Flagship Pioneering is betting $50 million that it can solve one of genetic medicine’s biggest problems — how to get the therapies to the right place in the body.
Its new startup, Mirai Bio, was unveiled Thursday with the goal of creating nanoparticles that deliver genetic medicines “to any tissue and cell type.” While the startup’s ambition is in line with many of Flagship’s past bold bets, its strategy isn’t. Rather than building a pipeline of its own drugs around its delivery platform, Mirai will focus entirely on partnering with other companies.
Mirai will design new lipid nanoparticles, optimize the genetic medicines they contain, and manufacture the drugs from preclinical to commercial scales. And while Mirai isn’t calling itself a contract development and manufacturing company, it isn’t denying that the label is at least partially accurate.
“What we are is an end-to-end nucleic acids platform company that will co-create medicines with other partners,” Mirai Bio’s founding president Hari Pujar told Endpoints News in an interview.
It’s a unique business model among Flagship startups, which usually pride themselves on expansive internal drug pipelines. Yet if successful, Mirai could start making money sooner than most through upfront fees, milestone payments and royalties on sales of future drugs. The company is already manufacturing medicines for preclinical studies and will start clinical manufacturing “in the not-too-distant future,” Pujar said.
A ‘virtuous cycle’ for nanoparticles
The company’s emphasis on manufacturing is partly an acknowledgment of the complex nature of cell and gene therapies, where being able to physically make a medicine at scale is increasingly seen as just as important as designing it.
“We want to walk the journey of development along with our partners, and work with a large number of partners,” Pujar said. He hopes to “create a virtuous cycle of learning” using artificial intelligence and machine learning models.
Hari PujarPujar said the company has started by improving lipid nanoparticles, or LNPs, used for vaccine delivery and liver delivery. Mirai is also working on LNPs for delivery to the eye, immune cells and the hematopoietic stem cells of the bone marrow.
Several other startups are also racing to unlock in vivo delivery to immune and blood cells, which could eliminate the need to remove cells and edit them in the lab. Some of these targets have even been a focus for other Flagship companies, including Tessera Therapeutics, which earlier this year reported promising results of LNPs that can target hematopoietic stem cells in mice.
“We’re building on all of that, and now creating an opportunity where delivery innovation can now be accessible to the outside world,” said Pujar, who is also the chief operating officer at Tessera.
Mirai is also trying to break new ground delivering LNPs to fat cells. Getting LNPs to target the central nervous system or skeletal muscle is a longer-term goal, too. Pujar said that if Mirai creates delivery vessels that “open up new tissues,” the startup may spin out those technologies into new startups focused on developing drugs.
Pujar provided few details about the scientific or technological underpinnings of Mirai. He said the startup is injecting many variants of nanoparticles in an animal at once, usually a monkey, and then seeing where in the body they end up by reading a genetic barcode delivered to the cells. It’s an approach that other companies and labs have taken as well.
Mirai will also help optimize the genetic cargo inside of nanoparticles. Thanks to redundancy in the genetic code, there are many ways to spell out an mRNA and get the same protein. Scientists at Moderna and other mRNA-focused companies have used AI to find the best sequence in a process called codon optimization.
Pujar wouldn’t say how Mirai plans to advance this technique, but he suggested that existing approaches to mRNA design may soon be viewed as “version 1.0 or maybe 1.5.”
“I think we can all imagine a future where mRNAs in 2040 will be designed very differently than mRNAs in 2020, and we want to be part of that journey,” he said.