November 1, 2018 (Vol. 38, No. 19)
We’ve become accustomed to new headlines about CRISPR nearly every week, touting gene editing’s potential to transform our world as we know it—from creating more resilient sources of food to saving coral reefs to improving how we treat cancer. Recently, however, new research has shown that in some instances, the gene editing tool known as CRISPR-Cas9 could unintentionally damage DNA during the editing process or unintentionally edit regions that were not targeted.
While these possibilities sound scary—and they could be—no one is in immediate harm. The good news is that the field is still in its infancy, and with enough research, there’s amazing potential for exciting life-changing advancements and discoveries.
In my view, however, we face a bigger obstacle than the potential downsides of CRISPR technology: Despite discovering CRISPR’s potential to tackle some of our biggest global challenges, there is not enough gene editing research being done today to address these challenges. The problem isn’t a lack of scientific knowledge, dedication to the field, or investment in research; rather, the problem lies in the limitations of the tools currently used in gene editing, in terms of both performance and access.
For example, performance and access issues are encountered with CRISPR nucleases, which are like molecular scissors that cut DNA. These nucleases, which include Cas9, are essential tools in gene editing research. However, if commercial researchers want to use Cas9, they are faced with paying high licensing fees, including, in many cases, reach-through royalties on anything discovered in their research. And although academic researchers may use Cas9 without such onerous terms, they are limited in the ability to commercialize their inventions. Once commercialized, academic inventions are subject to the same burdensome commercial terms. Unfortunately, many academics learn this harsh reality only after they try to commercialize products stemming from their academic work.
Sadly, the current reality is that existing restrictions around the use of Cas9 means many great ideas and innovations are left on the sidelines.
Imagine that you’re an academic researcher who has spent years laboriously conducting cancer research that makes use of Cas9. You’ve developed a breakthrough with great promise for treating patients. You may of course decide, for the betterment of medicine and those suffering, to take this to a pharmaceutical company for development, clinical trials, and ultimately mass production. Not so fast. Right now, the parties who own or have exclusively licensed the use of Cas9 can potentially claim a stake in the profits stemming from your invention or even block development of the product if it competes with their own development activities.
Additionally, even for those lucky enough to have unfettered access to the current CRISPR enzymes, the additional tools needed to use them in precision editing often do not work as well as required for important applications. Researchers need accurate tools to conduct the research they want—in a reliable, scalable, and cost-effective manner—to truly achieve the breakthroughs that CRISPR technology can enable.
Compare your smartphone today—with all of its applications and programs that allow you to leverage this remarkable technology—to the original mainframe computers of the past. The differences in performance and accessibility of smartphone technology are incredibly vast compared to those old mainframe computers.
Gene editing today is still at an early stage of the field, akin to the big, clunky mainframe in computing. But CRISPR technology has immense transformative potential and—like computing—may fundamentally change the way certain problems are approached and solved. More research in CRISPR editing is needed to achieve this reality, but the existing research tools are not advanced enough for forward-thinking biologists to realize CRISPR’s potential.
Biological researchers are being asked to solve some of the most complicated problems in the history of humankind; however, to try to do so without the innovative and sophisticated tools necessary is not viable much less optimal. In order to solve this dilemma, the scientific community—with support from business and technology leaders—must put effort behind both advancements in CRISPR research and advancements in the tools needed to do this research.
Once scientists have access to the tools and technology that they need, the pace and volume of genetic research will accelerate toward the advancements that will truly help humanity.
Kevin Ness, Ph.D., is the CEO of Inscripta, a company at the forefront of gene editing technology.