Drug intelligence / Profile preview

PAI-1 siRNA

Development stage
Preclinical
Lead developer
Hebei Medical University
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
Intranasal, Intraperitoneal, Intravenous
01

Overview

PAI-1 siRNA (plasminogen activator inhibitor-1 small interfering RNA) refers to a class of RNA interference (RNAi) therapeutics designed to selectively silence the expression of the SERPINE1 gene, which encodes PAI-1. PAI-1 is a key serine protease inhibitor that suppresses fibrinolysis by inhibiting tissue plasminogen activator (tPA) and urokinase plasminogen activator (uPA). Elevated levels of PAI-1 are associated with thrombosis, tissue fibrosis (such as pulmonary and ovarian fibrosis), obesity, and cellular senescence. By degrading PAI-1 mRNA, PAI-1 siRNA reduces PAI-1 protein levels, thereby promoting fibrinolysis, reducing fibrotic extracellular matrix accumulation, and potentially mitigating age-related pathologies. Various formulations, including lipid nanoparticles (LNPs) and polymeric nanoparticles, are being investigated in preclinical models for indications such as thrombosis, pulmonary fibrosis, and diminished ovarian reserve.

Other names
PAI-1 small interfering RNAPAI1 small interfering RNAPAI 1 small interfering RNASERPINE1 siRNASERPINE-1 siRNASERPINE 1 siRNAplasminogen activator inhibitor-1 siRNAplasminogen activator inhibitor-1 small interfering RNAsiPAI-1siPAI1siPAI 1
02

Targets

PAI-1 (Plasminogen activator inhibitor type 1)

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