The ongoing Covid-19 pandemic has highlighted the potential for mRNA therapies. The mRNA technology has experienced decades of research efforts from scientists and various biotechnology companies. Discovered in the 1960s as an option to convert DNA codes into proteins, mRNA took several decades to emerge as promising therapeutic modality for various medical conditions. mRNA medicines make use of the usual biological processes to extract proteins and create the required therapeutic effect, due to which the treatment of several diseases that cannot be achieved using current technologies is made easy. mRNA therapeutics have gained major attention following the development of new synthesis as well as delivery approaches. The advances in the generation, purification and cellular delivery of RNA have widened the scope of RNA-based therapeutics for a broad array of applications. The technology is marked by its compelling advantages over traditional methods in terms of turnaround times, production speed and cost savings. Various clinical and preclinical studies have indicated promising results for protein replacement therapies, prevention of infectious diseases, tumor immunotherapy and cancer vaccines. In the historic global race to develop a vaccine, mRNA based Covid-19 vaccine from Moderna and Pfizer stand out as frontrunners. The unique characteristics of mRNA has made it of great interest to vaccine developers. While traditional vaccines have used live or inactivated pathogens to elicit an immune response, mRNA is responsible for cellular processes, building structures, regulating functions, and building structural components. mRNA vaccines work by introducing a part of mRNA that reacts to the viral protein. It accelerates production of antibodies and trains the immune system to recognize the virus in the future, thereby offering protection. Essentially a chemical process, production of mRNA vaccines eliminates the need for growing proteins or viruses, a key reason why vaccines by Moderna and Pfizer/BioNTech quickly reached human trials. For instance, Moderna`s vaccine was created in just six weeks. Securing approval for emergency use, the two vaccines demonstrated around 95% efficacy in eliciting immune response against the COVID-19 virus during clinical trials.

Beyond Covid-19, mRNA-based therapies hold promise for combatting cancer, infectious diseases such as malaria and flu, and rare genetic diseases. Results from early animal testing demonstrates the ability of mRNA-based therapeutics to provide protection against viruses such as influenza, Zika and rabies. With the effectiveness of mRNA vaccines against Covid-19, there is heightened research interest in pursuing mRNA platform for treating infectious diseases, for example, BioNTech is preparing to launch the first vaccine for malaria based on mRNA technology with plans to conduct clinical testing by the end of 2022. Similarly, Moderna also announced plans to conduct human trials for an HIV vaccine. Currently, of the total 44 ongoing clinical trials of mRNA vaccines, 23 of them are aimed at infectious diseases. Cancer research has used mRNA platform to trigger the immune system to target specific cancer cells. Currently, there are many clinical trials underway utilizing mRNA vaccines for various cancers. Most of the mRNA cancer vaccine trials are targeting tumors such as melanoma and kidney cancer. An mRNA vaccine could be custom-made to match the genetic signature of an individual`s cancer. This personalized mRNA vaccine could be used following surgery to program the immune system to search out and destroy any residual cancer cells, and thus prevent the cancer from recurring. For instance, the pipelines of Moderna and BioNTech include drug trials for treating cancers of the brain, breast, prostate, pancreas, lung, skin and other tissues. The mRNA vaccines market is projected to receive notable contribution from the cancer segment that is estimated to claim the leading revenue share over the coming years. The segment`s growth is augmented by increasing incidence of cancer. The condition remains one of the major causes of mortalities globally, with the number of new cases annually exceeding 23.6 million by the year 2030.

mRNA is also being pursued to aid in rapid development of drugs for rare diseases. Recent advancements in the structural composition of mRNA have led the scientific community to swiftly embrace it as a new drug to deliver missing genes to organs. The pipelines of Moderna, CureVac and BioNTech, three major biopharmaceutical companies, cover heart diseases, metabolic diseases and immunomodulators for applications in immuno-oncology. The introduction of synthetic mRNA into human cells opens scope for a replacement therapeutic option for conditions known for defective or inadequate synthesis of key proteins. The approach holds significant potential over protein replacement and gene therapies owing to its lower risk, affordability and less frequent dosage. mRNA can be used as protein replacement therapy to treat diseases caused by a lack of protein, or by defective proteins. Many mRNA-based protein replacement therapies are under development for the heart, lungs and liver. For example, Cardior Pharmaceuticals, a Germany-based biotechnology company, is working on RNA drugs for treatment of heart failure. Translate Bio developed a protein replacement therapy for cystic fibrosis, which is currently undergoing Phase 1/2 clinical trial. In addition, mRNA is being explored as potential alternative to the gene therapy for treatment of intractable medical conditions like sickle cell disease. Increased scientific interest and funding brought on by the pandemic is expected to further boost advancements in mRNA vaccine and therapeutics technologies. The widespread recognition is leading to new uses of mRNA being developed and approved.