Validation of the first gene therapy platform in Canada


Québec is extremely well positioned to take advantage of the unique strengths of its university and hospital network to become a world leader in the emerging field of gene therapy, a treatment that applies primarily to cancer but will soon be a must for many other diseases. Gene therapy is a treatment strategy that involves the entry of genes into an individual’s cells either to replace defective cellular elements or to add functional elements. For its application in humans, the crucial step of producing viral vectors that deliver this genetic material under conditions of Good Manufacturing Practice (GMP) requires an important step of validation. The objective of the project is to validate viral production in our GMP environment in order to confirm the quality of production and thus produce a clinical and commercial level cellular material. This demonstration will be carried out under real conditions (not simulated) according to all the extremely strict rules of BPF. This involves the validation of equipment and procedures, as well as quality control testing. At the end of the project, we will be able to apply this viral vector production technology in real production conditions and so we will have confirmation that we can achieve a clinical viral vector production, which represents the ability to produce commercial level viral vectors for patients.