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Canada: Therapeutic / Stem Cell

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Highly Regulated

Gene therapy allowed but requires strict oversight and monitoring.

Gene therapy is permitted, but must be approved by Health Canada’s Biologics and Genetic Therapies Directorate (BGTD), which is responsible for overseeing all clinical trials. The BGTD applies the Food and Drugs Act, which includes the Food and Drug Regulations and the Medical Devices Regulations. The BGTD requires more detailed chemistry and manufacturing information for biological drugs to be approved than other drugs for human use.

The BGTD differentiates between high risk, moderate risk and low risk biological drugs. Every lot of a high-risk drug is tested before being sold. Moderate risk biological drugs are periodically tested. Manufacturers of low risk biological drugs are only required to contact Health Canada to certify that safety testing has been completed.

The reason for the tight oversight is because under Canada’s national health program, the cost of expensive gene therapy will almost certainly be borne by the public health-care system and not private drive insurance, and will be delivered in select hospitals.

The Canadian Agency for Drugs and Technologies in Health (CADTH), the independent organization that advises the provinces and territories on whether they should cover new drugs, puts new oncology drugs, including those developed through gene therapy, through an evaluation called the pan-Canadian Oncology Drug Review, which ends with a clear recommendation for or against public funding.

There are multiple gene therapy research programs being developed, including:

Products/Research

  • Urea cycle disorder: The University of Calgary Cumming School of Medicine (CSM) and Alberta Health Services (AHS) gave the first Canadian a gene therapy treatment for urea cycle disorder, a rare genetic disease that causes ammonia to build up in the body and can lead to brain damage or death.
  • Lipoprotein lipase deficiency: The ECOGENE-21 Clinical Research Center completed a clinical trial to treat lipoprotein lipase deficiency, a genetic disease that leads to problems with the pancreas and liver and can lead to diabetes.
  • Choroideremia: The University of Alberta conducted a clinical trial for ocular gene therapy for choroideremia, which causes progressive vision loss in males.
  • Cancer: Clinical trials have been completed and the CAR T-cell therapies for patients with certain types of leukemia and non-Hodgkin’s lymphoma have been approved.

Regulatory Timeline

2011: Food and Drug Regulations amended.

1985: Canada passes the Food and Drugs Act, which includes the Food and Drug Regulations.

NGO Reaction

None

Additional Resources

Click on a country (eg. Brazil, US) or region (eg. European Union) below to find which human / health products and processes are approved or in development and their regulatory status.

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Human / Health Gene Editing Index
Compare Regulatory Restrictions Country-to-Country

Gene editing regulations worldwide are evolving. The Gene Editing Index ratings below represent the current status of gene editing regulations and will be updated as new regulations are passed.

Colors and ratings guide
 

Regulation StatusRating
Determined: No Unique Regulations*10
Lightly Regulated8
Proposed: No Unique Regulations†6
Ongoing Research, Regulations In Development5
Highly Regulated4
Mostly Prohibited2
Limited Research, No Clear Regulations1
Prohibited0
Lightly Regulated: Gene and stem cell therapies regulated with minimal restrictions and requirements.
*Determined: No Unique Regulations: Gene and stem cell therapies regulated as phamaceuticals with no additional restrictions.

†Proposed: No Unique Regulations: Decrees under consideration for gene and stem cell therapies that would not require unique regulations beyond current restrictions on pharmaceuticals.

Therapeutic:
Gene editing of adult human cells, including gene therapy and stem cell therapy, that is used to treat and cure disease. Recent breakthroughs include CAR T-cell therapy, which uses patients’ own immune cells to treat their cancer.
Germline:
Gene editing of the human embryo or germline that results in genetic changes that are passed down to the next generation. This type of gene editing is the most controversial because changes are inherited and because it could theoretically be used to create “designer babies”. A Chinese scientist announced in 2018 that he had successfully edited twins that were brought to term. International backlash from the announcement has resulted in China and other countries working to clarify regulations on germline gene editing.

Rating by Country / Region
Click each column header and arrow to sort the countries / regions

Swipe right/left if all columns aren't visible

Country / RegionTherapeuticGermlineHuman Rating
Japan888
Brazil402
Canada402
Russia1057.5
Argentina513
Israel825
Australia402
China846
US402
Chile412.5
New Zealand402
Ukraine1057.5
Central America111
Paraguay111
Uruguay111
India402
UK444
Mexico804
EU402
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Gene editing is a set of techniques that can be used to precisely modify the DNA of almost any organism. It is being used for applications in human health, gene drives and agriculture. There are numerous gene-editing tools besides CRISPR-Cas 9, which gets most of the attention because it is a comparatively easy tool to use.

Gene editing does not usually involve transgenics – moving ‘foreign’ genes between species. It also refers to a specific technique in contrast to the general term GMO, which is scientifically ambiguous, as genetic modification is a process not a product. Most gene editing involves creating new products by deleting very small segments of DNA (sometimes in agriculture called Site-Directed Nuclease 1 or SDN-1 techniques), which can silence a gene or change a gene’s activity. Countries are evaluating whether or not to regulate this type of gene editing, since it is so similar to natural mutations. The GLP’s Gene Editing Index ratings reflect the regulatory status of SDN-1 techniques, which are the most liberally regulated and will generate most products in the near term.

To develop different products, gene editing can change larger segments of DNA or add DNA from other species (a form of transgenics sometimes in agriculture called SDN-2 or SDN-3 techniques). While many countries are not regulating or lightly regulating SDN-1 techniques, most are moving toward tightly regulating or even restricting SDN-2 and SDN-3.

For more background on the various gene editing SDN techniques, read background articles here and here.

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