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mRNA: The final frontier of genetic medicine

27 January 2021

GAM Investments’ Dr Jenna Denyes discusses the validation of mRNA technology and how it is set to drive the pace of innovation in the genetic medicine space.

Broken down to its most basic concepts, biology is reasonably straightforward, even when you are looking at something as complicated as genetic medicine, or an mRNA (messenger RNA) vaccine. The right job needs to be completed at the right time in the right place. When something in this simple concept goes wrong, disease occurs. For example, when infected with a virus, an individual’s immune system needs to recognise the virus and the cells infected by the virus, and eliminate the threat before the virus can do too much damage. Most of the jobs we need to get done in our cells are performed by proteins, and the information to make these proteins is stored in our DNA, the most important type of genetic material. Every single cell in our body has the same DNA; each has the information to make every single protein, but very clearly, the cells in our eyes work differently than the cells that grow our fingernails, or the cells that make up our immune system. How does this work? This is where RNA comes in. RNA does many things in our cells, more than DNA. For simplicity, we can think of it as the messenger step between DNA, which is the same in every cell, and getting the right proteins in the right place at the right time. When we talk about genetic medicine, we are referring to different ways we can use DNA and RNA to make sure the job gets done, and disease is treated or prevented from developing in the first place.

In 2020 both Moderna and Pfizer / BioNTech successfully developed vaccines against Covid-19 which use mRNA, a type of genetic medicine which biopharmaceutical companies have been developing for more than 20 years. In the past five years we have seen approvals for gene therapy, genetic medicine which works on the DNA level, and we have seen some different RNA therapies which work by interfering with protein production. However, this is the first successful development of a therapy or a vaccine which puts an mRNA into a patient, which gets turned into a protein, to make sure the right job gets done at the right time by the immune system. When the mRNA from the vaccine goes into the cell and is used to make the viral protein, this kicks off a cascade of important messages for the immune system which culminates in the immune system producing antibodies, memory B cells, and memory T cells to keep in its database, which lets it react quicker to the virus next time it is found. The clinical trial data is compelling; the vaccines are more than 94% effective while also being safe to use in a wide swath of the population. This validation of the mRNA technological platform, done at warp speed through the massive investment of capital and resources globally, brings us to the final frontier of genetic medicine.

Moderna and BioNTech are only two companies of many working on mRNA therapies and combined their pipelines include 43 named investigational drugs targeting diseases ranging from infectious through to cardiology, auto-immune and oncology. These therapies look to supply missing proteins to support cell function, add in a missing signal to let the immune system attack a tumour, or turn off a pathway which is running out of control and letting the body attack itself. This goes some way towards explaining why Scott Gottlieb, the former US Food and Drug Administration (FDA) commissioner, predicts that by 2025 the US will be approving 10 to 20 genetic therapies a year. Currently, most of these therapies are targeting rare diseases which usually mean small populations. However, with a lack of viable alternatives, countries quickly see the value-based argument for their price tags, similar to what we see with Zolgensma from Novartis, used to treat spinal muscular atrophy, with a one-time treatment costing approximately USD 2 million. Novartis expects Zolgensma to generate global sales of USD 2.5 billion by 2025. The total global market size in 2019 in terms of revenue for genetic medicines was approximately USD 500 million (mostly Zolgensma). In 2021, Moderna alone has already signed pre-orders totalling USD 11.7 billion. A number of companies will see this technological validation read through to their pipelines, which we anticipate will continue to drive the pace of innovation in the genetic medicine space, bringing both treatments to areas of unmet medical need as well as considerable commercial returns across the space.

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