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Article

ELX-02: an investigational read-through agent for the treatment of nonsense mutation-related genetic disease.

Expert opinion on investigational drugs - 1 Dec 2020

Kerem Eitan

Abstract excerpt

INTRODUCTION: ELX-02, an investigational compound that is structurally an aminoglycoside analog, induces read-through of nonsense mutations through interaction with the ribosome, through which full-length functional proteins can be produced. It is being developed as a therapy for genetic diseases caused by nonsense mutations such as cystic fibrosis (CF) and nephropathic cystinosis. In Phase 1 clinical trials, 105...

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Identifiers

PMID: 32972261
DOI: 10.1080/13543784.2020.1828862

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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