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NU-0129
- Development stage
- Phase 1
- Lead developer
- Northwestern University
- Modality
- Gold Nanoparticles → Inorganic Nanoparticles → Nanoparticles → Drug Delivery Systems, 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
- Intravenous
01
Overview
NU-0129 is an experimental nanotherapeutic drug developed at Northwestern University, composed of a spherical nucleic acid (SNA) gold nanoparticle formulation. It consists of small interfering RNAs (siRNAs) targeting the Bcl-2-like protein 12 (BCL2L12) sequence, covalently conjugated to gold nanoparticles. The drug is designed to cross the blood-brain barrier and suppress gene expression in tumor cells by silencing BCL2L12, an oncogene implicated in glioblastoma. This mechanism induces apoptosis (programmed cell death) in tumor cells. NU-0129 represents a novel class of systemically administered SNA drugs and has shown the ability to penetrate brain tumors and alter their genetic machinery in early clinical studies.
- Other names
- spherical nucleic acid nanoparticle NU-0129
02
Targets
BCL2L12
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