10 RNA Therapeutics Companies To Watch In 2019

(RTTNews) - After decades of research and development in RNA interference (RNAi), considered one of the most exciting discoveries in biology, the year 2018 saw the first-ever RNAi drug pass regulatory muster in the United States.

Alnylam Pharmaceuticals' (ALNY) Onpattro approved, in August of 2018, for the treatment of nerve damage caused by a rare disease called hereditary transthyretin-mediated amyloidosis (hATTR) is the first RNAi drug to hit the U.S. market.

RNA interference, or RNAi, is a natural mechanism present in living cells that selectively "turns off" or "silences" the expression of a specific gene, thereby affecting the production of a specific protein that contributes to disease. This cellular mechanism was discovered in 1998 by American biologists Andrew Fire and Craig Mello, and they were awarded the Nobel Prize in physiology or medicine in 2006 for their discovery. Scientists are leveraging this naturally occurring mechanism to prevent or treat diseases.

Another technology that targets RNA is known as antisense technology. In this technology, synthetic single-stranded strings of nucleic acids, known as antisense oligonucleotides, bind to RNA and thereby alter or reduce expression of the target RNA.

According to a recent report by Grand View Research, the global antisense and RNAi therapeutics market will register an 8.6% CAGR in terms of revenue, reaching $1.81 billion by 2025, from $945.8 million in 2016.

Below is a list of some of the biotechnology companies developing RNA-based therapeutics, and the latest update on their pipeline.