Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
eplontersen
- Development stage
- Approved
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Eplontersen is a transthyretin-directed antisense oligonucleotide designed for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. It works by binding to transthyretin (TTR) mRNA in hepatocytes, leading to its degradation via ribonuclease H1 and thereby reducing both mutant and wild-type TTR protein production. This reduction decreases the accumulation of amyloid deposits in tissues, slowing disease progression and improving neuropathy symptoms. Eplontersen is conjugated with triantennary N-acetyl galactosamine (GalNAc3), which enhances targeted delivery to liver cells. The drug was developed by Ionis Pharmaceuticals and AstraZeneca, with FDA approval granted in December 2023 for this indication[1][2][5][6][7][8].
- Brand names
- Wainua
- Other names
- AKCEA-TTR-LRxeplontersen
02
Targets
TTR (Transthyretin)
Beyond the preview
Go deeper on eplontersen.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on eplontersen.