Eplontersen
Eplontersen is a triantennary N-acetyl galactosamine (GalNAc3-7a) -conjugated antisense oligonucleotide targeting transthyretin (TTR) mRNA to inhibit production of both variant and wild-type TTR protein. Misfolded TTR induces amyloid fibrils formation in the heart and peripheral nerves, leads to amyloid TTR (ATTR) amyloidosis diseases[1][2][3].
Product Specifications
CAS Number
1637600-16-8
UNSPSC
12352208
Target
Transthyretin (TTR)
Type
Oligonucleotides
Related Pathways
Neuronal Signaling
Applications
Neuroscience-Neuromodulation
Field of Research
Neurological Disease
Assay Protocol
https://www.medchemexpress.com/eplontersen.html
Purity
96.92
Solubility
H2O : 100 mg/mL (ultrasonic)
Smiles
[Eplontersen]
Molecular Weight
8606.50
References & Citations
[1]Aimo A, et al. RNA-targeting and gene editing therapies for transthyretin amyloidosis. Nat Rev Cardiol. 2022 Mar 23. |[2]Diep JK, et al. Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis. Br J Clin Pharmacol. 2022 Jul 22. |[3]Prakash Thazha P, et al. Antisense oligonucleotides to hepatitis B virus RNA or transthyretin mRNA conjugated with N-acetylgalactosamine targeting moieties: World Intellectual Property Organization, WO2014179627[P]. 2014-11-06.
Shipping Conditions
Blue Ice
Storage Conditions
-20°C (Powder, sealed storage, away from moisture)
Scientific Category
Oligonucleotides
Clinical Information
Phase 3
Available Sizes
Frequently Asked Questions
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