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NNC0519-0130 (Novo Nordisk)

Development stage: Unknown
Lead developer: Novo Nordisk
Modality: Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Subcutaneous

Overview

NNC0519-0130 is an investigational small interfering RNA (siRNA) therapy developed by Novo Nordisk for the treatment of type 2 diabetes. Utilizing the GalXC technology platform (originally developed by Dicerna Pharmaceuticals), the drug is designed to specifically target and silence the expression of the *GYS2* gene in the liver. *GYS2* encodes glycogen synthase 2, the enzyme responsible for catalyzing the rate-limiting step of glycogen synthesis in hepatocytes. By reducing GYS2 levels through RNA interference, the therapy aims to modulate hepatic glucose metabolism and improve glycemic control in patients with diabetes. The drug is delivered via a N-acetylgalactosamine (GalNAc) conjugate, which facilitates targeted uptake by hepatocytes through the asialoglycoprotein receptor. It is currently being evaluated in Phase 1 clinical trials.

Other names: GYS2 siRNAGYS-2 siRNAGYS 2 siRNAGalXC-GYS2GalXC-GYS-2GalXC-GYS 2

Targets

GYS2 (Glycogen synthase 2)

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