David Schaffer: Research That Takes Risks Must be Supported

The Berkeley Changemaker is a Berkeley News series highlighting innovative members of the campus community engaged in work and research that tackles society’s most pressing issues.

David Schaffer remembers sitting on his father’s lap as a child, curiously delving into science books and crafting mnemonic phrases that instilled in him the building blocks of biology.

“He was a biochemist who would make up these silly rhymes that helped me remember microorganisms or parts of the body, like, ‘Your sternum can burn’em,’” Schaffer laughingly recalls. “It just made it kind of fun to learn, and I developed a natural interest in biology.”

Schaffer has since taken those lessons — along with major influence from his mother, a medical doctor from El Salvador — to heart over the years. As a UC Berkeley professor of bioengineering, chemical and biomolecular engineering, and molecular and cell biology, for more than 20 years he has made lab research and classes fun for his students. Many of them become members of his Schaffer Research Group, a campus lab that investigates biomedical problems focused on the engineering of stem cell and gene therapeutics.

A prominent researcher in his own right, Schaffer prides himself on being a shepherd for young Berkeley innovators and entrepreneurs, to help them understand “the business and joy of biotech.”

In 2021, Schaffer was named to the National Academy of Inventors. He holds over 50 research patents and has cofounded eight companies to commercialize stem cell and gene therapies to treat ailments such as blindness, degenerative disorders of the central nervous system and life-threatening cancer.

Recently, Schaffer was appointed the new executive director of QB3, a UC-systemwide group that supports California entrepreneurship and includes research divisions at UC Berkeley, UCSF and UC Santa Cruz.

Schaffer also leads the Bakar BioEnginuity Hub, which will hold 50 new biotechnology startup companies supported by the Bakar Labs incubator, and he directs Berkeley’s Bakar Fellows Program, a funding incubator that accelerates the application of discovery research.

Bakar Fellows, which is celebrating its 10th anniversary this year, is “worth its weight in gold,” said Schaffer. It has funded concepts from over 100 faculty and students — ideas that might have been viewed by others as too risky to support.

“From healthcare therapies to energy efficiency, our fellows apply discovery research to tackle some of society’s most daunting problems,” Schaffer said. “This program enables us to fund and train fantastic graduate students and postdocs willing to develop innovative technology and to take risks in this research.”

Berkeley News spoke with Schaffer recently about the impact the Bakar Fellows Program has had on researchers on campus and why Berkeley has become a leader in producing changemaking research and entrepreneurship.

Berkeley News: You have over 50 patents from your research that led to eight companies aimed at commercializing stem cell and gene therapies. How can researchers find this type of success in their own work?

David Schaffer: Pure discovery research is really important. There need to be people who ask very basic questions about how the world works to establish the foundation that others can then build on top of to create technologies that solve pressing societal problems.

In my own work, I tend to go out into the real world and identify not just a single such problem, but classes of problems that are related to each other. You don’t want to design a hammer that only hits one nail.

You want to invent a technology for a set of problems that you can then translate into a broad platform technology that can create a series of products to address multiple diseases.

What is the clearest way to demonstrate that your platform works?

If you’re for example developing a therapeutic, you identify the unmet clinical need where you think the technology can have the quickest and best chance of success. Then, if it works for that initial disease, you can broaden out and treat other diseases.

Our lab has worked, for example, on developing a platform technology to engineer better adeno-associated viruses for gene therapy, and our first application was to focus on delivery to the retina to help people with rare blinding diseases. Not only is there a clear need for therapies to treat these incurable conditions, but it’s relatively easy to measure improvement since the clinical endpoints used in human trials include some of the tests you take when you go to an optometrist. And, manufacturing the therapy is less of a challenge since the doses are low for a small tissue like the eye.

Once we were able to show the initial therapy was safe and effective, we expanded into more complex eye diseases and are now also running clinical trials in the heart and the lung.

We have taken similar approaches with other technology platforms: picking a lead clinical target that will hopefully give the fastest proof of concept, then expanding into a pipeline.

What are impediments to applying these technologies?

Without critical support and funding, young technologies — whether they be a new COVID-19 vaccine or a treatment for blindness — can get stuck or even lost in the early stages of lab research and never make their way into the hands of people who need them most.

One challenge is that it’s often hard to get funding before you’ve actually proven something works. You might have the most world-changing idea, but you sometimes have to already have strong proof of concept before a grant agency will invest in you.

So, where do you get the money when you want to conduct research that may seem risky to mainstream science?

UC Berkeley’s Bakar Fellows Program has been extremely valuable, and worth its weight in gold, in providing funding for concepts that are viewed as too risky for government agencies or foundations to support.

You are a former Bakar Fellow and now the program director. What makes this program unique from other funding sources?

It’s generous funding at a level that can make a major difference and allow individual projects, in a large number of research groups, to get to proof of concept.

