The science is still in early stages, but companies such as Remix say aiming chemical-based medicines at RNA molecules is the natural next step as drugmakers run out of proteins they can easily target with other methods.
RNA acts as a messenger inside cells, carrying instructions from DNA for protein production. Remix’s technology is designed to “tune” that process to attack the origin of a disease by degrading, boosting or altering RNA. The company aims to do that with small molecule drugs, rather than the nucleic acid therapies used by companies like Alnylam Pharmaceuticals and Ionis Pharmaceuticals.
The promise of using small molecules to target RNA has resulted in a flurry of deals between more established pharmaceutical makers and smaller companies.
Over the last two years, the Massachusetts biotech Arrakis Therapeutics has signed deals with both Roche and Amgen to develop drugs that modify or destroy RNA. In each case, the cash paid upfront was relatively modest, but the agreements hold the potential for “several billion dollars” in future payments if the research pans out.
Roche last year also joined forces with a North Carolina biotech called Ribometrix to target RNA molecules. In 2020, AstraZeneca teamed with Accent Therapeutics and Vertex announced plans to work with Skyhawk Therapeutics. Like the Arrakis deals, the Ribometrix, Accent and Skyhawk agreements are backloaded with big payments dependent on clinical success.
Remix, based in Cambridge Massachusetts, launched in December 2020 with $81 million in financing.