Dr. Guy Sauvageau. Photo credit: IRIC
Launched in June of 2015, ExCellThera is a clinical-stage biotechnology company making strides in developing robust and cost-effective processes for growing blood stem cells for therapeutic use, backed by award-winning proprietary technology.
The company was founded based on a key molecule discovered by Drs. Guy Sauvageau and Anne Marinier at Université de Montréal’s Institute for Research in Immunology and Cancer (IRIC), and a bioreactor developed by Dr. Peter Zandstra when he was at the University of Toronto’s Institute of Biomaterials and Biomedical Engineering (IBBME). Dr. Zandstra currently serves as CCRM’s Chief Scientific Officer and as Director of the University of British Columbia’s School of Biomedical Engineering. ExCellThera is CCRM’s first spin-off, brought into the world via a partnership between CCRM and the Institute for Research in Immunology and Cancer Commercialization of Research (IRICoR).
Cord blood (the blood left in the umbilical cord and placenta after a baby is born) is a good source of stem cells with wide ranging therapeutic applications, but the success rate of blood system transplantation is strongly correlated to the number of cells used, and cells are in short supply. ExCellThera’s stem cell expansion approach represents a valuable breakthrough for clinical applications, with a best-in-class process that improves the viability and success of cord blood stem cell transplantation.
In CCRM’s 2016 annual report (page 13), Dr. Sauvageau told of how it would have been difficult to launch the company without CCRM and IRICoR, because they “empowered the founders to maintain control of the research, while providing the financial and operational support” that they needed. We caught up with Dr. Sauvageau to find out how the technologies based on his research are progressing on the road to clinical adoption.
There are a lot of exciting technologies in development at ExCellThera that have the potential to change patients’ lives. How are these technologies progressing through the development pipeline?
For our cord blood technology, we are soon to transplant our 100th patient. We’re also progressing quite well for the indication we refer to as “donorless” patients. These patients represent 5-15 per cent of those needing a transplant. We are approximately 18 months away from a regulatory approval – we’re really quite close.
We’ve also invested quite heavily in bone marrow expansion. We’re still in the research and development phase and there are still aspects we want to improve, but that’s progressing really well. One of the key indications for this therapy is solid organ tolerization. It is significant for patients because it means that by getting bone marrow or mobilized blood from the solid organ donor, you can induce a state of tolerance in the patient who will receive the transplant, and hence they don’t need to take immune-suppressive therapy for the rest of their life. With this technology, we are approximately two years away from an initial clinical trial.
Has anything surprised you about the trajectory ExCellThera has taken compared to how you thought things would go back in 2015 when you co-founded the organization?
There are a lot of things that have surprised me; firstly, how unprepared we were to go to market. We underestimated the energy, time, effort and money that it takes to bring a cell therapy product to market. That’s something that really surprised us. We thought it was a lot more straightforward. We were able to make all this progress thanks to the strong team we have built over the years, starting with David Millette who is now my co-CEO and our COO.
I’ve also been surprised by the amount of interest that I’ve seen all over the world around our technology, with people interested in obtaining licencing for its use in a range of different applications. That’s not necessarily the business model that we had envisaged initially. Licensing is becoming a greater part of our business because our technology is dominant and has driven significant inbound interest from various bio-pharma companies. You need to decide what you focus on as a company, but you can’t ignore that there are other indications for your therapy as well. So that’s been quite involving and fun.
How about your personal trajectory, has your own career and scientific focus followed the path you had anticipated?
I must admit it has been a huge surprise. I never thought that I would co-lead a biotech company. That was not part of what I anticipated. When we started a company, I thought we would just hire someone and take off!
It has surprised me, but I must admit that it’s fulfilling and purposeful to witness a technology that you developed with colleagues help cure people from cancer. I’ve seen some of these patients myself because, as a practicing hematologist, I do bone marrow transplants every week. I’ve seen some of the UM171 [proprietary molecule] patients, namely people who were essentially condemned to a very nasty disease, who joined our trials and are still alive six years later. I guarantee there’s really something magic when you see this. A lot of the credit goes to our clinical specialists who know how to best position the technology for patients.
Having such a strong team with and around me also allows me to really focus on some of the key issues like the basic science and the clinical program of the company.
How did CCRM support ExCellThera’s journey in the early days?
CCRM, like IRICoR, was instrumental. In the case of CCRM, they invested some seed funding into the company. They really believed in our technologies and Drs. Michael May and Peter Zandstra were co-founders. They were important in saying, “hey guys, we need to get this going; it’s an important technology.” I’m not sure that without them I would have been part of developing a company, because you always think that you won’t be successful because you’ve never done it, or you don’t have the support to do it.
They brought the support, and they also brought the initial funds which were really critical in getting the company going. So CCRM in the seeding part was really essential, and Michael still serves as the chairman of the board of ExCellThera, bringing years of expertise in cell therapy and regenerative medicine. Michael is extremely active; he has been and continues to be very precious to ExCellThera.
And Peter, as a world-class bioengineer, is still a member of our scientific advisory board and brings important advice and connections to help the company in this field. CCRM, particularly through these two individuals, has been instrumental. Last but not least, CCRM has also provided a lot of scientific expertise for the manufacturing of the product and, in fact, the initial manufacturing process was in part developed at CCRM, which was very important.
Read about CCRM’s incubation program on the CCRM website, and keep up-to-date with ExCellThera’s journey at excellthera.com.
Cal Strode
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