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

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 fi...

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Autores: Merlo-Mas, Josep, Tomsen Melero, Judit|||0000-0002-2837-8107, Corchero Nieto, José Luis|||0000-0002-6109-144X, González Mira, Elisabet, Font, Albert|||0000-0003-3908-1111, Pedersen, Jannik N., García Aranda, Natalia|||0000-0003-3230-9881, Cristóbal-Lecina, Edgar, Alcaina Hernando, Marta, Mendoza Moreno, Rosa|||0000-0002-0488-4814, Garcia-Fruitos, Elena|||0000-0001-7498-4864, Lizarraga, Teresa, Resch, Susanne, Schimpel, Christa, Falk, Andreas, Pulido, Daniel|||0000-0002-2841-194X, Royo, Miriam|||0000-0001-5292-0819, Schwartz, Simon|||0000-0001-8297-7971, Abasolo, Ibane|||0000-0001-5970-6276, Pedersen, Jan Skov|||0000-0002-7768-0206, Danino, Dganit, Soldevila, Andreu, Veciana i Miró, Jaume|||0000-0003-1023-9923, Sala, Santi, Ventosa, Nora|||0000-0002-8008-4974, Córdoba, Alba
Formato: artículo
Fecha de publicación:2021
País:España
Recursos:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:247506
Acesso em linha:https://ddd.uab.cat/record/247506
https://dx.doi.org/urn:doi:10.1016/j.supflu.2021.105204
Access Level:acceso abierto
Palavra-chave:Quality by Design
DELOS
Scale-up
Protein-loaded liposomes
Fabry disease
α-galactosidase
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spelling Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp methodMerlo-Mas, JosepTomsen Melero, Judit|||0000-0002-2837-8107Corchero Nieto, José Luis|||0000-0002-6109-144XGonzález Mira, ElisabetFont, Albert|||0000-0003-3908-1111Pedersen, Jannik N.García Aranda, Natalia|||0000-0003-3230-9881Cristóbal-Lecina, EdgarAlcaina Hernando, MartaMendoza Moreno, Rosa|||0000-0002-0488-4814Garcia-Fruitos, Elena|||0000-0001-7498-4864Lizarraga, TeresaResch, SusanneSchimpel, ChristaFalk, AndreasPulido, Daniel|||0000-0002-2841-194XRoyo, Miriam|||0000-0001-5292-0819Schwartz, Simon|||0000-0001-8297-7971Abasolo, Ibane|||0000-0001-5970-6276Pedersen, Jan Skov|||0000-0002-7768-0206Danino, DganitSoldevila, AndreuVeciana i Miró, Jaume|||0000-0003-1023-9923Sala, SantiVentosa, Nora|||0000-0002-8008-4974Córdoba, AlbaQuality by DesignDELOSScale-upProtein-loaded liposomesFabry diseaseα-galactosidaseFabry 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. 22021-01-0120212021-01-01Articlehttp://purl.org/coar/resource_type/c_6501VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttps://ddd.uab.cat/record/247506https://dx.doi.org/urn:doi:10.1016/j.supflu.2021.105204reponame:Dipòsit Digital de Documents de la UABinstname:Universitat Autònoma de BarcelonaInglésengEuropean Commission https://doi.org/10.13039/501100000780 720942Ministerio de Ciencia e Innovación https://doi.org/10.13039/501100004837 CEX2019-000917-SMinisterio de Ciencia e Innovación https://doi.org/10.13039/501100004837 PID2019-105622RB-1085I00Agència de Gestió d'Ajuts Universitaris i de Recerca https://doi.org/10.13039/501100003030 2017/SGR-1439Agència de Gestió d'Ajuts Universitaris i de Recerca https://doi.org/10.13039/501100003030 2017/SGR-918Agència de Gestió d'Ajuts Universitaris i de Recerca https://doi.org/10.13039/501100003030 SLT006/17/270open accesshttp://purl.org/coar/access_right/c_abf2Aquest document està subjecte a una llicència d'ús Creative Commons. Es permet la reproducció total o parcial, la distribució, i la comunicació pública de l'obra, sempre que no sigui amb finalitats comercials, i sempre que es reconegui l'autoria de l'obra original. No es permet la creació d'obres derivades.https://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:ddd.uab.cat:2475062026-06-06T12:50:31Z
dc.title.none.fl_str_mv Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method
title Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method
spellingShingle Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method
Merlo-Mas, Josep
Quality by Design
DELOS
Scale-up
Protein-loaded liposomes
Fabry disease
α-galactosidase
title_short Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method
title_full Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method
title_fullStr Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method
title_full_unstemmed Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method
title_sort Application of Quality by Design to the robust preparation of a liposomal GLA formulation by DELOS-susp method
dc.creator.none.fl_str_mv Merlo-Mas, Josep
Tomsen Melero, Judit|||0000-0002-2837-8107
Corchero Nieto, José Luis|||0000-0002-6109-144X
González Mira, Elisabet
Font, Albert|||0000-0003-3908-1111
Pedersen, Jannik N.
