The FDA approved Onpattro last week, the first approved medicine to use small interfering RNA (siRNA) to prevent formation of disease causing proteins in the body. This class of therapeutics, known more generally as RNAi, is expected to create breakthroughs in the treatment difficult diseases caused by the deposition of abnormal proteins such as hereditary transthyretin-mediated amyloidosis (the indication for Onpattro) or other targets such as cancer, cardiovascular diseases, inflammatory diseases, and viral infections. To my mind the real news is the potential for anti-aging treatments, since buildup of abnormal proteins and protein fragments is known to play a role in cellular senescence (and subsequent aging). There are a few tricky problems to overcome, as RNAi medicines have so far occasionally triggered negative immune responses and Onpattro requires co-administration of a steroid, acetaminophen, and antihistamines to decrease the chance of having immune reactions. The medicine also requires a lipid delivery system, which makes the dose form an injectable, but that’s proven to be only a minor challenge for determined biohackers. The size of the market alone will guarantee commercial interest.

Text Copyright © 2018 Katrina Rogers