In the RNA

In August 2018, the FDA approved the first of a potentially game-changing class of drugs - small interfering ribonucleic acids (siRNAs). In a press statement announcing the approval of Onpattro (Patisiran), FDA Commissioner Scott Gottlieb announced, “New technologies like RNA inhibitors that alter the genetic drivers of disease have the potential to transform medicine so we can better confront and even cure debilitating illnesses (1).”

This new class of drugs are unique because they work at the RNA level to specifically silence the production of disease-causing proteins. Whilst initial research has found application in oncology and infectious disease, the technology is now being more broadly applied across a broader range of therapeutic areas.

DNA technologies including gene therapy or gene editing attempt to fix a dysfunctional gene or re-introduce an intact version of a gene. In contrast, RNA based approaches utilize a cell’s own internal machinery to alter the expression of genes. This broader range of applications gives RNA technology a significant advantage over DNA technologies.

RNA technologies have the potential to target the root causes of disease and help bring more novel drugs to patients. WuXi Xpress spoke with pioneering researchers and companies in the field as part of its Innovation that Matters series (2). Here, we summarise their discussions…