MRNA vaccine production has gained widespread attention since the COVID-19 pandemic began. These vaccines work by providing the body with the genetic code for a specific protein, allowing the immune system to recognize and respond to it. For the experts at contract development and manufacturing organization (CDMO) Samsung Biologics, the key question is: What other therapeutic applications does this technology hold beyond COVID-19?
“In October 2021, a total of 49 mRNA prophylactic vaccine candidates were in clinical development. Many of these vaccines target infectious diseases that have to date eluded effective vaccine solutions, including malaria, influenza, and HIV/AIDS,” explained Samsung Biologics’ Esther Yoo in a recent white paper. “There are also significant efforts underway to develop a universal flu vaccine. Many cancer vaccines based on mRNA are also progressing through clinical phases.”
Some of the many advantages of mRNA vaccine production include its easy scalability, the fast rate of vaccine testing and manufacture, and the ability to design and share information on mRNA quickly using digital technology.
With these and other advantages in mind, Samsung Biologics has invested in an end-to-end mRNA vaccine production suite. The CDMO can now produce the active mRNA drug substance, encapsulate and purify it, then transfer it to a final drug product via an aseptic fill/finish process. It also maintains the precise cold-chain storage conditions needed to ensure the stability of mRNA molecules.
How Does mRNA Vaccine Production Work?
Designing mRNA vaccines involves several steps. The first is identifying the target antigen, which is the specific protein that the vaccine will help the body recognize and fight against. Once the antigen is identified, the mRNA sequence for that protein is synthesized in the lab. The mRNA is then modified by adding a protective coating, which helps the mRNA avoid being degraded by the body’s immune system before it can enter cells. The final step is packaging the modified mRNA into lipid nanoparticles, which allow the mRNA to be easily taken up by cells in the body.
Quality control measures are critical at each stage to ensure the final product is safe and effective. This process requires specialized facilities and equipment. As Yoo explains, Samsung Biologics has invested in the equipment needed to produce mRNA at a variety of scales.
“The facility allows production from lab to commercial scale; from 5 to 200 liters. Furthermore, the equipment needed to produce three different types of lipid nanoparticles has been installed and configured to seamlessly select the optimal formulation for drug delivery. There is also a dedicated process development laboratory for characterizing processes before scale-up,” Yoo said.
The Advantages of mRNA
Once these facilities are installed, manufacturers like Samsung Biologics can take advantage of the unique upside that mRNA vaccine production has over traditional methods.
Traditional vaccine development typically involves growing large quantities of the virus or bacteria that causes the disease, then inactivating or weakening the pathogen before it is used to create the vaccine. This process can take years and is often hindered by the need to grow and manipulate the pathogen in a laboratory setting.
By contrast, mRNA vaccine production does not involve growing an actual virus or bacteria that causes disease. Instead, scientists only need the genetic sequence of the virus or bacteria, which can be obtained much more quickly than growing the pathogen.
As artificial intelligence technology develops, more scientists are using the technology to help identify the appropriate genetic sequence. Once the genetic sequence is obtained, it can be quickly synthesized in the lab.
Additionally, because mRNA vaccine production does not involve the use of live pathogens, the process does not carry the same safety concerns as traditional vaccines. This means that the safety testing required for mRNA vaccines is typically less extensive than for traditional vaccines, which can further accelerate the development process.
New mRNA Vaccine Applications
While much of the attention on mRNA vaccine production has focused on the COVID-19 vaccine, the technology holds promise for a wide range of therapeutic applications.
One under-discussed use of mRNA is as a treatment for ultrarare genetic disorders. By delivering functional mRNA to cells, mRNA therapy could potentially correct genetic mutations that cause these diseases, and the low cost of editing mRNA during the mRNA vaccine production process makes the technology an ideal fit for rare conditions. One example of this is an mRNA therapy in development to treat a rare genetic disorder called Crigler-Najjar syndrome, which causes a buildup of bilirubin in the blood.
The same idea applies to less-rare genetic disorders, such as Huntington’s disease. This disease occurs because of a specific genetic mutation, and mRNA could potentially instruct the body to correct this mutation.
Another potential use of mRNA therapy is in cancer treatment. mRNA vaccines could be designed to target specific cancer cells, helping the immune system to recognize and eliminate them. Early clinical trials have shown promising results in using mRNA vaccines to treat melanoma, colorectal cancer, and other widespread types of cancer.
In the white paper, Yoo said that “mRNA technology could potentially broaden treatment options for many diseases and disorders that smal-molecule and biologic drugs are not able to tackle.”
