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Moderna, teaming with a startup, wades further into gene editing

Before it rose to prominence as a vaccine maker, Moderna’s original ambitions were to use its messenger RNA technology — a way to prompt the body’s cells to produce helpful proteins — to make medicines.

As an emerging privately held biotech, the company raised billions of dollars through partnerships and venture rounds to support the effort. The company amassed a pipeline of dozens of potential programs and even dabbled in startup creation, with a short-lived experiment to hatch biotechs as wholly-owned drugmaking subsidiaries. Some of those programs, like a rare disease treatment Moderna worked on with Alexion Pharmaceuticals, were scrapped along the way.

All of that work faded into the background when Moderna shifted gears to become a vaccine developer, a decision that has paid off for the company and its investors. Moderna shares were worth less than $19 at the start of January 2020. They’re now worth nearly $344 apiece as its coronavirus shot has become one of the world’s top-selling pharmaceutical products and synthetic mRNA, in the process, has become a proven vaccine technology. Other Moderna vaccines for flu, cytomegalovirus, Zika and other infections are in clinical testing as well.

But Moderna hasn’t abandoned plans to make medicines, too. The company has a few experimental rare disease treatments in preclinical and early testing, and executives have made clear that gene editing tools could help broaden those ambitions. Gene editing biotechs like Intellia Therapeutics and Verve Therapeutics, for example, already use the type of delivery system — tiny fat bubbles known as lipid nanoparticles — that Moderna does for its vaccines and drugs. And thus far, Moderna has only dipped its toes in gene editing through an alliance with Vertex to develop drugs for cystic fibrosis.

“We think it’s the right time for us to start to expand in that direction,” said Moderna president Stephen Hoge, on a conference call in August, adding that the company believes its technology could help send gene editing drugs to a “range of different tissues.”

The deal with Metagenomi, while small, marks Moderna’s next step in the field. The startup possesses CRISPR-based technology that can be used to edit genes either outside, or inside the body, and landed a sizable investment last year from Bayer. So far, its research has led to very early inside-the-body programs for hemophilia and the liver disease primary hypoeroxaluria, as well as more complex cell-based treatments for cancer.

Moderna is interested in the inside-the-body approach, in which its lipid nanoparticles would be used to shepherd “curative” treatments for serious genetic diseases into cells, the company said. The strategy is similar to the method Intellia is using to develop a rare disease treatment whose early clinical success has become an important proof point for the field.