Recombinant adeno-associated viruses (rAAVs) are the safest and most effective gene delivery vehicles to drive long-term therapeutic gene expression in gene therapy. To date, generating vector quantities sufficient to meet the expanding clinical demand is still a hurdle when using current production systems.
The most common method of producing rAAVs is transient transfection of plasmid DNA in HEK293 cells. The main limitations to the current rAAVs production methods are their lack of scalability, high production costs, varying quality, and low predictability and reproducibility.
Currently developed NewBiologix technologies consist of stably engineered HEK that have integrated all genes necessary for rAAV production, with the following characteristics:
- Growth in suspension to high cell density
- Inducible gene expression to overcome the cellular toxicities of certain viral proteins
- Integration of viral vector genes in predefined genomic loci allowing for stable, safe and high rAAV production
- Production platform allowing different capsid proteins to optimize the vector serotype
These engineered HEK cell lines will be readily adaptable to meet the viral vector demands and requirements over a full range of therapeutic products and diseases.