Drug intelligence / Profile preview

AJ5939

Development stage
Preclinical
Lead developer
University of Ottawa
Modality
Small Molecules
01

Overview

AJ5939 is a brain-penetrant small molecule drug candidate designed to target brain tumor stem cells (BTSCs) in glioblastoma. It functions by directly impairing the Notch signaling pathway, which is critical for the self-renewal and maintenance of BTSCs. By altering the stemness signature and gene expression profiles related to the cell cycle and DNA repair, AJ5939 inhibits tumor growth and progression. Furthermore, it has been shown to sensitize BTSCs to ionizing radiation by suppressing DNA repair mechanisms. Preclinical studies indicate that AJ5939 treatment significantly prolongs lifespan in brain tumor models without affecting non-oncogenic neural stem cells.

02

Targets

NOTCH (Neurogenic locus notch homolog protein 3)

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