DB16199ApprovedInvestigationalGene TherapiesAntisense OligonucleotidesTransthyretin-directed RNA Interaction
Eplontersen is a transthyretin antisense oligonucleotide used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis. Eplontersen is a transthyretin-directed antisense oligonucleotide with 3 covalently linked e N-acetyl galactosamine residues for hepatic delivery.
- Mechanism
- Curator reviewed · 1 reference
Eplontersen is an antisense oligonucleotide-GalNAc conjugate that causes degradation of mutant and wild-type TTR mRNA through binding to the TTR mRNA, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.
- Primary indication
- Eplontersen is indicated for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.Curator reviewed · 1 structured indication
- First approval
- Canada, 2024 · United States, 2023 · European Union, 2025
- Also known as
- Akcea ttr lrx · AKCEA-TTR-LRx · Ion-ttr-lrx · Ionis-ttr-lrx
- Code names
- ION-682884 FREE ACID · ISIS-682884 FREE ACID
- Brand names
Resolves to
Compound
Clinical / RWD
Targets
What you can answer from here — as of September 12, 2026
2Protein targetsEach mapped to UniProt, with action and pharmacological actionListed above
Which drugs share a target with Eplontersen, and which of those have an active Phase 3 trial?
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