Efforts of university technology transfer offices to find the optimal path for commercializing discoveries have been well documented,1-3 but the offices are not set up to implement translational research directly. Indeed, a growing number of universities are creating translational programs that operate beyond the usual technology transfer track and aim to teach faculty how to advance the discoveries themselves.

In all cases, the biggest challenge is bridging the different mindsets of industry and academia. Those differences include structuring experiments to answer questions related to product concepts rather than to investigate therapeutic concepts in a more open-ended manner and working in teams that have predefined project goals rather than doing more exploratory research.

It should come as little surprise that many of the programs have been installed by universities with translational track records that are above average. These include Stanford University's SPARK program, the University of California, San Francisco Catalyst program and the Icahn School of Medicine at Mount Sinai's 4D Technology Development program.

The programs vary in size and scope but have the common thread of training academics in how to think about their discoveries in the context of a competitive landscape rather than in a strictly scholarly context. Key elements include defining the unmet need, evaluating the value of an idea versus currently available therapies and investigating the competition.

SPARK was one of the first such programs. It was started by Daria Mochly-Rosen in 2007 when she returned to Stanford after taking time out to found Kai Pharmaceuticals Inc., a peptide-based therapeutics company that was acquired by Amgen Inc. in 2012 for $315 million.

In 2008, former Kai colleague Kevin Grimes joined her to help run the program.

Mochly-Rosen is a professor of translational medicine in the Department of Chemical and Systems Biology at the Stanford University School of Medicine and is director of SPARK. Grimes is an associate professor of chemical and systems biology at the school of medicine and co-director of SPARK.

Mochly-Rosen started by securing undisclosed funding from the Dean's Office at the Stanford University School of Medicine, which continues to be the main contributor. She and Grimes since have obtained support from the school of medicine's Child Health Research Institute, otolaryngology department and cardiovascular institute.

Other money comes from NIH grants and support from Spectrum, which is a translational center partly funded by Stanford's Clinical and Translational Science Award from the National Center for Advancing Translational Sciences.

SPARK project manager Emily Egeler said that the original intention was to educate students to be better prepared to enter industry. The main vehicle to do that was to take on a few projects per year led by teams of university researchers and to help them set up target product profiles (TPPs) that would define the translational value of their discoveries and determine experimental strategies that included go/no-go decision points.

In the last few years, Mochly-Rosen and Grimes have expanded the scope to take on more projects and increase the emphasis on guiding programs to exits-licensing to an existing company or spinning out to a new startup. In addition, they teach graduate-level courses on drug development.

Now, SPARK supports about 10-12 new projects each year. Most projects last 2-3 years and on average receive $50,000 annually.

To be eligible, projects must address an unmet medical need, use a new approach and have the potential to advance to clinical testing or be a licensing option for industry within 2-3 years.

The funding is rarely used for salary and goes mostly toward supplies or CRO services. Funds are held centrally by SPARK's program managers, and any spending must relate directly to the project plan.

"You can stretch $50,000 a long way if you focus on the critical, value-adding or derisking experiments," Egeler said.

SPARK defines success as a project that is out-licensed and/or advances to clinical testing. The goal was a 50% success rate, and Egeler said that SPARK's current rate is 57%. That includes 22 projects in clinical testing, 9 licensed to existing companies and 11 spun out by the principal investigator as startups.

To date, SPARK has supported 90 projects, of which 51 have completed 2-3 years of funding.

Egeler said that for every dollar SPARK has spent on a project, there has been $5-$10 in external grants for follow-on activities for the project or for derivative projects.

One of the core assets of SPARK, according to Egeler, is a pool of more than 50 volunteer advisers from the local biotech community.

Advisers are invited to join weekly SPARK meetings at which university researchers present updates on their projects, discuss problems or strategy and receive guidance on what path to take.

"The real value comes in reducing the hurdles that a new project will experience on its translational path. We identify hurdles and address them," said Egeler.

Matthew Cooper, founder and CEO of Carmenta Bioscience Inc., said that researchers gain a lot of value from the expertise provided by the advisers, which ranges from venture funding and patent strategy to product development.

Carmenta is a startup developing a pre-eclampsia diagnostic that came out of one of the SPARK projects.

Steven Schow, an adviser on the program and VP of R&D at cancer company Telik Inc., said that in addition to helping academics navigate translational issues, SPARK gives them a chance to present their progress to people who "look at the programs and say, 'If this was our program, what would we do?' It's our job as collaborators to help them bridge what they don't know."

He added, "We don't necessarily all agree on something-as happens in industry-so they get to see the debate and get to see the different angles on a discussion in industry."

Advisory capacity

Like SPARK, Catalyst not only covers therapeutics and diagnostics but also has projects for devices and digital health. Its teams work as discrete entities that are guided by a project manager.

According to June Lee, director of early translational research at UCSF and head of Catalyst, the program has about 140 advisers from around the Bay Area who are matched to specific projects and brought in to help during the 4-5 month evaluation period.

