The Nobel Prize, CRISPR-Cas9 and Biological Threats

By Christine Parthemore

The Council on Strategic Risks (CSR) would like to congratulate Emmanuelle Charpentier and Jennifer A. Doudna for receiving the 2020 Nobel Prize in Chemistry, announced on October 7th. This prestigious prize was awarded for their work developing CRISPR/Cas9 genome editing technology.

Doudna—a champion for countering biological threats and improving science communications with policy makers—joined CSR and Sandia National Laboratories to speak at our first workshop focused on how to make bioweapons obsolete as a threat of mass destruction, in August 2019. While the discussion was under the Chatham House Rule and therefore this is not attributable to any individuals, this is an overview of what the group discussed regarding the CRISPR-Cas9 technology Doudna and Charpentier developed:

“CRISPR-Cas9, a breakthrough area of genome editing technology, can be used globally to precisely manipulate genomes through gene knock-out (gene inactivation). Within the next 10 to 15 years, any genome change, including a knock-in (substitution or insertion of DNA sequence information) will be possible. Uses include dialing up or down the expression of genes to create transient changes, detecting or responding to specific molecules in disease treatment via CRISPR enzymes, and more.

Technology today is limited by a lack of knowledge of what to edit. That is, we only know of some of the genes that play an obvious role in human health. Concerns such as using methods developed for personalized medicine to attack individuals may be further into the future. However, this knowledge will improve in the coming years, allowing (for example) analysis of large populations to identify a small group of people susceptible to a specific disease.

It is now easy to access genome editing tools globally in labs. However, this generally does not yet extend in significant ways outside of laboratory environments. While there have been breakthrough advances in genome editing, knowledge regarding what to edit for specific effects and what are the unintended off-target effects is still nascent today.

Use of synthetic biology to engineer organisms is a rapidly maturing one as evidenced by the large number of companies, both large and small, involved. Using large sequencing databases and bioinformatics tools, it is routine now to design and test thousands of genes for the purpose of making a novel molecule. 

Artificial intelligence will play a large role in future biotechnology advances. Advanced manufacturing and robotics technologies play key roles in how the bio-economy is evolving, and big-data analytical and prediction capabilities will be increasingly prominent. Automation allows scaling and enables lesser-skilled people to work more effectively while standardization can offer levels of control and security in biotech operations. Notably, these fields are progressing at different paces and that will carry implications for how they influence the biothreat environment and enable more effective responses to bio events. And though tools that might be used for enhancing or creating new bioweapons are advancing, commensurate changes in the technologies for delivering them effectively may not evolve at a commensurate rate.

While advances in biotechnologies represent a risk, these advances have been and continue to be critical for rapidly developing countermeasures, and to designing and synthesizing genes for more rapid prototyping and testing (emphasis added in this blog post).

When CRISPR-Cas9 emerged, many experts raised concerns about whether it would make it easier to produce biological weapons, including particularly novel ones. While those risks are real for any dual-use technologies, CRISPR-Cas9 and other genome editing approaches will be absolutely critical to rapidly responding to biological threats as they emerge. They will have a critical role in systemically halting diseases before they become widespread pandemics, whether they emerge naturally, by accident, or are deliberately deployed.

Congratulations again to Doudna and Charpentier for their Nobel Prize. We send our gratitude for their breakthrough work. 

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