Answer
Wainzua (eplontersen sodium) is an antisense oligonucleotide targeted to liver cells by its GalNAc conjugate, where it selectively binds transthyretin (TTR) messenger RNA and causes degradation of both mutant and normal TTR mRNA, preventing liver TTR synthesis and reducing secretion into the circulation.
Sources for this answer
- Wainzua 45 mg solution for injection in pre-filled penAstraZeneca UK Limited · emc · SmPC §5.1 Pharmacodynamic properties · Version 2Document updated
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