CRISPR-Cas9 tools have recently created a buzz in the global healthcare industry, with the development of numerous applications-focused solutions—and intensifying patenting disputes. The invention of CRISPR-Cas9 gene editing tools is one of the greatest scientific revolutions of this generation. Despite major controversies around patenting rights and ethical challenges, CRISPR-Cas9 tools have gained popularity with the scientific community and life science companies, primarily due to their ability to accurately cut the DNA sequence. CRISPR-Cas9 has entered commercial application in less than five years from its introduction—far earlier than expected by average technology life cycle theory. Although the phenomenon of experimentation with gene editing is not new, CRISPR’s heightened popularity highlights its spectacular success at solving the critical challenges.
CRISPR – A Guide RNA-Driven High Precision Gene Editing Tool
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats and Cas9 is a CRISPR-associated endonuclease (DNA cutting enzyme). CRISPR is capable of recognizing specific DNA sequences in human, plant and animal genomes and can precisely cut the targeted DNA sequence using the cutting enzyme (endonuclease Cas9). The edited region can also be filled with a new, preferred DNA sequence to induce the expression of desired traits in humans, plants and animals by altering their gene sequence.
Simply put, CRISPR-Cas9 provides scientists and clinicians with the ability to delete undesirable traits and add desirable traits with high precision and very limited risks of impact on nearby DNA sequences. For example, CRISPR could be used to edit or turn off one of the genes responsible for causing diabetes, resulting in decreasing beta cell death and increasing insulin production in the patient. Furthermore, the technology is very easy to use, is available at a very low cost and can be effectively used in experiments involving interactions with multiple genes. This capability could be very useful in treating various genetic and chronic diseases, such as different types of cancers, cystic fibrosis (CF), sickle-cell disease, beta thalassemias, and many more.
CRISPR Applications In Other Industries
Furthermore, CRISPR has applications in various other non-human health applications, such as agriculture and plant genomics, and animal biotechnology. In the case of agriculture and plant genomics, one of the most prominent applications is the development of gene-edited crops. The applications in animal technology are largely related to disease treatment and breeding.
Commercial Applications With Enormous Growth Opportunities
CRISPR can find applications in any organism on the planet which has DNA. In the case of human therapeutics, CRISPR solves the challenges related to curing chronic and genetic diseases. In the agricultural sector, CRISPR addresses the most important challenge—high productivity and low wastage.
Our recent study estimated the revenue potential from CRISPR-Cas9 tools at around $25-30 billion by 2030, which covers revenue from different applications, including human therapeutics, agriculture and plant genomics, animal biotech, and research tools.
CRISPR-based Therapeutics To Solve The Puzzle of Genetic Disorders
Rather than focusing on the treatment of diseases, companies can now aim to modify genomes to remove the genes responsible for the expression of those diseases, enabling patients to avoid repetitive and continuous programs of therapies. A number of specialist therapeutics companies, including Editas, Intellia, and CRISPR Therapeutics, have invested in the field and made notable progress. Large pharmaceutical companies, including GSK, Celgene, Bayer, Novartis, Regeneron Pharmaceuticals, and Allergan, have also invested through funding, JVs and partnerships with these specialist players. Most ongoing projects are focused on immuno-oncology, liver diseases, beta thalassemias, sickle-cell disease, cystic fibrosis and other hematopoietic diseases. Patients and providers can expect to see the launch of a few breakthrough treatments in the near future. Rare disease segments will be affected mostly in the long term.
However, ongoing patenting disputes and ethical considerations are critical challenges and need to be addressed with urgency, as they may have long-term implications. Changes in patent ownership will significantly impact the technology licensing status of commercial companies, and may affect the development of commercial applications.
Furthermore, various associations and independent organizations have urged the importance of considering the ethical challenges of gene editing. Numerous ethical issues have been highlighted in relation to the germline editing process using CRISPR. Editing human embryos may change the germline in a way which will affect future generations, their characteristics, and behavior patterns without their consent. Thus the associated developments need to be handled with due ethical consideration. Another key challenge is the possibility of triggering unintended mutations during the treatment process, which can pose serious concerns for patients. The scientific community has made tremendous progress in this respect, and it is expected that these possibilities of unintended mutations will be minimized as far as possible in the near future.
Extended Shelf Life For Crops, Eliminating The Possibility Of Food Shortage Globally
Gene edited crops using CRISPR-Cas9 represent another exciting area for companies, with CRISPR having potential applications in removing genetic disease, enhancing nutrient efficiency, improving drought resistance, and extending the shelf life of food crops. CRISPR-Cas9 tools let scientists modify the genetic structure of crops to induce desired features. DuPont, Monsanto, and BASF have already licensed the CRISPR tool for use in new crop and seed development. The limited clarity around regulations for transgenic and gene-edited crops, especially in European countries, represents a challenge for companies and scientists and has significantly impacted the commercialization process. It will be interesting to see how regulatory authorities maintain the balance between ethical issues and technological utility.
A Game Changer Or A Pandora’s Box?
Overall, CRISPR presents an opportunity to major organizations of using the convergence of biology and technology to improve human life at large. Like most scientific revolutions, this has also produced several pros and cons, with the pros thus far seeming to outweigh the cons. While the growth potential here is immense, the ethical considerations need be settled by legal and regulatory authorities. They should come up with guidelines for managing the interests of both human life and major organizations, and avoid any possible large-scale negative implications for future generations. In most countries, there are currently no guidelines on genetic modification in human reproduction. Those who do have such guidelines have only banned such activities (especially in Europe). Even the medical research community has not supported the idea of modifying embryos. However they are highly optimistic about the applications for rare and genetic diseases. Thus the larger picture has to be looked at, while taking such decisions on the basis of ethical considerations.
At the moment, the sector seems an exciting one, with numerous developments happening across the value chain. It will be interesting to see how different stakeholders take up different opportunities and challenges, as these come their way!