Limiting biotechnology innovation

CRISPR/Cas9 commercial licensing and royalty fees can reach millions of dollars and – more significantly for many applications – can take many months or years to negotiate. This makes the techniques inaccessible for many companies, but especially smaller enterprises and venture-backed start-ups that rely on speed to market. For the pharmaceutical industry, freedom to operate is a major barrier to future profits and growing research cost pressures. You can add to that the fact that navigating the IP landscape is complex and risky, which stymies innovation and limits the exploitation of biotechnology.

Our approach was developed within CC’s state-of-the-art lab-in-the-loop biotech laboratories and engineering workshops. The emerging method proved highly efficient and straightforward to use. In fact, in just a few weeks we had recapitulated classic gene editing experiments with identical sequences to those produced by commercial CRISPR/Cas9 kits with their heavily restrictive terms.

The progress has now brought us to a vision of an IP-free future for gene editing that puts its benefits into much sharper focus. Such technology has the power to accelerate innovation, improve precision in-organism edits and reduce industry costs. For the coming generation of CEOs, CTOs and founders, it opens the way for genetic tools to unlock transformative business value.

We plan to use these tools as part of a transdisciplinary programme to solve business problems, de-risk future investments and evaluate ways to embrace more sustainable bioengineered organisms and manufacturing processes. We’re particularly keen to explore how scaling our method can help train machine learning models to make better predictions in different genetic contexts – which may inform chassis engineering efforts in the future. We also plan on exploring the application of the method to other organisms beyond E. coli, where we believe there is room for further innovation and first-in-organism applications.

Gene editing in pharmaceuticals

Gene editing is a potent tool for innovation. In pharmaceuticals, it can enhance the speed and accuracy of drug development processes, leading to more effective therapies and personalised medicine getting to market faster. In agriculture, it can help develop more resilient crops, improve yields and create sustainable farming practices. Environmental science can benefit from advances in pollution control, ecosystem restoration and combating climate change. Industrial biotechnology, meanwhile, could be spurred by optimisation in manufacturing processes and the development of bio-based products, reducing reliance on petrochemicals.

For a bit more perspective, let me dig a little deeper into some real-world examples. I’ll start with human health, where a CRISPR gene edited drug, CASGEVY, was approved by the FDA in 2023 for treating sickle cell disease (SCD), a condition where sufferers produce a faulty type of haemoglobin that results in sickle shaped red blood cells.

CASGEVY is a cell therapy relying on a precise CRISPR gene edit to modify a patient’s own red blood cell. It targets the BCL11A gene in the cells with the aim of increasing the production of foetal haemoglobin, which can alleviate symptoms. Such treatments represent a paradigm shift away from large gene editing solutions that have high manufacturing costs. They also avoid the need for bone-marrow donors and the danger of donor-graft rejection.

Turning to agriculture now, and the centuries old practice of humans selectively breeding crops to maximise yields and establish them in new environments. With the world population growing, climate change is bringing adverse weather, diseases and pests that threaten the global food supply. It’s essential to apply great precision and accuracy in modifying food sources to withstand such pressures.

Tools like CRISPR are already enabling draught resistant maize and wheat, as well as crops such as false flax which is enhanced to produce omega-3 oils. This is good for human health and brings further benefits, such as slowing down browning on banana skins so that fewer are thrown away and wasted.

My third example is the microbiome, an area where our understanding is gathering momentum. The microbes in our gut have been described as a second brain – impacting on our health in symbiosis so that we thrive, rather than in dysbiosis, which is implicated in cancers and various other diseases.

Application of CRISPR

The application of CRISPR on the microbiome can not only enable health but also cosmetic benefits based on the thesis that entire ecosystems of microbes could be tailored to complement and enhance the way in which we feel and look. They could even modulate and control body odour and other consumer needs. Through modifying our very personalised biomes, CRISPR is set to play a major role in physical and mental health.

I think it maybe goes without saying that I and the rest of the team involved are thrilled to have identified an approach that allows us to securing long-term competitive advantage for our clients from new-to-the-world innovation that is hard to copy. However, getting to market faster without being weighed down by endless royalties is I think the most compelling part of the proposition . So – do please お問い合わせ if you’d like to discuss ways to navigate the complexities of gene editing and unlock its full potential. It’d be great to hear from you.