Christine Martin, Life Sciences Investment Manager at Cambridge Enterprise, will be on the panel at the next ATN event on 14 May 2019 discussing the unique relationship between investors and advanced therapies companies. In this blog, she talks about the privilege of working closely with Cambridge academics, the exciting investment space of advanced therapies and the compelling drive to invest in this promising sector.

Part of the Seed Funds team, Christine enables and supports University of Cambridge academics to commercialise their research. She has a DPhil in Chemistry from the University of Oxford. Before joining Cambridge Enterprise, she worked at Biotica, a Cambridge biotechnology spin-out for eleven years.

Working at Cambridge Enterprise

One of the most rewarding aspects of working here Cambridge Enterprise for me is the close relationship between academics and investors. Cambridge Enterprise was formed to help University staff and students commercialise the output of their research for benefit of society, which means we work very closely with our academics.

In the Seed Funds team we are lucky enough to have a pot of money we can use to support early stage companies. These opportunities obviously have to be credible as we need to recoup our investment to support other companies coming through the pipeline. That said, having an evergreen fund means that we can take more risk than some other investors. I think this is absolutely right. After all, if we’re not going to take a risk on a technology or opportunity from our own academics, then we can’t expect others to do the same.

Spinning out and seeding

As a team we review about 100 opportunities each year. Just under 50 per cent of these are in life sciences. They come in all shapes and sizes, from a sketch on paper to a fully-fledged business plan. Often, they come to us from Cambridge Enterprise’s Technology Transfer team –which means the opportunity might well have tangible IP and some consideration of commercial direction has been done—whilst other ideas might take a little more incubating.

Unlike many investors, we are aligned with the founding academic team from the start. Even if the plan isn’t fully developed, we work together to get over any hurdles and bring them to a seed round of investment. I really enjoy this way of working, and it is an absolute privilege to talk to the academics about their research and discuss their commercial aspirations.

Advanced therapies portfolio

This is an exciting time for the advanced therapies sector and for those investing in it. The government has demonstrated its support for the UK to establish itself as a manufacturing leader in advanced therapies through the Cell and Gene Therapy Catapult which is tremendously helpful.

We have a number of advanced therapies companies, at various stages of development, in our portfolio. Quethera, a spin-out from University of Cambridge, is a gene therapy company focussed on developing novel treatments for glaucoma and other eye diseases. Quethera first received investment from the University and others in 2015. This investment lead to a pre-clinical candidate that demonstrated good efficacy in pre-clinical models. Quethera was acquired by Astellas in 2018, thus putting this technology into an organisation with the expertise and capability to take this to the clinic.

Interestingly, two of our more established companies produce and sell technologies to support research or therapeutic work in the advanced therapies field. Qkine came out of the work of Professor Marko Hyvönen, from Cambridge’s Biochemistry department. The company uses protein engineering to optimise the manufacturing of growth factors and cytokines for stem cell, organoid and regenerative medicine applications. Provision of high quality reagents to the stem cell sector is important and, as this field continues to grow, and advanced therapies become more mainstream, Qkine is poised to meet this need for high quality reagents.

DefiniGEN arose from the work of Professor Ludovic Vallier’s group in the Stem Cell Institute. The DefiniGEN technology differentiates human induced pluripotent stem cells into high quality, functional and mature human cells including hepatocytes, pancreatic beta cells and intestinal cells for use in the pre-clinical research market. DefiniGEN had robust and reproducible methods to generate high yields of high quality cells, addressing current issues of variation in quality of cells available in the market.

Emerging advanced therapies portfolio

We also have a number of earlier stage companies in our portfolio that present exciting opportunities for advanced therapies. The company Bilitech has developed a technology to manufacture bioengineered bile ducts in the laboratory and aims to translate it to the clinic for use in patients. Currently the only treatments to manage bile duct disorders are liver transplants or complex operations. Based on the work of Fotios Sampaziotis and Mr Kourosh Saeb-Parsy from the Department of Surgery and co-founded with Professor Vallier, Bilitech is working with the Cell & Gene Therapy Catapult and is supported by Innovate UK as well as ourselves.

Cambridge Gene Therapy is based on the work of Professor Tim Cox and his colleagues in the University Department of Medicine. They are developing a gene therapy programme for Tay-Sachs and the related Sandhoff disease using a vector system based on adeno-associated virus (AAV). These hereditary disorders are cruel and relentless: they destroy neurones in nearly every part of the brain and spinal cord, so the unmet need remains very great. The aim is to treat babies and children with these hereditary neurodegenerative conditions with an early intervention that, in the absence of any approved treatments, will rescue the condition before irreparable damage has occurred.

Hepatotarget Therapeutics will use the same stem cell differentiation technology as DefiniGEN to manufacture mature hepatocytes as a cell therapy for liver disease.

Although it is early days for these emerging companies, they each have a very compelling plan with a clear market.

Challenges in commercialising advanced therapies

Our companies vary in terms of the support they need. Some are more self-starting with clear foundations whilst others need more help structuring the company. Advanced therapies companies tend to have more foundation work to do because the sector is young and there is not a well-trodden path to follow. We recognise the value of having a relationship with Cell & Gene Therapy Catapult and Innovate UK, who are already working with some of these opportunities. Part of our role is to make introductions to investors, funders, experts in the field who might become advisors and to help review business plans and funding proposals to make sure that the commercial opportunity comprises of the right elements to deliver the business plan for the proposed investment.

The earlier and more disruptive the technology, the more important it is to have mutual trust and transparency between the investor and the company. In the advanced therapy arena there are numerous challenges that we need to address. There are technical and manufacturing hurdles to overcome to ensure the quality and robustness of cell and gene therapy products.

The traditional, and sometimes lengthy, sequence of clinical trials may no longer be applicable in this sector because the motivation to get these new treatments approved and in use is high especially where there are no other options.

Reimbursement is another challenge and we anticipate that it may also need to take a different form, especially as we have don’t have any longitudinal data yet to demonstrate how long these therapies could last. A new therapy that bridges to a standard intervention, for example a transplant, or slows disease progression may be a relatively short-term therapy. But if the patient can be cured, or given many years of disease-free life then reimbursement may take a different shape.

Even though there is a lot of waiting and uncertainty, which is difficult for investors, we’re all investing in this area because we believe it has enormous potential.  We have to make sure we realise that potential to treat patients who currently have no options.

The drive to invest in advanced therapies

For Cambridge Enterprise I think it is different because we invest in what comes out of the University, and it means we have a different mindset about investment that is more aligned with our academics. The reason why advanced therapies are so exciting for our researchers and our scientists and particularly our clinicians is that they have the potential to cure people of diseases that, until this point, have been completely untreatable.

Once public money has supported researchers in understanding the disease and devising treatments, then we are ethically and societally driven to try to commercialise the research outcomes because that is the only way it can be made available to people.

For the therapeutics areas that may be addressed by advanced therapies, this sector may offer the opportunity to genuinely cure people of diseases and transform lives. For academic scientists, clinicians and, by extension, for us, that is incredibly motivating.

Christine will be on the panel at the next Advanced Therapies Network on 14 May  at the Silicon Valley Bank discussing the investment journey in the advanced therapies sector.