García Aranda, Natalia|||0000-0003-3230-9881
Cristóbal-Lecina, Edgar
Alcaina Hernando, Marta
Mendoza Moreno, Rosa|||0000-0002-0488-4814
Garcia-Fruitos, Elena|||0000-0001-7498-4864
Lizarraga, Teresa
Resch, Susanne
Schimpel, Christa
Falk, Andreas
Pulido, Daniel|||0000-0002-2841-194X
Royo, Miriam|||0000-0001-5292-0819
Schwartz, Simon|||0000-0001-8297-7971
Abasolo, Ibane|||0000-0001-5970-6276
Pedersen, Jan Skov|||0000-0002-7768-0206
Danino, Dganit
Soldevila, Andreu
Veciana i Miró, Jaume|||0000-0003-1023-9923
Sala, Santi
Ventosa, Nora|||0000-0002-8008-4974
Córdoba, Alba
author Merlo-Mas, Josep
author_facet Merlo-Mas, Josep
Tomsen Melero, Judit|||0000-0002-2837-8107
Corchero Nieto, José Luis|||0000-0002-6109-144X
González Mira, Elisabet
Font, Albert|||0000-0003-3908-1111
Pedersen, Jannik N.
García Aranda, Natalia|||0000-0003-3230-9881
Cristóbal-Lecina, Edgar
Alcaina Hernando, Marta
Mendoza Moreno, Rosa|||0000-0002-0488-4814
Garcia-Fruitos, Elena|||0000-0001-7498-4864
Lizarraga, Teresa
Resch, Susanne
Schimpel, Christa
Falk, Andreas
Pulido, Daniel|||0000-0002-2841-194X
Royo, Miriam|||0000-0001-5292-0819
Schwartz, Simon|||0000-0001-8297-7971
Abasolo, Ibane|||0000-0001-5970-6276
Pedersen, Jan Skov|||0000-0002-7768-0206
Danino, Dganit
Soldevila, Andreu
Veciana i Miró, Jaume|||0000-0003-1023-9923
Sala, Santi
Ventosa, Nora|||0000-0002-8008-4974
Córdoba, Alba
author_role author
author2 Tomsen Melero, Judit|||0000-0002-2837-8107
Corchero Nieto, José Luis|||0000-0002-6109-144X
González Mira, Elisabet
Font, Albert|||0000-0003-3908-1111
Pedersen, Jannik N.
García Aranda, Natalia|||0000-0003-3230-9881
Cristóbal-Lecina, Edgar
Alcaina Hernando, Marta
Mendoza Moreno, Rosa|||0000-0002-0488-4814
Garcia-Fruitos, Elena|||0000-0001-7498-4864
Lizarraga, Teresa
Resch, Susanne
Schimpel, Christa
Falk, Andreas
Pulido, Daniel|||0000-0002-2841-194X
Royo, Miriam|||0000-0001-5292-0819
Schwartz, Simon|||0000-0001-8297-7971
Abasolo, Ibane|||0000-0001-5970-6276
Pedersen, Jan Skov|||0000-0002-7768-0206
Danino, Dganit
Soldevila, Andreu
Veciana i Miró, Jaume|||0000-0003-1023-9923
Sala, Santi
Ventosa, Nora|||0000-0002-8008-4974
Córdoba, Alba
author2_role author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
author
dc.subject.none.fl_str_mv Quality by Design
DELOS
Scale-up
Protein-loaded liposomes
Fabry disease
α-galactosidase
topic Quality by Design
DELOS
Scale-up
Protein-loaded liposomes
Fabry disease
α-galactosidase
description 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.
publishDate 2021
dc.date.none.fl_str_mv 2
2021-01-01
2021
2021-01-01
dc.type.none.fl_str_mv Article
http://purl.org/coar/resource_type/c_6501
VoR
http://purl.org/coar/version/c_970fb48d4fbd8a85
dc.type.openaire.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv https://ddd.uab.cat/record/247506
https://dx.doi.org/urn:doi:10.1016/j.supflu.2021.105204
url https://ddd.uab.cat/record/247506
https://dx.doi.org/urn:doi:10.1016/j.supflu.2021.105204
dc.language.none.fl_str_mv Inglés
eng
language_invalid_str_mv Inglés
language eng
dc.relation.none.fl_str_mv European Commission https://doi.org/10.13039/501100000780 720942
Ministerio de Ciencia e Innovación https://doi.org/10.13039/501100004837 CEX2019-000917-S
Ministerio de Ciencia e Innovación https://doi.org/10.13039/501100004837 PID2019-105622RB-1085I00
Agència de Gestió d'Ajuts Universitaris i de Recerca https://doi.org/10.13039/501100003030 2017/SGR-1439
Agència de Gestió d'Ajuts Universitaris i de Recerca https://doi.org/10.13039/501100003030 2017/SGR-918
Agència de Gestió d'Ajuts Universitaris i de Recerca https://doi.org/10.13039/501100003030 SLT006/17/270
dc.rights.none.fl_str_mv open access
http://purl.org/coar/access_right/c_abf2
https://creativecommons.org/licenses/by-nc-nd/4.0/
dc.rights.openaire.fl_str_mv info:eu-repo/semantics/openAccess
rights_invalid_str_mv open access
http://purl.org/coar/access_right/c_abf2
https://creativecommons.org/licenses/by-nc-nd/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.source.none.fl_str_mv reponame:Dipòsit Digital de Documents de la UAB
instname:Universitat Autònoma de Barcelona
instname_str Universitat Autònoma de Barcelona
reponame_str Dipòsit Digital de Documents de la UAB
collection Dipòsit Digital de Documents de la UAB
repository.name.fl_str_mv
repository.mail.fl_str_mv
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