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

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  • Josep Merlo-Mas, NANOMOL TECHNOLOGIES SL
  • ,
  • Judit Tomsen-Melero, NANOMOL TECHNOLOGIES SL, CSIC - Institute of Materials Science of Barcelona, CIBER - Center for Biomedical Research Network
  • ,
  • José Luis Corchero, CIBER - Center for Biomedical Research Network, Autonomous University of Barcelona
  • ,
  • Elisabet González-Mira, CSIC - Institute of Materials Science of Barcelona, CIBER - Center for Biomedical Research Network
  • ,
  • Albert Font, Leanbio S.L
  • ,
  • Jannik N. Pedersen
  • ,
  • Natalia García-Aranda, CIBER - Center for Biomedical Research Network, Autonomous University of Barcelona
  • ,
  • Edgar Cristóbal-Lecina, CIBER - Center for Biomedical Research Network, CSIC - Institute for Advanced Chemistry of Catalonia
  • ,
  • Marta Alcaina-Hernando, NANOMOL TECHNOLOGIES SL
  • ,
  • Rosa Mendoza, CIBER - Center for Biomedical Research Network, Autonomous University of Barcelona
  • ,
  • Elena Garcia-Fruitós, CIBER - Center for Biomedical Research Network, Autonomous University of Barcelona
  • ,
  • Teresa Lizarraga, Leanbio S.L
  • ,
  • Susanne Resch, BIONANONET FORSCHUNGSGESELLSCHAFT MBH
  • ,
  • Christa Schimpel, BIONANONET FORSCHUNGSGESELLSCHAFT MBH
  • ,
  • Andreas Falk, BIONANONET FORSCHUNGSGESELLSCHAFT MBH
  • ,
  • Daniel Pulido, CIBER - Center for Biomedical Research Network, CSIC - Institute for Advanced Chemistry of Catalonia
  • ,
  • Miriam Royo, CIBER - Center for Biomedical Research Network, CSIC - Institute for Advanced Chemistry of Catalonia
  • ,
  • Simó Schwartz, CIBER - Center for Biomedical Research Network, Autonomous University of Barcelona
  • ,
  • Ibane Abasolo, CIBER - Center for Biomedical Research Network, Autonomous University of Barcelona
  • ,
  • Jan Skov Pedersen
  • Dganit Danino, Technion-Israel Institute of Technology
  • ,
  • Andreu Soldevila, Leanbio S.L
  • ,
  • Jaume Veciana, CSIC - Institute of Materials Science of Barcelona, CIBER - Center for Biomedical Research Network
  • ,
  • Santi Sala, NANOMOL TECHNOLOGIES SL
  • ,
  • Nora Ventosa, CSIC - Institute of Materials Science of Barcelona, CIBER - Center for Biomedical Research Network
  • ,
  • Alba Córdoba, NANOMOL TECHNOLOGIES SL

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.

OriginalsprogEngelsk
Artikelnummer105204
TidsskriftJournal of Supercritical Fluids
Vol/bind173
ISSN0896-8446
DOI
StatusUdgivet - jul. 2021

Bibliografisk note

Funding Information:
We also acknowledge the ICTS “NANBIOSIS”, more specifically the support from the Protein Production Platform of CIBER-BBN/IBB, at the UAB SepBioES scientific-technical service ( www.nanbiosis.es/unit/u1-proteinproduction-platform-ppp/ ), the Soft Materials Service linked to Biomaterial Processing and Nanostructuring Unit at ICMAB-CSIC ( www.nanbiosis.es/portfolio/u6-biomaterial-processing-and-nanostructuring-unit/ ) and the Peptide Synthesis unit at the IQAC-CSIC ( www.nanbiosis.es/portfolio/u3-synthesis-of-peptides-unit/ ).

Funding Information:
We acknowledge financial support to our research from Instituto de Salud Carlos III, through “Acciones CIBER”. The Networking Research Center on Bioengineering, Biomaterials, and Nanomedicine (CIBER-BBN) is 2008–2011, financed by the Instituto de Salud Carlos III with assistance from the European Regional Development Fund. Authors acknowledge financial support from the Agencia Estatal de Investigación-Ministerio de Ciencia e Innovación through the “Severo Ochoa” Programme for Centres of Excellence in R & D (CEX2019-000917-S). This work was also financed by 1084 the Ministerio de Ciencia e Innovación (PID2019-105622RB-1085 I00). We also thank the denomination of the 1101 consolidated group from Generalitat de Catalunya: 2017-1102 SGR-1439 (M.R.) and 2017-SGR-918 (J.V.).

Funding Information:
The authors acknowledge the financial support from European Commission through H2020 program of the Smart-4-Fabry project (ID 720942 ).

Funding Information:
N. A-G. is supported by a PERIS grant from the Catalan Government (SLT006/17/270).

Funding Information:
The authors acknowledge the financial support from European Commission through H2020 program of the Smart-4-Fabry project (ID 720942). We acknowledge financial support to our research from Instituto de Salud Carlos III, through ?Acciones CIBER?. The Networking Research Center on Bioengineering, Biomaterials, and Nanomedicine (CIBER-BBN) is 2008?2011, financed by the Instituto de Salud Carlos III with assistance from the European Regional Development Fund. Authors acknowledge financial support from the Agencia Estatal de Investigacio?n-Ministerio de Ciencia e Innovacio?n through the ?Severo Ochoa? Programme for Centres of Excellence in R & D (CEX2019-000917-S). This work was also financed by 1084 the Ministerio de Ciencia e Innovacio?n (PID2019-105622RB-1085 I00). We also thank the denomination of the 1101 consolidated group from Generalitat de Catalunya: 2017-1102 SGR-1439 (M.R.) and 2017-SGR-918 (J.V.). We also acknowledge the ICTS ?NANBIOSIS?, more specifically the support from the Protein Production Platform of CIBER-BBN/IBB, at the UAB SepBioES scientific-technical service (www.nanbiosis.es/unit/u1-proteinproduction-platform-ppp/), the Soft Materials Service linked to Biomaterial Processing and Nanostructuring Unit at ICMAB-CSIC (www.nanbiosis.es/portfolio/u6-biomaterial-processing-and-nanostructuring-unit/) and the Peptide Synthesis unit at the IQAC-CSIC (www.nanbiosis.es/portfolio/u3-synthesis-of-peptides-unit/). N. A-G. is supported by a PERIS grant from the Catalan Government (SLT006/17/270). J. T-M. is supported by a FI-AGAUR grant from the Catalan Government and the European Social Fund (ESF-Investing in your future) of the European Union. This work has been done in the framework of the JT-M. doctorate in Materials Science of the Universitat Aut?noma de Barcelona. The technical assistance of Inbal Ionita, Ella Kesselman and Mingming Zhang from Technion - Israel Institute of Technology; and Ramon Gonz?lez from ICMAB-CSIC is acknowledged.

Funding Information:
J. T-M. is supported by a FI-AGAUR grant from the Catalan Government and the European Social Fund (ESF-Investing in your future) of the European Union. This work has been done in the framework of the JT-M. doctorate in Materials Science of the Universitat Autònoma de Barcelona.

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© 2021

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