Sebastián Ezequiel Pérez* and Adriana Mónica Carlucci Pages 142-163 (22)
Method: The objective of this work is to review the main features that Gene Therapy Medicinal Product under current clinical evaluation present from a pharmaceutical technology point of view; it tries to bring up theoretical concepts that give scientific support to the interpretation of data obtained during pharmaceutical development process. It is basically focused on improving the translation from bench/theoretical concepts to bedside of non viral vectors carrying siRNA.
Results: The extensive presence of lipid-based nanoparticle non-viral systems in clinical stages is due to the advantages of their formulations. These include: safety, low immunogenicity, high degree of material properties control, function tuning and ability to impact pharmacokinetics and in vivo biodistribution. This work presents a pharmaceutical approach so as to improve the potential of success in siRNA delivery using liposomal systems.
Conclusion: Formulation design should be increasingly addressed with industrial criteria; it should be based on quality by design and on the estimation of critical attributes that affect product performance, and supported by a range of characterization techniques and appropriate analytical methods.
Gene therapy, antisense therapy, GTMP, siRNA nanodelivery systems, liposomal formulations, gene silencing.
Department of Pharmaceutical Technology, Faculty of Pharmacy and Biochemistry, University of Buenos Aires, Ciudad Autonoma de Buenos Aires, Argentina, Junin 956 (C1113AAD) Ciudad Autonoma de Buenos Aires, Department of Pharmaceutical Technology, Faculty of Pharmacy and Biochemistry, University of Buenos Aires, Ciudad Autonoma de Buenos Aires, Argentina, Junin 956 (C1113AAD) Ciudad Autonoma de Buenos Aires