These cells are produced from pluripotent stem cells, capable of transforming into any type of cell, or multipotent stem cells, which have a more limited differentiation potential. They can come from the patient themselves (autologous) or from an external donor (allogeneic).
This innovative therapy is under development for a wide range of medical applications. It offers promising solutions in regenerative medicine and for the treatment of various pathologies, including certain types of cancer, as well as rare diseases affecting immunology, hematology, neurology, cardiology, ophthalmology, and infectious diseases, among others.
Cellular therapy can also be combined with gene therapy. The latter aims to repair defective genes or introduce missing genes into cells to treat specific conditions. It can also be used to reprogram immune cells so that they can precisely target cancer cells, such as the “CAR T-cell” technology.
Cellular therapy in Quebec is a rapidly growing field, supported by cutting-edge research infrastructures and close collaborations between academic, clinical, and industrial institutions. Centers of excellence, such as the one at the Centre intégré universitaire de santé et de services sociaux de l’Est-de-l’île-de-Montréal (CIUSSS de l’Est-de-l’Île-de-Montréal), pioneers in the field, stand out with internationally recognized expertise. In parallel, networks such as ThéCell and CellCAN bring together researchers, clinicians, and experts to promote translational research and train highly skilled professionals, thereby facilitating the transfer of discoveries from the laboratory to clinical practice.
Quebec also benefits from solid financial support, both provincial and federal, which allows for the development of advanced infrastructures while ensuring compliance with the highest standards of quality and safety. For example, on November 16, 2020, the Quebec government allocated $6.2 million to the CIUSSS de l’Est-de-l’Île-de-Montréal, specifically to the Institut universitaire en hémato-oncologie et de thérapie cellulaire (IHOT) at the Maisonneuve-Rosemont Hospital (HMR), to equip the institute with essential tools for the production of anticancer cells and therapeutic vectors.
Finally, the integration of partners such as Héma-Québec, which manages the recruitment and qualification of stem cell donors, further strengthens Quebec’s healthcare system’s ability to innovate and offer tomorrow’s treatments to patients.
Key Sectors
Health Canada has approved several cell and gene therapies for the treatment of hereditary eye diseases, spinal muscular atrophy, and rare blood disorders such as leukemia and lymphoma. To support the development of these innovative therapies, the Quebec government invested over $6 million in 2020 to establish Canada’s first gene therapy production unit compliant with Good Manufacturing Practices. This investment was allocated to the IHOT at the CIUSSS, a center known for its expertise in cell therapy and rigorous quality standards.
Quebec’s centers of excellence are home to numerous internationally renowned researchers specializing in cell and gene therapy. Among them, Dr. Pierre Laneuville, medical director and head of the Cell Therapy Laboratory, is leading several research projects aimed at developing new cancer treatments, exploring cell immunotherapy, using stem cells for bone regeneration, and creating antitumor cell vaccines, as well as addressing conditions like cerebral palsy and reducing kidney transplant rejection.
A pioneer in the field of cell therapy, Dr. Denis-Claude Roy focuses his research on hematopoietic transplantation, graft-versus-host disease, and the use of stem cells in ocular and cardiac regenerative medicine. He is the director of IHOT and the Cell Therapy Research Unit at the Maisonneuve-Rosemont Hospital Research Centre (CRHMR). He also serves as the medical director of the Centre of Excellence in Cell Therapy (CETC) and as the scientific director of the C3i Centre, dedicated to cancer immunotherapy commercialization. He is also the CEO and a founding member of CellCAN, the Canadian network for regenerative medicine and cell therapy.
Dr. Jean-Sébastien Delisle is a hematologist-researcher and head of the Cancer Immunology – Transplantation Research Unit at CRHMR. He divides his time between laboratory research and his clinical practice in hematology, hematopoietic cell transplantation, and cell therapy. As the medical director of CETC, he supervises the preparation of therapeutic cellular products. His research covers the entire spectrum, from fundamental research to clinical applications in immunology and immunotherapy.
Dr. Sandra Cohen is a clinician-researcher and associate professor, participating in numerous clinical protocols in hematology and bone marrow transplantation. She is dedicated to umbilical cord blood transplantation, particularly in expanding stem cells with the UM171 molecule, while also working on lymphoproliferative syndromes and reducing post-allograft infections.