Advisers help both at a strategic level and with hands-on execution, and they often remain involved with the project during the year of the award or beyond, Lee told SciBX.

Catalyst funds 2 cycles of projects per year, with an average of about 30 projects per cycle. Projects receive up to $15,000 during the pilot stage and up to $100,000 during the full year of their term, although Lee is seeking additional sources to expand that dollar amount.

Catalyst was launched in 2010, and its money primarily comes from UCSF's Clinical and Translational Sciences Institute.

Catalyst projects have led to several startups and licensing deals, and other projects have received follow-on funding, Lee told SciBX. The numbers for each are undisclosed.

Another central theme at both SPARK and Catalyst is helping researchers define TPPs, which Cooper said is one of the big advantages of having industry advisers. In particular, advisers help researchers understand the market, find the competitive landscape of a product concept and discern whether a given discovery is likely to be commercially attractive.

"A lot of scientists presume their idea is venture backable and could be a stand-alone company. It's not the job of an academic to understand how to size a market or get financing," Cooper told SciBX.

Schow said that industry advisers also help academics tailor product profiles. For example, he said, "a researcher developing a drug for use in a Third World country might come with an idea based on a high-tech microfluidics test system, and we might suggest a colorimetric or fluorescent dipstick that could be held up to the sun."

According to Lee, the connection with industry advisers can play a tangible role in moving a project to a successful exit through targeted connections and specialized advice.

One example is a Catalyst program to make a biodegradable back-of-the-eye delivery device for use in diseases such as age-related macular degeneration (AMD). The technology resulted in the creation of Zordera Inc.

"The project had good preclinical data but didn't know where to go. So we connected them with the CEO and CMO of an eye company who connected them with the head of the eye division at the FDA. We helped them with a business model and the regulatory strategy and helped them set up a reasonable TPP," Lee said.

Building bridges

At least six universities in the U.S. and overseas have started translational programs modeled partially on SPARK. Another 10 have consulted with Mochly-Rosen and Grimes, and some are planning to launch translational programs.

One recently founded initiative is the 4D Technology Development program, which Geoffrey Smith and Scott Friedman are building at the Icahn School of Medicine at Mount Sinai after seeing SPARK and other translational programs in Silicon Valley.

Smith is director of the Mount Sinai Institute of Technology and a professor in the Department of Health Evidence and Policy at the Icahn School of Medicine. Friedman is dean for therapeutic discovery, a professor of medicine and chief of the division of liver diseases at the school of medicine.

A key question for the duo was whether the SPARK and Catalyst models were transferable outside of a biotech hub like the Bay Area. Although New York has seen an influx of biotech money and an increase in public-private partnerships in recent years, its ecosystem is not yet as well developed as that in San Francisco or Boston, Friedman said.

"We've used inputs in thinking about issues, but we need to build to the local environment," Smith told SciBX.

He said that building a committed cadre of volunteers, for example, would likely take longer in New York than San Francisco.

Friedman and Smith joined Mount Sinai within the last two years. Since then, the 4D program-the Ds stand for discover, develop, design and deliver-has established a six-month curriculum focused on teaching researchers to define go/no-go milestones, use Gantt charts to design project schedules and create budgets.

The teams have a principal investigator and usually are composed of postdocs and technicians.

In addition, Smith and Friedman aim to break down some of the assumptions and practices inherent in academic environments that can be obstacles to building commercially focused strategies. One of their early guidelines was that the team leader could not be the principal investigator, to help break down the hierarchy often found in academic labs.

Smith said that there still is an old guard in academia in the region that resists the influx of programs centered on commercialization.

However, he said, "there has been a change in the openness of the academic system to recognize the value of entrepreneurship and the importance of innovation. Schools are starting to think differently about funding options, partly due to challenges of getting federal funding. Entrepreneurship can provide other opportunities for financing. People recognize that they need to find other ways to solve problems."

Disclaimer: C. Simone Fishburn is an adviser to both the SPARK and Catalyst programs. She declares no conflict of interest.

Fishburn, C.S. SciBX 7(12); doi:10.1038/scibx.2014.333
Published online March 27, 2014


1.   Edelson, S. SciBX 6(4); doi:10.1038/scibx.2013.78

2.   Fishburn, C.S. SciBX 7(11); doi:10.1038/scibx.2014.303

3.   Fishburn, C.S. SciBX 7(3); doi:10.1038/scibx.2014.77


Amgen Inc. (NASDAQ:AMGN), Thousand Oaks, Calif.

Carmenta Bioscience Inc., Palo Alto, Calif.

Icahn School of Medicine at Mount Sinai, New York, N.Y.

National Center for Advancing Translational Sciences, Bethesda, Md.

National Institutes of Health, Bethesda, Md.

Spectrum, Palo Alto, Calif.

Stanford University, Stanford, Calif.

Stanford University School of Medicine, Stanford, Calif.

Telik Inc. (NASDAQ:TELK), Palo Alto, Calif.

University of California, San Francisco, Calif.

Zordera Inc., San Francisco, Calif.