Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method.

Autor: Merlo-Mas J; Nanomol Technologies S.L., 08193 Cerdanyola del Vallès, Spain., Tomsen-Melero J; Nanomol Technologies S.L., 08193 Cerdanyola del Vallès, Spain.; Institut de Ciència de Materials de Barcelona, ICMAB-CSIC, 08193 Bellaterra, Spain.; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain., Corchero JL; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Institut de Biotecnologia i Biomedicina (IBB-UAB), 08193 Cerdanyola del Vallès, Spain., González-Mira E; Institut de Ciència de Materials de Barcelona, ICMAB-CSIC, 08193 Bellaterra, Spain.; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain., Font A; Leanbio S.L., 08028 Barcelona, Spain., Pedersen JN; Department of Chemistry and Interdisciplinary Nanoscience Center (iNANO), Aarhus University, 8000, Aarhus, Aarhus C Denmark., García-Aranda N; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Functional Validation and Preclinical Research, Drug Delivery & Targeting, CIBBIM-Nanomedicina, Vall d'Hebron Institute of Research (VHIR), Universitat Autònoma de Barcelona (UAB), 08035 Barcelona, Spain., Cristóbal-Lecina E; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Institut de Química Avançada de Catalunya (IQAC-CSIC), 08034 Barcelona, Spain., Alcaina-Hernando M; Nanomol Technologies S.L., 08193 Cerdanyola del Vallès, Spain., Mendoza R; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Institut de Biotecnologia i Biomedicina (IBB-UAB), 08193 Cerdanyola del Vallès, Spain., Garcia-Fruitós E; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Institut de Biotecnologia i Biomedicina (IBB-UAB), 08193 Cerdanyola del Vallès, Spain., Lizarraga T; Leanbio S.L., 08028 Barcelona, Spain., Resch S; BioNanoNet Forschungsgesellschaft mbH, 8010 Graz, Austria., Schimpel C; BioNanoNet Forschungsgesellschaft mbH, 8010 Graz, Austria., Falk A; BioNanoNet Forschungsgesellschaft mbH, 8010 Graz, Austria., Pulido D; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Institut de Química Avançada de Catalunya (IQAC-CSIC), 08034 Barcelona, Spain., Royo M; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Institut de Química Avançada de Catalunya (IQAC-CSIC), 08034 Barcelona, Spain., Schwartz S Jr; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Functional Validation and Preclinical Research, Drug Delivery & Targeting, CIBBIM-Nanomedicina, Vall d'Hebron Institute of Research (VHIR), Universitat Autònoma de Barcelona (UAB), 08035 Barcelona, Spain., Abasolo I; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain.; Functional Validation and Preclinical Research, Drug Delivery & Targeting, CIBBIM-Nanomedicina, Vall d'Hebron Institute of Research (VHIR), Universitat Autònoma de Barcelona (UAB), 08035 Barcelona, Spain., Pedersen JS; Department of Chemistry and Interdisciplinary Nanoscience Center (iNANO), Aarhus University, 8000, Aarhus, Aarhus C Denmark., Danino D; CryoEM Laboratory of Soft Matter, Faculty of Biotechnology and Food Engineering, Technion - Israel Institute of Technology, 32000 Haifa, Israel., Soldevila A; Leanbio S.L., 08028 Barcelona, Spain., Veciana J; Institut de Ciència de Materials de Barcelona, ICMAB-CSIC, 08193 Bellaterra, Spain.; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain., Sala S; Nanomol Technologies S.L., 08193 Cerdanyola del Vallès, Spain., Ventosa N; Institut de Ciència de Materials de Barcelona, ICMAB-CSIC, 08193 Bellaterra, Spain.; Centro de Investigación Biomédica en Red - Bioingeniería, Biomateriales y Nanomedicina (CIBER-BBN), 28029 Madrid, Spain., Córdoba A; Nanomol Technologies S.L., 08193 Cerdanyola del Vallès, Spain.
Jazyk: angličtina
Zdroj: The Journal of supercritical fluids [J Supercrit Fluids] 2021 Jul; Vol. 173, pp. 105204.
DOI: 10.1016/j.supflu.2021.105204
Abstrakt: Fabry disease is a lysosomal storage disease arising from a deficiency of the enzyme α-galactosidase A (GLA). The enzyme deficiency results in an accumulation of glycolipids, which over time, leads to cardiovascular, cerebrovascular, and renal disease, ultimately leading to death in the fourth or fifth decade of life. Currently, lysosomal storage disorders are treated by enzyme replacement therapy (ERT) through the direct administration of the missing enzyme to the patients. In view of their advantages as drug delivery systems, liposomes are increasingly being researched and utilized in the pharmaceutical, food and cosmetic industries, but one of the main barriers to market is their scalability. Depressurization of an Expanded Liquid Organic Solution into aqueous solution (DELOS-susp) is a compressed fluid-based method that allows the reproducible and scalable production of nanovesicular systems with remarkable physicochemical characteristics, in terms of homogeneity, morphology, and particle size. The objective of this work was to optimize and reach a suitable formulation for in vivo preclinical studies by implementing a Quality by Design (QbD) approach, a methodology recommended by the FDA and the EMA to develop robust drug manufacturing and control methods, to the preparation of α-galactosidase-loaded nanoliposomes (nanoGLA) for the treatment of Fabry disease. Through a risk analysis and a Design of Experiments (DoE), we obtained the Design Space in which GLA concentration and lipid concentration were found as critical parameters for achieving a stable nanoformulation. This Design Space allowed the optimization of the process to produce a nanoformulation suitable for in vivo preclinical testing.
Competing Interests: The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: J.L-C., D.P., S.Sch., M.R., I.A., J.V., S.Sa. and N.V. are inventors of patent WO/2014/001509 licensed to Biopraxis Resarch AIE. J.V., S.Sa. and N.V. are inventors of patent WO/2006/079889 owned by Nanomol Technologies SL, and stock-owners in Nanomol Technologies SL. J.M-M., J.T-M., A.F., E.G-M., J-L.C, E.C-L., D.P., M.R., S.Sch., I.A., A.S., S.Sa., J.V., N.V. and A.C. are inventors of patent application EP21382062.4.
(© 2021 The Authors.)
Databáze: MEDLINE
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