A programming approach to biology – realising the opportunity for UK Life Sciences and novel cell therapies

Guest blog by Dr Mark Kotter, CEO and Founder of bit.bio, stem cell biologist and complex spine neurosurgeon, who speaks about the company and the importance of SynBio as part of the UK Life Sciences Vision.

Essentially Synthetic biology (SynBio) is rethinking biology and taking a software approach - where cells can be programmed with predictable outcomes. There’s already a lot of exciting work happening in this area when it comes to companies programming bacteria and other simple organisms to make drugs, and more recently new, sustainable materials.

But the concept is now moving into a new, more complex area where we are looking to program mammalian, including human, cells. This is what we dub SynBio2.0 and it’s a hugely exciting area. With the right regulatory frameworks and financial support the UK is well-positioned to take a leading, even preeminent, role in SynBio v2.0, particularly in the development of cell therapies and resulting new era of healthcare.

Our company, bit.bio, has technology that is unique in its ability to reprogram human stem cells into mature cell types. Our name represents the coming together of coding (bit) and biology (bio). Bits are building blocks of code, just as cells are building blocks of life. Our technology, called opti-ox, engineers code, in the form of a particular class of gene called transcription factors, into specific sites of the DNA in the stem cell. When activated, this code establishes a new program that converts the stem cell into a new identity. opti-ox is unique because it reprograms every cell in a culture dish - it’s a breakthrough in reprogramming that for the first time enables consistent manufacture of highly defined cell products at industrial scale.

We already have the code words for a variety of muscle cells and brain cells and are already producing vials of these cells. The use of our cells by researchers and in the context of drug discovery proves that these cells are real and that our approach is no longer science fiction. The other side of our business is using the power of big data, machine learning and mass experimentation to uncover the code words for every human cell type. Continuing on this journey we will eventually understand the operating system that governs human cells: LifeOS. In the words of Sydney Brenner, this ‘code script — is a fundamental feature of the living world and must form the kernel of biological theory’. bit.bio is in pole position in the race todiscover this knowledge and IP, which has the potential to define the life science industry in the 21st century.

So why is this ability to potentially manufacture every human cell at scale important? Every disease is a consequence of something going wrong in a set of particular cells in our bodies. Being able to access human cells will transform our understanding of disease and open up a new era of medicines. The most exciting application, however, is using cells themselves as medicines. Today cell therapies rely on donors or patients which means cells are a finite resource and in some cases we can’t access them at all – it is difficult and ethically questionable to get donated brain cells, for example. Also manufacturing treatments at scale, with consistency, and precision is a major challenge. As a consequence, existing treatments are one-offs, bespoke and costly. And whilst these early cell therapies are being approved because they are effective, without a revolution in how we manufacture cells, we will never make cell therapies accessible for all patients and all diseases that could benefit from them.

That’s where I think bit.bio will make a difference. And our investors agree. At the end of 2022, as part of our Series B funding round we raised GBP 103 million to accelerate the clinical development of opti-ox™. This funding round placed bit.bio in the top ten venture deals of 2021 as listed in the BIA report on biotech financing published in January.

As well as unblocking the major manufacturing hurdle faced by cell therapies, we believe our technology will also hugely increase the efficiency of drug development and commercialisation in line with the ambitions of the UK Life Sciences Vision, published last summer.

Getting the right regulatory environment and investment in place for this sort of frontier science depends on a close and trusted relationship between Government and our industry on a complicated basket of interrelated policy issues. During these historic times, where Ministerial ‘bandwidth’ is limited, the role of the BIA has been invaluable most recently around the Life Sciences Vision, concerns around the National Security and Investment Act and settlements in last autumn’s Spending Review. The BIA report on cell and gene therapies (Nov 2021) put forward four calls to Government, including amendments to HM Treasury accounting rules and improved data collection, to ensuring access and maintain UK’s position as a world leader.

bit.bio is playing its part through the BIA and directly informing initiatives such as the Taskforce on Innovation, Growth and Regulatory Reform of last year, led by George Freeman MP, who is now Science Minister and a leading champion for life sciences.

The new era for synthetic biology, allowing an industrial supply of human cells, will provide a great dividend for healthcare, tackling some of the most challenging medical questions, and providing an economic benefit through the development of new industry and the improvement of individual lives. The next ten years will be crucial to succeeding here, and government and industry will want to ensure every opportunity was taken to deliver success for the UK as a science (and SynBio) superpower.