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Nedosiran

Catalog No. T39802 CAS 2266591-83-5

Synonyms: DCR-PHXC

Nedosiran (DCR-PHXC) is a GalNAc-dsRNA conjugate designed for RNA interference (RNAi) targeting lactate dehydrogenase (LDH). It serves as a potential therapeutic option for patients with primary hyperoxaluria (PH) and end-stage renal disease (ESRD).

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Nedosiran, CAS 2266591-83-5

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25 mg	Inquiry	$ 1,520.00

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Description	Nedosiran (DCR-PHXC) is a GalNAc-dsRNA conjugate designed for RNA interference (RNAi) targeting lactate dehydrogenase (LDH). It serves as a potential therapeutic option for patients with primary hyperoxaluria (PH) and end-stage renal disease (ESRD).
In vitro	Nedosiran (DCR-PHXC), an investigational therapy utilizing ribonucleic acid interference (RNAi) technology, is a synthetic, double-stranded RNA oligonucleotide, specifically a small interfering RNA (siRNA). It is engineered to selectively target and silence the mRNA responsible for encoding LDHA, offering a targeted approach to therapy.
In vivo	Nedosiran, an RNAi therapy for primary hyperoxaluria, selectively reduces hepatic expression of lactate dehydrogenase (LDHA)[3] by specifically inhibiting its production in the liver. As a double-stranded siRNA molecule conjugated with GalNAc, it leverages the unique asialoglycoprotein receptor (ASGPR) delivery system in the liver for targeted action. Administered through monthly subcutaneous injections, Nedosiran has demonstrated the ability to decrease plasma oxalate levels in animal models[3].

Synonyms	DCR-PHXC
Molecular Weight	N/A
CAS No.	2266591-83-5

Storage

Powder: -20°C for 3 years | In solvent: -80°C for 1 year

References and Literature

1. Kevin Shee, et al. Nedosiran Dramatically Reduces Serum Oxalate in Dialysis-Dependent Primary Hyperoxaluria 1: A Compassionate Use Case Report. Urology.2021 Oct;156:e147-e149. 2. Gema Ariceta, et al. Hepatic Lactate Dehydrogenase A: An RNA Interference Target for the Treatment of All Known Types of Primary Hyperoxaluria. Kidney Int Rep. 2021 Feb 3;6(4):1088-1098. 3. Thomas A Forbes, et al. Therapeutic RNA interference: A novel approach to the treatment of primary hyperoxaluria. Br J Clin Pharmacol. 2021 May 22.

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Keywords

Nedosiran 2266591-83-5 DCR-PHXC inhibitor inhibit

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