The next chapter on MRNA has nothing to do with the COVID-19 vaccine

NSIt’s safe to say that before the development of Pfizer-BioNTech When modern Most people haven’t thought about messenger RNA or mRNA since the high school science class for the COVID-19 vaccine. Molecules play a vital role in the body and carry recipes for making different proteins to the parts of the cell that produce them. But until Pfizer-BioNTech and Moderna use the power of genetic material to teach the body to make some of the proteins on the surface of the COVID-19 virus, “mRNA” is not exactly a common phrase. was. Things, it was to attack.

Awesome Efficacy of mRNA-based COVID-19 vaccine Has created a lot of excitement about its potential use in Vaccines for other diseases.. And the vaccine may be just the beginning. Last month, researchers provided CRISPR gene editing technology that can permanently treat rare human genetic disorders using mRNA. Experts say that this advance goes far beyond treating a single condition.

Medical research using CRISPR —system This allows scientists to add, remove, or modify specific genetic information in the body. This has already made rapid progress in recent years.Researchers have shown the possibility of a reversal Blindness When Sickle cell anemia, And to treat hereditary diseases With animals..But a new job Explained In New England Journal of Medicine In June, researchers are calling for the first time that CRISPR was shown to treat hereditary diseases when administered directly to human patients.

In this case, this technique was applied to the treatment of transtyretin amyloidosis. Transtyretin amyloidosis is a hereditary disease that causes the patient’s liver to produce proteins that eventually reach toxic levels. NS Disease prevalence It depends on the patient’s demographics. This affects 1 in 100,000 European Americans, but in northern Portugal, for example, it affects 1 in 538 and could be passed on to future generations. There are drugs that can help patients manage their illness, but the goal of the new study was to stop the problem because of it.

“To imagine use [CRISPR] As a cure for people, you need to understand how to put these editing tools into the cells you are trying to modify. That’s where messenger RNA comes in, “says Daniel Anderson, a professor of chemical engineering at the Massachusetts Institute of Technology and co-founder of CRISPR Therapeutics, which develops medicines using CRISPR technology. Anderson was not involved in the study.

A research team led by Dr. Julian Gillmore, an amyloidosis expert at Royal Free Hospital in the United Kingdom, programmed mRNAs to direct gene editing to the liver and shut down the parts involved in the production of toxic proteins. After a single injection of the drug, three of the six who participated in the study confirmed that protein production was almost completely reduced. The remaining three low-dose did not show very dramatic results. It will take months to see if that achievement will help alleviate the symptoms, but early findings are promising. (This work was funded by the pharmaceutical companies Intellia Therapeutics and Regeneron, which manufacture injectable CRISPR drugs.)

Dr. John Leonardo, President and CEO of Intellia, said: “MRNA is a way to bring CRISPR gene editing to life. CRISPR is our flagship product. mRNA encodes it.”

Theoretically, the same common techniques can be used to treat conditions other than transthyretin amyloidosis. He is a senior professor of heart disease research at the Karolinska Institute in Sweden, a co-founder of Moderna Therapeutics, and research. “The most important aspect of this is that it means that technology can be reused.”

Chien has believed in the potential of mRNA drug development for over a decade. In fact, when Moderna was founded in 2010, its main goal was to develop mRNA-based drugs, not vaccines. (Chien hasn’t worked at Moderna and is now an advisor to pharmaceutical giant AstraZeneca.) He continues to research mRNA-based drugs that he hopes can ultimately cure heart disease.

According to Leonard, the strategy for delivering CRISPR-based therapies depends on the target, so the challenge is figuring out how to get the drug into different tissues. A new study provides a blueprint for liver-based conditions, and Leonard believes a similar approach can be used in the short term for bone marrow, nervous system and muscle disorders. The list will theoretically grow from there as long as the researcher can fine-tune the delivery.

“COVID [vaccines are] It’s a huge success for mRNA, and if it doesn’t do anything else, it’s great, “Chien says. “But I think the next chapter on mRNA will be as exciting as the story of mRNA vaccines.”

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Write to Jamie Ducharm jamie.ducharme@time.com..