Flagship Pioneering, which has founded over 100 bioplatform companies, introduced the latest, Mirai Bio, during the BioProcess International Conference in Boston. Mirai, whose founding CEO is Geoffrey von Maltzahn, PhD, features an open end-to-end platform to enable the co-creation of fully optimized genetic medicines, according to Hari Pujar, PhD, founding president of Mirai and operating partner at Flagship Pioneering who spoke during a conference presentation.
In a statement released before his presentation, Pujar noted that the life sciences are in “the age of information molecules. Yet, enormous technological challenges in the delivery, cargo design, and manufacturing of these molecules have hindered the speedy and full realization of their potential.”
“We created Mirai to solve these key limitations through AI trained on high quantities of quality in vivo data. By applying machine intelligence to the design of every atom within the medicine and opening this platform to the entire industry, we will have vast collective data points rolling through our optimization loops, allowing a greater innovation advantage to benefit each partner on the Mirai platform.”
Proprietary data algorithms and machine intelligence
Mirai’s machine intelligence-based, open platform uses proprietary data algorithms to unlock delivery to any tissue and cell type, optimize cargo design, and facilitate manufacturing, accelerating genetic medicines toward successful clinical translation for the company’s partners, continued Pujar, adding that Mirai plans to further advance its platform with Flagship’s initial commitment of $50 million.
Mirai’s platform is enabled by high throughput automation and in vivo multiplexing, generating vast amounts of data that fuel extensive optimization loops, he explained, while maintaining that the co-optimization of delivery, design, and manufacturing has the potential to enable a higher probability of success and a faster development timeline for any genetic medicine.
Pujar was asked about the high financial costs of genetic medicines, especially cell and gene therapy, to patients. What could Mirai do to address the cost issue?
“Two things,” he said. “One is we are transitioning from viral vectors to non-viral delivery. Non-viral medicines are easier to manufacture, far more scalable, and the process economics of non-viral medicines is decidedly more favorable compared to viral vectors.”
“The second factor is ex vivo versus in vivo. Some of the current genetic medicines are ex vivo, as for example, CAR T-cell therapy, effective for cancer. But it involves harvesting patient cells, bringing it to a manufacturing site, converting the cells to other viral vectors, bringing it back to the patient with a lot of exposure to plastic and freezing and thawing. This bespoke manufacturing is laborious and expensive, which is why the reach of these medicines has been limited.”
“If you imagine in vivo therapy where an individual just gets an IV injection, which is not customized to the patient, that would significantly reduce the cost of these medicines. So those are the two approaches that we’re imagining that will bring down.”
“Every new company with a payload idea faces the same challenge of either internally building high-quality, cost intensive delivery, design, and manufacturing capabilities, or externally sourcing these components, which can be fragmented and suboptimal,” pointed out Travis Wilson, executive chair at Mirai and growth partner, Flagship Pioneering.
“Leveraging learnings from semiconductors as a centralized resource model that fueled the rapid advancement of tech, we’ve developed a solution that’s been hiding in plain sight: an open platform to unlock genetic medicine development. Through aggressive investment in our own capabilities and expertise, and by creating long-term partnerships with biotech and pharma leaders in the space, Mirai’s hope is to become the partner of choice and help bring more medicines into the world than we would ever achieve alone.”