This past November, Professor Ronald E. Pearlman from York University’s Department of Biology gave a talk [1] at Osgoode Hall Law School to discuss the potential of the innovative CRISPR genome editing system. Central to the talk was the evolving nature of genome editing technology and the ethical concerns that come with its growing breadth of application.
What is CRISPR?
Some scientists believe the design and development of new biomolecules is as much an art as it is science. The CRISPR system discussed, and used, by Dr. Pearlman capitalizes on an adaptive immunity system found naturally in bacteria and archaea that uses clustered, regularly interspaced short palindromic repeat (CRISPER) DNA segments to fend off invading viruses. In naturally adapting to a virus invading the cell, CRISPR associated proteins (Cas proteins) will create a spacer unit of genetic code that is unique to the invading virus and incorporate this spacer into the CRISPR region of the cell’s genome. This unique spacer unit will then be transcribed (that is, converted from double stranded DNA to RNA), associate with Cas proteins to form a functional complex, and then target and inactivate the very same type of virus that led to the creation of the spacer unit.
In the laboratory, genome editing uses the functional complex found in this adaptive immunity mechanism to insert or remove genetic code from the genome of a cell. By attaching a synthetic, guiding portion of RNA (sgRNA) to Cas proteins they can be directed to a portion of the genome, through complimentary base pairing with the sgRNA, where Cas will recognize a portion of the genome and cut it to either insert a new region or to remove a portion and disrupt the expression of a gene. By cutting out sections of DNA a gene can be disrupted and lose its functional expression in the cell. In other words, it will no longer be able to produce the molecular products responsible for its former physical trait. By inserting new regions of DNA, the genome can be expanded to confer resistance to invading pathogens, such as viruses, or to express new protein products that can add or enhance the cell’s function. For example, a new portion of DNA may be inserted that codes for a digestive protein not normally found in the cell and, consequently, grant a new molecular digestive mechanism.
What does Genome Editing have to do with Law?
Dr. Pearlman noted that there has been an explosion of scientific literature covering the CRISPR system of genome editing since 2010 and it appears that the momentum will only grow in the coming years. The ability to edit genomes can allow for the expression of new protein products that can be of great commercial value as well as pave the way for new medical treatments that circumvent traditional pharmaceuticals. Additionally, Dr. Pearlman noted that the CRISPR system can be used to produce heritable traits – that is, changes that can be transferred from a parent to their offspring. With this sort of molecular modification becoming more pragmatic, it becomes paramount to have a thorough understanding of the biochemical expression pathways that govern genomic expression to keep an eye on the ethical implications of modification. If human genome editing were to become available, should those with advantageous genomic modifications be treated differently by public health systems? To whom should these technologies be made available, if ever? These questions are beyond the scope of current genomic technology but, with the growing pace of CRISPR methodologies, designs may soon start to reach more readily into the macroscopic domain.
What Makes Scientific Designs Different?
With the cost of biochemical research and development increasing and a billion-dollar entry fee for the drug and biomolecular development market it follows that when an industrially relevant molecule is finally created the developer should be able to recuperate their investment and benefit from their work. Normally, the boundaries of property rights require contextual understanding: what is the nature of comparable products, if the new product’s design is generic or obvious, and if the new product can have a place in its intended market. The differentiating criteria of the sciences become pronounced when considering the esoteric nature of the discipline. How can one reasonably expect a thorough consideration of the distinguishing criteria for obscure scientific concepts, like base pair fidelity, when the requisite knowledge is held only by a few people, like Dr. Pearlman, who have committed years, if not decades, to the study? The nuanced nature of genetics can make innovations in genome editing or CRISPR technology appear to be near imitation; however, the modification of a single nucleotide in the genetic code can have a profound impact on the success and possible application of a biotechnology.
Synthesis, Structure, and Industry
What amount of scientific knowledge is sufficient in legal practice? Some argue that a special breed of IP lawyer will arise to confront the high demands of contemporary science and technology patents. Considering the high financial stakes and the significant likelihood that a new molecule or molecular technique will fail the requisite safety tests at any of a multitude of stages, a lot of designs are left in the laboratory. A re-engineering of the approach or scrapping the project in its entirety may follow, meaning product patents should not be initiated until after the molecule has been proven safe for its regular use instead of when it is first designed or synthesized in the lab. Additionally, research and development can indirectly prioritize self-benefit over scientific collaboration since scientists rely on design details to learn about their ever-developing field and most details are kept secret until after a patent has been granted.
This is where innovation becomes conservative and structure becomes especially important. Does a single elemental substitution in the genetic code constitute a new product if the application remains the same? What about changing a single gene to modify a physical characteristic that relies on multiple genes? While certain business practices, such as non-competition deals, are commonly found outside of the sciences, unique loopholes can arise from small chemical modifications which effectively extend a patent beyond its expiry date through the issuing of a new patent for a highly similar molecule. Furthermore, patents may be sought for generic parts of biotechnology procedures that are nonessential to its action, prohibiting competitors from including strategies in their approach and significantly complicating, or even demolishing, a competing synthesis. Lastly, meeting the testing and safety demands of different communities poses an additional hurdle for introduction into a global market due to different national regulatory standards.
So, What Makes It My Molecule?
The same fundamental concepts that apply to patents outside of genome engineering also apply to those inside the discipline but with a stringent demand to understand the nuances of molecular design. An integration of mechanistic knowledge may prove to be key when evaluating possible distinguishing criteria among patents filed for similar compounds but it is ultimately up to practicing lawyers to integrate sufficient scientific knowledge to accurately capture the scope of their client’s designs.
Dominic Cerilli is the Content Editor for the IPilogue and a JD Candidate at Osgoode Hall Law School.
[1] Dr. Pearlman’s talk was organized by The York Collegium for Practical Ethics and The York Centre for Public Policy and Law under the leadership of Ian Stedman. Support for the event was provided by IP Osgoode, McLaughlin College, York University – Faculty of Health, York University – Faculty of Science, and York University’s Office of the VPRI.