Over recent years the pharmaceutical industry has gradually turned towards synthetic biology to assist with the discovery of new medicines. The ability to read and write DNA and RNA - the fundamental building blocks of molecular biology - underpins this pharmaceutical innovation. For example, with relative ease, researchers can now sample all of the RNA molecules in cells and this is opening up a wide range of new therapeutic possibilities.
For many years RNA has not been the focus of drug discovery, however, there is now increasing interest in using RNA as either a therapeutic or a drug target. In 2016 a team of researchers demonstrated that a small molecule could alter the messenger RNA of the gene SMN1, and therefore could be a treatment for spinal muscular atrophy, a progressive genetic disease that leads to muscle weakness and movement problems. This has fuelled the development of several new pharmaceutical start-ups, armed with synthetic biology tools, which are aiming to either ‘drug’ messenger RNAs or change the function of enzymes that modify RNA. These companies could open up whole new therapeutic areas. A good example of that potential came in August 2018, when the US FDA approved the first treatment, based on RNA interference (RNAi). This technology uses short RNA molecules to inhibit a gene’s function; Andrew Fire and Craig Mello received the 2006 Nobel Prize in Medicine and Physiology for its discovery.
Another area of the pharmaceutical industry where synthetic biology tools are set to help the discovery of new medicines, is biologics. These types of medicines, commonly an antibody, are engineered to bind specific target proteins that are involved in the development of a disease. To develop these, researchers need easy access to synthetic DNA that encodes antibodies. Gene variant libraries, where the DNA that encodes a protein is altered at specific regions, can also help to improve the binding specificity of antibodies.
Companies developing these new therapeutic options need access to high quality, reliable, DNA and RNA synthesis. This has traditionally been difficult to achieve and is a barrier to progress. Camena Bioscience’s next-generation technology, called gSynth, is a multi-enzymatic and modular assembly approach. Our technology is a reliable method for the accurate production of long synthetic DNA, RNA and XNA molecules, which will aid the development of new therapeutics.