The Canadian Regulatory System For Cell And Gene Therapies

Regenerative medicine is bringing about an entirely novel therapeutic approach to treating human diseases, many of which currently lack efficacious therapies. An increasing number of cell and gene therapy (CGT) products are approved by regulators around the world, including several therapies approved by Health Canada in recent years, as follows:

• Gilead’s Tecartus for mantel cell lymphoma (approved in 2021);
• Celgene’s Abecma for multiple myeloma (2021);
• Novartis’ Zolgensma for spinal muscular atrophy (2020);
• Spark Therapeutics’ Luxturna for a rare form of inherited vision loss (2020); and,
• Gilead’s Yescarta (2019) and Novartis’ Kymriah (2018), both CAR T cell-based gene therapies for the treatment of blood cancers.

In addition to these approvals, many CGT candidates are currently being evaluated in clinical trials in Canada.

Review how CGT products are regulated in Canada, the regulatory framework and environment, and how to navigate the Canadian regulatory system.