RNA-based Therapeutics

 

RNA is an essential molecule in cellular biology, translating genetic instructions encoded in DNA into the production of the proteins that enable cells to function. However, as protein production is also a central factor in most human diseases and disorders, RNA-based therapeutics have long been thought to hold the potential to treat a range of problems where conventional drug-based treatments cannot offer much help. The field has been slow to develop, however, with initial high hopes being dented by the sheer complexity of the effort and the need to better understand the variability of gene expression in cells.
Over the past year, there has been a resurgence of interest in this new field of biotech healthcare, with two RNA-based treatments approved as human therapeutics as of 2014. RNA-based drugs for a range of conditions including genetic disorders, cancer and infectious disease are being developed based on the mechanism of RNA interference, which is used to silence the expression of defective or overexpressed genes.
Extending the repertoire of RNA-based therapeutics, an even newer platform based on messenger RNA (mRNA) molecules is now emerging. Specific mRNA sequences injected intramuscularly or intravenously can act as therapeutic agents through the patient’s own cells, translating them into the corresponding proteins that deliver the therapeutic effect. Unlike treatments aimed at changing DNA directly, RNA-based therapeutics do not cause permanent changes to the cell’s genome and so can be increased or discontinued as necessary.
Advances in basic RNA science, synthesis technology and in vivo delivery are combining to enable a new generation of RNA-based drugs that can attenuate the abundance of natural proteins, or allow for the in vivo production of optimized, therapeutic proteins. Working in collaboration with large pharmaceutical companies and academia, several private companies that aim to offer RNA-based treatments have been launched. We expect this field of healthcare to increasingly challenge conventional pharmaceuticals in forging new treatments for difficult diseases in the next few years.