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SYH2070

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

SYH2070 is an investigational double-stranded small interfering RNA (siRNA) therapeutic being developed by CSPC Pharmaceutical Group for the treatment of hypertriglyceridemia and mixed hyperlipidemia. It is a GalNAc-conjugated siRNA designed for liver-targeted delivery and subcutaneous administration, where it selectively silences expression of angiopoietin-like protein 3 (ANGPTL3), a key regulator of triglyceride and cholesterol metabolism.[5][10] Through sequence optimization and chemical modification, SYH2070 aims to achieve ultra-long-acting reduction of ANGPTL3 with prolonged pharmacodynamic effects, potentially lowering residual cholesterol risk beyond standard lipid-lowering therapies.[3][5][10] Preclinical data have shown superior pharmacological activity, sustained efficacy, and a favorable safety profile compared with similar siRNA agents, and the program has entered phase 1 clinical evaluation in subjects with elevated triglycerides.[4][7][11]

02

Targets

ANGPTL3 (Angiopoietin-like protein 3)

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