The expanding world of RNA therapies

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Note from the editor

Genetic messenger molecules power the coronavirus vaccines developed by BioNTech and Moderna, training the body's immune system to detect and defend against disease and infection.

Known as messenger RNA, the molecules have for years been the focus of intense research and development efforts by drugmakers, drawing attention well before the spectacular successes of the two mRNA vaccines.

BioNTech, Moderna and others like CureVac and Translate Bio aim to use mRNA to design vaccines for viruses like cytomegalovirus, influenza and Zika, as well as in therapies for cancer, immune disorders and diseases of the lungs and metabolism.

More broadly, RNA, or ribonucleic acid, is squarely in drug developers' sights after the clinical success of multiple medicines that block gene expression by disrupting RNA. RNA-targeting therapies from Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Biogen and Sarepta Therapeutics are now approved for several nerve, liver and neuromuscular diseases.

Those drugs all use interfering strands of RNA to do their jobs. Over the past several years, a number of biotech companies have made strides in constructing small molecule drugs to target RNA, too — an approach long considered futile. Their work has led to a flurry of dealmaking, as large pharmaceutical companies buy into the field and expand their research. Roche, for example, won approval of the first such drug last year.

Read on for a look at the expanding world of RNA therapies, starting with a startup whose ambitions are even greater than Moderna's.

Ned Pagliarulo Lead Editor

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