The future (and the now) of mRNA therapeutics

The success of the mRNA vaccines, in terms of efficacy, safety, and the speed with which they were developed has been staggering. But they are just the beginning! The vignettes below argue for the near term future of mRNA as a therapeutic. As is the nature of scientific progress, we cannot imagine what lies beyond these solutions to human health.

Why mRNA? Perhaps you've heard of protein biologic medicines? Insulin is an early example, but any medicine with the ending "mab" is in a particular class of protein (antibody) biologic: Humira (adalimumab), Rituxan (rituximab), Keytruda (mepbrolizumab).

Remember "DNA makes RNA makes protein" from a biology class? Instead of producing these protein medicines in cultured cells or organisms, why not have our own cells make them (our cells are good at that!)? mRNA is a chemically simpler biologic that is easy to make, pure and when needed, in large quantities.

Vaccines. A variety of vaccines are following on the heels of (and were in development before) the COVID vaccines. Moderna has recently demonstrated a flu vaccine and was chosen by Time as the World's Most Impactful Company of 2026, but many other companies are active in this space. mRNA vaccines are readily adapted as new virus strains arise.

in vivo CAR-T cancer therapies. CAR-T therapy is a personalized medicine that trains your own white blood cells to recognize and destroy your cancer cells. In so-called ex vivo therapy, cells are harvested from a patient, re-trained and put back in the body. It works, but needless to say it is invasive and expensive. More recently, in vivo CAR-T therapy has proven to be much simpler - mRNA is used to train the T-cells directly in the body (see also mRNA cancer vaccines, another form of personalized medicine). There is little question that the future for fighting cancer is bright, but we need a system for preparing patient-specific (personalized) mRNA, quickly, safely, and at reasonable cost.

Personalized gene editing. This sounds like scary sci-fi stuff, but this new medical treatment (which requires mRNA, as well as a shorter sgRNA) is game changing for people with severe, inherited genetic diseases. Read about Baby KJ's success story to learn more.

Autoimmune diseases (e.g., inflammatory diseases like lupus). In approaches similar to the above, this mRNA therapeutic modality targets "flags" on malfunctioning B cells that cause autoimmune inflammation. The same benefits as above: an off-the-shelf therapy, rapid development, and substantially less expensive and less invasive than current approaches.