Drug intelligence / Profile preview

SRSD101

Development stage
Phase 1
Lead developer
Sirius Therapeutics
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, Small Molecules
Administration
Subcutaneous
01

Overview

SRSD101 is an investigational small interfering RNA (siRNA) therapeutic developed by Sirius Therapeutics for the treatment of dyslipidemia, specifically targeting primary hypercholesterolemia. The drug works by selectively inhibiting the expression of proprotein convertase subtilisin/kexin type 9 (PCSK9) mRNA, thereby reducing PCSK9 protein levels. PCSK9 is a key regulator of low-density lipoprotein cholesterol (LDL-C) metabolism; its inhibition leads to increased clearance of LDL-C from the bloodstream, offering a novel approach to lowering cholesterol levels in patients at risk for cardiovascular disease. SRSD101 is currently being evaluated in Phase 1 clinical trials in China[1][2][4][7][9].

02

Targets

PCSK9 (Proprotein convertase subtilisin/kexin type 9)

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