Standardisation of Gene Therapy Products: Practical Considerations and Perspectives

by Yuan Zhao, PhD and Robin Thorpe, PhD
Volume 3, Issue 6 (November/December 2004)

A prerequisite for producing medicinal products is ensuring their quality and safety. This requires appropriately controlled and standardised manufacture and testing procedures that result in consistent potency, safety, and efficacy. Assuring the quality and safety of gene therapy products in particular presents a great challenge because they are cell-based, multi-gene products which include viral and therapeutic proteins as well as modified cells. Although more than 860 gene transfer clinical trials are in progress and the first gene therapy product is already on the market (in China), the development of reference materials for gene therapy products is at an early stage with only a few accessible reference materials. Standardisation of gene therapy products to ensure their quality and safety is clearly necessary and has become increasingly important. Standardisation and other issues specifically related to gene therapy products are discussed in this article…

Zhao Y, Thorpe R. Standardisation of Gene Therapy Products: Practical Considerations and Perspectives. BioProcess J, 2004; 3(6): 21-25.