NEWS
Montreal, June 19, 2025 – Morphocell Technologies, a pioneering regenerative medicine company, is proud to announce its participation in a newly funded, multi-institutional research initiative aimed at transforming the treatment landscape for children suffering from urea cycle disorders (UCDs).
Co-sponsored by CQDM and the Stem Cell Network (SCN), the $727,507 project will be led at the CHU Sainte-Justine, in Montreal, a leading center for pediatric research, and will evaluate the application of Morphocell’s lead therapeutic, ReLiver®, in this devastating condition.
Children born with urea cycle disorders face life-threatening metabolic crises that can cause irreversible neurological damage or death. Current treatments remain insufficient, with liver transplantation as the only curative option—often available too late. This collaboration seeks to explore a novel alternative: ReLiver®, Morphocell’s off-the-shelf, stem cell-derived liver tissue designed to restore key liver functions and rapidly clear toxic metabolites without the need for immunosuppression.
“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. “By starting the development of ReLiver® for this new indication, we are building on our progress in liver failure to give these vulnerable patients a better chance to survive their first crises and reach future curative treatments such as gene therapy.”
The research, made possible by a $261,718 contribution from the Government of Quebec through CQDM (read press release), a $200,000 grant from the SCN, and $250,000 from Morphocell Technologies, brings together a world-class team. Alongside the Sainte-Justine-based research group, the project will benefit from the collaboration with Dr. Julien Baruteau of University College London and Great Ormond Street Hospital, in the United Kingdom, a leading global figure in metabolic liver disease, UCDs and gene therapy.
The study builds on Morphocell’s growing clinical momentum, with a Phase I/IIa trial of ReLiver® for acute liver failure set to begin shortly. If results from the new UCD-focused project are positive, Morphocell anticipates launching a dedicated clinical program within the next four years.
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This initiative is part of a broader $33 million national investment announced by the Stem Cell Network to advance regenerative medicine research across Canada (read the press release), reinforcing the country’s leadership in the development of next-generation therapeutic technologies.
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. Its flagship product, ReLiver®, is designed as an allogeneic, immunosuppression-free solution for acute liver failure and other liver-related conditions. The company is committed to transforming patient care by offering innovative, accessible alternatives to organ transplantation.
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.
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About CQDM
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 cqdm.org/ and join us on LinkedIn.
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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.
Media Contact:
Zaynah Ibrahim
Operations Manager
Phone: + 1 (514) 373-6444 ext. 106
Email: z.ibrahim@morphocell.com