The 60-year evolution of lipid nanoparticles for nucleic acid delivery

Jul 4, 2024Nature reviews. Drug discovery

Sixty years of developing tiny fat particles to deliver genetic material

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Abstract

Lipid nanoparticles (LNPs) have enabled the successful delivery of nucleic acid-based vaccines and therapeutics, exemplified by the COVID-19 mRNA vaccines and Onpattro.

Advances in lipid-based systems have addressed the challenge of delivering genetic information to target cells in vivo.
Clinically approved examples of LNPs include mRNA vaccines for COVID-19 and the small interfering RNA therapeutic Onpattro for amyloidosis.
Ongoing clinical development includes various promising LNP-enabled vaccines and gene therapies.
The success of LNPs is linked to research on lipoplexes with positively charged lipids and the use of ionizable cationic lipids in LNP design.
Insights from these research streams may provide delivery solutions for a wide range of gene therapies.

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Delivery of genetic information to the interior of target cells in vivo has been a major challenge facing gene therapies. This barrier is now being overcome, owing in part to dramatic advances made by lipid-based systems that have led to lipid nanoparticles (LNPs) that enable delivery of nucleic acid-based vaccines and therapeutics. Examples include the clinically approved COVID-19 LNP mRNA vaccines and Onpattro (patisiran), an LNP small interfering RNA therapeutic to treat transthyretin-induced amyloidosis (hATTR). In addition, a host of promising LNP-enabled vaccines and gene therapies are in clinical development. Here, we trace this success to two streams of research conducted over the past 60 years: the discovery of the transfection properties of lipoplexes composed of positively charged cationic lipids complexed with nucleic acid cargos and the development of lipid nanoparticles using ionizable cationic lipids. The fundamental insights gained from these two streams of research offer potential delivery solutions for most forms of gene therapies.

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The 60-year evolution of lipid nanoparticles for nucleic acid delivery

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