Also, when you develop an idea, either in an academic lab or as part of a company, the finished product rarely looks identical to the initial idea. It’s almost always the case that research evolves over time, and you may need to pivot or change directions.

There are some federal agencies that don’t give you the freedom to do that. Some are not going to fund something unless it’s already well developed. But Baker Fellows funding allows for risk-taking and is forgiving, if a change in direction is needed. That freedom enables fellows to innovate so that they can overcome problems and shift direction to get something to work really well.

Bakar Fellows is also a part of Berkeley’s greater academic and entrepreneurship community, which gives these researchers great opportunities to find collaborators, mentors and additional funding to further advance their technologies once their fellowship ends.

Once the Bakar Fellows program helps them establish proof of concept, researchers can for example, seek additional funding from the National Science Foundation or National Institutes of Health. Alternatively, if the work has already advanced far enough they can decide to create their own companies, to translate their technology toward products, with the aid of Berkeley’s world-class programs and facilities like the Bakar BioEnginuity Hub and Bakar Labs.

There is a whole ecosystem here for innovators and entrepreneurs to connect and make a difference in the world.

How important is it to have this thriving community for innovators and entrepreneurs on campus?

As executive director of QB3 and director of both the Bakar BioEnginuity Hub and Bakar Fellows program, community is something that I really emphasize and work on building at Berkeley and among Bay Area UC’s.

Having a supportive community is important for discovery research and for accelerating the translation of these technologies. No one has the knowledge necessary to solve all their own problems, so you need to learn from other people.

One of the great ways to learn is to be part of an incubator or accelerator since other people around you are facing similar challenges in starting their companies. So, we’re really trying to foster community within these programs so that people help one another.

At the same time as enhancing community among our small companies at Berkeley, we’re also building connections with leaders in the biosciences and bioentrepreneurship fields, such as Bayer, AbbVie, Genentech, Amgen, Lilly, venBio, The Column Group, Frazier and other successful partners. These industry affiliates support our programs and help our companies learn and grow.

It’s critical, as you grow and develop in your career, to recognize the value in the network of people around you, because they can help you solve problems faster than you would on your own. And you can try to return the favor.

As a leader in innovation, what makes Berkeley different from other universities that are producing world-changing research?

As a Stanford and MIT alumnus, I enjoyed the multifaceted education I received at those universities. But I did get a sense that the environment of a private institution was more insulated from the real world, and people, on average, were more financially privileged.

When I got a job at Berkeley over 20 years ago, I felt that, like at Stanford and MIT, I was becoming a part of a campus that had incredibly accomplished physical science, biological science and engineering departments; top-tier Nobel Prize-winning faculty; and strong academics across all fields of study.

And not only was I going to be working in the vibrant Bay Area, which is the birthplace of biotech, but I felt really good about being at the best public university in the world. At Berkeley, our goal is to educate the citizens of California and beyond, to give them access to a world-class education without students going into major debt.

Berkeley is a much more affordable place to attend than a private school. We have the most Pell Grant awardees of any university in the country, which means that Berkeley is more accessible to the full socioeconomic spectrum of students.

Another way in which Berkeley has become unique is that, in Bakar Labs, to my knowledge we now have the largest university-operated biotechnology incubator in the country. This enables us to translate university discoveries into companies that will create products that benefit society.

Is being collaborative a big part of producing world-changing research and companies?

Yes, it is. There is a collaborative culture present on our campus that is unlike other top universities. It has been both fun and surprisingly easy to form collaborations with others across campus.

As an engineer, I’ve collaborated with biologists, chemists, computational biologists and others. I’ve found numerous situations where one plus one is equal to three.

The campus also encourages interdisciplinary research on a larger scale by supporting the formation of new centers. Departments, which in some cases have been around for over a hundred years, are pillars of academic education in important disciplines. In addition, new centers are nimble in building connections among researchers across departments.

The Berkeley Stem Cell Center, which I directed for 10 years, didn’t exist until 2004, a time when people began to recognize that stem cell research was happening in molecular and cell biology, bioengineering, integrative biology and other programs, but the researchers in those different departments often didn’t know each other. That center was founded to start building bridges and connections across these departments and disciplines.

Sometimes a center will evolve into a new department, which is currently happening with neuroscience. This dynamic system encourages innovation and collaboration.

We’re also trying to build a culture of collaboration and collegiality within the programs that support entrepreneurship. Additionally, we are fostering a culture of entrepreneurship for societal good, rather than just for making money.

What is your advice for the next generation of researchers and entrepreneurs at Berkeley?

I would encourage people to find a really important problem and not be afraid of taking big risks to solve it.

Also, be open-minded to reaching out to and learning from others. We are blessed with many smart people around us at Berkeley.

Open yourself up to learning from other branches of knowledge and people with different expertise on campus — from science to business to law. It’s going to take a team of talented people to make new discoveries and to translate them into products that solve societal needs.