Drug intelligence / Profile preview

NP-PEI-siRNA

Development stage
Preclinical
Lead developer
University of Washington
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Biodegradable Polymers → Polymer-based Nanoparticles → Nanoparticles → Drug Delivery Systems, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intraperitoneal, Intravenous, Intracerebroventricular, Inhalation, Intranasal, Intratracheal, Topical, Ophthalmic, Oral, Endoscopic Gastrointestinal
01

Overview

NP-PEI-siRNA is a nanovector formulation designed for siRNA delivery. It consists of polyethylene glycol (PEG) coated superparamagnetic iron oxide nanoparticles (NPs) with a 12 nm core diameter, further modified with a polycationic coating of polyethylenimine (PEI), and covalently functionalized with siRNA oligonucleotides. This nanovector aims to deliver siRNA to target cells for gene silencing. While it demonstrated efficacy in delivering siRNA and gene silencing in rat C6 glioblastoma cells, in vivo studies revealed severe hemoincompatibility and damage to the liver and spleen, indicating safety concerns compared to a poly-l-arginine (pArg) coated counterpart.

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