Twenty years after the molecular mechanism of gene silencing was unraveled, researchers are making critical steps forward following the US Food and Drug Administration (FDA) approval of a new gene silencing therapeutic. The drug, Onpattro (Patisiran), licensed by Alnylam Therapeutics, is designed to treat patients with hereditary transthyretin-mediated (hATTR) amyloidosis, a rare but life-threatening disease that causes damage to the peripheral nerves. The drug works by targeting RNA before it has a chance to be translated into a disease-causing protein. Now that this drug approval barrier has been overcome, have doctors and researchers entered a new era of treating genetic diseases?