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).

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.

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].