A cross-disciplinary course on biotech’s “valley of death”

TabulaSynthase, the blog of Whitehead Institute, brings together ideas and perspectives from the Whitehead community and beyond. 

Basic biomedical research may be in its most fruitful period in history: each year, with new technologies helping scientists advance our understandings of the underlying basis of human disease. At the same time, it’s increasingly difficult to undertake the process of translating promising biomedical discoveries into new drugs or diagnostics, especially those that will help relatively small numbers of people. “The problem rests not in the science,” says Whitehead Institute Founding Member Harvey Lodish, “but with the lack of funding for early-stage development.”

The cost of developing basic science findings into tangible, testable products is considerable. And the period between making a discovery and obtaining clear evidence that it could be translated into an effective treatment is known colloquially as the Valley of Death. That is because few nonprofit research institutions have those kinds of resources. “It usually requires commercial funding, yet many investors are reluctant to risk their capital on the early-stage work,” explains Lodish, who is also professor of biology and professor of bioengineering at Massachusetts Institute of Technology (MIT).

Lodish knows what it takes to move past the Valley of Death. He’s been part of a series of successful start-ups that translate basic science discoveries into marketable therapeutics. Those successes range from Genzyme, launched in the 1980s to develop drugs based on recombinant human enzymes, to Rubius, created in 2015 to use red blood cells to deliver therapeutics for long periods of time. But Lodish knows, too, that his positive experiences bely the trend. “Too many good ideas wither on the vine, starved for funding,” he observes. “We need creative new ways of underwriting early-stage development—and that requires getting more smart, innovative people focusing on the problem from multiple perspectives.”

Earlier this year, Lodish and Andrew W. Lo—the Charles E. and Susan T. Harris Professor at the MIT Sloan School of Management and director of the MIT Laboratory for Financial Engineering—took a step in that direction. Together, they created and taught a new interdisciplinary course, The Science and Business of Biotechnology, designed to draw students from multiple backgrounds, including business management and finance, bioengineering and life sciences, healthcare, and public policy.

“More and more, it’s small companies making the big breakthroughs. But, no funding, no breakthroughs,” says Lo, who is also a member of Whitehead’s Board of Directors and a globally known thought-leader on subjects ranging from behavior of financial markets to the process of biomedical innovation. “Our central goal for the course was to help prepare a new generation of business leaders to succeed in bringing important new therapies and diagnostics to market.”

The course was built to introduce students to both sides of early-stage biopharmaceutical development, the science and the financing. Each class began with Lodish describing an area of bioscience that has underpinned new or potential therapies, such as therapeutic recombinant proteins, cancer immunotherapies, and gene therapies. Then Lo focused on specific challenges in financing biomedical research and clinical trials, and described some of the creative solutions that successful biotechs have employed. Most of the sessions then turned to guest speakers with deep experience in the translating discovery science and gathering financing for new ventures—including executives from Bluebird bio, developer of potential beta-thalassemia gene therapy treatments, Vertex, developer of cystic fibrosis treatments, and Editas, developer of potential gene editing-based therapies.

But conveying knowledge was not the instructors’ only purpose: Lodish and Lo wanted the students to learn to talk with each other. That objective is echoed by Robert K. Coughlin, president and CEO of the Massachusetts Biotechnology Council and one of the course’s guest speakers. “It is very difficult today getting researchers, business people, and venture funders to understand each other’s goals and ways of analyzing problems,” Coughlin says.

Connecting across professions was one of the biggest benefits for first-year chemistry graduate student Azin Saebi, who is considering a career in biotech. “I’d never taken a business class. This course got me out of the ‘chemistry bubble’ and will enable me to better understand the business people who may be my future colleagues,” she says. West Point graduate Daniel Shaffer, who served in an Army Infantry unit in Afghanistan and is now preparing for a business career, found the course similarly mind-opening: “It put me outside my ‘finance and management’ comfort zone—introducing me to biomedical science and encouraging me to seek more creative solutions for major problems.”

For Prerna Sekhri, who just earned both an MBA and a master of science degree in engineering and management, the course provided a welcomed jolt of optimism, “Going in, I was jaded about the possibilities of innovations reaching market,” she explains. “But professors Lo and Lodish showed it’s possible to overcome market barriers by combining imaginative financial engineering and great science.”

The students were not the only ones to learn from the class. “I need to learn more about financing biotech companies, too,” Lodish observes. “Listening to Andrew’s lectures—and those of the executives we brought into the class—was fascinating and hugely informative.”