The COVID-19 pandemic forever changed how researchers approach disease prevention and treatment development as messenger RNA (mRNA) became the strongest weapon against SARS-CoV-2. Beyond COVID-19, mRNA therapeutics have the potential to treat other infectious diseases, genetic diseases, and cancer.1
Conventional disease interventions typically employ small molecule drugs, which are predominantly synthetic organic compounds that deactivate or inhibit target proteins through competitive binding. However, these drugs collectively target only 2–5 percent of the protein-coding human genome, which leaves many human diseases undruggable. mRNA therapeutics are poised to solve this problem as they can be designed to make any protein in the genome. For example, these therapies can replace a patient’s absent or dysfunctional protein through injection of mRNA that produces a functional version.2,3
Most nucleic acid-based therapies use an indirect viral vector-mediated gene delivery method to introduce a transgene of interest into tissues. However, directly inserting RNA into cells abolishes the ...
