Cell & Gene Therapy in Canada

A pivotal moment
Canada has cultivated a system of world-class researchers who have conducted important clinical trials and generated landmark scientific results in cell and gene therapies.
Now we’re at a pivotal moment. There are several cell and gene therapies approved for use in Canada, but also thousands more in the pipeline.7
These therapies are already having a real, transformative impact on patient outcomes, including significant improvements in quality of life and, in some cases, potentially curing previously incurable diseases and disorders, including many rare diseases and disorders.8
One example of this is CAR T-cell therapies for certain blood cancers. These treatments involve extracting a patient’s T-cells (immune cells), then modifying them to target cancer cells before returning them to the patient’s body. CAR T-cell therapies have been shown to lead to more rapid recovery and long-term survival, all with a shorter treatment period than aggressive chemotherapy – outcomes that have been rare for these cancers in the past.9
Another example is gene therapy for retinal disease. An innovative treatment designed to restore vision in patients with inherited retinal conditions, this therapy delivers a healthy copy of the gene directly into the retina through a single injection, targeting the faulty genes at the cellular level. The therapy uses a viral vector – a specially modified virus used to deliver a working copy of a gene – to carry the gene into the retinal cells, enabling them to function properly and prevent further vision loss and, in some cases, even improve sight.10
What’s Next for Cell & Gene
Therapies in Canada?
The Canadian healthcare system must be ready to meet the demands of rapidly advancing cell and gene therapies. Navigating this new frontier in health technology requires a commitment to delivering these treatments in a manner that prioritizes timely and equitable access.
This is especially important for equity-seeking and geographically isolated groups who have historically lacked access to adequate care and, as a result, face poorer health outcomes.11
Canada should work towards establishing an environment that offers the necessary funding and regulatory mechanisms, health system capacity, and clinical expertise to provide access to this new generation of health therapies.
The unique and novel nature of these therapies demands new kinds of collaboration across sectors, specialties, and disease areas. It will take new approaches to our health systems, and budgets, payer reimbursement models, and patient and caregiver education.
For example, reimbursement models can consider the potentially significant improvements to quality and duration of life offered by cell and gene therapies. These therapies are often for rare diseases and disorders and those that currently lack effective treatments, as well as offering potential long-term savings despite high initial costs.12
By having these conversations and acting now, we can all benefit from this exciting and profound evolution in medical treatment.