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GMP-Grade Manufacturing and Quality Control of a Non-Virally Engineered Advanced Therapy Medicinal Product for Personalized Treatment of Age-Related Macular Degeneration

Affiliation
Group of Experimental Ophthalmology, University of Geneva, 1205 Geneva, Switzerland
Kropp, Martina;
ORCID
0000-0003-2644-5879
Affiliation
Group of Experimental Ophthalmology, University of Geneva, 1205 Geneva, Switzerland
Harmening, Nina;
Affiliation
Group of Experimental Ophthalmology, University of Geneva, 1205 Geneva, Switzerland
Bascuas, Thais;
ORCID
0000-0003-0028-2557
Affiliation
Department of Ophthalmology, University Hospital RWTH Aachen, 52074 Aachen, Germany
Johnen, Sandra;
Affiliation
Group of Experimental Ophthalmology, University of Geneva, 1205 Geneva, Switzerland
De Clerck, Eline;
Affiliation
3P Biopharmaceuticals SL, 31110 Noain, Spain
Fernández, Verónica;
Affiliation
BVI c/o Optikon 2000 S.p.A., 00138 Rome, Italy
Ronchetti, Mattia;
Affiliation
IGEA S.p.A., 41012 Carpi, Italy
Cadossi, Ruggero;
ORCID
0000-0002-6554-2113
Affiliation
BioAir S.p.A., 20016 Pero, Italy
Zanini, Cristina;
Affiliation
CNRS, Inserm, UTCBS, Université Paris Cité, F-75006 Paris, France
Scherman, Daniel;
ORCID
0000-0002-7803-6658
Affiliation
Division of Medical Biotechnology, Paul-Ehrlich-Institute, 63225 Langen, Germany
Ivics, Zoltán;
ORCID
0000-0001-9619-5700
Affiliation
CNRS, Inserm, UTCBS, Université Paris Cité, F-75006 Paris, France
Marie, Corinne;
ORCID
0000-0002-2053-2384
Affiliation
Max Delbrück Center for Molecular Medicine in the Helmholtz Association, 13125 Berlin, Germany
Izsvák, Zsuzsanna;
Affiliation
Group of Experimental Ophthalmology, University of Geneva, 1205 Geneva, Switzerland
Thumann, Gabriele

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.

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