Drug intelligence / Profile preview

SYH2061

Development stage
Phase 1
Lead developer
CSPC Zhongnuo Pharmaceutical
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

**SYH2061** is a double-stranded small interfering RNA (siRNA) drug independently developed by CSPC Pharmaceutical Group. It achieves liver-targeted delivery via N-acetylgalactosamine (GalNAc) conjugation and subcutaneously inhibits complement protein C5, reducing C5 levels and its downstream fragments (C5a, C5b-9) to block complement activation. Optimized sequencing and chemical modifications enable ultra-long-lasting gene silencing, outperforming similar siRNA products in preclinical studies with superior activity, duration, safety, and patient compliance. It is indicated for IgA nephropathy and other complement-mediated diseases, marking China's first independently developed siRNA targeting C5 to enter clinical trials.

02

Targets

C5

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