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Entering the RNA era of therapeutics

Article-Entering the RNA era of therapeutics

A remarkable opportunity for the developing world.

On 12 October 1768, the first smallpox vaccination was given by Edward Jenner to an era that would save billions of lives from life-threatening infections, reduce disability, and stir up an economic revolution. On 11 December 2020, the FDA allowed using the first mRNA vaccine, creating a paradigm shift no less critical than Edward Jenner’s act. Instead of introducing live attenuated, inactivated, or subunit of viruses, we can introduce an innocuous chemistry strip to produce a surface protein found in a virus to produce immunity to infection. The chemistry, the mRNA, does not enter the cell’s nucleus and cannot cause any harm to the body. (Figure 1)

Fig 1.jpg

Figure 1. Ab mRNA vaccine is a nucleotide sequence wrapped in a lipid coat of a nano size that allows quick penetration of the cell wall. Once it enters the cell, the lipid coat is removed, and the mRNA strip goes through a ribosome that produces the spike protein found on the surface of viruses. This protein is pushed out of the cell and returns to the Golgi apparatus if it had not already gone through it. The Golgi apparatus attaches a market (MHC class peptide) like a flagpole to invite the body’s immune system to attack the protein and produce long-term immunity. The mRNA strip simply gets destroyed. There is no impact on the DNA, or any genes present in the nucleus where the mRNA cannot enter.

It was the COVID-19 pandemic that forced the companies to create mRNA and the FDA to evaluate it. The safety and efficacy of the mRNA vaccines were way above any other vaccine ever produced.

All future vaccines will not be based on mRNA and prevent infections as the traditional vaccines do but to prevent hundreds of diseases of the immune system for which there are no therapies available or possible. This is truly a moment of revolution in the history of medicine. We will soon prevent Addison disease, Celiac disease-sprue (gluten-sensitive enteropathy), Dermatomyositis, Graves disease, Hashimoto thyroiditis, Multiple sclerosis, Myasthenia gravis, Pernicious anaemia, Reactive arthritis, Rheumatoid arthritis, Sjögren syndrome, Systemic lupus erythematosus, and Type I diabetes.

One question that is often asked is, “why was the mRNA vaccine not approved sooner?” While the RNA research dates back to the 1990s, it took 20 years to develop the technology of stable in vitro synthesis to produce RNA, to develop a technology to introduce the mRNA in the body by using nanoparticles in a lipid formulation and to establish formulations that will protect the highly unstable mRNA molecule. The big pharma, totally comfortable with their existing vaccine programmes, were in no hurry to invest in a new technology fearing regulatory constraints. This is all now history.

The safety of mRNA vaccines is assured because their production does not require toxic chemicals or cell cultures that could be contaminated with adventitious viruses, the common risks associated with all other vaccine platforms.

The RNA technology can be developed very fast; for example, Moderna developed the mRNA used in its COVID-19 vaccine within four days after receiving the genome sequence of the corona virus’s surface protein from the NIH that provides these data in the public domain.

A traditional path to develop an mRNA vaccine is shown in Figure 2.

Fig 2.png

Figure 2. The traditional process of mRNA vaccine development and commercial production. Growing the DNA in a bioreactor allows the production of an unlimited quantity of DNA at a meagre cost—one reason why mRNA vaccine can always be produced at a meagre price. The vaccine synthesis takes place in test tubes eliminating many risks of contamination with biological entities.

However, a newer approach uses PCR to produce sufficient mRNA to test the vaccine before entering the commercial stage that will significantly reduce the cost of development and time to market in the future (Figure 3).

Fig 3.png

Figure 3. A novel approach to develop mRNA vaccines. It involves using the PCR technology to eliminate several time and cost-consuming steps besides removing all possible contaminants. However, today, this technology is only available for small-scale production sufficient to produce enough mRNA to test safety and efficacy. Since the exact sequence of the mRNA is known, there is little risk in switching over to the commercial plasmid-based technology in the future. This dual-stage model is highly recommended for emerging companies to reduce their time to market significantly and at an extremely low cost. All steps described above can now be outsourced to several eligible CROs, eliminating the need to establish an in-house laboratory.

New vaccines

Some of the immediate targets for mRNA vaccines:

  • Influenza vaccine based on stalk domain as a long-term or permanent vaccine against influenza.
  • STD vaccine against HIV and HPV (contains multiple rRNAs)
  • Diabetes protection vaccine against the six strains of Coxsackie B (CVB) virus.
  • Antigens to produce antibodies against rogue antibodies: autoimmune disorders to prevent Parkinson’s disease, Alzheimer’s, and multiple sclerosis.

Advise to developing countries

Development and manufacturing of mRNA vaccines are straightforward and definitive because we can sequence the mRNA that we propose to us precisely and thus confirm the consistency of its batches—this had never been possible with any other vaccine in the past. With ample financial resources on hand, it should not take more than 24 months to develop and launch a new vaccine.

Today, the world is striving to get the mRNA vaccines against COVID-19. They will be aiming to get a vaccine to prevent diabetes. Given the ready technology, my suggestion is to all countries to initiate their mRNA vaccine production that does not require a significant capital investment and become self-sufficient, not just for the vaccine needs of today but for the future as the new era of medicines has just been opened.

Today, we have an opportunity to permanently eradicate the diseases that never had any treatment possible like autoimmune disorders; we have an opportunity to be self-sufficient in vaccine production without unaffordable investments of the past, and today, it is the day for the governments to make commitments to their citizens for a safe and productive life by producing their modalities and not depend on any other country to assist. COVID-19 is not the last pandemic—it pays to understand that we need to be ready for tomorrow.

References available on request

Dr Sarfaraz K. Niazi.JPG

Prof. Niazi is an adjunct professor at the University of Illinois in Chicago and Chairman of RNA Therapeutics, a U.S. company providing turnkey technology to produce mRNA vaccines. He owns more than 100 biotechnology patents and has authored over 100 research papers and 60 major technology books.

This article appears in the latest issue of Omnia Health Magazine. Read the full issue online today.

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