We finally know who Moderna has been courting behind the scenes to make the big jump into gene editing. The famed biotech has signed a research partnership with CRISPR gene editing company Metagenomi.
Both parties are keeping mum on a lot of the details. We don’t know what indications they’ll go after, how many targets they will develop, how much Moderna will put up for research funding, how much was paid upfront, what the milestone or royalty payments will end up being down the line or how much of an equity investment Moderna made through the transaction.
We do know that the partnership will involve in vivo treatment options for serious genetic diseases. Metagenomi will offer up access to its gene editing tools while Moderna will bring the expertise in mRNA and lipid nanoparticle delivery technologies.
Moderna CEO Stephane Bancel made waves in August when he told investors during second-quarter earnings that the company planned to finally spend some of its billions in COVID-19 vaccine revenue on a gene editing deal of some kind. President Stephen Hoge further clarified that Moderna is interested in improving delivery mechanisms in gene editing with its mRNA technology.
So how did Metagenomi rise to the top in the field of available gene editing companies?
Turns out the small biotech had an in with Eric Huang, Ph.D., the general manager and chief scientific officer of Moderna Genomics; he serves on Metagenomi’s science advisory board.
“When they presented the data to me, I just found it irresistible,” Huang said. “And then once Moderna decided to invest in genomic medicine in general, I brought this opportunity to Stephane.”
He jokingly said there was “no bias” in the selection, that of course Moderna conducted thorough due diligence. He admits he’ll likely need to resign the SAB position now that the companies are tied up. Huang said the two companies have complementary cultures, and a desire to “dream big—too big, but do good science.”
Huang would not disclose financial details either but called the partnership “very significant” in terms of cash and research funding as well as the equity investment.
“We would love to be part of their success,” Huang said of Metagenomi. “We're gonna be tied at the hip for a while.”
Metagenomi’s discovery platform finds DNA from natural samples that can be sequenced to create new tools for gene editing. Drug candidates are then identified using AI. The small biotech is backed by Bayer, which signed on to an initial $65 million funding round at the end of 2020. That round was pushed up to $75 million in April with the addition of new investment from VC shop RA Capital.
Metagenomi CEO Brian Thomas called the Moderna partnership a natural one and said his company has benefited from Huang’s input over the past three years.
“As we started to grow closer in our discussions about the broad toolbox that Metagenomi is building, it became really clear that the two companies' missions were starting to align,” Thomas said.
This is Metagenomi's first partnership, too, although Thomas said others have been interested. But the biotech was waiting to find a partner that could bring a better delivery mechanism to the deal.
Thomas and Huang were careful not to reveal the indications they’ll tackle, but they did say the underlying goal is to improve gene editing as a whole. As they work through the research partnership, they will select indications that seem promising along the way.
“We don't want to just do a simple gene knockout. We don't want to just do simple point mutation, single variant mutation,” Huang said. “We are dreaming bigger, creating different tools that scientists can think of that allow doctors to treat genetic diseases.”
The companies plan to deliver their medicine where other gene editing companies have already established a precedent—the liver, using delivery tech Moderna already has, Huang confirmed. He acknowledged that Intellia, the first company to present first-in-human data for a gene editing therapy this summer, is the “pioneer” in the space. That company’s technology targets the liver as well.
Moderna and Metagenomi will begin with building on technology that exists to get some candidates into the clinic as soon as possible, with a long-term goal of making broader improvements to gene editing.
“We have gene editing technology that's ready to go to the clinic right now. We plan to work with Moderna to move that into the clinic as fast as we can,” Thomas said. “But we also have a long-term vision to develop the best tools for in vivo gene editing, and that's going to be what we work on.”
Gene editing has so far focused on Cas9 technology, but Thomas thinks there’s more out there. The company recent unveiled data on its CRISPR-associated transposases system that can be used to precisely integrate large DNA fragments into genomes, allowing for new editing techniques beyond the currently available technology.
“We're just at the tip of the iceberg … there's no reason why evolution would stop at Cas9 and only give us one thing,” he said.
Huang pointed to Moderna’s well documented history of a company that can take the long game or immediately pivot when, for example, the world needs a life-saving vaccine. The company developed its first vaccine in 2013, back when mRNA technology seemed as though it was “nothing valuable.” So a one- or two-year deal would not have been “satisfactory,” he said.
“I hope in five to 10 years’ time, when people are thinking about in vivo gene editing, they think of Moderna and Metagenomi together,” Huang said.
The question remains whether this will be Moderna's only partnership in gene editing, or if they will pile on others down the line. Huang was, as expected, tight lipped, but still open to the prospect for other deals.
“We will be keeping our options open about complementary technologies that will be delivering or improving potencies in other aspects of genome editing, so Moderna will always keep an open mind and option."