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PhD Theses

Aida Carreño will defend her PhD Thesis on Fri, 23 Feb 2024 at ICMAB

The PhD researcher Aida Carreño from the Nanomol-BIO group at ICMAB-CSIC, will defend her PhD thesis on Friday, 23 Feb 2024 at 11:00 AM at ICMAB.

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05 February 2024

Advanced Characterization of Alpha-galactosidase Nanoliposomal Formulations for Fabry Disease Treatment

Aida Carreño from the Nanomol-BIO group at ICMAB-CSIC

Date: Friday, 23 Feb 2024
Time: 11:00 PM
Venue: Institut de Ciència de Materials de Barcelona (ICMAB, CSIC) - Sala d'Actes Carles Miravitlles

Abstract:

Nanopharmaceuticals represent a promising frontier in addressing the limitations of conventional drugs. Those based on nanocarriers as drug delivery systems offer advantages thanks to their size and tuneability, allowing to be designed to improve the drug half-life, biodistribution, and release profile. Despite these advantages, translating nanopharmaceuticals to an advanced stage of pharmaceutical development faces significant challenges, primarily related to ensuring adherence to stringent quality standards for preclinical (and further clinical) testing taking into account their complex multicomponent nature. In this thesis, an advanced characterization effort has been carried out to understand, guarantee and control the quality of a novel, under development, nanoliposomal formulation for Fabry disease treatment - a lysosomal storage disorder caused by the lack of α-galactosidase (GLA) enzyme. These nanoliposomes entrapping the GLA enzyme and prepared by the green-scalable DELOS-susp methodology yielded a patented product more effective at preclinical level than traditional marketed pharmaceuticals. To support the development of this nanoformulation to become a potential intravenous drug product candidate, further understanding and control of the molecular and supramolecular interaction between the liposomal nanocarrier and the GLA enzyme have been required. In this thesis, four recombinant GLA enzymes differing in purification tags and expression systems have been explored to investigate which GLA properties are the most favorable to generate stable, efficient and robust liposome-based nanoconjugates for Fabry therapy. Moreover, advanced methodologies have been developed, optimized and/or validated to characterize and control those nanoformulation attributes identified as critical to ensure the desired product quality, taking into account the regulatory compliance requirements.

Supervisors

  • Nora Ventosa Rull (ICMAB-CSIC)
  • Elisabet González Mira (ICMAB-CSIC)
  • José Luis Corchero Nieto (IBB-UAB)

PhD Committee:

  • President: Miriam Royo Expòsito, Institut de Química Avançada de Catalunya – Agència Estatal Consell Superior d’Investigacions Científiques (IQAC-CSIC)
  • Secretary: Ibane Abasolo Olaortua, Vall d’Hebron Institut de Recerca (VHIR)
  • Vocal: Thomas Birngruber, Joanneum Research (Austria)

University: Universitat Autònoma de Barcelona (UAB)
PhD Programme: Materials Science

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