https://diyhpl.us/~bryan/papers2/gene-therapy/Adeno-associated%20virus%20serotypes%20-%20vector%20toolkit%20for%20human%20gene%20therapy%20-%202006.pdf

Adeno-associated virus serotypes: vector toolkit for human gene therapy.

Adeno-associated viral (AAV) manufacturing at scale continues to hinder the application of AAV technology to gene therapy studies. Although scalable systems based on AAV–adenovirus, AAV–herpesvirus, and AAV–baculovirus hybrids hold promise for clinical applications, they require time-consuming generation of reagents and are not highly suited to intermediate-scale preclinical studies in large animals, in which several combinations of serotype and genome may need to be tested. We observed that during production of many AAV serotypes, large amounts of vector are found in the culture supernatant, a relatively pure source of vector in comparison with cell-derived material. Here we describe a high-yielding, recombinant AAV production process based on polyethylenimine (PEI)-mediated transfection of HEK293 cells and iodixanol gradient centrifugation of concentrated culture supernatant. The entire process can be completed in 1 week and the steps involved are universal for a number of different AAV serotyp...

Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale

A Simplified Baculovirus-AAV Expression Vector System Coupled With One-step Affinity Purification Yields High-titer rAAV Stocks From Insect Cells

Viral vectors and viral vaccines more and more play an important role in current medical approaches. Gene vectors like adenoviruses, adeno-associated viruses or retroviruses are the vehicles being developed for delivering genetic material to the target cell in gene therapy. Viral vaccines, like attenuated or inactivated rabies virus, influenza virus or hepatitis virus vaccines, are powerful tools to limit the number of serious viral infections and pandemics. Higher safety demands, that is, reduction of side effects, by regulatory authorities like Food and Drug Administration (FDA) and European Agency for the Evaluation of Medicinal Products (EMEA), nowadays force developers as well as manufacturers to improve their production and purification processes for viral vectors and vaccines. Like for influenza viral vaccines, manufacturers begin to switch from egg cultivation to mammalian cell culture systems. Also within the purification procedure, a clear trend from classical purification methods like sucrose gradient centrifugation towards more sophisticated techniques like tangential flow filtration and liquid chromatography can be observed.

Downstream processing of viral vectors and vaccines.

Recombinant adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great promise for human gene therapy. A major challenge for translation of promising research to clinical development is the manufacture and certification of AAV vectors for clinical use. This review summarizes relevant aspects of current Good Manufacturing Practice, focusing on considerations and challenges specific for recombinant AAV.

Manufacturing and characterizing AAV-based vectors for use in clinical studies.

A versatile and scalable two-step ion-exchange chromatography process for the purification of recombinant adeno-associated virus serotypes-2 and -5.

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A versatile and scalable two-step ion-exchange chromatography process for the purification of recombinant adeno-associated virus serotypes-2 and -5.