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Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics
The world raced to develop vaccines to protect against the rapid spread of SARS-CoV-2 infection upon the recognition of COVID-19 as a global pandemic. A broad spectrum of candidates was evaluated, with mRNA-based vaccines emerging as leaders due to how quickly they were available for emergency use w...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8876479/ https://www.ncbi.nlm.nih.gov/pubmed/35214130 http://dx.doi.org/10.3390/pharmaceutics14020398 |
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author | Semple, Sean C. Leone, Robert Barbosa, Christopher J. Tam, Ying K. Lin, Paulo J. C. |
author_facet | Semple, Sean C. Leone, Robert Barbosa, Christopher J. Tam, Ying K. Lin, Paulo J. C. |
author_sort | Semple, Sean C. |
collection | PubMed |
description | The world raced to develop vaccines to protect against the rapid spread of SARS-CoV-2 infection upon the recognition of COVID-19 as a global pandemic. A broad spectrum of candidates was evaluated, with mRNA-based vaccines emerging as leaders due to how quickly they were available for emergency use while providing a high level of efficacy. As a modular technology, the mRNA-based vaccines benefitted from decades of advancements in both mRNA and delivery technology prior to the current global pandemic. The fundamental lessons of the utility of mRNA as a therapeutic were pioneered by Dr. Katalin Kariko and her colleagues, perhaps most notably in collaboration with Drew Weissman at University of Pennsylvania, and this foundational work paved the way for the development of the first ever mRNA-based therapeutic authorized for human use, COMIRNATY(®). In this Special Issue of Pharmaceutics, we will be honoring Dr. Kariko for her great contributions to the mRNA technology to treat diseases with unmet needs. In this review article, we will focus on the delivery platform, the lipid nanoparticle (LNP) carrier, which allowed the potential of mRNA therapeutics to be realized. Similar to the mRNA technology, the development of LNP systems has been ongoing for decades before culminating in the success of the first clinically approved siRNA-LNP product, ONPATTRO(®), a treatment for an otherwise fatal genetic disease called transthyretin amyloidosis. Lessons learned from the siRNA-LNP experience enabled the translation into the mRNA platform with the eventual authorization and approval of the mRNA-LNP vaccines against COVID-19. This marks the beginning of mRNA-LNP as a pharmaceutical option to treat genetic diseases. |
format | Online Article Text |
id | pubmed-8876479 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-88764792022-02-26 Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics Semple, Sean C. Leone, Robert Barbosa, Christopher J. Tam, Ying K. Lin, Paulo J. C. Pharmaceutics Review The world raced to develop vaccines to protect against the rapid spread of SARS-CoV-2 infection upon the recognition of COVID-19 as a global pandemic. A broad spectrum of candidates was evaluated, with mRNA-based vaccines emerging as leaders due to how quickly they were available for emergency use while providing a high level of efficacy. As a modular technology, the mRNA-based vaccines benefitted from decades of advancements in both mRNA and delivery technology prior to the current global pandemic. The fundamental lessons of the utility of mRNA as a therapeutic were pioneered by Dr. Katalin Kariko and her colleagues, perhaps most notably in collaboration with Drew Weissman at University of Pennsylvania, and this foundational work paved the way for the development of the first ever mRNA-based therapeutic authorized for human use, COMIRNATY(®). In this Special Issue of Pharmaceutics, we will be honoring Dr. Kariko for her great contributions to the mRNA technology to treat diseases with unmet needs. In this review article, we will focus on the delivery platform, the lipid nanoparticle (LNP) carrier, which allowed the potential of mRNA therapeutics to be realized. Similar to the mRNA technology, the development of LNP systems has been ongoing for decades before culminating in the success of the first clinically approved siRNA-LNP product, ONPATTRO(®), a treatment for an otherwise fatal genetic disease called transthyretin amyloidosis. Lessons learned from the siRNA-LNP experience enabled the translation into the mRNA platform with the eventual authorization and approval of the mRNA-LNP vaccines against COVID-19. This marks the beginning of mRNA-LNP as a pharmaceutical option to treat genetic diseases. MDPI 2022-02-11 /pmc/articles/PMC8876479/ /pubmed/35214130 http://dx.doi.org/10.3390/pharmaceutics14020398 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Review Semple, Sean C. Leone, Robert Barbosa, Christopher J. Tam, Ying K. Lin, Paulo J. C. Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics |
title | Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics |
title_full | Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics |
title_fullStr | Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics |
title_full_unstemmed | Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics |
title_short | Lipid Nanoparticle Delivery Systems to Enable mRNA-Based Therapeutics |
title_sort | lipid nanoparticle delivery systems to enable mrna-based therapeutics |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8876479/ https://www.ncbi.nlm.nih.gov/pubmed/35214130 http://dx.doi.org/10.3390/pharmaceutics14020398 |
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