disorders**, where preclinical studies reported activity against nonsense mutations associated with **Usher syndrome**, **retinitis pigmentosa**, and **KCNJ13-related Leber congenital amaurosis**, with intravitreal delivery explored in ocular models. The program appears to have remained **preclinical** and later company pipeline materials emphasize other assets rather than ELX-03 specifically.

Other names

designer Eukaryotic Specific Ribosome Glycosideeukaryotic ribosomal selective glycoside