Bio-Production of Adeno-Associated Virus for Gene Therapy

Genetic diseases are caused by absent or defective genes. Gene therapy aims to treat these conditions by delivering a functional copy of the affected gene into a patient’s cells. Gene delivery can be performed by recombinant viruses, such as Adeno-Associated Virus (AAV). AAVs are favoured due to their safety, low pathogenicity and their ability to infect multiple tissue types. Currently three AAV-based therapies are approved for the treatment of genetic diseases.Genetic defects are usually present in every cell in a patient’s body. Therefore, gene therapy AAVs must be delivered to the majority of cells in an organ to achieve a therapeutic effect. As a result, AAV-based treatments require the administration of very high amounts of virus. Producing sufficient AAV, at an acceptable cost, without compromising safety is a major challenge in bio-production. Increasing or refining AAV production would enable the treatment of a wide variety of genetic diseases. Optimization strategies have focused on the genetic components needed for AAV assembly, the production cell lines, as well as omics-based approaches.