NNC9733-0001

NNC9733-0001 is an investigational small interfering RNA (siRNA) therapeutic developed by Novo Nordisk for the treatment of type 2 diabetes mellitus. Utilizing the GalXC-Plus (GalNAc-conjugated) technology platform acquired through the acquisition of 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 rate-limiting enzyme responsible for glycogen synthesis in hepatocytes. By inhibiting GYS2, NNC9733-0001 aims to modulate hepatic glucose and lipid metabolism, potentially reducing excessive glycogen accumulation and hepatic steatosis, thereby improving insulin sensitivity and glycemic control. The drug is currently in early-stage clinical development, with Phase 1 trials evaluating its safety, tolerability, and pharmacokinetics following single subcutaneous administrations in healthy volunteers and patients with type 2 diabetes.