| 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) is an investigational ribonucleic acid interference (RNAi) therapy[2]. Nedosiran is a synthetic, double-stranded RNA oligonucleotide (i.e., small interfering RNA [siRNA]) designed to target the mRNA encoding LDHA[2]. |
| In vivo | Nedosiran is a RNAi therapy for primary hyperoxaluria that selectively reduce hepatic expression of lactate dehydrogenase[3]. Nedosiran specially inhibits hepatic expression of lactate dehydrogenase (LDHA)[3]. Nedosiran 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 is administered by monthly subcutaneous injection. Nedosiran decreases plasma oxalate in animal models[3]. |
| Synonyms | DCR-PHXC |
| molecular weight | N/A |
| CAS | 2266591-83-5 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |
| 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. 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. |