Drug intelligence / Profile preview

SYH2053

Development stage
Phase 3
Lead developer
CSPC Zhongnuo Pharmaceutical
Modality
Small Molecules, 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

SYH2053 is an investigational small interfering RNA (siRNA) drug targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), developed for the treatment of hypercholesterolemia and related lipid disorders. It is designed to lower low-density lipoprotein cholesterol (LDL-C) by inhibiting PCSK9, a protein that regulates LDL receptor degradation, thereby increasing the clearance of LDL-C from the bloodstream. The drug is administered subcutaneously and is currently in Phase 1 clinical trials to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamics in subjects with normal or elevated LDL-C. SYH2053 was developed by CSPC Hebei Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd., a subsidiary of CSPC Pharmaceutical Group[1][2][3][7].

02

Targets

PCSK9 (Proprotein convertase subtilisin/kexin type 9)

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