MicroRNA (miRNA) therapeutics are being actively developed, with several candidates progressing to clinical and preclinical stages. Below are examples from each category: 

miRNA Therapeutics in Clinical Stages 

1.Miravirsen 

     ●      Function: A miR-122 inhibitor designed to treat hepatitis C virus (HCV) infection. 

     ●      Progress: Developed by Santaris Pharma, Miravirsen was the first miRNA inhibitor to enter clinical trials and has reached Phase II. 

     ●      Mechanism: Blocks miR-122, a host factor required for HCV replication, offering an innovative approach to antiviral therapy. 

2.CDR132L 

     ●      Function: A miR-132 inhibitor targeting heart failure. 

     ●      Progress: Developed by Cardior Pharmaceuticals, this therapy is undergoing clinical trials aimed at improving cardiac function by modulating miR-132. 

     ●      Mechanism: Inhibits miR-132, which is associated with adverse cardiac remodeling and fibrosis. 

miRNA Therapeutics in Preclinical Stages 

1.MRX34 

     ●      Function: A mimic of miR-34a, designed for cancer treatment. 

     ●      Progress: Developed by Mirna Therapeutics, MRX34 initially entered Phase I clinical trials but was halted due to immune-related adverse effects. It is now undergoing further preclinical evaluation. 

     ●      Mechanism: Restores tumor-suppressive miR-34a activity to inhibit oncogenic pathways. 

2.TTX-MC138 

     ●      Function: Targets miR-10b for the treatment of breast and pancreatic cancers. 

     ●      Progress: Developed by TransCode Therapeutics, TTX-MC138 has shown promising results in preclinical studies. It received FDA IND approval in 2024 and completed initial dosing in patients. 

     ●      Mechanism: Suppresses miR-10b, which promotes cancer metastasis, thereby inhibiting tumor progression. 

In the clinical stage, miRNA therapeutics like Miravirsen and CDR132L are advancing in addressing HCV and heart failure, respectively, showcasing the potential of miRNA inhibitors. Meanwhile, preclinical candidates such as MRX34 and TTX-MC138 are making strides in cancer research. Although promising, miRNA therapies face challenges like delivery efficiency and safety, which must be overcome to achieve broader clinical success.