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Nedosiran (DCR-PHXC) sodium is an RNA interference (RNAi) targeting lactate dehydrogenase (LDH). Nedosiran sodium represents an impactful potential therapeutic for primary hyperoxaluria (PH) with end-stage renal disease (ESRD). Nedosiran sodium is a GalNAc-dsRNA conjugate[1][2][3]. | [in vivo]
Nedosiran sodium is a RNAi therapy for primary hyperoxaluria that selectively reduce hepatic expression of lactate dehydrogenase[3].
Nedosiran sodium specially inhibits hepatic expression of lactate dehydrogenase (LDHA)[3].
Nedosiran sodium is a double-strand siRNA molecule that is conjugated with GalNAc, which takes advantage of the aforementioned unique asialoglycoprotein receptor (ASGPR) delivery system in the liver. Nedosiran sodium is administered by monthly subcutaneous injection. Nedosiran sodium decreases plasma oxalate in animal models[3]. | [References]
[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. DOI:10.1016/j.urology.2021.03.014
[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. DOI:10.1016/j.ekir.2021.01.029
[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. DOI:10.1111/bcp.14925 |
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