COVID mRNA Vaccines are a Form of Gene Therapy

Posted on 01/25/2021

Late last year, the Pfizer and Moderna COVID vaccines jabs were both licensed for emergency use only by the U.S. Food and Drug Administration (FDA). As the vaccinations begin to spread among the world population, the growth of other gene therapies as a type of vaccination could increase. Institutional investors like sovereign funds and biotech venture capitalists have invested in a wide range of companies engaged in these therapies. The vaccine was able to be made quickly because researchers already constructed an mRNA platform for cancer and other vaccines under trial. mRNA technologies were developed to fight cancer. mRNA has been around for decades, but there was little need to explore it further. Moderna took the mRNA idea seriously, its name – a new word combining modified and RNA. Synthetic mRNA is much easier and quicker to produce in the lab than it is to inactivate or attenuate a virus.

Did you know there is an estimated 10^31 virus-like particles that exist on the Earth and they are present in the blood, nose, mouth, lung, vagina, gastrointestinal tract, conjunctiva, skin, and the mammalian genome?

Form of Gene Therapy

The mRNA vaccinations are a form of gene therapy, according to its definition in many parts of the world, including Europe. Gene therapies remain under strict regulation and few gene therapeutics have been approved by health authorities because of safety concerns. Some experts are less concerned with the long-term risks of the mRNA vaccines, but more concerned about the efficacy of them, as mRNA is very fragile and if not properly stored, could be destroyed. These molecules can fall apart at room temperature. mRNA is a very fragile molecule, meaning it can be destroyed very easily compared to DNA.

Traditional vaccines revolved around injecting part of the pathogen, such as a protein or sugar, to induce an immune response. The COVID mRNA vaccine partly works by inducing local inflammatory reactions to trigger the immune system. The synthetic mRNA material, wrapped in an oily bubble coating made of lipid nanoparticles, delivers instructions to cells to make spike proteins to fight the virus. When synthetic mRNA enters the human patient, the material fuses to cells and cell’s molecules start to decode the genomic sequence to build the spike proteins. The immune system recognizes the spike protein as a foreign invader and produces antibodies against it. If the antibodies later encounter the actual coronavirus, they are ready to recognize and destroy it before it causes illness. Furthermore, the mRNA in the vaccine degrades in roughly 72 hours in order to not combine with human DNA.

According to the Mayo Clinic website, “Gene therapy involves altering the genes inside your body’s cells in an effort to treat or stop disease.

Genes contain your DNA — the code that controls much of your body’s form and function, from making you grow taller to regulating your body systems. Genes that don’t work properly can cause disease.

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body’s ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.”

In an introduction of the National Center for Biotechnology Information (NCBI) research paper, “Gene Therapy Leaves a Vicious Cycle”, which was published online on April 24, 2019, “Gene therapy appears simple in principle but involves identification of affected gene(s), cloning and loading of a wild type or recombinant healthy version in a suitable vector for optimal delivery and expression in the target cells or tissue and thus has seen its fair share of hurdles. Because it often uses repurposed viruses to deliver therapeutic genes, gene therapy has been caught in a vicious cycle for nearly two decades owing to immune response, insertional mutagenesis, viral tropism, off-target activity, unwanted clinical outcomes (ranging from illness to death of participants in clinical trials), and patchy regulations (23, 28–31). This led to a sharp decline in research funding for basic, preclinical development and vector production via individual investigators grants such as R01 and program grants. Thus, with limited information of preclinical data and vector production, the number of clinical trials conducted worldwide did not rise steadily from 1999 to 2015 (32). Furthermore, funding of the actual clinical trial was not guaranteed even vectors have been produced and certified for human use at significant cost. The American Society of Gene Therapy has taken lead in fixing this fragmented funding method by making many recommendations including the elimination of redundant regulatory processes and establishment of the National Gene Vector Laboratories (NGVL) to review vector production and toxicology. Now, with new technological advances in gene delivery and editing methods, increased enthusiasm of clinicians and drug companies, the advent of several viral-based drugs in the market, and the potential to provide a one-time treatment option without corrupting the genetic code, gene therapy is breaking free of this cycle. Undoubtedly, the resurgent interest in offering gene therapy-based treatments is one of the most defining developments in the pharmaceutical industry and is expected to have far-reaching implications on curing dangerous diseases in the future. With an estimated US $11 billion market in the next 10 years, both clinical trials and pharmaceutical industry are anticipated to benefit immensely from gene therapy. Here, we describe popular viral vectors used in gene therapy and gene therapy drugs available in the market.”

The paper also says, under its “Risks Associated With Viral Vectors” section, “The general concerns with viral vectors are the risks of an immune response, off-target effects, inflammation, and insertional mutagenesis. An immune response could make a viral treatment less efficient, or the resulting creation of antibodies could preclude a second dosage of the same virus (244–248). Inflammation was seen as a worst-case scenario in the 1999 death of Jesse Gelsinger caused by a very high dosage of adenovirus (249). Tailoring the viral dose to the patient, however, can better control this risk. Also, insertional mutagenesis is a major obstacle that the gene therapy field must overcome. The risk of inserting a gene into a tumor suppressor gene or activating an oncogene is present for the vectors that integrate into the unwanted locations of the genome, such as retrovirus. To counter this, vectors can be used that do not integrate readily into the genome.”


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