Ontology highlight
ABSTRACT:
SUBMITTER: Leubitz A
PROVIDER: S-EPMC8451797 | biostudies-literature | 2021 Aug
REPOSITORIES: biostudies-literature
Leubitz Andi A Vanhoutte Frederic F Hu Ming-Yi MY Porter Kaela K Gordon Efrat E Tencer Kathleen K Campbell Kathleen K Banks Kate K Haverty Tom T
Clinical pharmacology in drug development 20210119 8
ELX-02 is an investigational compound being developed as a therapy for genetic diseases caused by nonsense mutations such as cystic fibrosis. Structurally, ELX-02 is an aminoglycoside analogue that induces read-through of nonsense mutations through interaction with the ribosome, resulting in the production of full-length functional proteins. This phase 1 multiple-ascending-dose trial evaluated the safety and pharmacokinetics of ELX-02 in 62 healthy volunteers. ELX-02 plasma exposure was dose pro ...[more]