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Product Description
– 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].
Web ID
– HY-148089
Shipping
– Room temperature
Applications
– Neuroscience-Neuromodulation
Molecular Formula
– N/A
References
– [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.
CAS Number
– 1637600-16-8
Molecular Weight
– N/A
SMILES
– [Eplontersen]
Clinical Information
– Phase 3
Research Area
– Neurological Disease
Solubility
– H2O : 100 mg/mL (ultrasonic)
Target
– Transthyretin (TTR)
Pathway
– Neuronal Signaling
Product type
– Oligonucleotides
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