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| Item Type: | Article |
|---|---|
| Title: | GMP-grade manufacturing and quality control of a non-virally engineered advanced therapy medicinal product for personalized treatment of age-related macular degeneration |
| Creators Name: | Kropp, M. and Harmening, N. and Bascuas, T. and Johnen, S. and De Clerck, E. and Fernández, V. and Ronchetti, M. and Cadossi, R. and Zanini, C. and Scherman, D. and Ivics, Z. and Marie, C. and Izsvák, Z. and Thumann, G. |
| Abstract: | The introduction of new therapeutics requires validation of Good Manufacturing Practice (GMP)-grade manufacturing including suitable quality controls. This is challenging for Advanced Therapy Medicinal Products (ATMP) with personalized batches. We have developed a person-alized, cell-based gene therapy to treat age-related macular degeneration and established a vali-dation strategy of the GMP-grade manufacture for the ATMP; manufacturing and quality control were challenging due to a low cell number, batch-to-batch variability and short production duration. Instead of patient iris pigment epithelial cells, human donor tissue was used to produce the transfected cell product ("tIPE"). We implemented an extended validation of 104 tIPE productions. Procedure, operators and devices have been validated and qualified by determining cell number, viability, extracellular DNA, sterility, duration, temperature and volume. Transfected autologous cells were transplanted to rabbits verifying feasibility of the treatment. A container has been engineered to ensure a safe transport from the production to the surgery site. Criteria for successful validation and qualification were based on tIPE's Critical Quality Attributes and Process Parameters, its manufacture and release criteria. The validated process and qualified operators are essential to bring the ATMP into clinic and offer a general strategy for the transfer to other manufacture centers and personalized ATMPs. |
| Keywords: | Personalized Medicine, Cell Therapy, Advanced Therapy Medicinal Product (ATMP), Good Manufacturing Practice (GMP), Quality Control, Non-Viral Gene Therapy, Age-Related Macular Degeneration (AMD), Iris Pigment Epithelial Cells (IPE), Animals, Rabbits |
| Source: | Biomedicines |
| ISSN: | 2227-9059 |
| Publisher: | MDPI |
| Volume: | 10 |
| Number: | 11 |
| Page Range: | 2777 |
| Date: | November 2022 |
| Official Publication: | https://doi.org/10.3390/biomedicines10112777 |
| PubMed: | View item in PubMed |
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