Canadian Rare Disease Symposium: Preparing for Clinical Trials – International Regulatory Insights, and cGMP Gene Therapy Manufacturing Capabilities in Canada

Start Date: October 1, 2025
Time: 1:00 – 4:00 PM ET
Location: Virtual (Zoom)

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The Stem Cell Network (SCN) and the National Research Council Canada (NRC) are excited to contribute to the national conversation on cell and gene therapy for rare diseases. Building on the 2024 event titled “Canadian Rare Disease Ecosystem Symposium: Genetic Therapies for Rare Disease”, SCN and NRC are pleased to invite you to the Canadian Rare Disease Symposium: Preparing for Clinical Trials – International Regulatory Insights, and cGMP Gene Therapy Manufacturing Capabilities in Canada–a focused half-day event dedicated to advancing gene and cell therapy development in Canada through regulatory alignment and manufacturing readiness.

This virtual symposium will convene international regulatory authorities, Canadian manufacturing experts, and rare disease developers to explore two critical enablers of translational success: clinical trial readiness and cGMP manufacturing capacity.

What to Expect:

Session 1: Preparing for Clinical Trials – International Regulatory Insights for Canadian Developers

• Hear directly from national and international regulators (Japan, Europe, Canada) on how their jurisdictions are supporting the development and approval of rare disease therapies, including evolving policies, expedited pathways, and opportunities for cross-border collaboration. This session will also feature the global experience of a regulatory strategist who will highlight similarities and differences across regions.

Session 2: Overview of Canadian Manufacturing

• Gain a clear view of Canada’s cGMP manufacturing landscape, with flash presentations from companies and national labs producing cGMP vectors for gene therapy. This session will highlight current capabilities, and the role of these facilities in supporting the development and commercialization of gene and cell therapies.

Whether you’re an scientific researcher, biotech innovator, or policymaker, this symposium will offer practical insights and strategic context for navigating the rare disease therapy landscape in Canada and beyond.

Who Should Attend?

The event is primarily targeted to academics, clinicians and industry representatives; and government researchers and funding and regulatory agencies who work in the field of cell and gene therapies for rare diseases, as well as their trainees (post-docs, graduate students, research associates, or technicians).

Symposium Learning Objectives:

By the end of the symposium, participants will be able to:

• Describe key regulatory expectations and considerations for advancing gene and cell therapies into clinical trials, for rare diseases.
• Compare international regulatory approaches to identify opportunities for early engagement and understand strategies for global clinical trial deployment of rare disease therapies.
• Understand the current capabilities and future potential of Canada’s GMP manufacturing infrastructure for gene and cell therapies.

Speakers:

Session 1: 

• Yoshiaki Maruyama, Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency, Japan
• Omar Tounekti, Manager, Gene Therapies and Radiopharmaceuticals Division, Health Canada, Canada
• Ilona Reischl, Quality Assessor, Austrian Medicines and Medical Devices Agency; Chair, European Medicines Agency Committee for Advanced Therapies, Austria
• Sarmitha Sathiamoorthy, President & Principal Consultant, AspireBio Consulting, Canada
• Moderated by Patrick Bedford, Founder & Managing Director, weCANreg Consulting Group

Session 2:

• Jessica Madigan, Director, Business Development, BIOVECTRA
• Minh-Luan Tran, Director, Clinical Trial Material Facility, Human Health Therapeutics Research Centre, National Research Council Canada
• Jennifer Quizi, Director, Biotherapeutics Manufacturing Centre- Virus Manufacturing Facility, Ottawa Hospital Research Institute
• Lisa Dreolini, Research Assistant, BC Cancer
• Jessica Wilson, Production Coordinator, Quality Management Systems, Canada’s Michael Smith Genome Sciences Centre
• Dennis-Claude Roy, Chief Scientific Officer, C3i
• Jessica Tate, Vector Platform Technologies, CCRM
• Moderated by Kelley Parato, Program Director, Health Challenge Program National Research Council Canada

For further information on this symposium or for application related enquiries, please email researchSCN@stemcellnetwork.ca.