August 11, 2022

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Has the door to mRNA therapy opened?​​

Has the door to mRNA therapy opened?​​


Has the door to mRNA therapy opened?​​  After the COVID-19vaccines of Moderna and Pfizer-BioNTech went on the market, two mRNA therapeutic drugs appeared on the market, and they were the only two.

Has the door to mRNA therapy opened?​​

At least four years before the pandemic, Moderna and BioNTech were called mavericks. Their mRNA technology was completely unproven, but their market value was also high. Especially Moderna can raise a lot of funds from investors. In January 2017, it had approximately $1 billion in cash, which included a global health partnership with the Bill and Melinda Gates Foundation and a large grant from the Biomedical Advanced Research and Development Agency (BARDA) , But there were no products on the market at that time.

It was not until January 2017 that the company publicly released the latest information on its 12 mRNA development candidates, including vaccines and therapeutics for infectious diseases, immuno-oncology and cardiovascular diseases. Most of BioNTech’s efforts in mRNA are in the field of oncology. Until now, mRNA therapy is still promising, and no one doubts it.

Katalin Kariko has been working in his hometown of Hungary since the 1970s, engaged in the research of mRNA therapy. She and her family left Hungary in 1985 for the United States, worked at Temple University in Philadelphia, and then moved to the School of Medicine at the University of Pennsylvania. However, her research is considered too radical and risky.

Eventually, despite resistance from funders, she continued to work. She and colleagues at Drew Weissman University of Pennsylvania developed a method using synthetic mRNA. Now this is the basis of the COVID-19 vaccine, and Kariko is the senior vice president of BioNTech.

Stephane Bancel spoke in a podcast created by Silicon Valley venture capital firm Andreessen Horowitz. He described how he has been working in infectious diseases throughout his professional life and how he was “watching the development of the epidemic.” Therefore, one thing I want to do is to read the Wall Street Journal and the Financial Times every morning when I get up. Between Christmas and New Year (2019) last year, I noticed an article saying that a new pathogen in China has pneumonia-like symptoms, and nothing more. “

He regularly works with Barney Gram, who works at the National Institute of Allergy and Infectious Diseases (NIAID), and sent him an email asking him. He was told that what was little known at the time might be a virus, a new coronavirus, and within a few days, the Chinese will publish the sequence of the virus on the Internet.

Bancel said: “On January 11, 2020, the Chinese published the sequence, and our team at Moderna used the sequence to design a vaccine. Other teams did the same thing. When they shared these studies 48 hours later , They designed the exact same vaccine.”

The importance of this cannot be underestimated. They did not use the digital information of the virus genome, and did not actually get a sample of the virus, but were able to design a vaccine within 48 hours!

Bancel said: “We think this is computer technology. We have never been exposed to viruses. We designed the vaccine. We set up two teams at NIH and Moderna because we are very worried about making a mistake in vaccine design, as people can imagine. So, when the team compared the vaccine designs two days later, they were exactly the same, we were very happy. Because this is a virus outbreak, we know that every day is important. At the same time, we started Produce clinical-grade products and enter the first phase. What is really eye-catching is that the FDA reviewed the vaccine on December 17, which is exactly the same as the vaccine we designed in January. We have never changed an atom, it is The exact same molecule.”

Now that mRNA technology has ended the worst global pandemic in a century in record time, it has broken through the barriers of proof-of-concept, and researchers are looking for the next step in the development of mRNA therapeutics. As early supporters of the study believed, this seems to be the limit.

The Yale University research team recently applied for a similar RNA-based method to vaccinate against malaria. Pfizer said that because of its ease of editing, they plan to use mRNA for its seasonal flu vaccine. As mentioned earlier, BioNTech is studying cancer therapies, which are personalized therapies that can produce proteins related to specific tumors, thereby helping the body’s immune system to resist advanced cancer. Among other methods, mRNA treatment has slowed and reversed multiple sclerosis in mouse models.

Özlem Türeci, chief medical officer of BioNTech, told The Atlantic Times: “I believe more than ever that mRNA can achieve a wide range of transformations.” “In principle, everything that protein can do can be replaced by mRNA.”

This is not to say that there are no limits. As both Pfizer BioNTech vaccine and Moderna vaccine have proven, mRNA may be very fragile and currently requires very cold storage and transportation temperatures. Other diseases may also have major limitations. Infectious diseases are often regarded as the killer of mRNA therapy and are more easily applied.

John Mascola, director of the NIAID Vaccine Research Center, told the Atlantic Times: “This must be the upsurge of mRNA.” “In the scientific world, RNA technology may be the most important story of the year. We don’t know if it’s effective. Now we do. Arrived.”

However, before we start a new era of drug development, it is worth remembering that it took more than 40 years for the two products to reach their goals.

Although generally optimistic, Türeci pointed out: “I do not claim that mRNA is everything. We will find that mRNA for some diseases is surprisingly successful, and some diseases are not. We have to prove this one by one for each infectious disease.



(source:internet, reference only)

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