Dr. Guy Sauvageau, scientific founder of the Institute of Immunology and Cancer Research (IRIC) and former director for 12 years, has made significant contributions to molecular genetics research on stem cells. He is also the founder of IRICoR, the commercialization center for IRIC research, and ExCellThera, where he leads the clinical and commercial development of the UM171 molecule in cell and gene therapy.
Dr. Anne Marinier is a principal researcher and director of medicinal chemistry at IRIC. She played a key role in creating what is now the largest medicinal chemistry group in academia in Canada. Leading the Institute’s Drug Discovery Unit, she directs a team of 60 chemists and biologists dedicated to developing new biologically active chemical compounds with therapeutic potential. She contributed to the discovery of the UM171 molecule mentioned above. Dr. Anne Marinier is the CEO of RejuvenRx, a biotechnology company specializing in the development of precision stem cell-based therapies aimed at combating cancer and aging. She was also the technology director at ExCellThera, an advanced clinical-stage company specializing in cell expansion and engineering.
Key Players
Our Industry Stars
C3i
C3i is a company offering comprehensive solutions in the cell and gene therapy development chain. C3i is a contract pharmaceutical organization focused on partnerships and driven by patient needs. They provide cost-effective cell and gene therapy manufacturing services, from process development to Phase III, for the U.S., Canadian, and European markets. C3i aims to become a leading organization, making cell and gene therapies accessible to all those in need. Their goal is to develop and manufacture cutting-edge cell and gene therapies at reduced costs, making them accessible to people in need.
CORDEX
Cordex is a company specializing in allogeneic cell therapy from umbilical cord blood. The company is preparing to commercialize a cell expansion therapy based on its proprietary UM171 technology. Its mission is to improve the quality of life for patients with blood diseases by developing and providing innovative and curative stem cell-based therapies. With its expertise in cord blood-derived cell therapy and its ongoing commitment to research and development, Cordex positions itself as a key player in the field of allogeneic cell therapies. Its goal is to treat and cure blood diseases, thus improving the lives of millions of people worldwide.
ExCellThera
ExCellThera is an advanced clinical-stage company specializing in cell expansion and engineering. It provides molecules and bioengineering solutions to amplify and modify various cell lines for use in innovative curative therapies for patients with malignant hematological tumors and other diseases. ExCellThera’s most advanced technology, ECT-001 cell therapy, combines a proprietary molecule, UM171, with an optimized culture system. UM171 cell therapy is now integrated into Cordex Biologiques Inc., a wholly owned subsidiary of ExCellThera. To offer better treatments to patients, the company is developing its cell expansion and engineering platform while supporting leading clinical trials.
Morphocell
Morphocell Technologies is a Montreal-based company specializing in preclinical regenerative medicine, focusing on the development and commercialization of cell and engineered tissue therapies aimed at treating severe organ deficiencies. Morphocell is developing an extensive platform based on its engineered tissues derived from allogeneic stem cells for organ replacement therapy. Morphocell’s first therapeutic product, ReLiver®, enables allogeneic treatment of acute and acute-on-chronic liver failure. The temporary implantation of ReLiver® improves survival, treats hepatic encephalopathy, and accelerates liver regeneration in the recipient, all without the need for immunosuppression.
RejuvenRx
RejuvenRx is a biotechnology company specializing in the development of precision therapies based on stem cells to combat cancer and aging. It is developing a major asset consisting of a series of small molecules that stimulate tissue regeneration, aiming to treat cancers, age-related frailty, and other diseases. Its expanding pipeline relies on a platform for identifying synergistic molecules targeting cancer stem cells, access to diverse libraries of small molecules, and strong expertise in drug optimization. They address cellular aging by modulating the epigenetic control of stem cells.
StemAxon
StemAxon is a company aiming to become the global leader and go-to reference in research and development in the field of neurodegenerative diseases, primarily sporadic Alzheimer’s disease and macular degeneration. StemAxon offers a new platform to the pharmaceutical industry to test their candidate compounds against Alzheimer’s disease. This unique platform significantly increases the efficiency of validating existing molecules for drug and treatment development. StemAxon has also developed a molecular diagnostic and detection method for Alzheimer’s disease targets. The company is currently in the final preclinical phase for its universal macular transplantation program in translational medicine.
