Gene and engineered cell therapies hold great promise for treating rare genetic disorders, as well as chronic diseases, such as cancer. However, these therapies raise a number of challenges including regulatory barriers, equity in access to treatment, and affordability.
Somatic gene therapy alters or eliminates genes that cause disease in a patient without imparting these changes to future generations. This assessment will examine the challenges related to approval and use of these therapies, focusing on the latter stages of therapy development including drug approval, market introduction, and use.
National Research Council
The CCA’s Board of Directors has approved the assessment and preliminary background research has begun. The chair and expert panel members are being identified with guidance from the CCA’s Scientific Advisory Committee and input from its Academies ― the Royal Society of Canada, the Canadian Academy of Engineering, and the Canadian Academy of Health Sciences. It is anticipated the final report will be released in 2020.