NEW YORK – In May, the National Institutes of Health launched a new prize competition to develop delivery systems for targeted genome editing technology.
Up to $6 million is available for contestants, who must first submit a written proposal as part of phase 1. Up to 10 winners will get $75,000 each and move on to phase 2, where they will submit preliminary results from experiments. Entries that move on to phase 3, the delivery of reagents and protocols for use in validation and animal testing, will each receive $250,000. Finally, winners in two target areas will receive a top prize of $625,000.
The launch of the Targeted Genome Editor Delivery Challenge illustrates both an overall rise of the use of this disbursal mechanism by NIH and the increasingly complex challenges the agency seeks to address with it.
Over the last decade, approximately three quarters of the institutes have combined to sponsor 88 challenges, with $50 million in total direct funding. For comparison, NIH's total budget for extramural funding in 2019 alone was $29.47 billion.
"We are, amongst the feds, one of the more active utilizers of this mechanism and the largest within the Department of Health and Human Services," said Taylor Gilliland, senior advisor for innovation programs at the National Institute of Biomedical Imaging and bioengineering. "It's still a very small piece of the overall NIH budget, however, we find that there's increasing interest across the agency."
The idea of a competition to solve technological challenges in biology was likely inspired by similar efforts by private foundations in the past, notably the X Prize Foundation, whose Archon X Prize for Genomics in 2006 promised $10 million to the first team to sequence 100 human genomes in 10 days for less than $10,000 per genome. In light of rapidly improving sequencing technology, those goals shifted in 2011 to help the clinical adoption of genome sequencing, and the competition was eventually canceled in 2013.
The XPrize Pandemic Alliance, on the other hand, awarded five companies a total of $6 million in 2021 as part of the Rapid COVID Testing competition.
Prize competitions were authorized by Congress as part of the America Competes Act of 2010. NASA and the Department of Defense were early adopters, but overall, federal agencies have slowly but steadily increased their use of them, Gilliland said.
NIH is on track to launch 12 prize competitions, or "challenges," in 2023, down from 18 in 2022; however, overall, the amount of funding available for these has increased over the past five years.
Prize challenges won't be replacing the U-type grant mechanism for large consortia anytime soon, but they bring certain advantages to the government. "It's a tool within a toolkit to advance agency mission," Gilliland said. "It's like a hammer, [it] can't be used to cut a piece of wood. A tool that has a unique approach can't solve all problems, but no one tool can."
Challenges help in "engaging non-traditional participants, people who wouldn't typically apply for a grant or contactor agreement," he said, such as undergraduate or even high school students. "Do you know who the solver is in advance? If you don't, perhaps an open innovation [funding] mechanism is the way to go."
That's a stated goal of the genome editor delivery challenge, which seeks entries from "delivery technology developers who have not previously delivered genome editors but have delivered other macromolecules," said Marko Zitko, communications manager at Freelancer, a contractor that runs prize competitions for several government agencies, including NIH and NASA.
But the format also shifts some of the risk from the funding agency to the entrants. Rather than give a grantee money and hope that they can deliver certain outcomes, the agency gets to define the outcomes and have the solutions come to it.
Many early challenges addressed the development of digital technologies, such as predictive algorithms and an integrated chemistry database for the National Center for Advancing Translational Sciences (NCATS).
But the genome editor delivery challenge is among several that are focusing on the "wet lab" side. Cosponsored by NCATS, the National Institute of Neurological Disorders and Stroke, the National Heart, Lung, and Blood Institute, and the BRAIN Initiative, it seeks a system to deliver genome editing machinery to specific tissues or cell types as well as a non-viral delivery system capable of crossing the blood-brain barrier.
Challenges are also increasingly seeking novel diagnostics. Gilliland is involved in an $8 million maternal health-related challenge, for example, that is part of the Rapid Acceleration of Diagnostics (RADx) program, which seeks to help provide diagnostic devices to improve postpartum healthcare, especially in areas where maternity care is difficult to find.
In addition to courting non-traditional participants, challenges offer some stimulation for private sector investment. For organizations that are already taking on risks, the rewards can be tangible. In addition to the cash prizes, which can provide nondilutive capital, being a winner can help companies secure additional financing. "We've seen companies compete and go on to raise additional rounds," Gilliland said.
"I've been Impressed by the willingness of innovators to take on that risk," he said. "Not just for the reward but to contribute to the mission. If they win, they not only get the prize but validation of their idea or approach."
The hardest part about challenges is often getting the word about them out to potential entrants, Gilliland said. A conference paper coauthored by Freelancer and NASA researchers suggested that the NASA brand was key to incentivizing engagement.
One other benefit of the prize format is that it is conducive to press coverage. "It all goes to solving a problem," said Steven Benowitz, a science writer at the NCATS Office of Policy, Communications, and Education. "So there are a lot of good stories out there about innovation and discovery, which play into topics that journalists would like to cover."