Key Players
Our Institutional Leaders
Centre intégré universitaire de santé et de services sociaux de l’Est-de-l’Île-de-Montréal (CIUSSS)
The CIUSSS brings together two hospitals, two university institutes, eight local community service centers (CLSCs), and fifteen long-term care and rehabilitation centers (CHSLDs). With 17,500 employees, the CIUSSS offers a comprehensive range of healthcare and social services to the population.
A pioneer in medical advancements, the Maisonneuve-Rosemont Hospital (HMR) of the CIUSSS of the East Island of Montreal made history in Quebec in 1980 with the first stem cell transplant. Now known as the University Institute for Hemato-Oncology and Cell Therapy (IHOT), HMR has gained global recognition and receives patients from across the country to benefit from its innovations. On April 2, 1980, Dr. Yvette Bonny, a retired hematologist and then-director of the pediatric bone marrow transplant unit at HMR (1980-1998), performed the first bone marrow transplant in Quebec. This achievement saved the life of Sonia Sasseville, a 12-year-old patient.
The clinicians and researchers at IHOT have built internationally recognized expertise, particularly in hematology-oncology, bone marrow transplantation, and the use of cells as medicines. Thanks to these major advances, the IHOT team primarily cares for patients with blood disorders such as leukemia, lymphoma, myeloma, and other cancers. At the same time, it develops innovative cellular therapies aimed at treating other conditions, including those affecting the eyes, kidneys, immune system, as well as cardiometabolic and neurological diseases.
The Research Center of the CHUM (CRCHUM)
The Research Center of the Centre hospitalier de l’Université de Montréal (CRCHUM) is one of the largest and most modern biomedical and healthcare research centers in Canada. It brings together fundamental, clinical, and population health research under one roof. Recognized as a leading institution, it received the “exceptional” mention from the Fonds de recherche du Québec — Santé in its 2020-2026 strategic planning.
CRCHUM stands out for its internationally renowned scientific output and its commitment to fostering a diverse, equitable, and inclusive environment for training the researchers of tomorrow. Among its areas of expertise is cell therapy, with researchers such as Dr. Sophie Lerouge, whose research team is developing new biomaterials and hydrogels for treating vascular diseases, and Dr. Nicolas Noiseux, who works on using stem cells to treat ischemic cardiovascular diseases. Dr. Nicolas Noiseux’s research team conducts clinical studies assessing the effect of stem cells for treating ischemic cardiomyopathies and investigates the cellular and molecular mechanisms of cell therapy.
Montreal Clinical Research Institute (IRCM)
The IRCM is a leading scientific hub affiliated with the University of Montreal and McGill University. With over 400 members in its community, it houses about thirty laboratories and three specialized clinics, covering a wide range of biomedical disciplines. Its research activities are driven by around fifty scientists, including several internationally renowned figures, as well as hundreds of students and professionals from around the world.
Among its researchers, Dr. Mohan Malleshaiah, director of the Stem Cell and Cellular Reprogramming Research Unit, has developed ultra-versatile stem cells, paving the way for new innovative therapies. Specifically, they discovered that it is possible to reprogram pluripotent stem cells into totipotent stem cells. Dr. Yasaman Aghazadeh, director of the Regenerative Medicine for Diabetes Research Unit, specializes in cell therapy applied to diabetes. Her laboratory uses stem cells to study the mechanisms of type 1 diabetes and develop regenerative treatments.
McGill University Health Centre Research Institute (RI-MUHC)
The RI-MUHC is a world-renowned healthcare research center in Montreal, affiliated with McGill University. It offers a dynamic multidisciplinary research environment that fosters collaboration and leverages discoveries aimed at advancing precision health. The RI-MUHC supports various types of research, from fundamental biomedical research to applied clinical research, including healthcare systems and population health, while considering societal and cultural aspects.
The Cell Therapy Laboratory (CTL) at the RI-MUHC was created to enhance stem cell research capabilities. Located at the Centre for Innovative Medicine (CIM), the CTL collects and stores cells and tissues from healthy donors. It provides screening services, supports basic research, adapts discoveries for clinical use, and administers cells to patients participating in clinical trials.
The CTL supports various research needs, including clinical trials for Crohn’s disease, solid cancer tumors, esophageal and stomach tumors, chronic myelogenous leukemia, and several orphan diseases. It has the capacity to isolate and freeze cells from apheresis, adipose tissue, whole blood, and umbilical cord. The CTL’s biobank stores hematopoietic and mesenchymal stem cells and tissues. The CTL is approved by the MUHC Research Ethics Committee and operates in compliance with Health Canada and the Foundation for the Accreditation of Cellular Therapy (FACT).
