Science News

RNAi Gene Therapy: Gene Silencing Drug ‘Speaks Up’ Following FDA Approval

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?