Drug intelligence / Profile preview

PHmiR-SM101X

Development stage
Preclinical
Lead developer
Pharmahungary Group
Modality
RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

PHmiR-SM101X is a microRNA-based therapeutic candidate (ProtectomiR) being developed for cardioprotection against ischemia-reperfusion injury following acute myocardial infarction. Identified in a translational large-animal model of myocardial infarction, PHmiR-SM101X has demonstrated dose-dependent cardioprotective effects in neonatal rat cardiomyocytes and human AC16 cells under simulated ischemia-reperfusion conditions. Bioinformatic analyses indicate that PHmiR-SM101X modulates gene networks involved in survival and stress signaling pathways, with predicted targets including SMAD2, DAPK2, and SOD2, implicating TGF-beta signaling, apoptosis, and redox regulation. It is being evaluated with polymer-based and lipid nanoparticle (LNP) delivery systems to optimize myocardial uptake.

02

Targets

DNM1L (Dynamin-related protein 1)SMAD2 (Mothers against decapentaplegic homolog 2)Misfolded SOD1

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