Drug intelligence / Profile preview

QN-247

Development stage
Discontinued
Lead developer
Qualigen Therapeutics
Modality
RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous (preclinical/anticipated)
01

Overview

QN-247 is a novel investigational anti-cancer drug candidate developed by Qualigen Therapeutics in collaboration with the University of Louisville. It is a new chemical entity consisting of the anti-nucleolin DNA aptamer AS1411 covalently bonded to a gold nanoparticle (GNP). This design leverages the tumor-targeting and anti-proliferative properties of oligonucleotide aptamers and enhances them with the pharmacokinetic advantages of gold nanoparticles. The mechanism centers on inhibiting nucleolin—a multifunctional regulatory protein overexpressed on cancer cell surfaces—thereby selectively targeting and inhibiting proliferation in cancer cells while sparing normal tissue. Preclinical studies have demonstrated robust efficacy against multiple cancer types including triple negative breast cancer (TNBC) and acute myeloid leukemia (AML), with favorable safety profiles observed in animal models[1][4][5][9]. Clinical development has been discontinued for AML, neuroblastoma, pancreatic cancer, and TNBC[2].

02

Targets

NCL (Nucleolin)

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