Deep Dive #18: Non-Viral Gene Delivery
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Overview
Non-viral gene delivery modalities have been around for decades, entering the clinic during the early 2000s, and further developing year by year. The technology took off during the SARS-CoV-2 pandemic with the Moderna and Pfizer/BioNTech mRNA lipid nanoparticles (LNP) vaccine that significantly reduced the severity of COVID and hospitalizations. The FDA approval of the LNP vaccine sets the precedent and opens doors for future non-viral gene delivery innovations. This space is becoming of increased interest with intersections across various indication spaces. Furthermore, the technologies allow for “plug-and-play" with nucleic acid/gene cargos ranging from mRNA, DNA, CRISPR-Cas9, miRNA, siRNA, CAR constructs, and many more. The non-viral gene delivery market has been growing and expected to exponentially increase. Translational development of non-viral gene delivery will have broad impacts given the potential for high loading, low immunogenicity, multiple dosing, cost, stability, and tissue/cell targeting.