As cell and gene therapies and other advanced therapeutics develop rapidly around the world, Canada faces an urgent question: how can we ensure promising Canadian innovations move from lab to clinic to market — and to Canadian patients — faster, more predictably, and more strategically?
In June 2026, the Stem Cell Network convened nearly 50 leaders from across Canada’s regenerative medicine ecosystem for a two-day workshop, Strengthening Canada’s Innovation Ecosystem for Cell and Gene Therapies for Small Populations. This was the second workshop in SCN’s Access, Affordability, and Adoption series, building on a successful workshop in June 2025. Participants included researchers, clinicians, patient partners, industry, manufacturers, regulators, health technology assessment and reimbursement organizations, health charities, non-profit organizations, and federal policy leaders.
The full report will be released later this year. In advance of that report, several high-level takeaways have emerged.
1. Canada needs to do things differently
Canada has strong science, clinical expertise, emerging infrastructure, a skilled talent base and a publicly funded health system committed to equitable access.
What Canada lacks is a coordinated pathway.
Today, advanced therapies often move through a linear sequence: research, translation, regulation, evidence generation, manufacturing, health technology assessment, pricing, reimbursement, procurement, and clinical delivery. For many advanced therapies — especially those for small patient populations — this model is too slow, too fragmented and too uncertain.
Workshop participants consistently pointed to the need for a more coordinated approach that connects these functions earlier and more deliberately. Without that shift, promising Canadian innovations may move offshore, public investments may not translate into domestic economic benefit, and patients may wait too long for therapies developed from Canadian science.
2. Patient access must remain the starting point
The workshop opened with a patient and caregiver perspective from Charmain Brown, whose family’s experience accessing a gene therapy helped ground the discussion in what is truly at stake.
For patients and families, access is shaped by diagnosis, geography, affordability, clinical readiness, navigation, family burden, and time. Delays are not just system inefficiencies; they are lost opportunities and life years. Advanced therapy pathways must be designed around the full journey required to deliver therapies safely, equitably, and sustainably.
3. Small-population therapies challenge the current system
The workshop tested Canada’s current system through three Canadian case studies:
- a gene therapy being developed in Ottawa for an ultra-rare, fatal neonatal lung disease;
- a patient-specific tissue-engineered skin substitute developed in Laval for severe burns; and
- an antisense oligonucleotide (ASO) therapy developed by a non-profit for a rare paediatric neurodevelopmental disorder being tested in an N-of-1 clinical trial at the Hospital for Sick Children in Toronto.
Although the therapies differed, they revealed a common challenge: small-population regenerative medicines often do not fit the standard path from academic discovery to commercial sponsorship, large clinical trials, market authorization, health technology assessment, price negotiation, provincial reimbursement and routine clinical delivery.
Some therapies may be highly specialized, patient-specific, hospital-linked, academically developed, or intended for very small numbers of patients, and may not have a conventional commercial pathway, even when the patient need is significant.
4. Canada needs to activate existing tools and build missing supports
A central theme from the workshop was the need for a prioritized Made-in-Canada pathway for Canadian-developed advanced therapeutics. This would help high-priority Canadian innovations access coordinated advice and support earlier, including regulatory guidance, manufacturing expertise, translational funding, clinical trial support, streamlined research ethics review, health technology assessment planning, commercialization assistance, and early adoption planning.
Participants also heard from an international expert regulatory panel, which highlighted progress in other jurisdictions, including more coordinated regulatory pathways, earlier engagement with innovators, flexible evidence approaches, and models that better connect regulation, assessment, reimbursement and adoption. Canada can learn from these approaches while adapting them to its own publicly funded health system and federal-provincial context.
Participants identified the Advanced Therapeutic Products (ATP) pathway as a key near-term tool for cell and gene therapies that do not fit neatly into existing drug or device frameworks. However, the pathway has not yet been activated in a practical, precedent-setting way for these therapies.
Canada should also revisit previous rare disease policy work and the current National Strategy for Drugs for Rare Diseases to support a formal orphan/ultra-rare designation for advanced therapeutics serving small patient populations.
5. Innovators need navigation, de-risking, and manufacturing support
Academic innovators and early-stage Canadian biotechs are often expected to navigate regulatory strategy, manufacturing requirements, evidence expectations, intellectual property, commercialization, health technology assessment, payer expectations and clinical adoption without the infrastructure of a large industrial sponsor.
Participants identified the need for a not-for-profit concierge or navigation function to help innovators ask the right questions earlier, connect to the right expertise, and design with the end in mind. This support should complement, not replace, Health Canada, CDA-AMC/INESSS or payer decision-making.
Manufacturing was also identified as a recurring barrier. Canada has invested in biomanufacturing infrastructure, but facilities and equipment alone will not create national capability. Advanced therapies require early manufacturing planning, quality systems, Good Manufacturing Practices (GMP) readiness, technology transfer, specialized workforce capacity, and clinical trial batch production.
For rare, ultra-rare and patient-specific therapies, demand can be unpredictable. Canada needs approaches that stabilize and de-risk manufacturing capacity so specialized facilities, staff and quality systems are available when patients need them.
6. Evidence and access models must fit small populations
Evidence expectations must remain rigorous, but they also need to be reasonable. Large traditional clinical trials may not be feasible or affordable for ultra-rare, small-population or N-of-1 therapies.
Canada needs proportionate evidence frameworks that make appropriate use of natural history data, registries, real-world evidence, common outcome measures, patient- and caregiver-reported outcomes, long-term safety monitoring and reassessment over time.
At the same time, approval alone does not equal access. Canada needs national adoption, reimbursement, stewardship and full care-pathway funding models that account for diagnostics, administration, hospital readiness, monitoring, travel, data infrastructure, long-term follow-up, and cross-provincial access — so therapies are not approved but unfunded, funded but unavailable, or accessible only through ad hoc workarounds.
Looking ahead
The workshop made clear that Canada has the science, talent and infrastructure to lead in regenerative medicine, but a more coordinated pathway is needed to translate that strength into patient access, health-system readiness and economic benefit.
The full workshop report, expected later this year, will provide a more detailed synthesis of the discussions and identify practical opportunities for action. Early themes include the need to better align regulatory, evidence, manufacturing, reimbursement and adoption pathways; support innovators earlier; strengthen manufacturing readiness; and develop access models that reflect the realities of small patient populations.
Canada helped build the field of regenerative medicine. Now it must build the pathway to deliver it.
