Harnessing the promises of cell therapy, gene therapy, and regenerative medicine in Québec, Canada

Jean-Sébastien Delisle^1,2,3,4*Diane Fournier⁵Kim Santerre⁴Samuel Rochette⁵Ma'n H. Zawati^4,6Maude Dumont-Lagacé^7,8*Simon Turcotte^10,4,9Jason Robert Guertin^11,12,13,4Patrick Vermette^14,15,4Jacques P. Tremblay^16,17,4Maxime Parisotto¹⁸Christian Beausejour^19,20,4Louisa Petropoulos²¹Natasha Kekre²²Stéphanie Michaud²³Mélanie Dieudé^24,25,26*

• ¹Centre de recherche Hopital Maisonneuve-Rosemont, Montreal, Quebec, Canada
• ²Institut d’hématologie, oncologie et thérapie cellulaire (iHOT), Hôpital Maisonneuve-Rosemont, Montreal, Canada
• ³Département de Médecine, Faculté de Médecine, Université de Montréal, Montreal, Quebec, Canada
• ⁴Québec Cell, Tissue, and Gene Therapy Network, Montreal, Canada
• ⁵Transfusion Medicine, Héma-Québec, Montreal, Canada
• ⁶Centre of Genomics and Policy, Department of Human Genetics, Faculty of Medicine and Health Sciences, McGill University, Montreal, Canada
• ⁷Héma-Québec, Montreal, Canada
• ⁸Borealis Medical Writing Inc, Montreal, Ontario, Canada
• ⁹Cancer Axis, Centre de recherche du Centre hospitalier de l'Université de Montréal, Montreal, Ontario, Canada
• ¹⁰Hepatopancreatobiliary Surgery & Liver Transplantation Service, Centre hospitalier de l'Université de Montréal, Montreal, Canada
• ¹¹Axe santé des populations et pratiques optimales en santé, Centre de recherche du Centre hospitalier universitaire de Québec-Université Laval, Québec, Canada
• ¹²Département de médecine sociale et préventive, Faculté de médecine, Université Laval, Québec, Canada
• ¹³Centre de recherche en organogénèse expérimentale de l'Université Laval/LOEX, Québec, Canada
• ¹⁴Department of Chemical and Biotechnological Engineering, Laboratoire de bio-ingénierie et de biophysique de l'Université de Sherbrooke, Université de Sherbrooke, Sherbrooke, Canada
• ¹⁵Faculté de médecine et des sciences de la santé, Centre de recherche du Centre hospitalier universitaire de Sherbrooke, Sherbrooke, Canada
• ¹⁶Centre de recherche du Centre hospitalier universitaire de Québec-Université Laval, Québec, Canada
• ¹⁷Département de médecine moléculaire, Université Laval, Québec, Canada
• ¹⁸adMare BioInnovations, Montreal, Canada
• ¹⁹Centre de Recherche, Centre Hospitalier Universitaire Sainte-Justine, Montréal, Canada
• ²⁰Département de pharmacologie et physiologie, Faculté de médecine, Université de Montréal, Montréal, Canada
• ²¹Centre C3i, Montréal, Canada
• ²²Division of Hematology, Department of Medicine, The Ottawa Hospital, Ottawa, Canada
• ²³BioCanRx, Ottawa, Canada
• ²⁴Medical Affairs and Innovation, Héma-Québec, Montréal, Canada
• ²⁵Axe immunopathologie, Centre de recherche du Centre hospitalier de l'Université de Montréal, Montréal, Canada
• ²⁶Département de microbiologie, infectiologie et immunologie, Faculté de médecine, Université de Montréal, Montréal, Canada

The last decade has witnessed tremendous progress in the fields of cell and gene therapy and regenerative medicine (CGT/RM). However, these advances came with their own challenges and opportunities, which may vary among jurisdictions depending on infrastructures, laws, regulations, access to funding, and socioeconomic, political and cultural aspects.In September 2023, the Québec cell, tissue and gene therapy network (ThéCell) and Héma-Québec held a symposium on the opportunities and challenges of CGT/RM in Québec, Canada. We collected and synthetized the views of scientists, CGT/RM manufacturers, business representatives, and experts in ethics and law, pharmacoeconomics, and regulatory affairs expressed during and after this symposium.Comments were grouped into seven strategic orientations: (1) a framework should be built for the development of CGT/RM products based on principles that will enable fair and timely access for all;(2) governments should spur private and public research investments in CGT/RM; (3) the skillsets of developers should be mobilized to foster the development and production of CGT/RM products in the academic and industrial settings, and the training of Québec's workforce could be better aligned with industry and population needs to facilitate the industrialization of the sector, with the aim of reducing production costs and improving accessibility to patients; (4) Québec should ensure that the province's production and healthcare capacity is aligned with current and future needs in CGT/RM products, considering the rapidly evolving landscape of CGT/RM; (5) regulatory awareness may be improved among developers through outreach approaches and early consultations; (6) the regulations governing the development of CGT/RM could be streamlined and adapted to the needs of these emerging products; (7) ongoing efforts to reform the clinical reimbursement framework could be continued in Québec's public, single-payer healthcare system. This symposium provided guidance addressing current limitations and taking advantage of opportunities in CGT/RM in Québec. These considerations should help guide the development of new policies for CGT/RM products in Québec, keeping with principles of universal healthcare in Canada. We surmise that other jurisdictions face similar challenges, and the global CGT/RM community could benefit from these shared experiences to promote the optimal development and access to these promising therapies.