4th of February 2021

Vaccinations with mRNA-based medicinal products

How do mRNA-based medicinal products work and how safe are they?

Referring to the public discussion on the currently introduced mRNA vaccines, our pharmaceutical industry consulting company would like to make its contribution to clarification and knowledge transfer. For many years, we have been supporting research-based companies and manufacturers of novel therapies. We advise and support them in their conception and regulatory compliant implementation of the development and marketing of advanced therapy medicinal products, including cell and gene therapy approaches. These therapies are summarized under the term ATMP (Advanced Therapy Medicinal Products), which also includes mRNA-based medicinal products.

mRNA-based medicinal products are innovative therapeutics that have only been developed in recent years. However, many research papers have already been published on basic mechanisms and tolerability. We know they can, among other things, stimulate the immune system, which is then "primed" for a specific immune response. Research on immune stimulation has been ongoing for 30 years. The focus is on the treatment of civilization diseases, in particular the development of cancer therapies. In Germany, several clinical studies for therapy of highly malignant tumors in the indication of skin and breast cancer have been and are currently being conducted by the German company BioNTech (https://clinicaltrials.gov/ct2/results?term=biontech). The results of the studies are remarkable but not comparable to the breakthrough success of the promising Covid-19 mRNA vaccine BNT162b2 from BioNTech/Pfizer (Polack et al., 2020).

mRNA-based medicinal products use mRNA as a carrier for information by means of which body cells make proteins that can fight or prevent disease. The new mRNA vaccine BNT162b2 from BioNTech/Pfizer contains the construction plan for viral envelope proteins of SARS-CoV-2 (antigen) packaged in a lipid nanoparticle for injection into patients. Body cells of the patients take up the mRNA and generate the vaccine antigen. Those cells present this antigen to the immune system and this engenders the desired immune response. As a vaccinated person comes into contact with the virus, the immune system recognizes the antigen and fights the virus. Production period of mRNA vaccines is much shorter and also much safer than of conventional vaccination systems. BioNTech/Pfizer is also taking the approach of packaging multiple mRNA information into one vaccine. The new mRNA vaccine BNT162b2 also contains different mRNAs that code for different antigens of the virus. This has the advantage that a sufficient immune response can be triggered in patients even in the presence of viral mutations.

Syringe with mRNA vaccine

mRNA-based vaccines are currently the subject of much debate, but are still more heavily regulated to ensure quality, safety and efficiency.

mRNA-based medicinal products, in addition to CAR-T cells, are a promising new approach in cancer therapy to treat diseased patients individually, effectively, rapidly, and safely. mRNA target sequences could be derived from the NY-ESO-1 gene in skin cancer and from ErBB2 in breast cancer (Thomas et al., 2018; Waks and Winer, 2019).

mRNA-based medicinal products, like all other medicinal products worldwide, are strictly regulated and controlled by regulatory agencies for their safety. Whenever new medicinal products or biologically active substances are developed, their composition and functionality must be specified and their identity, safety and functionality must be proved during manufacturing. These specifications already apply to clinical trials, which must also meet a correspondingly high standard of quality. The already high quality standards in the clinical trials made it possible that results of the mRNA vaccines could be published surprisingly quickly and successfully.

For the future, we hope that the cultural achievements of vaccination and the scientific progress of such medicinal products will be regarded as outstanding developments of our time. While scientific findings may and must be constantly questioned and require control, a trust in innovative developments and their regulations, which are very familiar to our GMP consultants team, is also a basic prerequisite for the containment and suppression of the Corona pandemic. Lastly, however, it should be emphasized that other mRNA-based ATMPs may be equally important to us. Strictly speaking, they represent, for example, "cancer vaccines" that make an important contribution to the fight against difficult-to-treat diseases. We are looking forward to supporting such impressive innovations with you and for you.

Look here for more information specially for ATMPs: GMP for ATMPs | Valicare GmbH

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Author: Dr. Melanie Zimmermann, GMP Documentator, Valicare


Polack FP, Thomas SJ, Kitchin N, Absalon J, Gurtman A, Lockhart S, Perez JL, Pérez Marc G, Moreira ED, Zerbini C, Bailey R, Swanson KA, Roychoudhury S, Koury K, Li P, Kalina WV, Cooper D, Frenck RW Jr, Hammitt LL, Türeci Ö, Nell H, Schaefer A, Ünal S, Tresnan DB, Mather S, Dormitzer, PR, Sahin U, Jansen KU, Gruber WC, C4591001 Clinical Trial Group. „Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine .“ N Engl J Med, 2020: 383(27):2603-2615.

Thomas R, Al-Khadairi G, Roelands J, Hendrickx W, Dermime S, Bedognetti D, Decock J. „NY-ESO-1 Based Immunotherapy of Cancer: Current Perspectives.“ Fron Immunol, 2018: 9:947.

Waks AG, Winer EP. „Breast Cancer Treatment: A Review.“ JAMA, 2019: 321(3):288-300.

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