Drug intelligence / Profile preview

SYH2068

Development stage
Phase 2
Lead developer
CSPC Pharmaceutical Group
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
01

Overview

SYH2068 is an investigational small interfering RNA (siRNA) therapeutic developed by CSPC Pharmaceutical Group for the treatment of hyperlipoproteinemia (a). It is designed to specifically target and silence the mRNA encoding apolipoprotein(a) [Apo(a)], a key structural component of lipoprotein(a) [Lp(a)]. By utilizing the RNA interference (RNAi) pathway to degrade the target mRNA in hepatocytes, SYH2068 inhibits the production of Apo(a), thereby significantly reducing circulating levels of Lp(a). Elevated Lp(a) is a genetically determined and independent risk factor for cardiovascular diseases, including atherosclerosis, coronary heart disease, and aortic stenosis. SYH2068 typically employs a GalNAc-conjugation delivery platform to ensure targeted delivery to the liver and is currently being evaluated in early-phase clinical trials.

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