RNA technology provides an innovative approach for developing new drugs for rare or difficultto-treat diseases. Since 2014, several drugs been approved to treat macular degeneration, Duchenne muscular dystrophy (DMD), polyneuropathy, and amyotrophic lateral sclerosis. Drugs to treat many other diseases, including cancer, hepatic and renal diseases, cardiac diseases, metabolic diseases, blood disorders, respiratory diseases, and autoimmune diseases are currently in various stages of clinical studies, some showing promising results.