Bayer AG products

Traditionally, many small molecule drugs follow the same basic mechanism of action to treat diseases: They inhibit the function of a target protein that has an active role in a disease. To do so, they require a tractable active binding site to occupy, which will result in protein function inhibition like blocking its enzymatic activity. But traditional small molecules can only block one single domain function of the protein, which means that potential other functions remain intact and can continue to fuel disease processes. This is one reason why it is assumed that traditional small molecule drugs can sufficiently address only a small percentage of the disease-causing proteins in the human proteome via the described mode of action. The great majority of proteins remains in the so called “undruggable space”. Novel chemical modalities with a different mode of action such as PROTACs^®, however, could offer a means to minimize this “undruggable space” .

Small molecules (SMOLs) have long been in the focus of modern medicine. Over the last century, their advent revolutionized healthcare and improved the lives of patients all over the world. Most of today´s SMOLs exert their function by binding to proteins, thereby modulating their ability to catalyze biochemical reactions. However, a lot of proteins are difficult targets for SMOLs, since they do not possess defined binding sites that can be used for SMOL drug candidates and this is making them hard to drug or even “undruggable”. A new and promising approach to solve this challenge may become possible in the future. The development of new technologies has recently unlocked a new class of target molecules for the application of SMOLs: ribonucleic acid, or in short: RNA.