exaluren

Exaluren (ELX-02) is a small molecule eukaryotic ribosomal selective glycoside (ERSG) developed by Eloxx Pharmaceuticals. It is designed to treat genetic diseases caused by nonsense mutations, which introduce premature termination codons (PTCs) into mRNA, leading to truncated, non-functional proteins. Exaluren works by binding to the decoding site on the 18S ribosomal RNA (rRNA) of the eukaryotic ribosome. This binding reduces the fidelity of translation specifically at the PTC, allowing the insertion of a near-cognate tRNA and enabling the ribosome to "read through" the stop signal to produce a full-length, functional protein. Unlike traditional aminoglycoside antibiotics, exaluren is engineered for high selectivity for the eukaryotic ribosome over the mitochondrial ribosome to minimize toxicity, such as ototoxicity and nephrotoxicity.

Other names

1375073-94-1EXALUREN SULFATE