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Evaluating how cationic lipid affects mRNA-LNP physical properties and biodistribution
Eur J Pharm Biopharm. 2023 Aug 12:S0939-6411(23)00205-9. doi: 10.1016/j.ejpb.2023.08.002. Online ahead of print.ABSTRACTRNA therapeutics represents a powerful strategy for diseases where other approaches have failed, especially given the recent successes of mRNA vaccines against the coronavirus disease 2019 (COVID-19) and small interfering (siRNA) therapeutics. However, further developments are still required to reduce toxicity, improve stability and biodistribution of mRNA-LNPs (lipid nanoparticles). Here, we show a rational combinatorial approach to select the best formulation based on a new cationic lipid molecule (IM21...
Source: European Journal of Pharmaceutics and Biopharmaceutics - August 14, 2023 Category: Drugs & Pharmacology Authors: Claire Gu éguen Thibaut Ben Chimol Margaux Briand Kassandra Renaud M élodie Seiler Morgane Ziesel Patrick Erbacher Malik Hellal Source Type: research

Lipid nanoparticle-mediated drug delivery to the brain
Adv Drug Deliv Rev. 2023 May 5:114861. doi: 10.1016/j.addr.2023.114861. Online ahead of print.ABSTRACTLipid nanoparticles (LNPs) have revolutionized the field of drug delivery through their applications in siRNA delivery to the liver (Onpattro) and their use in the Pfizer-BioNTech and Moderna COVID-19 mRNA vaccines. While LNPs have been extensively studied for the delivery of RNA drugs to muscle and liver targets, their potential to deliver drugs to challenging tissue targets such as the brain remains underexplored. Multiple brain disorders currently lack safe and effective therapies and therefore repurposing LNPs could po...
Source: Advanced Drug Delivery Reviews - May 7, 2023 Category: Drugs & Pharmacology Authors: Purva Khare Sara X Edgecomb Christine M Hamadani Eden E L Tanner Devika S Manickam Source Type: research

Use of Iontophoresis Technology for Transdermal Delivery of a Minimal mRNA Vaccine as a Potential Melanoma Therapeutic
Biol Pharm Bull. 2023;46(2):301-308. doi: 10.1248/bpb.b22-00746.ABSTRACTmRNA vaccines have attracted considerable attention as a result of the 2019 coronavirus pandemic; however, challenges remain regarding use of mRNA vaccines, including insufficient delivery owing to the high molecular weights and high negative charges associated with mRNA. These characteristics of mRNA vaccines impair intracellular uptake and subsequent protein translation. In the current study, we prepared a minimal mRNA vaccine encoding a tumor associated antigen human gp10025-33 peptide (KVPRNQDWL), as a potential treatment for melanoma. Minimal mRNA...
Source: Biological and Pharmaceutical Bulletin - February 1, 2023 Category: Drugs & Pharmacology Authors: Rabab A Husseini Naoko Abe Tomoaki Hara Hiroshi Abe Kentaro Kogure Source Type: research

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

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