DB14713ApprovedInvestigationalGene TherapiesAntisense OligonucleotidesTransthyretin-directed RNA Interaction
Inotersen is an antisense oligonucleotide used to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis. It was FDA approved in October 2018.
- Mechanism
- Curator reviewed · 1 reference
Inotersen is an antisense oligonucleotide 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
- Inotersen is a transthyretin-directed antisense oligonucleotide indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.Curator reviewed · 2 structured indications
- First approval
- Canada, 2018 · United States, 2018 · European Union, 2020
- Code names
- GSK-2998728 · ISIS-420915
- Brand names
Resolves to
Compound
Clinical / RWD
Targets
What you can answer from here — as of July 17, 2026
2Protein targetsEach mapped to UniProt, with action and pharmacological actionListed above
Which drugs share a target with Inotersen, and which of those have an active Phase 3 trial?
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