Genetic Engineering & Biotechnology News

NOV1 2018

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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Page 30 of 57 | Genetic Engineering & Biotechnology News | NOVEMBER 1, 2018 | 29 Kevin Ness, Ph.D. We've become accustomed to new head- lines about CRISPR nearly every week, tout- ing 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 dam- age DNA during the editing process or unin- tentionally 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 ob- stacle than the potential downsides of CRIS- PR technology: Despite discovering CRIS- PR'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 prob- lem lies in the limitations of the tools cur- rently used in gene editing, in terms of both performance and access. For example, performance and access issues are encountered with CRISPR nucle- ases, which are like molecular scissors that cut DNA. These nucleases, which include Cas9, are essential tools in gene editing re- search. However, if commercial researchers want to use Cas9, they are faced with pay- ing 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 com- mercial terms. Unfortunately, many academ- ics 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 research- er who has spent years laboriously conduct- ing 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 pharma- ceutical 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 stem- ming from your invention or even block de- velopment 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 need- ed 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 man- ner—to truly achieve the breakthroughs that CRISPR technology can enable. Compare your smartphone today—with all of its applications and programs that al- low you to leverage this remarkable technol- ogy—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 exist- ing 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 prob- lems 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 di- lemma, the scientific community—with sup- port 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 to- ward the advancements that will truly help humanity. We Won't Know without More Research Translational Medicine Eliminate host cell DNA and RNA in biological and vaccine products DENARASE ® Contact c-LEcta for a free sample! Email: Made in Germany • Full cGMP Compliance • Animal Product Free Production • Endotoxin-Free Production Strain • Highly Active endonuclease from Serratia marcescens • High Purity ≥ 99%y The cGMP Nuclease that cleaves all forms of DNA and RNA Do CRISPR Risks Outweigh Rewards? Kevin Ness, Ph.D., is the CEO of Inscripta, a company at the forefront of gene editing technology. Website:

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