Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX‐02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers

• Published in: Clinical pharmacology in drug development Vol. 8; no. 8; pp. 984 - 994
• Main Authors: Leubitz, Andi, Frydman‐Marom, Anat, Sharpe, Neal, Duzer, John, Campbell, Kathleen C.M., Vanhoutte, Frédéric
• Format: Journal Article
• Language: English
• Published: United States Wiley Subscription Services, Inc 01.11.2019
• Subjects: aminoglycoside; ELX‐02; genetic disorders; healthy volunteers; Mutation; nonsense mutations; Pharmacokinetics; phase 1; safety; single ascending dose; translational readthrough; single ascending dose; aminoglycoside; phase 1; translational readthrough; genetic disorders; safety; healthy volunteers; ELX-02; pharmacokinetics; nonsense mutations

ELX‐02 is an investigational synthetic eukaryotic ribosome–selective glycoside optimized as a translational read‐through molecule that induces read through of nonsense mutations, resulting in normally localized full‐length functional proteins. ELX‐02 is being developed as a therapy for genetic disea...