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Drug intelligence / Profile preview
RN-032
- Development stage
- Unknown
- Lead developer
- Rege Nephro
- Modality
- Small Molecules
01
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
02
Targets
MSI1 (RNA-binding protein Musashi homolog 1)MSI2 (Musashi RNA-binding protein 2)
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