세미나

Many disease-associated targets have traditionally been considered "undruggable" due to their lack of well-defined binding pockets or their essential roles in cellular processes, making direct inhibition challenging. To overcome these limitations, new drug discovery approaches are being developed that extend beyond conventional small-molecule inhibitors. Among them, RNA-targeting small molecules and RIBOTACs (Ribonuclease-Targeting Chimeras) have emerged as promising strategies for modulating gene expression at the transcript level, offering new opportunities to tackle previously inaccessible disease mechanisms. RNA-targeting small molecules provide a means to selectively interact with structured RNA elements within mRNAs and non-coding RNAs, influencing their stability, translation, or splicing. Expanding on this concept, RIBOTAC technology represents a next-generation RNA-targeting strategy that induces selective degradation of disease-related RNAs. By recruiting endogenous ribonucleases, RIBOTACs drive the catalytic degradation of pathogenic transcripts, thereby offering a highly specific and sustained therapeutic effect. In this study we will highlight recent advances, mechanistic insights, and the future potential of RNA-targeted drug discovery in overcoming critical challenges in medicine.