Drug intelligence / Profile preview

RN-032

Development stage: Unknown
Lead developer: Rege Nephro
Modality: Small Molecules

Overview

RN-032 is a small molecule inhibitor of the RNA-binding proteins **Musashi-1 (MSI1)** and **Musashi-2 (MSI2)**. It specifically binds to the RNA-binding domain 1 (RBD1) of Musashi-1, thereby disrupting its interaction with target mRNAs such as *Numb*, which is a negative regulator of the Notch signaling pathway. By inhibiting Musashi activity, RN-032 promotes the expression of Numb, leading to the downregulation of Notch and other oncogenic pathways like p21/WAF1. This mechanism has shown potential in preclinical models for inhibiting the growth and survival of various cancer types, including glioblastoma and colorectal cancer, where Musashi proteins are often overexpressed and contribute to stemness and chemoresistance.

Other names: RN032RN-032RN 032

Targets

MSI2 (Musashi RNA-binding protein 2)MSI1 (RNA-binding protein Musashi homolog 1)

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