While the COVID-19 vaccines introduced many people to RNA-based medicines, RNA oligonucleotides have already been on the market for years to treat diseases like Duchenne Muscular Dystrophy and amyloidosis. RNA therapies offer many advantages over traditional small molecule drugs, including their ability to address almost any genetic component within cells and to guide gene editing tools like CRISPR to their targets. However, the promise of RNA is currently limited by the fact that rapidly growing global demand is outpacing the industry's ability to manufacture it.
The standard method of chemically synthesizing RNA was invented in the 1980s, and requires specialized equipment and labor-intensive processes. Chemical synthesis methods are also limited in terms of the range of nucleotide building blocks that they can incorporate into RNA molecules, and they produce metric tons of toxic chemical byproducts that create environmental hazards and limit factories' production capacity. These problems will only increase as RNA production ramps up in response to demand.
A team of scientists at the Wyss Institute at Harvard University and Harvard Medical School (HMS) has created a solution to this problem: a new RNA synthesis process that expands the RNA therapeutic design space and unlocks the potential for rapid scale-up that chemical synthesis cannot achieve Their novel method can produce RNA with efficiencies and purities comparable to current industry standards using water and enzyme.