Targeting RNA in Disease with Novel Technologies

Targeting RNA in Disease with Novel Technologies (TRDNT) is the latest initiative from the NIH’s Common Fund’s Venture Program, which supports short-term projects with significant impact. RNA-targeting therapies have the potential to treat diseases that have no, or few, effective treatments, and could be life-changing medications for patients suffering from these conditions.  Including RNA as a target for treatment could have an enormous impact on the number of treatments and the number of diseases that can be treated.  RNAs have been linked to many diseases, including rare diseases like myotonic dystrophy, neurological disorders like Parkinson’s disease, certain aggressive forms of cancer, such as triple negative breast cancer, and heart failure, among others.  Expanding research to target specific RNA molecules could have an enormous impact on how we prevent or treat many diseases that currently have few or no treatment options.  If RNA is targeted, it is estimated that the druggable space could be expanded 3-10-fold.  This initiative will create new RNA-targeting treatments for the American people and will result in publicly shared and/or commercially sold products for the benefit of the American public.