Drug intelligence / Profile preview

siRNA + amiloride

Development stage
Preclinical
Lead developer
Yonsei University
Modality
Small Molecules, 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
Administration
Oral, Intravenous, Topical, Inhalation
01

Overview

**siRNA + amiloride** is an experimental combination therapy, where **small interfering RNA (siRNA)** is co-administered with **amiloride**. siRNA is a class of synthetic double-stranded RNA molecules that utilize the RNA interference (RNAi) pathway to silence specific genes through the targeted degradation of mRNA, thus inhibiting the translation of disease-related proteins. Amiloride is a small molecule diuretic known for its inhibition of epithelial sodium channels, and has been investigated for its role in modulating endocytosis pathways and enhancing cellular uptake of siRNA therapies. The combination is studied mainly for cancer treatment, aiming to overcome barriers to siRNA delivery and improve therapeutic efficacy via synergistic gene silencing and chemotherapeutic effects. Amiloride can increase the efficiency of siRNA transfection in target cells by modulating cellular mechanisms such as inhibition of macropinocytosis and sodium channel blockade, assisting endosomal escape and cellular uptake.

02

Targets

Endosomal sodium-dependent uptake pathwayENaC

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