RNA therapies and medicines – general overview and pharmacological challenges

Recent advances in the science of RNA production, purification, and intracellular delivery have enabled the development of RNA therapeutics. RNA therapies represent a rapidly expanding category of medicinal products that will change the standard treatment of many diseases, redefining the concept of personalized medicine. They may have great therapeutic potential in hereditary, oncological, and neurological diseases, metabolic diseases, etc. RNA therapies may provide better opportunities to target the underlying pathophysiological mechanisms of diseases, which in turn may lead to better therapeutic results. These medications are cost-effective to develop, relatively easy to manufacture, and hold potential for many presently incurable conditions. They are rapidly gaining traction in clinical practice. In this comprehensive review, we discuss the general concepts of the different classes of RNA drugs and their pharmacological properties. Furthermore, we provide an overview of RNA drugs that have already received approval from regulatory bodies such as the FDA and EMA, shedding light on their pivotal role in modern medicine.