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Directing the Way —Receptor and Chemical Targeting Strategies for Nucleic Acid Delivery
AbstractNucleic acid therapeutics have shown great potential for the treatment of numerous diseases, such as genetic disorders, cancer and infections. Moreover, they have been successfully used as vaccines during the COVID-19 pandemic. In order to unfold full therapeutical potential, these nano agents have to overcome several barriers. Therefore, directed transport to specific tissues and cell types remains a central challenge to receive carrier systems with enhanced efficiency and desired biodistribution profiles. Active targeting strategies include receptor-targeting, mediating cellular uptake based on ligand-receptor in...
Source: Pharmaceutical Research - September 15, 2022 Category: Drugs & Pharmacology Source Type: research

The role of lipid components in lipid nanoparticles for vaccines and gene therapy
Adv Drug Deliv Rev. 2022 Jul 1:114416. doi: 10.1016/j.addr.2022.114416. Online ahead of print.ABSTRACTLipid nanoparticles (LNPs) play a key role in mRNA vaccines against COVID-19. In addition, many preclinical and clinical studies, including the siRNA-LNP product, Onpattro®, highlight that LNPs unlock the potential of nucleic acid-based therapies and vaccines. To understand how this 'key' works, we need to learn about the building blocks that constitute LNPs. In this review, we discuss what each lipid component adds to the LNP delivery platform in terms of size, structure, stability, apparent pKa, nucleic acid encapsulati...
Source: Advanced Drug Delivery Reviews - July 5, 2022 Category: Drugs & Pharmacology Authors: Camilla Hald Albertsen Jayesh Kulkarni Dominik Witzigmann Marianne Lind Karsten Petersson Jens B Simonsen Source Type: research

Regulatory guidelines and preclinical tools to study the biodistribution of RNA therapeutics
Adv Drug Deliv Rev. 2022 Mar 26;184:114236. doi: 10.1016/j.addr.2022.114236. Online ahead of print.ABSTRACTThe success of the messenger RNA-based COVID-19 vaccines of Moderna and Pfizer/BioNTech marks the beginning of a new chapter in modern medicine. However, the rapid rise of mRNA therapeutics has resulted in a regulatory framework that is somewhat lagging. The current guidelines either do not apply, do not mention RNA therapeutics, or do not have widely accepted definitions. This review describes the guidelines for preclinical biodistribution studies of mRNA/siRNA therapeutics and highlights the relevant differences for...
Source: Advanced Drug Delivery Reviews - March 30, 2022 Category: Drugs & Pharmacology Authors: P Vervaeke S E Borgos N N Sanders F Combes Source Type: research

Delivery Strategies for mRNA Vaccines
AbstractThe therapeutic potential for messenger RNA (mRNA) in infectious diseases and cancer was first realized almost three decades ago, but only in 2018 did the first lipid nanoparticle-based small interfering RNA (siRNA) therapy reach the market with the United States Food and Drug Administration (FDA) approval of patisiran (Onpattro ™) for hereditary ATTR amyloidosis. This was largely made possible by major advances in the formulation technology for stabilized lipid-based nanoparticles (LNPs). Design of the cationic ionizable lipids, which are a key component of the LNP formulations, with an acid dissociation constan...
Source: Pharmaceutical Medicine - January 30, 2022 Category: Drugs & Pharmacology Source Type: research

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