Quality evaluation of gene therapy products
We provide a wide range of quality evaluation services for ex vivo gene therapy products, including CAR-T therapy ones.
Features
We can perform analytical method development and method validation, in compliance with pharmaceutical regulations, such as GMP and the Japan’s Reliability Criteria *1.
Potency testing with cell cultures are also available.
- *1: Article 43 of the Regulation for Enforcement of the Act on Securing Quality, Efficacy and Safety of Products Including Pharmaceuticals and Medical Devices
Test items
| Evaluation items | Test items | Analytical techniques |
|---|---|---|
| General tests | Osmolarity, pH, Sampling volume, Insoluble particulate matter, etc. | pH analysis, Sampling volume, etc. |
| Content | Cell count, cell viability, Number of cells with the desired function | Cytometry (Hemocytometer/Automated cytometry apparatus), Flow cytometry |
| Identity | Appearance/Description, Target cell markers, Cytokine production capacity, Transgene confirmation, Vector persistence, etc. | Visual observations, Cellular immunostaining, Flow cytometry, quantitative PCR (qPCR), Droplet Digital PCR (ddPCR), ELISA, Electrophoresis |
| Purity | Off-target cells, etc. | Cell immunostaining, Flow cytometry, qPCR |
| Process-related impurities | Process-related impurities (Serum albumin, Antibiotics, Raw material-derived vectors, etc.) | ELISA, LC/MS, GC/MS, ICP-MS, qPCR, ddPCR, etc. |
| Safety | Mycoplasma, Endotoxin, Sterility, etc. | Microbiological tests, Rapid sterility test |
| Virus | qPCR | |
| Proliferative virus | qPCR | |
| Chromosomal aberrations | PCR | |
| Karyotype Analysis (G-band) | ||
| Biological activity and potency | Setting for each product (e.g., protein expression, secretory capacity of bioactive substances, cell phenotype, cell proliferation capacity) | ELISA, cellular assays, cellular immunostaining, morphological observations, flow cytometry, etc. |
| Characterization | Localization and differentiation studies of cells | Immunostaining (fluorescent microscopy) using organization sections. |
| Study of cell differentiation and evaluation of storage stability | Live-cell image analysis | |
| Confirmation of transgene sequence and vector sequence | DNA sequence identification (Sanger sequencer) |
Instruments
(ChemoMetec NC-3000™)
(Bio-Rad QX200)
(Applied Biosystems™
SeqStudio™ Genetic Analyzer)
Precautions regarding test samples
Please provide us with the following information in advance.
If samples should be handled under Biosafety level (BSL) 3 or higher or if the safety of samples cannot be ensured, we may not accept them.
- Information on infectivity of samples and pathogenic micro-organisms (origin, BSL, the methods that shows non-pathogenicity, status of inactivation, etc.)
- Whether β-lactam antibiotics are used or not
- Whether it is a genetically modified organism or not
Points to consider in providing samples derived from human cultured cells
When sending samples obtained from the human body to us, please review and get approval from your ethical review committee, in accordance with the "Ethical Guidelines for Medical and Biological Research Involving Human Subjects", and take necessary measures required by this guideline.
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