
Aldevron Breakthrough Blog
Perspectives: Where do we go from here?
April 5, 2023 / by Aldevron
Wide-ranging potential for mRNA vaccines
Overnight success is often the result of years of effort and setbacks, which could be the life story of an mRNA pioneer, Dr. Katalin Karikó, now with the Perelman School of Medicine at the University of Pennsylvania. Karikó spent decades focusing her research on how mRNA could be used for therapies, which ultimately helped lead to the development of COVID-19 vaccines.
In an interview with The Telegraph, a news service in the United Kingdom, Karikó says the potential of mRNA is limitless, including treatments for conditions from cancer and malaria to cystic fibrosis. From the developments I’ve seen over the past two years, and happening right now, that evaluation seems on target.
The impact of mRNA COVID vaccines is well documented and has paved the way for other mRNA-based vaccines, such as for influenza and respiratory syncytial virus (RSV); and even the possibility of a multivalent vaccine immunizing against COVID and/or influenza and/or RSV. That is an exciting prospect and serves to highlight the potential of mRNA technologies to impact global healthcare.
Many traditional vaccines, including the flu vaccine, are manufactured via a process using chicken eggs to generate and produce the vaccine antigen. This process is laborious, with each step requiring several weeks:
- to prepare and verify the hybrid vaccine strain,
- to optimize growth conditions in the egg, and
- to grow and purify the vaccine antigen.
mRNA-based vaccine manufacturing is not constrained by these, offering the potential to reduce the overall manufacturing time required.
Beyond infectious disease
Karikó mentioned mRNA cancer vaccines as well, which is something that is now being heavily pursued by academia, biotech and pharma. However, there are a number of obstacles to overcome. mRNA encapsulated LNPs preferentially localize to the liver. This property is of benefit for treating liver diseases and cancer. The question is, how do you get target and direct the LNP to other tissue types? This is not an easy problem to solve.
An alternative approach to LNP targeting is to modulate and control tissue specific expression, thereby enabling expression only in appropriate cells or tissues. Regardless, any mRNA cancer vaccine would likely be one part of a patient’s overall treatment plan and would likely be utilized in combination with traditional chemotherapeutics.
Karikó’s outlook and vision regarding the potential of mRNA Tx and Vx is encouraging. mRNA has the potential dramatically improve human health care in the near term. Not only infectious disease prevention and cancer treatments. The implications for patient’s suffering from rare and ultra-rare diseases are also exciting. These examples are just a few reasons that show why mRNA development is worth pursuing.