Startup Supported by Institute for Applied Life Sciences Lands Government Contract Worth up to $54.5 Million

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L-R: Peter Reinhart, Sarah Perry, Craig Martin, and Karen Utgoff

This story was first published by the UMass News Office.

With its 25 state-of-the-art research facilities and cutting-edge equipment, the Institute for Applied Life Sciences (IALS) at the University of Massachusetts Amherst provides seed funding, incubation space and venture mentoring to nurture fledgling companies with promising tech developed at the university as they work to attract outside investors.

A sterling example of the success of the IALS approach is Waterfall Scientific, built upon research led by Craig Martin, emeritus professor of chemistry, and Sarah Perry, Gary R. Lapidus Professor in the Department of Chemical and Biomolecular Engineering. Waterfall was recently awarded up to $54.5 million by the Advanced Research Projects Agency for Health (ARPA-H) to lead a six-company consortium to revolutionize the production of medical-grade messenger RNA (mRNA)—an achievement which could not have occurred without IALS and the associated Center for Bioactive Delivery (CBD).

Martin’s specialty is synthesizing the kind of RNA that is the backbone of recent advances in medical therapeutics—such as the recent treatment of “Baby KJ’s” genetic disorder with a customized CRISPR gene editing therapy—and he has been a pioneer in the chemistry needed to do this for more than 35 years. But how to synthesize mRNA at the scale needed by industry while dramatically reducing the impurities and high costs associated with traditional methods?

“I make microscale devices—think of a complex chemical refinery, but with equipment that is the size of a human hair. My lab is inventing the machines that will be able to automatically generate long RNA,” says Perry, an expert in microfluidics, a crucial technique for precisely engineering the flow of chemicals at the microscale.

Back in 2018, Perry was teaching her class, “Microfluidics and Microscale Analysis in Materials and Biology,” in which her students develop microscale technologies for real-world needs—and Martin’s mRNA work was one of the class’s case studies.

Together, Martin’s chemistry and Perry’s chemical engineering led to the pair to invent and refine a technique for synthesizing mRNA with unmatched efficiency, reliability and cost effectiveness.

RNA therapeutics have the potential to transform medicine as we know it—but the current multi-step techniques for making mRNA are far too expensive and slow for the therapy to realize its full potential, which includes the ability to tailor therapies to individual patients and particular tumor cells. This is why ARPA-H recently released $125 million for the research and development of made-to-order genetic medicines, including the up to $54.5 million to Waterfall Scientific and its team.

And yet, it is the rare multi-million-dollar idea that actually turns into a multi-million-dollar investment—this is where IALS comes in.

Founded in 2014 with a more than $150 million from the State of Massachusetts through the Massachusetts Life Sciences Center and UMass Amherst, IALS, in addition to housing more than 25 state-of-the-art research facilities with cutting-edge equipment, incubates and accelerates the translation of basic research into innovative product candidates, technologies and services.

“There are three main ways that we bridge the gap between academic research and a marketable solution,” says Peter Reinhart, IALS’s founding director. “We offer bespoke entrepreneurial mentoring tailored to the specific idea and the shape of the market niche it could occupy. We also maintain a robust network of business and patent attorneys, seed-funding organizations, venture capital groups—everyone that can play a role in launching a successful company. And we provide seed funding ourselves, to help shepherd start-ups—like Waterfall Scientific—through the ‘valley of death’: that dry stretch in the transition from brilliant idea to financially viable product, which many great ideas can’t survive.”

“I was initially clueless about how to start a company,” says Martin who has been working with IALS for years to develop Waterfall Scientific, and has been the recipient of multiple seed-funding awards, including a Manning/IALS Innovation Award and an IALS Translation Seed Award. “Perry and I were continuing to advance our technology with federal grants, and at the same time IALS was helping connect me with investors, schooling me in how to pitch them and build Waterfall from a company on paper to one with a leadership team, employees and a lab space in Worcester at the Massachusetts Biomedical Initiative incubator—one of the few wet-lab spaces in western Massachusetts that offers spaces for new start-ups. Now, with this contract from ARPA-H, we can build a truly novel system for producing the mRNA that the medical industry desperately needs.”

When asked about the role that IALS played in the long journey of Martin and Perry’s work from research idea to multimillion dollar grant, Reinhart points to the idea of “de-risking.” Truly innovative approaches—like Martin and Perry’s—are by their nature unproven. Few investors have the stomach to back something that has never been done before, especially when the idea comes from a first-time founder. It’s just too risky, and the idea often dies.

“When we significantly reduce that risk, through mentoring and seed funding, we can help create a company that is much more attractive to investors,” says Reinhart. “It can be a long process, but with companies like Waterfall, we’re seeing that initial investment from the State and from UMass Amherst pay off.”

In addition to Waterfall’s stunning achievement, Reinhart points to the more than 75 startup ideas that IALS has helped get off the ground, an increasing number of which can now stand on their own. Those emerging businesses are starting to make a real impact on the western Massachusetts economy. “We have helped create approximately 90 jobs in the region to date,” says Reinhart.

“Traditional batch-based mRNA manufacturing was never designed to meet the demands of personalized medicine,” says Inna Shcherbakova, CEO of Waterfall Scientific. “The government’s call for decentralized, on-demand therapeutic production requires a fundamental paradigm shift. Our continuous-flow platform, developed from technology invented at UMass Amherst, drastically reduces manufacturing footprints while ensuring ultra-high purity. With the support of ARPA-H and our consortium partners, Waterfall is uniquely positioned to deliver the agile infrastructure needed to make bespoke genetic medicines scalable, accessible, and affordable.”

“Part of what is so validating about this $54.5 million award is that it signifies that we all—IALS, the federal government, private industry and academic researchers—want the same thing: significant change in the way mRNA is manufactured,” says Martin. “Right now, Massachusetts is the is the world’s epicenter of mRNA therapeutics, but China is rapidly gaining ground. It is particularly import to the state that we continue to enable mRNA companies to make their products better, faster and cheaper.” 

“Ultimately,” Martin concludes, “this is all about changing lives, whether by preventing or treating diseases, crafting personalized therapeutics, training the Massachusetts workforce or building economic resilience out here in Western Massachusetts.”