(Attachment) Draft: Evaluation Items for Quality and Safety
Information on the origin and history of the Master Virus Bank (MVB), the culturing methods of the MVB and the Working Virus Bank (WVB), media and reagents used for manufacturing, results of microbiological tests (e.g., sterility tests, mycoplasma exclusion tests), in vivo and in vitro tests for infectious agents such as viruses, tests to confirm the absence or limitation of replication-competent viruses, endotoxin and impurity tests, structural analyses of gene therapy vectors, as well as data and information on storage and management methods should be clearly documented.
For gene therapy vectors or genetically modified cells to be used in clinical research, it is necessary to conduct appropriate final product testing. These product tests should include:
Infectious agent tests (e.g., sterility tests, mycoplasma exclusion tests, adventitious virus tests) from the perspective of ensuring safety;
Purity tests (e.g., for endotoxins and process-related impurities);
Tests to evaluate the characteristics of the vectors;
In some cases, additional analyses such as:
Viability assays for genetically modified cells;
Biological activity assays;
Viral titer assays.
Depending on the circumstances, purity analysis of the cells may also be required, and such tests should be considered as part of the quality control strategy.
When setting up the tests, it is necessary to define limits, ranges, or other specifications. However, these specifications are expected to evolve alongside the progress of clinical research, and provisional values may be established at the start of clinical trials. Likewise, it is desirable that test items themselves be re-evaluated and refined as the clinical research progresses.
Furthermore, if the final product consists of genetically modified cells, the characteristics and quality test results of the viral or non-viral vectors used in the cell preparation must also be clarified.