Science & Pipeline

The Phase I, randomized, double-blind, placebo-controlled, single-dose, and multiple-dose escalation study is designed to evaluate the safety, tolerability, pharmacokinetic and pharmacodynamic characteristics of SGB-3403 when administered subcutaneously in healthy participants and participants with elevated Low Density Lipoprotein Cholesterol (LDL-C).

SGB-3403, an siRNA- N-acetylgalactosamine (GalNAc) conjugate targeting PCSK9, is delivered to liver using Sanegene's novel GalNAc platform technology to inhibit the synthesis of PCSK9 in the liver through RNA interference. The advantages of GalNAc platform have been also fully verified, including safety, effectiveness, and stability. SGB-3403 has been shown in preclinical studies with good safety and tolerability, superior efficacy than benchmark products in multiple animal disease models (including the hyperlipidemic cynomolgus monkey model), and it can reduce LDL-C by up to 40% - 70% even with lower dose and less frequency of administration, showing its potential to be the best-in-class siRNA drug targeting PCSK9.