Lipid nanoparticles beyond Covid vaccines

The pandemic has accelerated progress in the field of lipid nanoparticles (LNP), and now CRISPR-based treatments are set to reap the benefits.

An old saying in the gene therapy field is that there are only three problems: delivery. delivery, delivery. One could also say that the challenges are three: manufacturing, manufacturing, manufacturing. Indeed, in addition to designing increasingly efficient and safe systems to deliver the molecules needed to correct DNA in the target organs and tissues, we need to be able to produce them on a large scale in order to make the treatments available to a potentially large number of patients.

The good news is that recent progress in this direction has been remarkable. In their pandemic effort, Pfizer and Moderna scaled LNP manufacture in less than one year and delivered billions of doses of their mRNA vaccines, meaning that LNP-CRISPR-treatments can hopefully be scaled as well. Please see the numbers of Pfizer’s light-speed approach in Nature Biotechnology.

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