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There is, however, an application besides vaccines, where brief exposure to messenger RNA could have effects that last for years or even a lifetime.
At the end of 2019, before covid-19, the United States National Institutes of Health and the Bill and Melinda Gates Foundation ad they would spend $ 200 million to develop affordable gene therapies for use in sub-Saharan Africa. The main targets: HIV and sickle cell disease, which are widespread there.
Gates and the NIH didn’t say how they would make these cutting edge treatments cheap and easy to use, but Weissman told me the plan might depend on using messenger RNA to add instructions for the tools. editing genes like CRISPR in a person’s body. , making permanent changes to the genome. Think of mass vaccination campaigns, Weissman says, except with gene editing to correct inherited diseases.
Today, gene therapy is complex and expensive. Since 2017, several types have been approved in the United States and Europe. One, a treatment for blindness, in which viruses carry a new gene to the retina, costs $ 425,000 per eye.
A start-up called Intellia Therapeutics is testing a treatment that conditions CRISPR into RNA, then into a nanoparticle, with which it hopes to cure painful hereditary liver disease. The goal is to make the gene scissors appear in a person’s cells, remove the problematic gene, and then disappear. The company tested the drug on a patient for the first time in 2020.
It is no coincidence that Intellia treats liver disease. When released into the bloodstream intravenously, the lipid nanoparticles tend to all end up in the liver, the body’s cleansing organ. “If you want to treat liver disease, all the better – everything else, you’ve got a problem,” Weissman says.
But Weissman says he figured out how to target nanoparticles to end up in the bone marrow, which constantly makes all of the red blood cells and immune cells. It would be an extremely valuable tip – so valuable that Weissman wouldn’t tell me how he does it. It’s a secret, he says, “until we get the patents filed”.
He intends to use this technique to try to cure sickle cell anemia by sending new instructions into the body’s blood plant cells. He’s also working with researchers who are ready to test in monkeys whether immune cells called T cells can be engineered to go on a search-and-destroy mission after HIV and cure this infection once and for all.
All of this means that the fatty particles of messenger RNA can become a means of editing genomes on massive scales and inexpensively. A drip drug that enables engineering of the blood system could become as important a public health boon as vaccines. The burden of sickle cell disease, an inherited disease that shortens life by decades (or, in poor areas, kills in childhood), weighs most heavily on blacks in Equatorial Africa, Brazil and the United States . HIV has also become a persistent scourge: around two-thirds of people living with or dying with the virus are in Africa.
Moderna and BioNTech sell their covid-19 vaccines for between $ 20 and $ 40 a dose. What if it was also the cost of genetic modification? “We could fix sickle cell disease all at once,” Weissman says. “We believe this is a revolutionary new therapy.”
There are fantastic fortunes to be made in mRNA technology. At least five people connected to Moderna and BioNTech are now billionaires, including Bancel. Weissman is not one of them, although he is entitled to patent royalties. He says he prefers academia, where people are less likely to tell him what to look for – or, equally important, what not to do. He’s always on the lookout for the next big scientific challenge: “It’s not that the vaccine is old news, but it was obvious it was going to work.” Messenger RNA, he says, “has an incredible future.”
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