16 March 2021

Matrix RNA on the rise

Money and talent are flowing into the biotechnology sector after the success of the COVID-19 vaccine

Igor Chernenkov, Finversia based on materials Reuters.

The success of COVID-19 vaccines based on matrix RNA (mRNA) has had a positive impact on the possibilities of using the new technology not only in other vaccines, but also in various difficult-to-treat diseases.

Scientists say that mRNA can fight diseases that cannot be treated with conventional drugs.

Moderna Inc's emergency authorization of vaccines in the US, one from partners Pfizer Inc and BioNTech SE – and their gene-based manufacturing processes have shown that the Food and Drug Administration (FDA) is open to wider use of previously untested technologies, according to interviews with eight leading experts in the field mRNA.

Based on these inflated expectations, billions of dollars of capital have flowed into companies focused on the use of mRNA technology – hundreds of millions in the last few months alone. According to experts, with the approval of the FDA technology, this area attracts the best scientific talents.

"More and more attention is being paid to mRNA now, as this technology has proven its effectiveness," he said Glenn Hunzinger, head of the American consulting firm PricewaterhouseCoopers in the field of pharmaceuticals and life sciences.

The regulator's approval for the use of vaccines "accelerates the process" for other mRNA treatments by providing real evidence that it is possible to secure fragile mRNA components during production and distribution, he said Josh Kellar, co-head of the COVID-19 group at Boston Consulting Group.

This technology is often compared to an operating system on a computer that allows drug manufacturers to change their target by inserting a new genetic code into an artificial form of mRNA, a natural chemical intermediary that instructs the body to produce certain proteins.

The advantage of this approach in vaccines is versatility and speed compared to standard technology, which requires a long time to produce and purify proteins and create a vaccine.

According to the Indian research company Roots Analysis, in 2020, given the urgent need that arose in connection with the coronavirus pandemic, more than $5.2 billion was invested in companies developing mRNA vaccines and therapeutics, compared with $596 million in 2019.

In just the past three months, mRNA company CureVac NV has raised $518 million, Arcturus Therapeutics Holding has raised $150 million, and Gilead Sciences Inc has agreed to a deal to develop a therapeutic HIV vaccine worth up to $785 million with Gritstone Oncology, an mRNA manufacturer.

According to Roots Analysis, more than 150 mRNA vaccines and therapeutic agents are being developed worldwide. Most of them are still undergoing early animal testing, but more than 30 of them have already been tested in humans.

mRNA technology is quite difficult to work with, which makes the future success of the treatment uncertain.

Instructions from mRNA are fleeting and non-specific as to where it gets to, which works well when cells force a harmless fragment of coronavirus to trigger an immune response. It is more difficult to send these orders to a specific tissue, such as the lungs or heart muscle, which require other delivery or encapsulation methods to protect the fragile mRNA molecule.

Most of last year's funding was directed to projects related to COVID-19, but allowed companies to promote other categories of diseases as well.

Moderna, for example, works on the treatment of heart disease, cancer and rare genetic diseases. Its most advanced non–COVID program is a vaccine against cytomegalovirus, which is the leading cause of birth defects in the United States.

According to the CEO Ron Renaud, Translate Bio Inc. could become the first company to offer mRNA treatment if its inhaled cystic fibrosis drug is shown to successfully deliver instructions for delivering a protein known as CFTR to the lungs.

The company expects to receive interim test results in the second quarter of this year. If the results on safety and efficacy are encouraging, the company will conduct a larger trial and then possibly apply for approval of the treatment in the United States.

People with cystic fibrosis, a life-shortening lung disease, have mutations in the CFTR gene that cause protein malfunction or its complete absence, which leads to the accumulation of thick mucus, which causes lung infections and other serious health complications.

"Most medications treat the side effects of the disease... mRNA is designed to make up for what is missing," he said Joe Payne, CEO of Arcturus, which is working on the development of mRNA vaccines against COVID-19 and influenza, as well as drugs for liver disease and cystic fibrosis.

Doctor Drew Weissman, professor of infectious diseases at the University of Pennsylvania's Perelman School of Medicine (Penn Med), is one of two scientists credited in 2005 with the revolutionary discovery of how to change the molecular structure of mRNA so that it remains stable enough to overcome protective obstacles in the body. 

According to Weissman, over the past nine months, 20 companies working in the field of mRNA have asked him to join their boards of directors, and the number of laboratories willing to cooperate with Penn Med in working with mRNA has almost tripled.

As fields such as cell therapy begin to develop, scientists are turning to mRNA companies, he said Jacob Becraft, CEO of Strand Therapeutics, which is working on technology to monitor the activity of mRNA treatments.

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