Institute for Research in Immunology and Cancer (IRIC)
Founded in 2003 to combat cancer, IRIC stands out with an innovative research model that brings together 27 researchers with complementary expertise, a dedicated technology transfer team, and 10 state-of-the-art technological platforms. Its Drug Discovery Unit, the largest of its kind in an academic setting in the country, consists of 60 chemists and biologists specialized in medicinal chemistry.
The Institute combines fundamental research, training the next generation with unique programs in systems biology, and strategic collaborations with the clinical sector and industry. These partnerships have led to Phase I and II clinical trials with several Canadian hospitals and universities, including Jewish General Hospital, Maisonneuve-Rosemont Hospital, McMaster University Medical Centre, Laval University Health Centre, and the University of British Columbia.
IRIC positions itself as a key player in the development of cell therapies. In 2014, researchers Anne Marinier and Guy Sauvageau discovered the molecule UM171, which allows for the multiplication of stem cells derived from umbilical cord blood. This innovation has already treated over 100 patients with blood cancers. Furthermore, the institute has recently secured $3.7 million in funding for a collaborative project between the teams of Sauvageau and Marinier, who is now the CEO of RejuvenRx. Supported by the Quebec Consortium for Drug Discovery (CQDM), the Government of Quebec, RejuvenRx, the Canadian Institutes of Health Research, RejuvenRx, and the University of Montreal Partnership Fund, this project aims to optimize molecules developed at IRIC to combat cellular aging. This is a promising breakthrough for treating age-related diseases such as Alzheimer’s or Parkinson’s.
Key Players
Other Structuring Organizations
CellCAN
CellCAN is a Canadian non-profit network, established in 2014 and a member of the Networks of Centres of Excellence of the Government of Canada. Its mission is to improve the quality, safety, and feasibility of cell and gene therapies by optimizing production practices and strengthening collaborations between key players such as production centers, regulatory experts, bioengineering professionals, and ethicists.
Recognized for its major events, CellCAN organizes the biennial Strategic Forum, workshops at the International Society for Cell and Gene Therapy, Till & McCulloch meetings, and the GMP Hands-On Workshop, an immersive three-day training on cell and gene therapy production. CellCAN has also launched a National Training Institute with a solid and standardized training program.
Its main objective is to accelerate the development of stem cell-based treatments for previously incurable diseases such as cancer, diabetes, and neurological disorders, by transforming scientific discoveries into concrete clinical solutions. By facilitating dialogue between researchers, industry professionals, clinicians, patients, and government decision-makers, CellCAN actively contributes to the creation of a collaborative ecosystem capable of tackling the field’s challenges and providing solutions for patients.
Centre of Excellence in Cell Therapy (CETC)
The CETC is Canada’s largest cell therapy center. It brings together highly specialized teams from the Cell Therapy Laboratory and the C3i Centre, which manipulate, multiply, and transform human cells to treat patients across Quebec.
The CETC is Canada’s largest cell therapy center. It brings together highly specialized teams from the Cell Therapy Laboratory and the C3i Centre, which manipulate, multiply, and transform human cells to treat patients across Quebec.
CETC’s activities are carried out according to Good Manufacturing Practices (GMP), ensuring compliance with the highest production standards. It is the only center in Canada to simultaneously meet the regulations of Quebec, Canada, the United States, and Europe.
Since its opening in 2013, CETC has focused on three major missions: preparing standard cellular products used for patient treatment; manipulating cells to generate stem cells, as well as cells with anti-infectious and anti-cancer properties; and developing new treatments derived from advanced regenerative medicine research, particularly in gene therapy.
In 2020, CETC received funding from the Government of Quebec to create the first clinical-level gene therapy production unit in the country. It is also the first cell therapy center in Canada to obtain certification from the Foundation for the Accreditation of Cell Therapy (FACT, USA) for extensive cell manipulation.
