Merck Invests in RNA Startup to Target Cancer

Pharmaceutical giant Merck has announced a $200 million licensing agreement with Moderna Therapeutics, a startup based in Cambridge, Massachusetts, that is developing therapies based on RNA. Merck wants to combine its expertise in cancer immunotherapy with Moderna’s personalized cancer vaccines so that the two treatments together can more effectively target cancer.

Moderna hopes to have its tailored cancer vaccines ready for clinical trials in 2017. The company’s novel therapeutics are essentially modified molecules of messenger RNA, or mRNA, which translates the genetic code of DNA into proteins. Rearranging the nucleotides, or letters, in mRNA allows Moderna to create antibodies and other proteins that are useful in therapies for a wide range of conditions.

Theoretically, therapeutic mRNA should allow for the production of virtually any protein in the body. But if it sounds simple, there’s a reason it didn’t gain traction before. If straight mRNA is injected into the bloodstream, the body attacks it in an immune reaction, mistaking it for a virus. Moderna’s treatments overcome this challenge by swapping two of the mRNA letters for natural analogues that don’t trigger a viral immune response.

There are several other companies with similar mRNA-based therapeutics in the works. Shire announced work on using mRNA for a cystic fibrosis treatment in 2014, but it has remained silent on the development ever since. And the German biotech company Ethris is working on mRNA therapy for rare diseases.

Most of the competition in RNA therapy comes from companies working with a different technology: RNA interference, or RNAi, which can block the activity of naturally occurring mRNA. The idea is to halt the production of harmful proteins or the growth of cancer cells. Alnylam, also based in Cambridge, Massachusetts, has two RNAi therapies in late-stage human trials and several more in early clinical testing.