While gene editing itself is not that new, the CRISPR/Cas9 technology represents one of the most important breakthroughs in the field in the last decade. Scientists are now able not only to read but also to write genes. This could have major implications for health, wealth and humanity.
Thanks not only to advances in genetics, but also to a general convergence in technologies, including computing, today it is possible to precisely edit DNA mutations in the three billion DNA base pairs of the human genome. CRISPR/Cas9 was first developed relatively recently, in 2021, as a therapeutic agent. It represents a massive leap forward in gene technology, and the scientists behind it won the Nobel Prize in chemistry in 2020. What makes CRISPR/Cas9 such an important development is that it is easy to use, cheap and versatile, putting other gene-editing technologies in the shade.
The potential applications for this technology are immense. CRISPR/Cas9 enables the development of one-off treatments that can replace life-long therapies. Of course, this applies to rare genetic disorders, but also other, more complex diseases. We highlight diabetes as an example, where pancreas cells grown from the patient’s own stem cells could be developed to produce insulin. Another example of the technology’s use is in treating liver disease, with the potential dual benefit of also averting affections to the heart which such liver conditions can cause. Cancer treatment could also be a major beneficiary from CRISPR/Cas9-based therapies, as T cells (the immune cells that fight cancer cells) can be edited to target the cancer, obviating the need for arduous treatments such as chemotherapy.
Progressing from such ambitions to adoption of CRISPR/Cas9 is not straightforward. There are development and delivery challenges and costs, although the latter will decrease over time. Regulation is, without a doubt, a further issue, although currently the FDA and the EMA are supportive of getting new, effective treatments out to patients, knowing that they can reduce healthcare costs.
CRISPR/Cas9 also represents a business challenge: its “one-and-done” treatment approach could have a massively disruptive effect on more traditional market players. For big pharma, CRISPR/Cas9 could, for example, wipe out the antibody market for cancer treatments, and insulin producers would find themselves in trouble with the development of artificial pancreases. Then there is the issue of speed, as CRISPR/Cas9 could greatly reduce the time-to-market of new therapies.
There is, of course, an ethical dimension too, as CRISPR/Cas9 has the potential to enhance the human body, and even to create a new species. Currently, only somatic cell lines are being edited (i.e. the edited genes are not passed on to offspring), rather than germline cells (i.e. egg and sperm cells, and embryos), meaning that genetic diseases can still be passed onto progeny.
The opportunity for the adoption and commercialisation of CRISPR/Cas9 therapies is exciting, being a potential game-changer for human health for decades to come. However, it also entails possible moral conflicts and uncertain outcomes, begging the question of how best to use the technology and ensure it is only for good.