Announcement
Montreal, June 19, 2025 – CQDM and the Stem Cell Network (SCN) join forces to fund a cutting-edge collaborative research project. It aims to evaluate the potential of an encapsulated liver tissue, developed by Morphocell Technologies, to treat urea cycle disorders in children. This research and development project, worth a total of $727,507, is made possible by a $261,718 grant from the Government of Quebec, awarded by CQDM, a $200,000 grant from the SCN, and a $250,000 contribution from Morphocell Technologies.
Children with urea cycle disorders are at risk of acute metabolic crises that can lead to irreversible brain damage and even death. Current treatments are limited, and liver transplantation, the only curative option, often comes too late. The project, led by Dr. Paganelli at CHU Sainte-Justine in collaboration with Quebec-based Morphocell Technologies, aims to fill this gap with ReLiver®, Morphocell’s flagship product. It is a bioengineered liver tissue composed of stem cell-derived organoids and tailored biomaterials. Designed as an off-the-shelf, allogeneic and immunosuppression-free therapy, ReLiver® rapidly replaces liver functions and clears toxins like ammonia, offering a transformative alternative to liver transplantation. This new project will explore the potential of this technology in other critical clinical contexts, such as urea cycle disorders.
“This project represents a crucial step forward for children suffering from hyperammonemic crises due to urea cycle disorders, especially newborns and infants, who often face devastating neurological outcomes,” said Dr. Claudia Raggi, Chief Scientific Officer at Morphocell Technologies. “By accelerating the development of ReLiver® for this new indication, we are building on our clinical progress in liver failure and giving these vulnerable patients a real chance to survive their first crises and reach future curative treatments such as gene therapy.”
If the results are conclusive, Morphocell Technologies plans to launch a clinical trial in four years’ time, building on the expertise acquired in an upcoming Phase I/IIa trial in acute liver failure. This innovative treatment could represent a breakthrough in pediatric regenerative medicine, offering new hope to children affected by these serious diseases, by giving them a real chance of survival and access to curative therapies in complete safety.
“We are proud to once again support Morphocell Technologies and Dr. Paganelli’s work on this innovative project, which addresses a critical medical need for which transplantation remains the only option today. Our mission is to support bold, high-risk projects that offer hope and impact, right up to the doors of clinical trials. This type of initiative is a powerful illustration of our ecosystem’s potential to offer solutions with global reach.”, affirms Véronique Dugas, President and CEO of CQDM.
The collaboration also involves Dr. Julien Baruteau, a world-leading expert in urea cycle disorders and gene therapy from University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children. Dr. Baruteau will contribute by providing advanced disease models, specialized analytics, and translational expertise, further enhancing the scientific strength of the project. His involvement underscores the global potential of ReLiver® as a novel therapeutic option for metabolic liver diseases.
“The Stem Cell Network (SCN) is pleased to collaborate with CQDM to support this innovative project led by Dr. Paganelli and his team at CHU Sainte-Justine and Morphocell Technologies. To date, SCN has invested nearly $1.4 million to advance cutting-edge research aimed at finding new therapeutic approaches to treat liver disease, and we’re excited to see how Dr. Paganelli’s team will continue to push the science forward.” explains Cate Murray, President & CEO, SCN.
“Collaborative research fuels incredible breakthroughs in cutting-edge healthcare fields such as regenerative medicine. The project announced today holds great promise, and I can’t wait to see what comes of it. Our government is proud to contribute in such a meaningful way to the advancement of science in Québec,” says Christopher Skeete, Minister for the Economy, Minister responsible for the Fight against Racism and Minister responsible for the Laval region.
This project is part of a broader investment announced on June 16 by the SCN totalling more than $33 million to support 36 regenerative medicine research projects and clinical trials across the country, 14 of which are taking place in Quebec – a powerful example of the momentum building across Canada’s life sciences ecosystem. Read the press release.
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About the Centre de recherche Azrieli du CHU Sainte-Justine
The project mentioned in this announcement was carried out at the Centre de recherche Azrieli du CHU Sainte-Justine. The Centre de recherche Azrieli du CHU Sainte-Justine is a leading mother-child health research institution affiliated with the Université de Montréal. It is an integral part of CHU Sainte-Justine, the largest mother-child health centre in Canada with its 7,054 employees, including 1,846 in nursing and cardio-respiratory care; 526 physicians, dentists and pharmacists; 200 volunteers; and 4,300 interns and students in a wide range of disciplines.
Driven by a passion for excellence, the Centre de recherche Azrieli du CHU Sainte-Justine aims to develop true precision health by leveraging the full potential of data and of the most advanced technologies. It brings together 295 research investigators, including more than 160 clinician-scientists, and over 580 graduate and postgraduate students, all focused on advancing knowledge, finding innovative means for prevention, developing faster and less invasive diagnostic methods and treatments, and optimizing patients’ care trajectories. For more information, consult our website recherche.chusj.org and follow us on LinkedIn.
About CQDM
Biopharma Innovation Facilitator
CQDM is a not-for-profit biopharmaceutical research consortium whose mission is to support and facilitate collaborative multi-stakeholder research and development aimed at accelerating the translation or transformation of innovative technologies into solutions to unmet medical needs, while generating significant benefits for the Quebec and Canadian economy. For more information, consult our website www.cqdm.org and join us on Twitter/X @CQDM_Canada and LinkedIn.
About Morphocell Technologies
Morphocell Technologies is a Montreal-based regenerative medicine company developing cell therapies and engineered tissues for the treatment of severe organ deficiencies. The company is committed to advancing groundbreaking solutions to address critical patient needs. For more information, visit morphocell.com or follow us on LinkedIn.
About the Stem Cell Network
The Stem Cell Network (SCN) is a Canadian not-for-profit that supports stem cell and regenerative medicine research; training the next generation of highly qualified personnel; enabling knowledge mobilization of research; and enhancing the commercialization readiness of stem cell and RM innovations. From the lab to the clinic, SCN’s goal is to power life-saving therapies and technologies through regenerative medicine research for the benefit of all. Created in 2001, with support from the Government of Canada, the Network has funded over 280 research projects and 30 clinical trials, cumulatively driven by experts from 350 research groups across Canada. Since its inception, over 28 biotech companies have been catalyzed or enhanced, and more than 7,900 highly qualified personnel have been trained. In 2023, the Government of Canada announced additional funding for SCN through the Strategic Science Fund that will support SCN activities and research through to the end of the decade.
For further information:
Julia Serafino
CQDM Marketing and Communications Manager
438-543-9498
[email protected]
Samantha Rae Ayoub
Vice-President, Communications & Knowledge Mobilization
Stem Cell Network
613-402-3974
[email protected]
