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NUP98 siRNA
- Development stage
- Preclinical
- Lead developer
- Cleveland Clinic
- 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, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous, Intratumoral
01
Overview
NUP98 siRNA is an experimental small interfering RNA (siRNA) construct designed to silence the expression of the Nucleoporin 98 (NUP98) gene or its oncogenic fusion variants, such as the NUP98-NSD1 fusion transcript. In the context of acute myeloid leukemia (AML), particularly in pediatric patients with cytogenetically normal AML, NUP98-NSD1 siRNA is formulated into lipid nanoparticles (LNPs) for in vivo delivery to reduce fusion gene expression and prolong survival. In glioblastoma research, NUP98 siRNA has been utilized to demonstrate that NUP98 maintains glioblastoma stem cell (GSC) proliferation and therapeutic resistance by modulating the transcriptional control of the homologous recombination DNA repair pathway, including genes such as BRCA1, BRCA2, and RAD51. The drug is currently in the research and preclinical stages, involving academic groups and technology contributions from companies like Precision NanoSystems and Axolabs.
- Other names
- siNUP98siNUP-98siNUP 98NUP98-NSD1 siRNANUP-98-NSD1 siRNANUP 98-NSD1 siRNA
02
Targets
NUP98NUP98–NSD1 (Nucleoporin 98–Nuclear receptor binding SET domain protein 1 fusion mRNA)
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