Chapter 18. The Transfer of Technology from the Laboratory to the Clinic: In Process Controls and Final Product Testing

Summary

The development of novel gene delivery systems and applications in gene therapy is raising new regulatory issues. In the USA, production and safety testing are regulated by the Food and Drug Administration. Gene therapy products are characterized with regard to safety, potency, identity, purity and stability. This includes definition of the reagents used for production as well as control of key intermediates, such as cell banks. As a product moves from early development towards and through clinical trials, safety issues and regulatory requirements for product characterization may vary: regulatory agencies in the US sponsor a gradual approach to allow for continuous refinement. The establishment of master and working banks for cells, viruses and plasmids represents part of the transition from research to a manufacturing program. Clinical products undergo stringent testing, preclinical toxicology and pharmacokinetic studies but ultimately only the widespread application for human use will provide an accurate safety assessment.