Ionis Pharmaceuticals and budding gene editing company Metagenomi have penned a multi-target research collaboration that could total almost $3 billion in biobucks.
The gargantuan financial deal is astounding for two companies that are not heralded as large pharmas, but nonetheless, the announcement shows just how much the industry is betting on gene editing. The agreement is essentially split into two groups of four possible genetic targets—Ionis is paying $80 million for the first batch of four, with two of the targets being co-developed with Metagenomi.
If or when the first of those targets is submitted to regulators to enter clinical trials, Ionis will have the chance to sign up to four additional targets, Metagenomi Chief Investment Officer and SVP of Strategy Simon Harnest explained in an interview with Fierce Biotech. The upfront cost to sign onto all four additional targets would be $120 million. In total, there are almost $3 billion in biobucks available spanning all eight potential targets, which, for comparison, exceeds recent deals from some of the world’s largest pharma companies
For context, Bristol Myers Squibb linked up with GentiBio for a deal in August worth up to $1.9 billion for three targets. Last month, Lilly tagged Schrödinger for a drug discovery pact worth $425 million in biobucks; Bayer’s Vividian also went on a shopping spree, offering a potential $930 million payday for four of Tavros’ oncology targets. And just recently, Sanofi locked arms with AI-driven Insilico for six targets worth up to $1.2 billion in milestone payments.
For Metagenomi CEO Brian Thomas, such a massive opportunity—even if much of it hinges on future buy-in and clinical progress—is centered on the ability to utilize Ionis’ RNA capabilities to find optimal gene editing targets outside of well-known regions like the liver.
“I'm also really excited because they bring 30 years of experience in RNA chemistry...as well as targeting,” Thomas told Fierce Biotech in an interview. “And so we're really excited to work with them to try to solve that problem.”
While the two companies weren’t ready to disclose exact targets, the first wave of four will likely hone in on familiar areas like the liver, while the second group would expand to new territory, Thomas added.
The deal itself came together extremely quickly, according to Thomas, who met with Ionis' CEO Brett Monia, Ph.D., Chief Scientific Officer Frank Bennett, Ph.D., and Dave Ecker, Ph.D., VP of Strategic Innovation, in the first quarter of 2022. Immediately, Thomas “was just really impressed about how aligned” the two groups were.
This is at least the third collaboration Metagenomi has signed since launching in 2018, with Moderna and Affini-T Therapeutics also typing up deals (an earlier research pact with Vor Biopharma never materialized into an option for targets). Those agreements, paired with an oversubscribed $175 million series B announced in January, give the company significant momentum heading into next year. At that point, the company is expected to have 170 employees, up from the 150 working there now.
Much of Metagenomi’s early hype centers on its AI cloud computing platform that screens from trillions of base pairs to find novel nucleases that could be used for future gene editing therapies. By diversifying the ways it can edit genes, Metagenomi is betting it can also diversify the range of diseases it can treat.
With some of the first gene editing companies, like CRISPR Therapeutics, just now gearing up for approval submissions, Harnest emphasized that it’s been a bit of an advantage to not be first in the space. It means Metagenomi is able to take the regulatory feedback passed along to competitors and apply it to their early development work, like testing the potential for off-target edits. Right now, the company is on track to ask regulators to enter the clinic with its first solely owned assets sometime in 2024 or 2025. But Thomas wouldn’t be surprised if one of Metagenomi’s partnered assets is clinic-ready earlier than that.
“The partnerships that we have with the cell therapy companies...Affini-T, and we have some others that are in the works there...they’re actually thinking about targeting the clinic very rapidly,” he said. “There’s a very good possibility that the first time our technology sees the clinic is with a cell therapy partnership.”