Before the pandemic, Moderna and BioNTech were each working on using mRNA for therapeutic purposes. Moderna paired with AstraZeneca on an mRNA therapy to regenerate heart tissue patients with heart failure. Their mRNA encodes a protein called vascular endothelial growth factor A, which promotes new blood-vessel growth. A phase 1 trial completed in early 2019 showed the mRNA, after being injected into the skin of men, caused a localized production of the protein without severe side effects. Last month they reported positive early results from a Phase 2 trial.
As Mr. Rossi recognized a decade ago, mRNA also offers the potential to reprogram cells. “We have shown that mRNA can be used to take a blood cell and generate a stem cell,” Dr. Sahin says. “This opens up the potential to address various diseases including aging and tissue repair.” Future mRNA uses could include stimulating the production of cartilage to ease arthritis and collagen to reduce wrinkles.
Autoimmune diseases, in which the immune system attacks parts of the body, are another promising area of research. BioNTech this year published a study that showed an mRNA vaccine has potential to treat multiple sclerosis without suppressing the immune system like existing therapies do.