Héma-Québec
Héma-Québec is a non-profit organization founded in 1998, whose mission is to efficiently meet the needs of the Quebec population for blood and other human biological products. It ensures the collection, processing, and distribution of essential blood products to hospitals in Quebec, including blood, plasma, and platelets, as well as specialized products such as breast milk, umbilical cord blood, stem cells, and human tissues. Between 2012 and 2013, Héma-Québec provided over 500,000 labile blood products and more than 340,000 stable blood products to meet patient needs.
In the field of stem cells, Héma-Québec is responsible for donor recruitment and qualification, as well as managing the Quebec Stem Cell Donor Registry. The organization coordinates the entire process, from searching for compatible donors to transplantation. With a bank of more than 10,000 umbilical cord blood units and a community of over 60,000 registered donors, Héma-Québec is well-equipped to meet the needs of transplant centers. Its registry, certified to the highest international standards, is integrated into the global network of the World Marrow Donor Association under the ION-6912 code, ensuring optimal global collaboration.
Québec Cell, Tissue, and Gene Therapy Network (ThéCell)
ThéCell’s mission is to promote translational research in cell, gene, and tissue therapy in Quebec while ensuring its international visibility.
Founded in 2009, ThéCell brings together more than 140 regular researchers, along with numerous associate members and students. It aims to facilitate Phase I and II clinical trials to make cutting-edge technologies accessible to the population. To achieve this, it highlights the technological platforms of Quebec universities and their partners. Funded by the Fonds de recherche du Québec – Santé (FRQS), ThéCell acts as a catalyst by mobilizing and coordinating access to infrastructures and highly skilled personnel in the field of cell and tissue therapy.
Its main objectives are to develop collaborative research in regenerative medicine and cell, gene, and tissue therapy. It also aims to increase and optimize the use of dedicated resources and infrastructures. Furthermore, it focuses on addressing clinical issues and facilitating the transfer of discoveries from the laboratory to clinical practice. Maintaining and developing infrastructures suited to advancements in the sector is also a priority. Finally, ThéCell is committed to fostering the training of highly qualified personnel and researchers in cell, gene, and tissue therapies.
Montreal Cancer Institute
The Montreal Cancer Institute is a foundation that has funded fundamental and clinical research for 75 years to accelerate the discovery of new treatments and conquer cancer. By supporting innovative projects and providing financial assistance to researchers and their teams, the Institute plays a vital role in advancing knowledge in oncology.
Guided by a board of directors composed of leaders from various business sectors, the Institute evolves with a commitment to its values of dedication, collaboration, integrity, excellence, and entrepreneurship.
Its mission is to promote excellence in cancer research at CRCHUM (Research Centre of the University of Montreal Hospital). To achieve this, it invests in funding scientific projects, acquiring cutting-edge equipment, providing scholarships, and organizing scientific events and specialized training.
The Institute and CRCHUM have made cell therapy a priority research focus. This approach aims to exploit immune cells found in tumors that are naturally inactive by selecting and reactivating them in a specialized CHUM laboratory. These cancer-fighting cells are amplified to billions of units before being reintroduced to the patients.
Driven by the work of Dr. Simon Turcotte and the significant support from the Institute and its partners, these efforts led to the launch of a clinical trial in 2021.
Biomedical Technology and Cell Therapy Research Laboratory
Founded in 2001 at McGill University, the Biomedical Technology and Cell Therapy Research Laboratory (BTCTRL) is part of the Faculty of Medicine and the Department of Biomedical Engineering. Globally recognized for its multidisciplinary approach, it combines fundamental research with therapeutic innovations. Its team, composed of professors and researchers with diverse expertise, forms a unique collaborative ecosystem dedicated to the health of tomorrow.
BTCTRL focuses on developing new cell- and microbiome-based therapies and designing innovative medical devices such as stents, artificial tissues for wound healing, and targeted drug delivery systems. Its research covers a broad range of areas, including the microbiome, probiotics, regenerative medicine, artificial cells, nanomedicine, and the optimization of medical devices. In recent years, the laboratory has significantly contributed to the advancement of biotherapeutic products and medical technologies in these fields, solidifying its role as an international reference.
Some figures on cell therapies in Quebec and worldwide
Over 7,000
rare diseases worldwide
500,000
Quebecers are estimated to be affected by a rare disease
100
The Quebec-based UM171 therapy, discovered in 2014 and still in clinical trials, has already been used to treat more than 100 patients with blood cancers.
2,848
gene and cell therapies are in development worldwide as of the first half of 2024.