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Eplontersen

Catalog No.GC71015 Copy One-Click Copy Product Info

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.

Products are for research use only. Not for human use. We do not sell to patients.

Eplontersen Chemical Structure

Cas No.: 1637600-16-8

Taille Prix Stock Qté
1 mg
252,00 $US
En stock

Tel:(909) 407-4943 Email: sales@glpbio.com


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Based on customer reviews.

Sample solution is provided at 25 µL, 10mM.



Description of 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.

Eplontersen mediates N-acetylgalactosamine moiety targeting the oligonucleotide to cells bearing an asialoglycoprotein receptor[3].

Eplontersen (682884) (0.6, 2, 6 mg/kg; s.c.; once a week for 3 weeks) inhibits TTR protein expression in a dose-dependent manner in vivo, without affecting normal growth in transgenic C57BL/6 mice expressing human transthyretin (TTR)[3].

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.

Chemical Properties of Eplontersen

Cas No. 1637600-16-8 SDF
Formula M.Wt 8606.5
Solubility H2O : 100 mg/mL (11.62 mM; Need ultrasonic) Storage -20°C, sealed storage, away from moisture
General tips Please select the appropriate solvent to prepare the stock solution according to the solubility of the product in different solvents; once the solution is prepared, please store it in separate packages to avoid product failure caused by repeated freezing and thawing.Storage method and period of the stock solution: When stored at -80°C, please use it within 6 months; when stored at -20°C, please use it within 1 month.
To increase solubility, heat the tube to 37°C and then oscillate in an ultrasonic bath for some time.
Shipping Condition Evaluation sample solution: shipped with blue ice. All other sizes available: with RT, or with Blue Ice upon request.

Complete Stock Solution Preparation Table of Eplontersen

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1 mg 5 mg 10 mg
1 mM 116.2 μL 581 μL 1.1619 mL
5 mM 23.2 μL 116.2 μL 232.4 μL
10 mM 11.6 μL 58.1 μL 116.2 μL
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Method for preparing DMSO master liquid: mg drug pre-dissolved in μL DMSO ( Master liquid concentration mg/mL, Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug. )

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Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.

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3. All of the above co-solvents are available for purchase on the GlpBio website.

Product Documents

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Review for Eplontersen

Average Rating: 5 ★★★★★ (Based on Reviews and 30 reference(s) in Google Scholar.)

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