Lipid-based siRNA Nanodelivery Systems: A Learning Process for Improving Transfer from Concepts to Clinical Applications

Current Clinical Pharmacology
Sebastián Ezequiel Pérez, Adriana Mónica Carlucci

Abstract

The molecular mechanism of silencing genes using small interference RNA is as particular and innovative phenomenon as the proposed delivery systems to release them. Recent advances in RNAi have resulted in the development of multiple siRNA candidates that are currently being evaluated in preclinical / clinical instances. SNALP®, Atuplex® and Rondel® technologies stand out; they are mainly based on polymers, cyclodextrins or lipids. 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. 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 pharmaceutica...Continue Reading

Citations

Nov 7, 2019·Chemistry and Physics of Lipids·Marcel ScheidelerRuth Prassl
Nov 13, 2019·International Journal of Pharmaceutics·Aurélie Paganin-GioanniMuriel Golzio

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