Aiden Courtney is CEO of Roslin Cells Ltd. He is an experienced commercial/operational senior executive of early-stage technology and property companies, having held the roles of CEO, CFO and Commercial Director
Phacilitate: Roslin Cells is recognised for its involvement in partnerships and collaborations aimed at advancing the cell therapy area. Can you summarise those activities (the participants and the main goals and priorities) and comment on future plans or requirements for other such initiatives?
Aidan: Roslin Cells’ activities fall into two areas. The manufacture of ATMP cell therapy products, which takes place in our clean room facilities in Edinburgh, and the production of induced pluripotent stem cells for research use, which we do in Edinburgh and Cambridge.
Our largest collaborative project is focused on establishing a central facility for research-grade IPS cells that are being produced in Europe and making them available for academic and commercial research across Europe and beyond. The project is funded by the Innovation Medicines Initiative to create the European Bank for Induced Stem Cells (EBiSC) and involves a consortium of 26 partners, 6 of which are pharmaceutical companies. In this large consortium, we are working with the same community of researchers that are developing new cell therapy products - leading academics looking at the differentiation of stem cells into neuronal and hepatocyte and pancreatic lineages - but we're looking very much at the industrialisation of the production. I think this is absolutely essential if we're going to advance cell therapy.
It was clear to me when we were assembling the consortium that the scale of this project would lead to important developments in cell processing, which would advance our capacity to manufacture cell therapies at scale. And in fact, the EBiSC project is a tremendous proving ground for many of the challenges of developing a new cell therapy, such as consent, manufacture at scale, quality control testing and management of the chain of custody. It is also highlighting data related issues which any cell-based product must address. Whether you are producing cells for research use or for therapy, maintaining the data-set associated with the cell-based product and controlling access to it will be of critical importance.
On the other side of the business - manufacturing cell therapy products - we are in a number of collaborations with academics across Europe, mainly funded by the European Commission through Framework 7 and Horizon 2020. One in particular which stands out is a project entitled Repair-HD, led by Cardiff University with ourselves and several other academic players, which is developing a cell-based treatment for Huntington's disease.
In that project and others, we have three roles to play. Firstly, we are able to supply the clinical-grade starting material. We have our own clinical-grade embryonic stem cell lines which we've made in our facility, and we are working with the Cell Therapy Catapult in London to produce clinical-grade induced pluripotent stem cells.
Our second role is to work with researchers to translate their processes into clinical-grade protocols - so really industrialising the research that will have already taken place in an academic setting. This involves converting the entire process to robust standard operating procedures and very tight control over the reagents used and quality control testing; in short, moving the process to a Good Manufacturing Practice format.
Finally, we have our clean rooms, so having developed a GMP protocol we can then move it in-house and do the manufacturing ourselves for a phase I/II clinical trial.
We provide the same manufacturing service to companies that have already moved into a clinical setting, or that are looking for a contract manufacturing operation to do their clinical manufacture for their trial. For those companies, we're also able to provide support in looking at how to optimise the manufacturing process, or how to move it to a larger scale.
Phacilitate: It is increasingly apparent that the hESC and iPSC areas are two particular fields of research where Europe has a real opportunity to cement its status as genuine world leaders. What will be needed to realise this given strong competition from the likes of Japan and North America?
Aidan: It's a real challenge. There are many well funded programmes in the United States to develop stem cell-based therapies, several involving embryonic stem cells. While in Japan, there is a clear commitment to developing induced pluripotent stem cell therapies and use induced pluripotent stem cells as a game changer in a variety of areas within life sciences. So I think we should start by recognising that while many European researchers have great capabilities in basic science, they need to move fast otherwise those others reach the clinic first.
The inability to secure patents in Europe for products or inventions which involve the destruction of an embryo, because of the Biotech Directive, is a real challenge and I think it will impact the number of embryonic stem cell therapies that are developed here. But for therapies based on induced pluripotent stem cells, I think that there is a real opportunity for Europe to play on the same field as Japan and North America.
The key is support, particularly financial support. I think that some of the major research charities, such as the Wellcome Trust and the British Heart Foundation to name two in the UK, will have an important role to play in that translational space, as do agencies such as Cell Therapy Catapult and the Fraunhofer Institutes in Germany. The lack of private sector funding for early-stage development means that these intermediate agencies will be very important for the tremendous basic science coming out of the science base in Europe to be translated into the new cell therapies.
Phacilitate: The translation of such technologies from research to the therapeutic setting is just beginning to gain real traction on the continent and worldwide – are there any specific trailblazers whose approaches and therapeutic product candidates offer particular promise in your eyes?
Aidan: Well, I think to start with there are the two therapies that we're helping manufacture. The first of those involves ReNeuron, which has a cell therapy for treatment of stroke and ambitions to also move into treatment of critical limb ischemia. I think what's really impressive about ReNeuron is their persistence: it's a company that's been going a long time. They have really knocked down the barriers in working out how to actually move a very clever and promising cell therapy through the research phase to the clinic, which will be for the benefit of others moving forward. We're now manufacturing the product which is being used in clinical trials here in Scotland. I would definitely identify ReNeuron as a trailblazer.
We're also manufacturing the retinal pigmented epithelial cells for Pfizer, which will shortly be going into clinical trial. I think that this is another very interesting convergence of support for basic science towards the development of a particular cell therapy. It is led by Pete Coffey from UCL, with charitable funding in London and then the very early engagement of Pfizer to support the project, which is now moving through to clinical trial. I think that certainly, I'd identify Pfizer as a trailblazer within the pharmaceutical community for wanting to support and drive forward regenerative medicine cell therapies.
Beyond that, I think the other area which is clearly important at the moment is the CAR T-cell therapy space. It's not an area we are manufacturing in at present but it's clearly one that has really gained a lot of momentum over the last couple of years and is probably the most exciting area in cell therapy just now.
Phacilitate: Roslin Cells has a stated goal of helping drive the development of the stem cell sector in Scotland. Can you tell us more about the sector and its key players and stakeholders?
Aidan: When Roslin Cells was originally set up as a spin-out from the Roslin Institute, we were supported very substantially by the local development agency, Scottish Enterprise, and because of this we identified a stated goal of actually developing the stem cell sector in Scotland generally.
In terms of players in Scotland, the University of Edinburgh's MRC Centre for Regenerative Medicine is the academic jewel in the crown which is also located here in Edinburgh BioQuarter. Scotland also has a lot of support services for life sciences, such as BioOutsource, Charles River, Sistemic etc., that provide support for the life sciences sector as a whole.
Then finally, I think the other really important player is the Scottish National Blood Transfusion Service. SNBTS has been very active in not just the use of blood products, but also other cell therapies including the very active islet transplant programme. It is a key player in what we're doing in this space as well.