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, Corchero, José-Luis, González-Mira, Elisabet, Font, Albert, Pedersen, Jannik N., García-Aranda, Natalia, Cristóbal-Lecina, Edgar, Alcaina-Hernando, Marta, Mendoza, Rosa, Garcia-Fruitos, Elena, Lizarraga, Teresa, Resch, Susanne, Schimpel, Christa, Falk, Andreas, Pulido, Daniel, Royo, Miriam, Schwartz Jr., Simó, Abasolo, Ibane, Pedersen, Jan Skov, Danino, Dganit, Soldevila, Andreu, Veciana, Jaume, Sala, Santi, Ventosa, Nora, Córdoba, Alba
Tipo de recurso: artículo
Fecha de publicación:2021
País:España
Institución:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
Repositorio:Recercat. Dipósit de la Recerca de Catalunya
OAI Identifier:oai:recercat.cat:20.500.12327/1599
Acceso en línea:http://hdl.handle.net/20.500.12327/1599
https://doi.org/10.1016/j.supflu.2021.105204
Access Level:acceso abierto
Palabra clave:577
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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, JuditCorchero, José-LuisGonzález-Mira, ElisabetFont, AlbertPedersen, Jannik N.García-Aranda, NataliaCristóbal-Lecina, EdgarAlcaina-Hernando, MartaMendoza, RosaGarcia-Fruitos, ElenaLizarraga, TeresaResch, SusanneSchimpel, ChristaFalk, AndreasPulido, DanielRoyo, MiriamSchwartz Jr., SimóAbasolo, IbanePedersen, Jan SkovDanino, DganitSoldevila, AndreuVeciana, JaumeSala, SantiVentosa, NoraCórdoba, Alba577Fabry 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.info:eu-repo/semantics/publishedVersionElsevierProducció AnimalProducció de Remugants2021info:eu-repo/semantics/article15http://hdl.handle.net/20.500.12327/1599https://doi.org/10.1016/j.supflu.2021.105204reponame:Recercat. Dipósit de la Recerca de Catalunyainstname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)InglésJournal of Supercritical FluidsEC/H2020/720942/EU/Smart multifunctional GLA-nanoformulation for Fabry disease/Smart-4-FabryMICIU-AEI/Programa Estatal de generación del conocimiento y fortalecimiento científico y tecnológico del sistema I+D+I y Programa Estatal de I+D+I orientada a los retos de la sociedad/PID2019-105622RB-I00/ES/PROCESAMIENTO DE MOLECULAS PARA CREAR MATERIALES HIBRIDOS ESTRUCTURADOS JERARQUICAMENTE PARA APLICACIONES BIOMEDICAS/Attribution 4.0 Internationalhttp://creativecommons.org/licenses/by/4.0/info:eu-repo/semantics/openAccessoai:recercat.cat:20.500.12327/15992026-05-29T05:05:01Z
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
577
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
Corchero, José-Luis
González-Mira, Elisabet
Font, Albert
Pedersen, Jannik N.
García-Aranda, Natalia
Cristóbal-Lecina, Edgar
Alcaina-Hernando, Marta
Mendoza, Rosa
Garcia-Fruitos, Elena
Lizarraga, Teresa
Resch, Susanne
Schimpel, Christa
Falk, Andreas
Pulido, Daniel
Royo, Miriam
Schwartz Jr., Simó
Abasolo, Ibane
Pedersen, Jan Skov
Danino, Dganit
Soldevila, Andreu
Veciana, Jaume
Sala, Santi
Ventosa, Nora
Córdoba, Alba
author Merlo-Mas, Josep
author_facet Merlo-Mas, Josep
Tomsen-Melero, Judit
Corchero, José-Luis
González-Mira, Elisabet
Font, Albert
Pedersen, Jannik N.
García-Aranda, Natalia
Cristóbal-Lecina, Edgar
Alcaina-Hernando, Marta
Mendoza, Rosa
Garcia-Fruitos, Elena
Lizarraga, Teresa
Resch, Susanne
Schimpel, Christa
Falk, Andreas
Pulido, Daniel
Royo, Miriam
Schwartz Jr., Simó
Abasolo, Ibane
Pedersen, Jan Skov
Danino, Dganit
Soldevila, Andreu
Veciana, Jaume
Sala, Santi
Ventosa, Nora
Córdoba, Alba
author_role author
author2 Tomsen-Melero, Judit
Corchero, José-Luis
González-Mira, Elisabet
Font, Albert
Pedersen, Jannik N.
García-Aranda, Natalia
Cristóbal-Lecina, Edgar
Alcaina-Hernando, Marta
Mendoza, Rosa
Garcia-Fruitos, Elena
Lizarraga, Teresa
Resch, Susanne
Schimpel, Christa
Falk, Andreas
Pulido, Daniel
Royo, Miriam
Schwartz Jr., Simó
Abasolo, Ibane
Pedersen, Jan Skov
Danino, Dganit
Soldevila, Andreu
Veciana, Jaume
Sala, Santi
Ventosa, Nora
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.contributor.none.fl_str_mv Producció Animal
Producció de Remugants
dc.subject.none.fl_str_mv 577
topic 577
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 2021
dc.type.none.fl_str_mv info:eu-repo/semantics/article
format article
dc.identifier.none.fl_str_mv http://hdl.handle.net/20.500.12327/1599
https://doi.org/10.1016/j.supflu.2021.105204
url http://hdl.handle.net/20.500.12327/1599
https://doi.org/10.1016/j.supflu.2021.105204
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Journal of Supercritical Fluids
EC/H2020/720942/EU/Smart multifunctional GLA-nanoformulation for Fabry disease/Smart-4-Fabry
MICIU-AEI/Programa Estatal de generación del conocimiento y fortalecimiento científico y tecnológico del sistema I+D+I y Programa Estatal de I+D+I orientada a los retos de la sociedad/PID2019-105622RB-I00/ES/PROCESAMIENTO DE MOLECULAS PARA CREAR MATERIALES HIBRIDOS ESTRUCTURADOS JERARQUICAMENTE PARA APLICACIONES BIOMEDICAS/
dc.rights.none.fl_str_mv Attribution 4.0 International
http://creativecommons.org/licenses/by/4.0/
info:eu-repo/semantics/openAccess
rights_invalid_str_mv Attribution 4.0 International
http://creativecommons.org/licenses/by/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv 15
dc.publisher.none.fl_str_mv Elsevier
publisher.none.fl_str_mv Elsevier
dc.source.none.fl_str_mv reponame:Recercat. Dipósit de la Recerca de Catalunya
instname:Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
instname_str Varias* (Consorci de Biblioteques Universitáries de Catalunya, Centre de Serveis Científics i Acadèmics de Catalunya)
reponame_str Recercat. Dipósit de la Recerca de Catalunya
collection Recercat. Dipósit de la Recerca de Catalunya
repository.name.fl_str_mv
repository.mail.fl_str_mv
_version_ 1869422452353794048
score 15,811543

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