Patisiron (Onpattro), a RNA-drug based on gene silencing was cleared by the U.S. Food and Drug Administration (FDA) regulations recently. This approval will pave the way for new generation drugs by targeting disease causing genes.
The mechanism of RNA interference which involves silencing of target gene by short stretch of complementary RNA molecule has been fascinating the scientific fraternity ever since its discovery. Two scientists viz., Andrew Z. Fire and Craig C. Mello shared the Noble Prize in Physiology or Medicine (2006) for their path breaking finding about RNAi which is the control mechanism that determines the flow of genetic information.
RNAi is outcompeting other small molecule drugs because of its higher targeting capacity for almost all the genes. This technique can be utilized for treatment of various diseases and disorders. Although it is enthralling for scientists, they have a formidable task ahead of them in terms of safe and effective delivery of RNA-based drug in the target tissues. Researchers from University of Massachusetts took up the challenge of developing specific carriers and came up with lipid-based carriers targeting the liver tissues for metabolic disease like hypercholesterolemia, hepatic steatosis and hyperlipidemia. Further, chemical modification of these drugs proved to be safe, effective, stable and potent that aids in reducing the immunogenic response of RNA drug in clinical trials. Approximately every other person in the world is affected by obesity/diabetes mellitus, these therapeutics offers an attractive alternatives for their treatment.