Manufacturing and QC

The latest advances and successes of gene therapy as evidenced by the conditional approval of uniQure’s adeno-associated vectors (AAV) for the treatment of Lipoprotein Lipase Deficiency (Glybera®) as well as projects for the treatment of patients affected by neuromuscular disorders such as Duchenne Muscular Dystrophy require the production of large quantities of viral vectors to satisfy the needs of the market but also the requirements for clinical trials using locoregional and systemic administration. Ideally, the production of large quantities of vectors takes place in bioreactors which allow easy scale up to the production scale. Although this is possible for AAV and adenovirus vectors because the bioreactor processes have been developed, it is not yet the case for lentiviral vectors which are still produced at a small scale by transfection of producer cells with plasmids.

Except for this specific problem, manufacturing processes for clinical batches of vectors are subject to the same quality specifications and are carried out under a GMP (Good Manufacturing Processes) regime. Vector batches are produced by using cell cultures, the vectors are harvested and purified, and the final products are rigorously characterized by performing dozens of quality control tests in order to demonstrate safety and the absence of potential contaminants.

Even if the primary goal of this preindustrial consortium is to develop platforms for production and testing of viral vectors for gene therapy, new and more efficient methods for production, purification and quality control will be developed in the framework of this project.