The concept of RNA as a medicine is not new. The act of using ribonucleic acid molecules to treat or prevent diseases by affecting biological pathways has been pursued for almost 50 years. Antisense oligonucleotides were first synthesized in the 1970s; aptamers first described in the 1990s; the first use of exogenous mRNA to induce the expression of a protein in vivo occurred in the 1990s; and microRNA was discovered in 1993.
These efforts and proofs of concept have been translated into actual commercial products. Since 2004, 18 RNA therapeutics or vaccines have been launched, mainly antisense and RNAi therapies. The latest...